Archive for March, 2021

An estimated 88 million Americans have prediabetes, and just over 1 in 10 have been diagnosed with type 2 diabetes. In Kentucky, nearly 12 percent of the adult population is affected by this disease. Week after week, Kentucky physicians care for patients with type 2 diabetes and the potentially debilitating complications it can cause. Unfortunately, it is also well-documented that pre-existing conditions such as diabetes can put patients at high-risk for complications should they contract COVID-19.

March 23, is American Diabetes Alert Day, and the Kentucky Medical Association (KMA) and its thousands of physician members are encouraging Kentuckians to learn more about their risk factors and family history of the disease, and to talk to their doctor about prevention and treatment options.

Dr. Dale Toomey

Diabetes is the condition in which the body does not properly process food for use as energy. Most of the food we eat is turned into glucose, or sugar, for our bodies to use for energy. The pancreas makes a hormone called insulin to help glucose get into the cells of our bodies. When you have diabetes, your body either doesnt make enough insulin or cant use its own insulin as well as it should. This causes too much sugar to build up in your blood. In those with type 1 diabetes, the body completely stops producing insulin, and this usually occurs in childhood or early adulthood. With type 2 diabetes, the body produces insulin, but the cells dont respond to insulin the way they should.

Prediabetes occurs when a patient has a higher-than-normal blood sugar level. Its not high enough to be considered type 2 diabetes yet, but without lifestyle changes, adults with prediabetes are more likely to develop type 2 diabetes. More than 8 in 10 American adults with prediabetes dont know they have it. These patients are also at risk for developing other serious health problems like heart disease and stroke.

The first step in preventing or delaying type 2 diabetes and related health problems is to screen and test for prediabetes. Patients are encouraged to talk to their physician during their annual well-visits about being screened for type 2 diabetes. Your physician can order a simple blood test for patients with risk factors for prediabetes and type 2 diabetes, which include anyone 45 years of age or older, those who are overweight or obese, people with a family history of type 2 or gestational diabetes, and those who are physically active less than three times per week.

Patients diagnosed with prediabetes can be referred to the National Diabetes Prevention Program (DPP) lifestyle change program to prevent or delay type 2 diabetes. Programs are conducted by lifestyle coaches who are trained on an evidence-based curriculum. The coach can engage and guide you through makingand sticking withlifestyle changes. When patients join the program, they get a full year of support to make their new, healthy habits stick and keep them from slipping back into old habits. Kentucky also offers diabetesself-management education and support (DSME) groups, which can help you learnto manage your diabetes as part of your daily life. More information on these programs is available here.

Type 2 diabetes prevention is a priority of Kentucky physicians. As a focus of its AIM for Better Care: Administrative Improvements in Medicine initiative, KMA seeks to eliminate barriers to care for diabetes and to provide patients with the most updated resources and education to help prevent and manage this disease.

On American Diabetes Alert Day, consider this a wake up call to talk to your physician about diabetes prevention and treatment and get on the right path to a healthier tomorrow.

Dale Toney, M.D. is President of the Kentucky Medical Association. He is a board-certified internist in Lexington and is employed by the University of Kentucky Albert B. Chandler Hospital, is an Associate Professor at the University of Kentucky College of Medicine and serves as the Division Chief of General Internal Medicine and Womens Health.

More here:
Dale Toney: A 'wake up call' for diabetes talk to your doctor about prevention and treatment - User-generated content

MILFORD Pelvic exams, in vitro fertilization and childbirth typically aren't comfortable procedures for any woman, but Dr.Mitchell Bellucci seeks toensure that patients are secure and that he is positive but especially calm.

So calm thatpatients sometimes askhim if he'sabout to head out for vacation after work.

Everyone would always say, Well, geez, you look like youre going on vacation, said Bellucci during an interview in his office on Friday afternoon, wearing a loose-fitting button-up shirt withthe top fourbuttons undone. For me, it was convincing myself that everything was cool, even if sometimes it wasnt.

Hes been the doctor to more than 10,000 patients and has helped deliver more than 3,500 babies in his career, he said.Known for his cowboy boots, Bellucci, 66, also frequently wore flip flops and casual Hawaiian shirts.

He has a faded 1988 newspaper clipping from the Daily Newsof himself wearinga pair offull-quill ostrich boots. Those cost him around $400, he said. He wore cowboy boots so much, he ended up developing plantar fasciitis, which causes a lot of pain in the heel.

Thecowboy boots have been retired, and Bellucci is poised to do the same.

More: U.S. Rep. Clark aims to build child care into infrastructure debate

Bellucci is retiring next month after 40 years as an OB/GYN. He'splanning to close hiswomens health care practice at 192 West St. after opening it 33 years ago. He's selling the space, which is part of the Westview Professional Building.

Over four decades, Bellucci has made his mark in advancingwomens health care, including setting up the first bone density unit at Milford Regional Medical Center, bringing womens health innovations to local television, patenting medical devices for newborns, training medical doctors on advanced laparoscopyand establishing scholarships for nurses and pre-med students.

He's gone from the swimming pool into the delivery room a few times, too.

Ive delivered no less than two dozen kids in my Speedos... well, they were swim trunks, said Bellucci, who moved to Milford to be close to the hospital (and his practice) if he was needed immediately. He has a pool athome, and has raced from there to Milford Regional several timeswith just enough time to towel off.

It's a career field he never intended to enter, but he's glad he did, he said.

Training as a medical studentwas a pivotal time for Bellucci. While growing up onLong Island, New York, knew he wanted to be surgeon, but didn't want to docancer surgery, which he thought was depressing.

From there, it was process of elimination. He thought he would be an orthopedist performing bone surgery, but found most of his time would be spentdoing hip surgery for elderly patients who stayed at the hospital for weeks, he said. Hand surgery was too long to train forand general surgery seemed too boring, he said. He considered urology, but "didnt want to look at penises and prostates all day."

Then he tried obstetrics and gynecology.

Reproduction and bringing new life into the world especially intrigued Bellucci, who has a daughter, Laura, who's 33 and a bartender in New Orleans. But on abulletin board in the hallway of his office, she's a child, peeringinto the lensof anunderwater camera. Surrounding her are photos of dozens of other babies Belluccihas helped deliver, some also now in their 30s.

Youre there at the most important timeof their lives, man. And youre part of it, he said. What other doctor gets to be with a woman in pain for eight hours, she pushes for two hours, you catch the baby, and youre the hero?Its such a neat thing to experience.

Plus, most if not all of the patients he sees are healthy and have insurance.

I like explaining things, and you have to be direct and have to not overreact, he said about making patients feel comfortable and reassured. The office even has a therapy dog "Charlie," a cockapoo who comes in occasionallyand is adored by patients, said Bellucci.

He also has aletter froma patient, written in 1994, that still makes him smile.

Hehelped a woman deliver her first baby, but she moved and delivered her second baby elsewhere. That experiencewas very upsetting, she wrote him, and said her doctor lacked a sympathetic bedside manner.

But her experience with Bellucci in Milford,by contrast, was wonderful, she wrote.She said he remained positive throughout the ordeal and joked with her to make her feel more relaxed. She wrote that she and her husbandstill laugh years later recalling when a nurse in the delivery room strapped a maxi pad to Belluccis forehead to soak up some of his sweat.

Opinion: 2020 saw great strides for women, but it also exposed inequities we should fix in 2021

When I went into it, there were five guys to one woman. Now theres five women and no guys, said Bellucci.

In the 1970s, about 7% of American gynecologists were female, according to the American College ofObstetricians and Gynecologists.Today, about 82%of OB/GYN residents throughout the nation are women.

About a decade ago, Bellucci wasapproached by Bryant University in Rhode Island to teach male physician assistant students who weredenied access to learn inside other women's health care practices. For about nine years, he taught one student a month.

Moremen are turning away fromobstetrics and gynecology, said Bellucci, and its still difficult for them togain accessinto that space. The upside is that physician assistants aren't going into delivery rooms anymore with positions like midwives and nurse practitioners. Rather, male physician assistants today are going into settings like emergency rooms or family practices, he said.

More healthcare news: Dr. Dennis Plante, who grew up in Worcester, dies of COVID-19 in Texas

When most women hit age 50 many of his patients are about thatage their estrogen drops, he said. He realized bone density was an important component to womens health, because while estrogenis the key regulator ofbonemetabolism in both men and women, menopause leads to decreased estrogen, which is associated with decliningbonemineraldensity.

Milford Regional Medical Center didnt have a bone density unit at that time, so he helped create one, he said, and he taught primary care doctors the value of testing for it.

He also helped developthe CO2 Surgical Laser,designed to treat pre-cancerous conditions like warts and lesions, and is known for his Mona Lisa Touch.

That machine, created inItaly, is a laser treatment for symptoms somewomen experience after menopause, such as painful sex. It's an alternative to hormone treatment, which can be risky for some.

A lot of women who have these problems have had breast cancer, and what do (many doctors)do? They put you on anti-estrogen and they save your life, he said. But then you say, 'Well, what about my sex life?' I think we should address that.

If you didn't see "Dr. B" at his medical practice, you might have seen him on "Pasta Playoffs," a cooking show that aired on community access television.

I used to say, If you think you can cook good food, you should go one-on-one against Grandma," said Bellucci, who enjoys cooking Italian food. "And that was the premise of the show."

Various "grandmas" competed against Bellucci in making a pasta sauce, which was thenjudged by three randomly selected customers at Caff Sorrento in Milford, said Bellucci. The showlasted five episodes, and eventually morphed into "Cooking Against Cancer," specificallybreast cancer.

Opinion: We need to help women during the pandemic and beyond

Bellucci isexcited aboutretirement and has had a rewarding career, but said itll be a tough goodbye to his practice. After closing and selling it, he's moving toRhode Island to be with his girlfriend Mary, where hell spend most of his time boating, fishing, possibly offering consulting services in the medical device industry and writing screenplays drawing from his medical knowledge.

And finally, the next time he wears a Hawaiian shirt and pair of flip flops, he really will be on vacation an extended one.

Lauren Young writes about business and pop culture. Reach her at 774-804-1499 orlyoung@wickedlocal.com. Follow her on Twitter @laurenwhy__.

Continue reading here:
Milford OB/GYN to close womens health care practice after over 30 years - Milford Daily News

Gov. Andy Beshear signed 13 health bills into law Monday, highlighting one that caps insulin costs for about 30% of Kentuckians who need the life-preserving hormone.

The limit is $30 for a 30-day supply. This is the right thing to do and its a game-changer for those who rely on insulin to live, Beshear said. Until now, a single dose of insulin which cost between $2 and $7 to manufacture could sell for an average wholesale price of around $300 per vial.

Beshear noted that in a lawsuit he filed against insulin companies while attorney general over their unconscionable overpricing,revealed that over 10 years, the price of one insulin product went up 311%, and another rose 285%, including a rise from $325 per package in 2011 to $530 in 2017.

While these companies work to increase bottom lines and sustain market shares, he said. Kentucky families hit hardest by this price gouging can be paying more than $1,000 a month on insulin just to stay alive.

House Bill 95, co-sponsored by Reps. Danny Bentley, R-Russell, and Patti Minter, D-Bowling Green, will cap the monthly out-of-pocket cost for a 30-day supply of insulin at $30 for Kentuckians covered by state-regulated employer health plans or plans purchased on the marketplace exchange.

Minter, whose son has Type 1 diabetes,has saidthe bill will cover about 30% of insulin-dependent Kentuckians. It doesnt cover Medicare, Medicaid or self-funded employer plans except the one for state employees.

I want to be very clear, this is only a first step, Minter said. There is much more work to do but today is a very big deal. It will save lives and it will give people hope. She said that for many families, it will make the difference between bankruptcy and keeping a child alive.

In a video, Angela Lautner,Kentucky #insulin4alllegislative lead, expressed her gratitude to the lawmakers for the bill, but she too warned that more work needs to be done, saying it will help about 22,000 people.

Much more has to be done, butHB 95is a step forward, said Lautner, My chapter is here for insulin for all and thats why we must continue this momentum into the next session with urgency, with priority.

More than half a million Kentuckians have diabetes, and Kentucky ranks seventh-highest in the U.S. for diabetes prevalence. Lautner said more than one in four insulin-dependent people ration insulin due to cost.

The price cap will apply regardless of the amount or type of insulin the person with diabetes needs.

In addition to capping the price, HB 95requires health plans to provide the equipment, supplies and outpatient training and education needed to help diabetics stay healthy, and forbids any reductions from this coverage by others involved in coverage.

Thefiscal impact statementattached to the bill says it will increase premiums for health benefit plans, not including the state employee plan, by about 80 cents a month.

Bentley said thats a small price to pay to make sure diabetics keep getting their insulin because without it they can suffer amputations, loss of vision, neuropathy, ketoacidosis and even death. The costs on the medical side is much more than the cost that were going to have by helping people with insulin, he said.

Beshear also signed bills addressing these health topics:

HB 140, sponsored by Rep. Deanna Frazier, R-Richmond, will permittelehealth servicesthat were allowed to expand due to the pandemic to remain in place. The bill requires reimbursement for telehealth to be equivalent to reimbursement for the same service provided in person. I think its one of the most important bills that have been passed, Beshear said.

HB 219, sponsored by Bentley, allows pharmacies to sell hypodermic syringes and needles without a prescription. The aim is to increase access to clean supplies for people who inject drugs, which will help to decrease the risk of blood-borne diseases such as hepatitis C and HIV/AIDS.

SB 55, sponsored by Sen. Stephen Meredith, R-Leitchfield, abolishes co-payments required by Medicaid.

HB 183, sponsored by Rep. Brandon Reed, R- Hodgenville,will allowKentucky hospitals to get more money from Medicaid, based on an average commercial rate instead of the current Medicaid rate, which is often below that amount. The program would not cost the state anything, because Kentuckys hospitals have agreed to cover the cost. To get the money, hospitals will have to abide by higher quality standards that are still being decided by theKentucky Hospital Associationand theCabinet for Health and Family Services.The bill is expected to help many of the states rural hospitals; arecent reportshows that 16 of them are at risk of closing.

HB 108, sponsored by Melinda Gibbons Prunty, R-Belton (Muhlenberg County), codifies current Medicaid coverage of colorectal cancer, including screenings starting at 45 for most people and genetic cancer-risk testing, to align Medicaid and commercial coverage.

HB 50, sponsored by Rep. Kim Moser, R-Taylor Mill, will make health-insurance plans comply with a 2008 federal law that requires them to treat mental health conditions and substance use disorders the same as physical health. It also requires health insurers to file annual reports with the state to show how they are complying with federal law.

HB 276, sponsored by Moser, allows Kentuckians trained as temporary COVID-19 personal-care attendants under an executive order to apply their supervised training toward their Registered Nurse Aide certification. About 300 personal-care attendants work in Kentucky long-term care facilities, Moser said while presenting the bill to the House in February.

SB 154, by Sen. Tom Buford, R-Nicholasville, lets advanced practice registered nurses and physician assistants prescribe and supervise home-health services, as federal law has allowed them to do in the pandemic. An emergency clause ensures there would be no gap in care if the federal rule ended.

HB 448, sponsored by Rep. Bill Wesley, R-Ravenna, expands the definition of qualified mental-health professional so that it fits the states juvenile code, allowing those who work in private agencies to testify in child-welfare hearings, especially around issues of emotional injury.

SB 74, by Sen. Ralph Alvarado, R-Winchester,createsbut does not fund a dementia services coordinator in the heath cabinet to manage theAlzheimers Disease and Related Disorders Council, the state plan to address Alzheimers in Kentucky, and apply for federal funding.

HB 75, sponsored by Rep. Shawn McPherson, R-Scottsville, prohibits insurance companies from increasing rates on organ donors or dropping their coverage. It would also encourage the cabinet to develop educational materials relating to organ donation.

SB 163, sponsored by Sen. Alice Forgy Kerr, R-Lexington, expands the definition of charitable health care provider to include those that provide invasive or surgical procedures. This change was needed to allow the non-profit surgery program Surgery on Sunday in Lexington to be reimbursed for liability insurance premiums.

Beshear alsovetoed five billsthat would strip power from the governor or the executive branch. None were related to health.

(Kentucky Health Newsis an independent news service of the Institute for Rural Journalism and Community Issues, based in the School of Journalism and Media at the University of Kentucky, with support from the Foundation for a Healthy Kentucky.)

See more here:
Beshear ceremonially signs 13 health bills, including one that caps price of insulin - Hoptown Chronicle

Statistics Canada. Table 13-10-0096-01 Health characteristics, annual estimates. 2019. https://www150.statcan.gc.ca/t1/tbl1/en/tv.action?pid=1310009601. Accessed 27 Nov 2019.

Vinturache A, Moledina N, McDonald S, Slater D, Tough S. Pre-pregnancy Body Mass Index (BMI) and delivery outcomes in a Canadian population. BMC Pregnancy Childbirth. 2014;14:110.

Article Google Scholar

Wei YM, Yang HX, Zhu WW, Liu XY, Meng WY, Wang YQ, et al. Risk of adverse pregnancy outcomes stratified for pre-pregnancy body mass index. J Matern Neonatal Med. 2015;29:22059.

Article Google Scholar

Kulie T, Slattengren A, Redmer J, Counts H, Eglash A, Schrager S. Obesity and womens health: an evidence-based review. J Am Board Fam Med. 2011;24:7585.

PubMed Article PubMed Central Google Scholar

Steinig J, Nagl M, Linde K, Zietlow G, Kersting A. Antenatal and postnatal depression in women with obesity: a systematic review. Arch Womens Ment Health. 2017;20:56985.

PubMed Article PubMed Central Google Scholar

Cunningham SD, Mokshagundam S, Chai H, Lewis JB, Levine J, Tobin JN, et al. Postpartum depressive symptoms: gestational weight gain as a risk factor for adolescents who are overweight or obese. J Midwifery Womens Heal. 2018;63:17884.

Article Google Scholar

LaCoursiere DY, Barrett-Connor E, OHara MW, Hutton A, Varner MW. The association between prepregnancy obesity and screening positive for postpartum depression. BJOG. 2010;117:10118.

CAS PubMed Article PubMed Central Google Scholar

Schofield Z, Kapoor D. Pre-existing mental health disorders and pregnancy. Obstet Gynaecol Reprod Med. 2019;29:749.

Article Google Scholar

Binder EB, Newport DJ, Zach EB, Smith AK, Deveau TC, Altshuler LL, et al. A serotonin transporter gene polymorphism predicts peripartum depressive symptoms in an at-risk psychiatric cohort. J Psychiatr Res. 2010;44:6406.

PubMed Article PubMed Central Google Scholar

Galea LAM, Frokjaer VG. Perinatal depression: embracing variability toward better treatment and outcomes. Neuron. 2019;102:136.

CAS PubMed Article PubMed Central Google Scholar

Guille C, Newman R, Fryml LD, Lifton CK, Epperson CN. Management of postpartum depression. J Midwifery Womens Health. 2013;58:64353.

PubMed PubMed Central Article Google Scholar

Gude NM, Roberts CT, Kalionis B, King RG. Growth and function of the normal human placenta. Thromb Res. 2004;114:397407.

CAS PubMed Article PubMed Central Google Scholar

Napso T, Yong HEJ, Lopez-Tello J, Sferruzzi-Perri AN. The role of placental hormones in mediating maternal adaptations to support pregnancy and lactation. Front Physiol. 2018;9(AUG):139.

Google Scholar

Bridges RS. Neuroendocrine regulation of maternal behavior. Front Neuroendocrinol. 2015;36:17896.

CAS PubMed Article PubMed Central Google Scholar

Freemark M. Placental hormones and the control of fetal growth. J Clin Endocrinol Metab. 2010;95:20547.

CAS PubMed Article PubMed Central Google Scholar

Sonagra AD, Biradar SM, Murthy J. Normal pregnancya state of insulin resistance. J Clin Diagnostic Res. 2014;8:CC01-3.

Google Scholar

Kampmann U, Knorr S, Fuglsang J, Ovesen P. Determinants of maternal insulin resistance during pregnancy: an updated overview. J Diabetes Res. 2019;2019:19.

Article CAS Google Scholar

Sumption LA, Garay SM, John RM. Low serum placental lactogen at term is associated with postnatal symptoms of depression and anxiety in women delivering female infants. Psychoneuroendocrinology. 2020;116:104655.

CAS PubMed Article PubMed Central Google Scholar

Janssen AB, Capron LE, ODonnell K, Tunster SJ, Ramchandani PG, Heazell AEP, et al. Maternal prenatal depression is associated with decreased placental expression of the imprinted gene PEG3. Psychol Med. 2016;46:29993011.

CAS PubMed PubMed Central Article Google Scholar

Vakili H, Jin Y, Menticoglou S, Cattini PA. CCAAT-enhancer-binding protein (C/EBP) and downstream human placental growth hormone genes are targets for dysregulation in pregnancies complicated by maternal obesity. J Biol Chem. 2013;288:2284961.

CAS PubMed PubMed Central Article Google Scholar

Jin Y, Vakili H, Liu SY, Menticoglou S, Bock ME, Cattini PA. Chromosomal architecture and placental expression of the human growth hormone gene family are targeted by pre-pregnancy maternal obesity. Am J Physiol Endocrinol Metab. 2018;315:E43545.

CAS PubMed Article PubMed Central Google Scholar

Muralimanoharan S, Maloyan A, Myatt L. Mitochondrial function and glucose metabolism in the placenta with gestational diabetes mellitus: role of miR-143. Clin Sci (Lond). 2016;130:93141.

CAS Article Google Scholar

Sibiak R, Jankowski M, Gutaj P, Mozdziak P, Kempisty B, Wender-Oegowska E. Placental lactogen as a marker of maternal obesity, diabetes, and fetal growth abnormalities: current knowledge and clinical perspectives. J Clin Med. 2020;9:1142.

CAS PubMed Central Article Google Scholar

Dam P, Cherubini K, Goveia P, Pena G, Galliano L, Faanha C, et al. Depressive symptoms in women with gestational diabetes mellitus: the LINDA-Brazil Study. J Diabetes Res. 2017;2017:7341893.

PubMed PubMed Central Article Google Scholar

Molyneaux E, Poston L, Ashurst-Williams S, Howard LM. Obesity and mental disorders during pregnancy and postpartum: a systematic review and meta-analysis. Obstet Gynecol. 2014;123:85767.

PubMed PubMed Central Article Google Scholar

Rogan SC, Payne JL, Meltzer-Brody S. Relationship between depressive mood and maternal obesity: implications for postpartum depression. In: Nicholson W, Baptiste-Roberts K, editors. Obesity during pregnancy in clinical practice. London: Springer; 2014. p. 99120.

Google Scholar

Azami M, Badfar G, Soleymani A, Rahmati S. The association between gestational diabetes and postpartum depression: a systematic review and meta-analysis. Diabetes Res Clin Pract. 2019;149:14755.

PubMed Article PubMed Central Google Scholar

Roos LL, Brownell M, Lix L, Roos NP, Walld R, MacWilliam L. From health research to social research: privacy, methods, approaches. Soc Sci Med. 2008;66:11729.

PubMed Article PubMed Central Google Scholar

Jutte DP, Roos LL, Brownell MD. Administrative record linkage as a tool for public health research. Annu Rev Public Health. 2011;32:91108.

PubMed Article PubMed Central Google Scholar

Fryar CD, Kruszon-Moran D, Gu Q, Ogden C. Mean Body Weight, Height, Waist Circumference, and Body Mass Index Among Adults: United States, 19992000 Through 20152016. 2018. https://www.cdc.gov/nchs/data/nhsr/nhsr122-508.pdf. Accessed 27 Nov 2019.

Centers for Disease Control and Prevention. 2017 Data Release. National Health Interview Survey. 2017. https://www.cdc.gov/nchs/nhis/nhis_2017_data_release.htm. Accessed 27 Nov 2019.

Ghaedrahmati M, Kazemi A, Kheirabadi G, Ebrahimi A, Bahrami M. Postpartum depression risk factors: a narrative review. J Educ Health Promot. 2017;6:60.

PubMed PubMed Central Google Scholar

Gelaye B, Rondon MB, Araya R, Williams MA. Epidemiology of maternal depression, risk factors, and child outcomes in low-income and middle-income countries. Lancet Psychiatry. 2016;3:97382.

PubMed PubMed Central Article Google Scholar

Silverman ME, Reichenberg A, Savitz DA, Cnattingius S, Lichtenstein P, Hultman CM, et al. The risk factors for postpartum depression:a population-based study. Depress Anxiety. 2017;34:17887.

PubMed PubMed Central Article Google Scholar

Fransoo R, Mahar A, The Need to Know Team, Anderson A, Prior H, Koseva I, et al. The 2019 RHA Indicators Atlas. Winnipeg, Manitoba; 2019. http://mchp-appserv.cpe.umanitoba.ca/reference//RHA_Report_web.pdf. Accessed 27 Nov 2019.

Shingo T, Gregg C, Enwere E, Fujikawa H, Hassam R, Geary C, et al. Pregnancy-stimulated neurogenesis in the adult female forebrain mediated by prolactin. Science (80-). 2003;299:11720.

CAS Article Google Scholar

Lucas BK, Ormandy CJ, Binart N, Bridges RS, Kelly PA. Null mutation of the prolactin receptor gene produces a defect in maternal behavior. Endocrinology. 1998;139:41027.

CAS PubMed Article PubMed Central Google Scholar

Pawluski JL, Lonstein JS, Fleming AS. The neurobiology of postpartum anxiety and depression. Trends Neurosci. 2017;40:10620.

CAS PubMed Article PubMed Central Google Scholar

Newbern D, Freemark M. Placental hormones and the control of maternal metabolism and fetal growth. Curr Opin Endocrinol Diabetes Obes. 2011;18:40916.

CAS PubMed Article PubMed Central Google Scholar

Nicklas JM, Miller LJ, Zera CA, Davis RB, Levkoff SE, Seely EW. Factors associated with depressive symptoms in the early postpartum period among women with recent gestational diabetes mellitus. Matern Child Health J. 2013;17:166572.

PubMed Article PubMed Central Google Scholar

Putnick DL, Sundaram R, Bell EM, Ghassabian A, Goldstein RB, Robinson SL, et al. Trajectories of maternal postpartum depressive symptoms. Pediatrics. 2020;146:e20200857.

PubMed Article PubMed Central Google Scholar

Hinkle SN, Buck Louis GM, Rawal S, Zhu Y, Albert PS, Zhang C. A longitudinal study of depression and gestational diabetes in pregnancy and the postpartum period. Diabetologia. 2016;59:2594602.

CAS PubMed PubMed Central Article Google Scholar

Nyberg ST, Batty GD, Pentti J, Virtanen M, Alfredsson L, Fransson EI, et al. Obesity and loss of disease-free years owing to major non-communicable diseases: a multicohort study. Lancet Public Heal. 2018;3:4907.

Article Google Scholar

Edqvist J, Rawshani A, Adiels M, Bjrck L, Lind M, Svensson A-M, et al. BMI and mortality in patients with New-Onset Type 2 diabetes: a comparison with age- and sex-matched control subjects from the general population. Diabetes Care. 2018;41:48593.

PubMed Article PubMed Central Google Scholar

Kim SS, Zhu Y, Grantz KL, Hinkle SN, Chen Z, Wallace ME, et al. Obstetric and neonatal risks among obese women without chronic disease. Obstet Gynecol. 2016;128:10412.

PubMed PubMed Central Article Google Scholar

Scott-Pillai R, Spence D, Cardwell CR, Hunter A, Holmes VA. The impact of body mass index on maternal and neonatal outcomes: a retrospective study in a UK obstetric population, 20042011. BJOG. 2013;120:9329.

CAS PubMed Article PubMed Central Google Scholar

Shields M, Connor Gorber S, Tremblay MS. Estimates of obesity based on self-report versus direct measures. Heal Rep. 2008;19:6176.

Google Scholar

Gutin I. In BMI we trust: reframing the body mass index as a measure of health. Soc Theory Health. 2018;16:25671.

PubMed Article PubMed Central Google Scholar

Ortega FB, Sui X, Lavie CJ, Blair SN. Body mass index, the most widely used but also widely criticized index: would a criterion standard measure of total body fat be a better predictor of cardiovascular disease mortality? Mayo Clin Proc. 2016;91:44355.

PubMed PubMed Central Article Google Scholar

Bliddal M, Pottegrd A, Kirkegaard H, Olsen J, Srensen TIA, Nohr EA. Depressive symptoms in womens midlife in relation to their body weight before, during and after childbearing years. Obes Sci Pract. 2016;2:41525.

CAS PubMed PubMed Central Article Google Scholar

See the article here:
A potential role for insulin treatment during pregnancy in reducing postpartum psychological distress in maternal obesity: an administrative...

KENILWORTH, N.J. & WOODCLIFF LAKE, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Eisai today announced the first presentation of investigational data from the pivotal Phase 3 KEYNOTE-775/Study 309 trial in an oral plenary session (Plenary Session #10191) at the virtual Society of Gynecologic Oncology (SGO) 2021 Annual Meeting on Womens Cancer. The trial evaluated the combination of KEYTRUDA, Mercks anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, for the treatment of certain patients with advanced, metastatic or recurrent endometrial cancer following one prior platinum-based regimen in any setting.

The study met the dual primary endpoints of progression-free survival (PFS), as assessed by blinded independent central review (BICR) per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, and overall survival (OS) as well as the secondary efficacy endpoint of objective response rate (ORR), as assessed by BICR per RECIST v1.1, in the all-comer population (mismatch repair proficient [pMMR] and mismatch repair deficient [dMMR]) and in the pMMR subgroup. Median follow-up was 11.4 months for both the all-comer population and the pMMR subgroup. A statistically significant and clinically meaningful improvement in PFS was seen in the all-comer population, in which KEYTRUDA plus LENVIMA (n=411) reduced the risk of disease progression or death by 44% (HR=0.56 [95% CI: 0.47-0.66]; p<0.0001), with a median PFS of 7.2 months (95% CI: 5.7-7.6; number of events=281) versus 3.8 months (95% CI: 3.6-4.2; number of events=286) for patients who received chemotherapy (treatment of physicians choice [TPC] of doxorubicin or paclitaxel; n=416). Additionally, a statistically significant and clinically meaningful improvement in OS was seen in the all-comer population, in which KEYTRUDA plus LENVIMA reduced the risk of death by 38% (HR=0.62 [95% CI: 0.51-0.75]; p<0.0001), with a median OS of 18.3 months (95% CI: 15.2-20.5; number of events=188) versus 11.4 months (95% CI: 10.5-12.9; number of events=245) for patients who received TPC. The safety profile of KEYTRUDA plus LENVIMA was generally consistent with the established safety profiles of the individual monotherapies.

Patients diagnosed with endometrial cancer, the most common type of gynecologic cancer in the U.S., face low survival rates when diagnosed at an advanced stage or at recurrence, especially once the disease progresses after prior platinum-based therapy and is not amenable to curative surgery or radiation, said Dr. Vicky Makker, Principal Investigator and Medical Oncologist, Memorial Sloan Kettering Cancer Center. With a 38% reduction in risk of death regardless of mismatch repair status, KEYTRUDA plus LENVIMA significantly improved overall survival compared with chemotherapy in the all-comer group of patients with advanced, metastatic or recurrent endometrial carcinoma, which is very encouraging, as this arm included an investigational patient population for which more data have been sought after by the gynecologic oncology community.

In the all-comer population, the secondary efficacy endpoint of ORR was 31.9% (95% CI: 27.4-36.6), with a complete response (CR) rate of 6.6% and a partial response (PR) rate of 25.3%, for patients who received KEYTRUDA plus LENVIMA versus 14.7% (95% CI: 11.4-18.4), with a CR rate of 2.6% and a PR rate of 12.0% for patients who received TPC (ORR difference versus TPC: 17.2 percentage points; p<0.0001). For patients who responded, the median duration of response (DOR) was 14.4 months (range: 1.6-23.7) for patients who received KEYTRUDA plus LENVIMA versus 5.7 months (range: 0.0-24.2) for patients who received TPC.

In this confirmatory Phase 3 study, KEYTRUDA plus LENVIMA demonstrated statistically significant improvements in progression-free survival, overall survival and objective response rate versus chemotherapy, said Dr. Gregory Lubiniecki, Vice President, Oncology Clinical Research, Merck Research Laboratories. We are encouraged by these results that reaffirm Mercks and Eisais commitment to explore the potential of the combination to help more patients with difficult-to-treat types of cancer.

The positive results seen in KEYNOTE-775/Study 309 help confirm the currently approved use of the KEYTRUDA plus LENVIMA combination in certain patients with advanced endometrial carcinoma, said Dr. Takashi Owa, Chief Medicine Creation Officer and Chief Discovery Officer, Oncology Business Group at Eisai. As this stage of disease has been notoriously difficult to treat, Eisai and Merck remain committed to addressing the unmet need of advanced endometrial carcinoma. We are grateful to the patients and healthcare providers whose participation and persistence amid a global pandemic have made this milestone possible.

Results were similar across the all-comer population and the pMMR subgroup. In the pMMR subgroup, KEYTRUDA plus LENVIMA reduced the risk of disease progression or death by 40% (HR=0.60 [95% CI: 0.50-0.72]; p<0.0001), with a median PFS of 6.6 months (95% CI: 5.6-7.4; number of events=247) versus 3.8 months (95% CI: 3.6-5.0; number of events=238) for patients who received TPC. KEYTRUDA plus LENVIMA reduced the risk of death by 32% (HR=0.68 [95% CI: 0.56-0.84]; p=0.0001), with a median OS of 17.4 months (95% CI: 14.2-19.9; number of events=165) versus 12.0 months (95% CI: 10.8-13.3; number of events=203) for patients who received TPC. The secondary endpoint of ORR was 30.3% (95% CI: 25.5-35.5), with a CR rate of 5.2% and a PR rate of 25.1%, for patients who received KEYTRUDA plus LENVIMA versus 15.1% (95% CI: 11.5-19.3), with a CR rate of 2.6% and a PR rate of 12.5%, for patients who received TPC (ORR difference versus TPC: 15.2 percentage points; p<0.0001). For patients who responded, the median DOR was 9.2 months (range: 1.6-23.7) for patients who received KEYTRUDA plus LENVIMA versus 5.7 months (range: 0.0-24.2) for patients who received TPC.

In the all-comer population, in the KEYTRUDA plus LENVIMA arm (n=406), treatment-emergent adverse events (TEAEs) of any grade led to discontinuation of KEYTRUDA in 18.7% of patients, of LENVIMA in 30.8% of patients, and of both in 14.0% of patients. In the TPC arm (n=388), TEAEs of any grade led to discontinuation of chemotherapy in 8.0% of patients. Grade 5 TEAEs of any cause occurred in 5.7% of patients in the KEYTRUDA plus LENVIMA arm and in 4.9% of patients in the TPC arm. Grade 3 TEAEs occurred in 88.9% of patients in the KEYTRUDA plus LENVIMA arm and in 72.7% of patients in the TPC arm. In the KEYTRUDA plus LENVIMA arm, the most common TEAEs of any grade occurring in at least 25% of patients were hypertension (64.0%), hypothyroidism (57.4%), diarrhea (54.2%), nausea (49.5%), decreased appetite (44.8%), vomiting (36.7%), weight decrease (34.0%), fatigue (33.0%), arthralgia (30.5%), proteinuria (28.8%), anemia (26.1%), constipation (25.9%) and urinary tract infection (25.6%). In the TPC arm, the most common TEAEs of any grade occurring in at least 25% of patients were anemia (48.7%), nausea (46.1%), neutropenia (33.8%), alopecia (30.9%), and fatigue (27.6%). Median treatment duration was 231 days (range: 1-817) with KEYTRUDA plus LENVIMA and 104.5 days (range: 1-785) with TPC.

KEYNOTE-775/Study 309 is the confirmatory trial for KEYNOTE-146/Study 111, which supported the U.S. Food and Drug Administrations (FDA) 2019 accelerated approval of the KEYTRUDA plus LENVIMA combination for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation.

KEYNOTE-775/Study 309 Trial Design (Plenary Session #10191)

KEYNOTE-775/Study 309 is a multicenter, randomized, open-label, Phase 3 trial (ClinicalTrials.gov, NCT03517449) evaluating KEYTRUDA in combination with LENVIMA in patients with advanced endometrial cancer following one prior platinum-based regimen in any setting. The dual primary endpoints are PFS, as assessed by BICR per RECIST v1.1, and OS. Select secondary endpoints include ORR, as assessed by BICR per RECIST v1.1, and safety/tolerability. Of the 827 patients enrolled, 697 patients had tumors that were pMMR, and 130 patients had tumors that were dMMR. Patients were randomized 1:1 to receive:

About Endometrial Cancer

Endometrial cancer begins in the inner lining of the uterus, which is known as the endometrium and is the most common type of cancer in the uterus. In 2020, it was estimated there were more than 417,000 new cases and more than 97,000 deaths from uterine body cancers worldwide (these estimates include both endometrial cancers and uterine sarcomas; more than 90% of uterine body cancers occur in the endometrium, so the actual numbers for endometrial cancer cases and deaths are slightly lower than these estimates). In the U.S., it is estimated there will be more than 66,000 new cases of uterine body cancer and nearly 13,000 deaths from the disease in 2021. The five-year survival rate for metastatic endometrial cancer (stage IV) is estimated to be approximately 17%.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,400 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications in the U.S.

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS 10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Endometrial Carcinoma

KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Triple-Negative Breast Cancer

KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Selected Important Safety Information for KEYTRUDA

Severe and Fatal Immune-Mediated Adverse Reactions

KEYTRUDA is a monoclonal antibody that belongs to a class of drugs that bind to either the programmed death receptor-1 (PD-1) or the programmed death ligand 1 (PD-L1), blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions.

Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Early identification and management are essential to ensure safe use of antiPD-1/PD-L1 treatments. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction. In general, if KEYTRUDA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis. The incidence is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.4% (94/2799) of patients receiving KEYTRUDA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (0.9%), and Grade 2 (1.3%) reactions. Systemic corticosteroids were required in 67% (63/94) of patients. Pneumonitis led to permanent discontinuation of KEYTRUDA in 1.3% (36) and withholding in 0.9% (26) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Pneumonitis resolved in 59% of the 94 patients.

Pneumonitis occurred in 8% (31/389) of adult patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients. Patients received high-dose corticosteroids for a median duration of 10 days (range: 2 days to 53 months). Pneumonitis rates were similar in patients with and without prior thoracic radiation. Pneumonitis led to discontinuation of KEYTRUDA in 5.4% (21) of patients. Of the patients who developed pneumonitis, 42% of these patients interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis, which may present with diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (1.1%), and Grade 2 (0.4%) reactions. Systemic corticosteroids were required in 69% (33/48); additional immunosuppressant therapy was required in 4.2% of patients. Colitis led to permanent discontinuation of KEYTRUDA in 0.5% (15) and withholding in 0.5% (13) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Colitis resolved in 85% of the 48 patients.

Hepatotoxicity and Immune-Mediated Hepatitis

KEYTRUDA as a Single Agent

KEYTRUDA can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.4%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 68% (13/19) of patients; additional immunosuppressant therapy was required in 11% of patients. Hepatitis led to permanent discontinuation of KEYTRUDA in 0.2% (6) and withholding in 0.3% (9) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Hepatitis resolved in 79% of the 19 patients.

KEYTRUDA with Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider monitoring more frequently as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased alanine aminotransferase (ALT) (20%) and increased aspartate aminotransferase (AST) (13%) were seen, which was at a higher frequency compared to KEYTRUDA alone. Fifty-nine percent of the patients with increased ALT received systemic corticosteroids. In patients with ALT 3 times upper limit of normal (ULN) (Grades 2-4, n=116), ALT resolved to Grades 0-1 in 94%. Among the 92 patients who were rechallenged with either KEYTRUDA (n=3) or axitinib (n=34) administered as a single agent or with both (n=55), recurrence of ALT 3 times ULN was observed in 1 patient receiving KEYTRUDA, 16 patients receiving axitinib, and 24 patients receiving both. All patients with a recurrence of ALT 3 ULN subsequently recovered from the event.

Immune-Mediated Endocrinopathies

Adrenal Insufficiency

KEYTRUDA can cause primary or secondary adrenal insufficiency. For Grade 2 or higher, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold KEYTRUDA depending on severity. Adrenal insufficiency occurred in 0.8% (22/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.3%) reactions. Systemic corticosteroids were required in 77% (17/22) of patients; of these, the majority remained on systemic corticosteroids. Adrenal insufficiency led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.3% (8) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Hypophysitis

KEYTRUDA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Hypophysitis occurred in 0.6% (17/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.2%) reactions. Systemic corticosteroids were required in 94% (16/17) of patients; of these, the majority remained on systemic corticosteroids. Hypophysitis led to permanent discontinuation of KEYTRUDA in 0.1% (4) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Thyroid Disorders

KEYTRUDA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Thyroiditis occurred in 0.6% (16/2799) of patients receiving KEYTRUDA, including Grade 2 (0.3%). None discontinued, but KEYTRUDA was withheld in <0.1% (1) of patients.

Hyperthyroidism occurred in 3.4% (96/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (0.8%). It led to permanent discontinuation of KEYTRUDA in <0.1% (2) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. Hypothyroidism occurred in 8% (237/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (6.2%). It led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.5% (14) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. The majority of patients with hypothyroidism required long-term thyroid hormone replacement. The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC, occurring in 16% of patients receiving KEYTRUDA as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in 389 adult patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.2%) and Grade 2 (10.8%) hypothyroidism.

Type 1 Diabetes Mellitus (DM), Which Can Present With Diabetic Ketoacidosis

Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold KEYTRUDA depending on severity. Type 1 DM occurred in 0.2% (6/2799) of patients receiving KEYTRUDA. It led to permanent discontinuation in <0.1% (1) and withholding of KEYTRUDA in <0.1% (1). All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Immune-Mediated Nephritis With Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Immune-mediated nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.1%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 89% (8/9) of patients. Nephritis led to permanent discontinuation of KEYTRUDA in 0.1% (3) and withholding in 0.1% (3) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Nephritis resolved in 56% of the 9 patients.

Immune-Mediated Dermatologic Adverse Reactions

KEYTRUDA can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome, drug rash with eosinophilia and systemic symptoms, and toxic epidermal necrolysis, has occurred with antiPD-1/PD-L1 treatments. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate nonexfoliative rashes. Withhold or permanently discontinue KEYTRUDA depending on severity. Immune-mediated dermatologic adverse reactions occurred in 1.4% (38/2799) of patients receiving KEYTRUDA, including Grade 3 (1%) and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 40% (15/38) of patients. These reactions led to permanent discontinuation in 0.1% (2) and withholding of KEYTRUDA in 0.6% (16) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 6% had recurrence. The reactions resolved in 79% of the 38 patients.

Other Immune-Mediated Adverse Reactions

The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received KEYTRUDA or were reported with the use of other antiPD-1/PD-L1 treatments. Severe or fatal cases have been reported for some of these adverse reactions. Cardiac/Vascular: Myocarditis, pericarditis, vasculitis; Nervous System: Meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barr syndrome, nerve paresis, autoimmune neuropathy; Ocular: Uveitis, iritis and other ocular inflammatory toxicities can occur. Some cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada-like syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss; Gastrointestinal: Pancreatitis, to include increases in serum amylase and lipase levels, gastritis, duodenitis; Musculoskeletal and Connective Tissue: Myositis/polymyositis, rhabdomyolysis (and associated sequelae, including renal failure), arthritis (1.5%), polymyalgia rheumatica; Endocrine: Hypoparathyroidism; Hematologic/Immune: Hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% of 2799 patients receiving KEYTRUDA. Monitor for signs and symptoms of infusion-related reactions. Interrupt or slow the rate of infusion for Grade 1 or Grade 2 reactions. For Grade 3 or Grade 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Fatal and other serious complications can occur in patients who receive allogeneic HSCT before or after antiPD-1/PD-L1 treatment. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute and chronic GVHD, hepatic veno-occlusive disease after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between antiPD-1/PD-L1 treatment and allogeneic HSCT. Follow patients closely for evidence of these complications and intervene promptly. Consider the benefit vs risks of using antiPD-1/PD-L1 treatments prior to or after an allogeneic HSCT.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with an antiPD-1/PD-L1 treatment in this combination is not recommended outside of controlled trials.

See original here:
KEYTRUDA (pembrolizumab) Plus LENVIMA (lenvatinib) Significantly Improved Progression-Free Survival and Overall Survival Versus Chemotherapy in...

While some people whove had abortions in West Virginia regret them, and others say those decisions were best for them, they agree on the importance of accurate information.

But a bill working its way through the West Virginia Legislature would require health care professionals to provide them with one piece of information that isnt supported by research and could amount to, in the words of one state medical group, unmonitored experimentation on West Virginians seeking abortions.

Under House Bill 2982, those people would hear that it may be possible to stop a medical abortion after taking the first of two pills required for such an abortion. The bill doesnt require they be told that the assertion isnt supported by research, that its based on taking a medication that isnt approved by the U.S. Food and Drug Administration for the purpose of stopping abortions, or about the potential harm of attempting to stop an abortion.

Supporters of the bill, including some people whove had abortions, say the bill is needed because some women have regrets after beginning the procedure.

Opponents of the bill, also including people whove had abortions, say that its important for people seeking abortions to understand theyre making permanent decisions.

The bill, if it becomes law, would tell people they dont have to make a decision, that they can be wishy-washy about it, said Hunter Starks, who had an abortion while in an abusive relationship.

And you cant, Starks said. You have to make that decision, whether thats easy or hard for you. You have to make that decision.

Starks, a transgender non-binary person, said if they hadnt had that abortion, they would have been tied to that abuser for life.

Opponents of the bill also warn that its based on the unproven idea that taking progesterone after the first pill in a medication abortion can reverse the process. They note the FDA has not approved progesterone for this use, and that limited research suggests patients health and safety could be put at risk.

What does the bill do?

When a patient seeks a medical abortion, health care providers provide them with two pills. Mifepristone, the first pill, blocks the hormone progesterone. This thins the uterine lining, so the embryo wont stay implanted and continue growing. Misoprostol, the second pill, causes the uterus to contract and expel the embryo through the vagina.

The original version of House Bill 2982, sponsored by Delegate Kayla Kessinger, R-Fayette, said that providers had to tell patients that their abortion could be reversed. Lawmakers in the House of Delegates Health and Human Resources Committee changed that wording last week, so the bill now would require health care providers to tell patients that it may be possible to stop an abortion.

Delegate Todd Longanacre, R-Greenbrier and a bill co-sponsor, said the bill would help a young lady [who] realizes the error of her ways, by taking that first pill to terminate a babys life.

Its an opportunity for that young lady who may have, in a knee-jerk, emotional state-of-mind decided to take an RU-486 pill, and then woke up the next morning and thought, Oh, my gosh, what have I done? he said. This is her opportunity to save that babys life.

Its not a perfect bill, Longanacre said. But you know what, an old general from the World War II era once said and that guys name was General Patton he said a less-than-perfect plan violently executed now is better than the perfect plan executed next week. People are dying; babies are dying. This is an opportunity to save lives.

House Bill 2982 is based on the unproven idea that taking progesterone after the first pill will reverse its effects.

Research on progesterone for this use is so weak, according to a statement from the West Virginia American College of Obstetricians and Gynecologists chapter, that administering it to patients in an effort to reverse or stop abortions potentially subjects women to unmonitored experimentation, which is in direct violation of a physicians oath to care.

The bill is supported by West Virginians for Life and the National Right to Life Committee. It is opposed by both the national and West Virginia chapters of the American College of Obstetricians and Gynecologists, Planned Parenthood and the state-level reproductive rights group, WV Free.

Democrats in the House committee last week noted that a study on the effectiveness of progesterone in stopping an abortion mid-procedure was halted early because several women experienced severe bleeding, possibly because they had taken the first pill but not the second.

Researchers aimed to determine whether progesterone could be used to reverse the effects of mifepristone, preventing pregnancy termination, but three patients experienced severe hemorrhaging and had to be taken to the hospital, according to the study, published in a 2019 edition of Obstetrics & Gynecology, the official publication of the American College of Obstetricians and Gynecologists.

Delegate Barbara Fleischauer, D-Monongalia, wanted to amend the bill in committee to warn patients about possible side effects, but that amendment was rejected.

Delegate Ric Griffith, D-Wayne and a pharmacist who said he is anti-abortion, was concerned enough to oppose the bill, saying he worried taking the first pill, but not the second, could lead to birth defects.

In our desire to protect the unborn, we are potentially causing harm to the unborn, he said.

No medical professionals were called to testify during the meeting.

Karen Cross, who is from West Virginia and lobbies for the National Right to Life Committee, was present but did not testify.

Ive had two abortions, and I regret mine, Cross said in an interview following the meeting. I know so many women, who are involved in the pro-life movement even, actively, because of the decision they made to abort their children, so that they can help other women not make that mistake.

Delegate Heather Tully, R-Nicholas and an anti-abortion family nurse practitioner, voted for the bill during that meeting, but said in an interview that she did have concerns.

I would be very concerned about a non-approved FDA medication, but obviously, this procedure has been done in some places, maybe not necessarily within the state of West Virginia, or within the confines of the border, she said.

Tully, who noted that she doesnt work in reproductive medicine, said abortion pills also come with risk. Shes had patients change their minds about other procedures, she said. And patients have a right to withdraw consent, she noted.

I think that this bill, it really quite honestly strengthens the informed consent process for a patient that may change their mind, she said.

Ten states have passed similar legislation, and others are working on their own bills, according to Cross. Courts have blocked implementation in several states. Six states have the law in place, according to a March 1 statement from the pro-abortion rights Guttmacher Institute.

The House of Delegates Judiciary Committee, where delegates would normally discuss the legality of bills, passed the bill with no discussion of those court cases Thursday.

The bill is up for second reading in the House on Tuesday, during which time delegates may offer amendments. Its tentatively scheduled for a final House vote on Wednesday, and would then have to pass the state Senate and get the approval of Gov. Jim Justice to become law.

Lived experiences

While some West Virginians whove had abortions made the right decision for them and others have regrets, several people interviewed agreed on the important of being informed.

But while the bill does require doctors to tell patients about an option, it doesnt tell them anything about the lack of research supporting that option and the potential harmful effects of it.

Suzi Bragg, of Morgantown, still supports the bill. She regrets her two abortions, and would rather anti-abortion lawmakers become involved than rely on doctors.

Im 63 and to this day, I do not trust when doctors tell me things, she said.

Bragg said when she was 16 and in foster care, a young man blackmailed her into sex. She said her social workers took her to Pittsburgh for the abortion when she was about 18 weeks pregnant.

She said they injected saline into her abdomen, which was extremely painful, and held her down when she yelled for them to stop. She said the experience was extremely traumatic, because I wasnt told the truth about what would occur.

She said she was told the experience would be no more painful than menstrual cramps, and the fetus was a clump of cells. She didnt realize until seeing a Newsweek magazine later on that a fetus is more developed than that at about 18 weeks.

I remember just screaming, They lied to me, she said.

Bragg said she went on to develop post-traumatic stress disorder. She didnt want to eat. She didnt want to socialize with friends. She said the trauma was due in part to being coerced into both the act of sex, and the abortion. During a second abortion in her 20s, she declined pain medication. She felt like she deserved the pain.

Back then, information about abortion was less readily available to women, she said.

Hunter Starks, of Charleston, was 19 and in an abusive relationship when they sought their first of two abortions in 2012.

Starks, who was assigned female at birth, took pain pills for the first abortion, which was medical, so pain wasnt significant, they said, and they experienced no pain for the second, which was surgical.

Starks said they were in a fragile mindset at the time and it was mainly their partners decision, but they still dont regret the choice.

While Longanacre, the bill co-sponsor, said some abortions may be knee-jerk reactions, ACOG-WV and some West Virginians whove had abortions say the bill itself would actually encourage people to see abortion as something they dont have to be sure about.

In their statement, leaders of the state OB/GYN group wrote that if the bill becomes law, it would create an environment of confusion and stigma.

Starks already felt confused and scared going into the abortion, without doctors telling them that the decision could be stopped or reversed.

Everyone should have agency over their own decisions with their body, and doctors shouldnt be making that any more confusing or difficult, they said.

Starks does regret one thing going to the clinic alone. Protestors screamed and name-called.

Maggie McCabe, whose mother worked to start the last remaining abortion clinic in West Virginia, the Womens Health Center in Charleston, was 16 when her mother flew her to Washington, D.C., for an abortion in the days before the U.S. Supreme Court legalized abortion in its Roe v. Wade decision in 1973.

A professor at BridgeValley Community and Technical College who worked for a health insurance provider for 28 years, McCabe was just getting ready to start her life.

She said she wouldnt have wanted wishy-washy information when seeking an abortion, and said patients deserve concrete, appropriate information.

My abortion had no negative effect on me, she said. Its made me a stronger woman, because I believe in helping others in any way that I can. Telling my story is my contribution to helping other women in their decisions.

McCabe, of Charleston, criticized lawmakers for involving themselves in health care decisions and called abortion bills a form of sex discrimination.

She noted people with fewer resources and less support couldnt have flown to D.C., like she did.

The right-to-lifers are using a very offensive, very un-Christian campaign to keep women oppressed, and to keep women in a lower class controlled, she said.

Jamie Miller, an abortion rights advocate who lives in South Charleston, was almost 17 when she received an abortion.

She had already gotten approval from a mental health professional for the abortion to avoid telling her family, and saved up money.

Miller said if after all that, she arrived at her appointment and providers told her the process could be stopped or reversed, she would have wondered whether she could trust them and if she needed to attempt a possibly deadly abortion on her own.

I would have felt like they werent on my side, she said.

We are making critical coverage of the coronavirus available for free. Please consider subscribing so we can continue to bring you the latest news and information on this developing story.

Here is the original post:
West Virginians considering abortion need good information about the procedure; bill might keep them from getting the facts - Beckley Register-Herald

Although many people think otherwise, bacterial vaginosis (BV) isnt a sexually transmitted infection (STI).

Any person who has a vagina can develop it, and there are a number of factors that may lead to such an infection.

Yes, exposure to a new sexual partner is one of them. But the list also includes things like smoking and douching.

So theres no way that anyone can definitively say BV is linked to cheating.

No, BV isnt considered to be an STI even though some people have reported being told this by a clinician.

The confusion likely comes from the fact that BV can be associated with sexual activity.

For example, penetrative sexual activity can affect the natural bacterial balance in your vagina, leading to extra bacterial growth and eventually BV.

But theres little evidence that the infection can be passed between people through sexual contact, so it isnt on the STI list.

However, BV can increase your chances of contracting an STI, as the bacterial changes may lower the vaginas natural defenses.

The exact cause of BV is unknown, but its characterized by an unbalanced bacterial balance in the vagina.

However, experts have found a number of factors that may increase your risk of developing it.

This includes anything that affects the vaginas pH levels, such as douching or using irritating vaginal products.

Youre also more likely to develop BV if:

Unfortunately, there isnt an easy answer to this. Theres still much more for researchers to learn about the infection.

From using an IUD as contraception to taking up smoking or even changing the way you clean your genital area, all of these factors can lead to BV.

Because of this, theres a chance that you may not know why or even when youve suddenly developed it.

BV can go away on its own after a few days.

But if you need medical treatment, youll likely have to take a weeklong course of antibiotics. If the infections persistent, your provider may prescribe a second round.

Half of people with BV dont have any symptoms, so you may have little to deal with.

But strong-smelling vaginal discharge and irritation when urinating are typical symptoms of the infection.

Although you should seek medical advice from a doctor or other healthcare professional, you can try the following at home to lessen symptoms:

If your partner has a penis, its unlikely that theyll need treatment.

But the infection can be passed between people who have vaginas.

So if your partner has a vagina, its worth seeking medical advice for the both of you.

As doctors arent sure how bacterial vaginosis occurs or spreads, its hard to say how to prevent a recurring infection.

But there are a few simple steps you can take to help reduce your risk of developing a second bout of BV. (Most of these steps are similar to the ones you may have taken to relieve symptoms at home.)

First, its advisable to avoid putting anything that may cause irritation in or around your vagina.

This includes douches, deodorants, and perfumed cleansing products.

Instead, use water and plain soap to clean the area, sticking to showers rather than baths where possible.

When it comes to your underwear, stick to breathable, moisture-wicking fabrics, such as cotton, to avoid unwanted bacterial growth.

And wash underwear using a mild detergent, rather than a strong formula.

Finally, when having intercourse or any kind of sexual activity, ensure sex toys are clean before contact and use condoms or dental dams.

Unfortunately, recurrence is quite common, but it wont hurt to follow the above tips.

If youre worried about STIs, its better to book a test to put your mind at rest.

Symptoms to look out for include:

Thinking that your partner has been unfaithful is a little more complex.

Its natural to want to confront them, but try to take some time to think things through.

After all, your worries could be nothing more than a misunderstanding.

If you do want to speak with your partner, its often a good idea to write down the kinds of things you want to say beforehand.

You may also want to think about whether youd like to try and move forward if it turns out they have been unfaithful, or whether the relationship will have to end.

Speaking with a neutral person who has little connection to you or your partner can also help you get things straight.

When youre ready to talk, let your partner know that youd like to discuss something thats concerning you.

Try to set the conversation up in an environment that suits the both of you, whether thats in private or in public.

Start off by talking about how much the relationship means to you, as well as honesty and trust.

You can then say that you feel there might be a problem in the relationship, bringing up specific examples if needed.

Try not to be accusatory and listen to what your partner has to say. But if something doesnt feel right, dont be afraid to press them on it.

If the shoes on the other foot and your partner thinks that youre the guilty party, try to stay calm.

We tend to get defensive when were being confronted with something thats not true.

But try to put yourself in their shoes and realize that theyre likely only acting this way because they care about the relationship.

Let them talk through the issue and then attempt to understand why they think the way they do.

For example, have you been paying them less attention than usual?

Or is there something going on in their life that could be affecting their emotional state?

I hear you is a good way to start off your end of the conversation. It lets them know that youre listening and understanding where theyre coming from.

At the same time, dont be afraid to let them know if theyve upset you with such an accusation. Remember, its important for both of you to be open and honest.

Asking if youre able to move past the issue is often a good way to end things.

Itll leave you both with an understanding of where youre currently at and clear steps to take to improve the relationship if needed.

If their concern is about contracting an STI, explain that BV isnt an STI. And if theyd still like an STI test, be supportive.

Offer to go with them and get one too if youre comfortable doing so.

Most doctors recommend booking an appointment if you think you have BV, even though it can go away on its own.

This is because, if left untreated, BV can lead to pregnancy complications, pelvic inflammatory disease, or an increased risk of STIs.

So any unusual discharge, itching, burning, swelling, or soreness around the genital area warrants a call.

A healthcare professional can test vaginal discharge and fluid for the infection and prescribe the right treatment, if necessary.

Treatment usually involves a course of antibiotics, either in a pill, capsule, or cream form.

Although much more research is needed into BV, the infection is most definitely not a clear-cut sign of cheating.

So if you or a partner do experience it, try not to blame yourself or others. The cause may have nothing to do with your sex life.

Lauren Sharkey is a U.K.-based journalist and author specializing in womens issues. When she isnt trying to discover a way to banish migraines, she can be found uncovering the answers to your lurking health questions. She has also written a book profiling young female activists across the globe and is currently building a community of such resisters. Catch her on Twitter.

Originally posted here:
BV Isn't a Sign of Cheating Here's Why - Healthline

CARLSBAD, Calif., March 18, 2021 (GLOBE NEWSWIRE) -- Qualigen Therapeutics, Inc. (NASDAQ: QLGN), a biotechnology company focused on developing novel therapeutics for the treatment of cancer and viral diseases, announced today that Michael Poirier, President, Chief Executive Officer and Chairman of Qualigen, and Amy Broidrick, EVP, Chief Strategy Officer will present on March 24, 2021 at the Benzinga Biotech Small Cap Conference being held March 24-25, 2021.

Qualigen Therapeutics, Inc. Presentation Details are as follows:

Investors and others invited to attend this conference event may request one-on-one meetings with representatives of the Company through the respective conference hosts or via email to Tony Schor, tony@investorawareness.com or David Kugelman, dk@atlcp.com.

About Qualigen Therapeutics, Inc.

Qualigen Therapeutics, Inc. is a biotechnology company focused on developing novel therapeutics for the treatment of cancer and infectious diseases, as well as maintaining and expanding its core FDA-approved FastPack System, which has been used successfully in diagnostics for 20 years. The Company's cancer therapeutics pipeline includes ALAN (AS1411-GNP), RAS-F and STARS. ALAN (AS1411-GNP) is a DNA coated gold nanoparticle cancer drug candidate that has the potential to target various types of cancer with minimal side effects. The foundational nucleolin-targeting DNA aptamer of ALAN, AS1411, is also a drug candidate for use in treating COVID-19 and other viral-based infectious diseases. RAS-F is a family of RAS oncogene protein-protein interaction inhibitor small molecules for preventing mutated RAS genes proteins from binding to their effector proteins; preventing this binding could stop tumor growth, especially in pancreatic, colorectal and lung cancers. STARS is a DNA/RNA-based treatment device candidate for removal from circulating blood of precisely targeted tumor-produced and viral compounds.

Because Qualigen's therapeutic candidates are still in the development stage, Qualigen's only products that are currently commercially available are FastPack System diagnostic instruments and test kits, used in physician offices, clinics and small hospitals around the world. The FastPack System menu includes rapid point-of-care diagnostic tests for cancer, men's health, hormone function, vitamin D status and antibodies against SARS-CoV-2. Qualigen's facility in Carlsbad, California is FDA and ISO Certified and its FastPack product line is sold worldwide by its commercial partner Sekisui Diagnostics, LLC.

For more information on Qualigen Therapeutics, Inc., please visit https://www.qualigeninc.com/.

Forward-Looking Statements

This news release contains forward-looking statements by the Company that involve risks and uncertainties and reflect the Company's judgment as of the date of this release. Actual events or results may differ from the Companys expectations. For example, there can be no assurance that any clinical trials will be approved to begin by or will proceed as contemplated by any projected timeline; that the Company will successfully develop any drugs or therapeutic devices; that preclinical or clinical development of the Company's drugs or therapeutic devices will be successful; that future clinical trial data will be favorable or that such trials will confirm any improvements over other products or lack negative impacts; that any drugs or therapeutic devices will receive required regulatory approvals or that they will be commercially successful; that patents will issue on the Companys owned and in-licensed patent applications; that such patents, if any, and the Companys current owned and in-licensed patents would prevent competition; that the Company will be able to procure or earn sufficient working capital to complete the development, testing and launch of the Companys prospective therapeutic products; that the Company will be able to maintain or expand market demand and/or market share for the Companys diagnostic products generally, particularly in view of COVID-19-related deferral of patients physician-office visits and FastPack reimbursement pricing challenges; that adoption and placement of FastPack PRO System instruments (which are the only FastPack instruments on which the Company's SARS-CoV-2 IgG test kits can be run) will be widespread; that the Company will be able to manufacture the FastPack PRO System instruments and SARS-CoV-2 IgG test kits successfully; that any commercialization of the FastPack PRO System instruments and SARS-CoV-2 IgG test kits will be profitable; or that the FDA will ultimately approve an Emergency Use Authorization for the Companys SARS-CoV-2 IgG test. The Companys stock price could be harmed if any of the events or trends contemplated by the forward-looking statements fails to occur or is delayed or if any actual future event otherwise differs from expectations. Additional information concerning these and other risk factors affecting the Company's business (including events beyond the Company's control, such as epidemics and resulting changes) can be found in the Company's prior filings with the Securities and Exchange Commission, available at http://www.sec.gov. The Company disclaims any intent or obligation to update these forward-looking statements beyond the date of this news release, except as required by law. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

Investor Relations:

David Kugelman, President and CEOAtlanta Capital Partners, LLC(404) 856-9157 Office(866) 692-6847 Toll Free - U.S. & Canadadk@atlcp.com

Tony Schor, PresidentInvestor Awareness, Inc.(847) 971-0922tony@investorawareness.com

See the rest here:
Qualigen Therapeutics, Inc. to present at the Benzinga Biotech Small Cap Conference - GlobeNewswire

Minoxidil (Rogaine) is a popular product for people with thinning hair. The product comes as a gel or a foam, and is meant to be applied topically to your scalp on a daily basis.

When people first start using minoxidil to restore their hair, some notice that they actually start losing more of it at least for a short period of time.

There are plenty of clinical trials and medical evidence that support the use of minoxidil for moderate hair loss caused by alopecia. But how do you know if its actually working, especially if it looks like youre losing more hair than before?

Lets cover hair loss caused by Rogaine so you can understand how common it is, what causes it, and whether you should be concerned.

Minoxidil was a drug originally developed to treat hypertension.

Researchers observed that people who had alopecia and used minoxidil for hypertension experienced hair regrowth, and the worlds most popular over-the-counter treatment for alopecia was born. People have been using minoxidil to treat hair loss since 1986.

The way that minoxidil works isnt completely clear. Whats apparent is that minoxidil decreases hair loss in some people while also increasing hair growth. It doesnt work for everyone.

Minoxidil is also classed as a vasodilator, meaning that it dilates your blood vessels so that blood flows more easily where its applied. An increase in blood circulation to your scalp could be part of why minoxidil increases hair growth.

Your hair follicles go through four phases of growth. Not every follicle is in the same phase at once. Minoxidil is believed to affect two stages of hair growth.

The anagen phase of hair growth is its growing phase. This is when the hair is being pushed out from the root. Applying minoxidil may extend the length of the anagen phase.

The telogen phase of your hair is its resting phase, when its done growing but not yet ready to fall out. In clinical trials on rats, minoxidil shortened the telogen phase of hair from 20 days to 1 to 2 days.

Minoxidils side effects are typically mild. Common side effects include mild itching and burning as well as flaky skin. Minoxidil can also cause your hair to shed, especially when you first start using it.

As minoxidil speeds up the resting phase of your hair, sometimes it falls out more quickly than it normally would.

However, minoxidil also extends the growth phase of your hair. That means that even though some hair shedding is to be expected at first, new hair growth should soon replace the hair that youve lost.

Not everyone will experience shedding as a side effect of minoxidil, while some may experience it severely. There arent statistics currently available that explore how common this particular side effect is.

You cant do much to prevent minoxidil-related shedding or even predict if youll experience it when you first start using the product.

One thing to be aware of is that the higher the concentration of minoxidil you use, the more powerful the side effect is likely to be. Using a foam with 2 percent concentration of minoxidil, for example, could cause fewer side effects than with a 5 percent concentration.

If youre seeing a lot of hair loss, you might want to switch to a less powerful dose of minoxidil. If youre concerned about hair loss and havent started using minoxidil yet, start with a lower concentration and work up to a higher one if you need it.

Results of minoxidil vary from person to person. Generally speaking, it takes about 8 weeks of consistent use to start to see results with minoxidil. After 4 months of use, you should start to see the end of hair loss and start to see hair growth.

If its been 4 months and youre still seeing hair shedding, it may not be related to minoxidil. Its also possible that minoxidil isnt the right product for you. If you see a lot of your hair falling out after 4 months of use, speak with your doctor about alternative treatments.

Minoxidil does have some other side effects in addition to hair shedding. Side effects may include:

If youre experiencing strong side effects as a result of minoxidil, call your doctor and discontinue use.

If youve been using minoxidil for several weeks and youre still seeing more hair loss than growth, see your primary care doctor or a dermatologist. They may be able to determine if hair loss is being caused by another underlying health condition.

You should always seek medical attention if you experience the following:

Some shedding is normal when you start using any topical product that contains minoxidil. If youre concerned about hair loss, this might be alarming, but its typically no cause for concern.

If shedding doesnt stop within 4 months of starting a hair regimen with minoxidil, discontinue use and speak with your doctor about other hair regrowth options.

See more here:
Minoxidil and Shedding: Why it Happens and What to Expect - Healthline

Lets talk about a painful subject. I am of the opinion and Im far from alone that the most reliable way to determine if a possible therapy has any usefulness is a randomized, double-blinded controlled clinical trial. I can be a bit more specific than that, even: lets make that a trial that is run with sufficient statistical power to have a good chance of providing a meaningful readout.

The worldwide coronavirus pandemic has featured some well-run trials that have truly advanced our knowledge of the disease and how to treat it. But it has featured far, far more garbage. That word was chosen deliberately. There have been too many observational trials, too many uncontrolled (or poorly controlled) ones, too many open-label ones, and above all, there have been way too many trials whose number of patients would be insufficient to tell us much of anything even if everything else had been run properly.

I am not revealing any hidden tricks of the trade here. Clinical trial design is a subject with a very large literature, and there are any number of people and organizations who can provide useful guidance on both its theoretical and practical aspects. Among these aspects are the calculations that should be made for how many patients a trial is likely to need to be well-powered enough for a clean read on its clinical endpoints. You can start to learn the basic outlines of the subject online. Now, thats not to say that its an easy subject to get ahold of. Youre going to have to estimate some of your key parameters as well as you can, among them what you think the effect size of your treatment might be, what the patient-to-patient variability might be like, the time course of treatment that might be needed, and more. Just picking the proper clinical endpoints is a subject all in itself (and its one that can have a huge effect on a trials design and on its chances for success). And at the other end of things, your inclusion criteria and patient enrollment process is a place for serious thought, too. Who should be evaluated (or definitely not evaluated) in your trial, and how long will it take you to round those people up? Where are you thinking about doing all this, anyway?

There are a wide variety of trial designs out there as well, and you can find yourself sorting through some that are clearly inappropriate to the problem at hand, some that would be great if you had about ten times as much money and time as you do, and several that at first glance look like they could all work out, but which have real-world differences that its crucial that you be aware of. You would be well advised to consult with experience practitioners before you start, to make sure youre on the right track.

Unfortunately, underpowered, badly-run, and badly designed trials have been with us for a long time. Here are some well-justified concerns from 2002, for starters, and various fields of clinical research undergo periodic bouts of soul-searching over the years about these issues. But the pandemic year has really made some of our problems more obvious. Not only do we have trouble with badly run trials, but mixing in with that is a bandwagon effect. Clinicians all over the world just piled onto some of the coronavirus ideas, and kept piling on for months and months and months.Think, for example, about the hydroxychloroquine situation. Now, I still get messages condemning me as an implacable, irrational foe of the One True Coronavirus Therapy. But its worth remembering that I started out as a Huh, I dont know how that would work, but lets look into it person, which I really think should be the default setting. And in that spirit, I was all for running trials and getting more hard data.

But what did we get? A search through clinicaltrials.gov for hydroxychloroquine|coronavirus gives you 113 trials. Whats more, thirty-six of those are still listed as recruiting patients. This is ridiculous, but its not amusing. There are some large, well-controlled data sets available that indicate that HCQ is very likely not a useful therapy, but as you can see, there are also dozens of other smaller ones that say Yes! No! Maybe! Sorta! Kinda! Kinda Not! Depends! Could Be! Who Knows? And that adds up not just to a lack of knowledge, it turns into an actual hindrance to knowledge as you try to sort through the data. The heap of fuzzy indeterminate results also fuels the extrascientific political and cultural arguments about the drug, since everyone can find some sort of support for whatever opinion they might have.

You have to think that there were other therapies that deserved a look in the clinic as compared to the forty-third, sixty-seventh, or ninety-eighth hydroxychloroquine study. Youll recall that for a while, HCQ ended up mixed into other clinical trials just because everyone wanted it or imagined that it was some sort of standard of care, and that did no one much good, either. Now, HCQ isnt the only offender, but its a big one, and I think it illustrates what we should try not to do next time.

How, then, should we try not to do that? (Update: some thoughts here on this problem from a distinguished team of authors with exactly the same concerns). Its not like the US (to pick a big example) has a National Clinical Trial Authority that passes judgment on these things. To be honest, the downsides of having such an agency might worry me even more. But letting everyone go into Headless Poultry Mode and pile up overlapping crap in the clinic isnt such a good way to go, either. You would hope for a little more coordination among major medical research centers, and youd also hope for some local university/research hospital review boards to be aware that greenlighting the East Porkford Covid-19 Treatment Study with 47 patients isnt really going to advance medical science very much. Especially when its covering the same ground as the trials kicking off in Mashed Potato Falls, Rancho Malario, and Kidneystone Pass. But Im being unfair to East Porkford some of these lackluster trials were conducted at larger institutions that should have known better. The way were set up, its down to the review boards and the sources of funding to police things better, and to keep their heads while all about them are losing theirs.

And its also down to the NIH and the CDC to lead the way more than they did during 2020. The RECOVERY trial in the UK has been an example of what can be accomplished in that line. The NIH has helped run some good trials, but weve had nothing that comprehensive in the US as compared to the UK effort, and I really wish we had. I fear that some day, eventually, were going to have a chance to do better, and I hope that we take it.

More:
The Problem with COVID-19 Clinical Trials | In the Pipeline - Science Magazine

Most genetic tests focus on your odds of developing certain diseases or health conditions. But some known as pharmacogenomic (or pharmacogenetic) tests can reveal how your body may respond and react to different medications. To date, researchers have identified more than 400 genetic variations known to affect the metabolism of numerous drugs, including some that help lower cholesterol or prevent blood clots (see "Pharmacogenomics of common heart drugs").

In theory, knowing how people metabolize specific drugs could help doctors choose the safest, most effective treatment for their patients. But in practice, it's not that straightforward, says Dr. Jason Vassy, assistant professor of medicine at Harvard Medical School and a primary care physician at the VA Boston Healthcare System.

Subscribe to Harvard Health Online for immediate access to health news and information from Harvard Medical School.

New subscriptions to Harvard Health Online are temporarily unavailable. Click the button below to learn about our other subscription offers.

Learn More

Link:
Genetic testing to tailor heart drug prescriptions? - Harvard Health - Harvard Health

This article was originally published here

J Surg Oncol. 2021 Mar 18. doi: 10.1002/jso.26372. Online ahead of print.

ABSTRACT

BACKGROUND: Genetic testing for hereditary breast cancer has implications for breast cancer decision-making. We examined genetic testing rates, factors associated with testing, and the relationship between genetic testing and contralateral prophylactic mastectomy (CPM).

METHODS: Patients with breast cancer (2000-2015) from The Health of Women Study were identified and categorized as low, moderate, or high-likelihood of the genetic mutation using a previously published scale based on period-relevant national guidelines incorporating age and family history. Genetic testing and CPM rates were compared using univariate and multivariate logistic regression.

RESULTS: Among 4170 patients (median age 56-years), 38% were categorized as high-likelihood of having a genetic mutation. Among high-likelihood women, 67% underwent genetic testing, the odds of which were increased among women of higher-education and White-race (p < .001). Among 2028 patients reporting surgical treatment, 385 (19%) chose CPM. CPM rate was highest among mutation-positive women (41%), but 26% of women with negative tests still underwent CPM. Independent of test result, genetic testing increased the odds of CPM on multivariate analysis (adjusted-OR: 1.69; 95% CI: 1.29-2.22).

CONCLUSIONS: Genetic testing rates were higher among women at high-likelihood of mutation carriage, but one-third of these women were not tested. Racial disparities persisted, highlighting the need to improve testing in non-White populations. CPM rates were associated with mutation-carriage and genetic testing, but many women chose CPM despite negative testing, suggesting that well-educated women consider factors other than cancer mortality in selecting CPM.

PMID:33735483 | DOI:10.1002/jso.26372

Read more here:
Genetic testing and surgical treatment after breast cancer diagnosis: Results from a national online cohort - DocWire News

This article was originally published here

J Genet Couns. 2021 Mar 21. doi: 10.1002/jgc4.1413. Online ahead of print.

ABSTRACT

Clinical guidelines recommend that women at high risk of breast cancer should consider various risk-management options, which remain widely underutilized. We conducted semi-structured, qualitative interviews with 50 high-risk women to understand how financial constraints affect use of genetic counseling, genetic testing, and further risk-management decisions. Inductive analyses revealed three categories of health-related financial constraint: (a) lack of insurance, (b) underinsurance, and (c) other financial constraints (e.g., medical debt, raising children, managing comorbidities). Various breast cancer risk-management actions were limited by these financial constraints, including genetic counseling, genetic testing, enhanced screening, and prophylactic surgeries. Womens narratives also identified complex relationships between financial constraint and perceptions of healthcare providers and insurance companies, particularly as related to bias, price transparency, and potential genetic discrimination. Results from this study have implications for further research and expansion of genetic counseling services delivery to more economically and racially diverse women.

PMID:33749063 | DOI:10.1002/jgc4.1413

Excerpt from:
Financial constraints on genetic counseling and further risk-management decisions among U.S. women at elevated breast cancer risk - DocWire News

Decisive Markets Insights publishes detailed report on Global Genetic Testing Market. Experts predict the market to grow exponentially from its earlier record of US$XX billion in 2020 to an estimated value of US$XX billion by 2027 with an annual compound growth rate of 5.6% over the next seven years. Nevertheless, the global COVID-19 pandemic, business has registered steady growth and there are huge prospects of investment opportunities.

Kindly click on the link below if you intend to make a direct purchase [emailprotected]https://decisivemarketsinsights.com/genetic-testing-market/23470160/buy-now

Report Summary

The Genetic Testing report is a one-stop station for providing industry research reports. The experts have used their intelligence and experiences in building the report. This report will be helpful for clients who are looking for solutions related to the industry. The report consists of a special section for consultation and recommendations regarding the industry. It is specially dedicated to the clients so they can feel more delighted after getting the solutions, that they are looking for. The report has made a special focus on the analysis of the global trend. A detailed study of product type, application, geography, and market segmentation is done as well. During the forecast period, a high amount of growth is expected regarding the industry. Significant statistics on the market status of the leading market players of the industry are provided in the report. In this way, the customers can be well aware of the threats and opportunities of the industry.

To avail Sample Copy of report, visit [emailprotected] https://decisivemarketsinsights.com/genetic-testing-market/23470160/request-sample

Key Companies Operating in this MarketAbbott LaboratoriesBio-Rad Laboratories, Inc. (RainDance Technologies, Inc.)Myriad Genetics, Inc. (Myriad RBM, Inc.)Danaher Corporation (Cepheid)F. Hoffmann-La Roche Ltd.Eurofins ScientificIllumina, Inc.Qiagen N.V.Thermo Fisher Scientific, Inc.CSL Ltd

Market By TypePredictive & Presymptomatic TestingCarrier TestingPrenatal & Newborn TestingDiagnostic TestingPharmacogenomic TestingOthers

Market By TechnologyCytogenetic TestingBiochemical TestingMolecular Testing

Market By ApplicationCancer diagnosisGenetic Disease DiagnosisCardiovascular Disease DiagnosisOthers

A competitive analysis is done in the report. This competitive analysis provides an insightful data on the market leaders in the industry. The intension is to help the clients to know about the existing market players as well as potential market entrants of the industry. The report is made in a way such that the clients can not only make proper decisions regarding the industry but also can make stable growth in the industry in the long run. The aim is to direct the clients towards the steady progress of their growth in the industry. There are many contents in the report like networking and distribution strategies, revenue shares in the market,companies with their profiles and comprehensive profiles of their offerings and many more.

In order to make a pre-order inquiry, kindly click on the link below:-https://decisivemarketsinsights.com/genetic-testing-market/23470160/pre-order-enquiry

The primary motive is to track the evolution of the growth path of the market from the year 2021 to the year 2027. The Genetic Testing report also offers an extensive assessment of manufacturing scenario and demand supply structures based on the segmental performance of the industry and its key dynamics. Through this report, the buyers of this industry are contributed with a full picture of the players that are highly influential regarding the industry. Besides this, there are different attributes based on the parameters like gross margin, profit, performance of the companies and their strategic movements. All these are well explained through various resources like graphs, tables and charts.

To inquire about the discount, kindly fill the form by clicking on the link below:-https://decisivemarketsinsights.com/genetic-testing-market/23470160/request-discount

Reasons to Buy a Full Report

An insight into the market size and growth 2020-2027 CAGR: 2020 to 2027, calculating 2019 as the base year Detail information about the dominant players in this segment Demand and supply chain mapped to clearly evaluate the market Apart from primary and secondary research methodology, data triangulation method is used for a clear understanding of the report Analysis by Industry expert

Kindly contact us and our expert will get back to you within 30 minutes:Decisive Markets InsightsSunil KumarSales HeadEmail [emailprotected]US +18317045538UK +441256636046

See the original post here:
Genetic Testing Market to Observe Positive Growth | Players Illumina, Inc., Qiagen NV, Thermo Fisher Scientific, Inc., CSL Ltd KSU | The Sentinel...

ROCKVILLE, Md., March 24, 2021 /PRNewswire/ --To support quality improvement (QI) projects in colorectal cancer, the Association of Community Cancer Centers (ACCC) has joined with Pfizer Global Medical Grants to issue a Request for Proposals (RFP) with the intent of funding QI initiatives that focus on the integration of biomarker testing into the treatment planning for patients with metastatic colorectal cancer (mCRC). The RFP seeks individual grant requests up to a maximum of $150,000. In total, Pfizer will provide $1.5 million in funding for these quality improvement grants.

"Efficient processes for the timely integration of molecular biomarker and genetic testing is increasingly recognized as a component of metastatic colorectal cancer care," said Advisory Committee Member Al B. Benson III, MD, FACP, FASCO, Professor of Medicine; Associate Director for Cooperative Groups Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL. "Through this grant program, ACCC member programs and practices are offered a significant opportunity to submit proposals to expand understanding and explore approaches for patient care in colorectal cancer."

In a survey of practitioners conducted by ACCC to assess the status of biomarker testing in patients with unresectable or mCRC, over 70% of respondents reported that more than half of their patients undergo biomarker testing. Fifty-two percent of respondents (52%), however, indicated that their cancer program has no standard biomarker testing protocol for patients with unresectable or mCRC. Over 40% of respondents reported that patients with mCRC who have had biomarker testing are treated with systemic medical therapy "frequently" or "almost always" before all biomarker test results are available.

Survey respondents reported that challenges to the optimal use of biomarker testing in this population include patient factors such as patients' general health and physical fitness, patient preference, insurance coverage, clinical trial eligibility, and age. There are also practice-level factors including insufficient tissue for testing, poor tissue quality, long turn-around time, patient refusal, quality of in-house testing, difficulty getting reimbursed, lack of availability of in-house testing, no access to molecular tumor board(s), and inadequate staffing.

Access the survey summary on the ACCC website at http://accc-cancer.org/colorectal-survey-summary.

The opportunity to submit a proposal in response to the RFP is only available to ACCC-member cancer programs and practices. Grant requests should describe concepts and ideas for design and implementation of systems or programs that will close clinical practice gaps related to biomarker testing in patients with mCRC through establishment of education and support mechanisms for community providers.

The RFP process has two stages. Stage one is submission of a three-page letter of intent (LOI). If the LOI is selected, the applicant will be invited to submit a full proposal. Deadline for LOI submission is May 12, 2021.

For more information and to view the RFP, visit https://www.accc-cancer.org/projects/colorectal-cancer/overview.

About the Association of Community Cancer Centers

The Association of Community Cancer Centers (ACCC) is the leading education and advocacy organization for the cancer care community. Founded in 1974, ACCC is a powerful network of 28,000 multidisciplinary practitioners from 2,100 hospitals and practices nationwide. As advances in cancer screening and diagnosis, treatment options, and care delivery models continue to evolve - so has ACCC - adapting its resources to meet the changing needs of the entire oncology care team. For more information, visit accc-cancer.org or call 301.984.9496. Follow us on Facebook, Twitter, and LinkedIn; read our blog, ACCCBuzz; and tune in to our podcast, CANCER BUZZ.

SOURCE ACCC

Read the original post:
Association of Community Cancer Centers and Pfizer Offer Grant Opportunities Focused on Improving Quality of Metastatic Colorectal Cancer Care Through...

Syndicate Market ResearchsNew ExclusiveResearch on Predictive Genetic Testing & Consumer/Wellness Genomics Market Report Demandgrows Rapidly as Our Research Analyst covers the key parameters Required for your Research Need.This Predictive Genetic Testing & Consumer/Wellness Genomics Market Report coversglobal, regional, and country-level market size,market shares,market growth rate analysis (include Reseaon of highest and lowest peak Market analysis),product launches, recent trend, the impact of covid19 on worldwide or regional Predictive Genetic Testing & Consumer/Wellness Genomics Market. The Report also includes Key competitors/players/Manufactures/vendors in recent market trends areARUP Laboratories, Bio-Rad Laboratories Inc., BGI, Genesis Genetics, Thermo Fisher Scientific Inc., 3andMe Inc., Abbott Laboratories, F. Hoffmann-La Roche Ltd, Myriad Genetics Inc, Agilent Technologies, Illumina Inc., Counsyl Inc. Syndicate Market Research AnalysesResearch Methodology overview consists ofSecondary Research,Primary Research,Company Share Analysis,Model ( including Demographic data, Macroeconomic indicators, andIndustry indicators i.e. Expenditure, infrastructure, sector growth, and facilities, etc),Research Limitations and Revenue Based Modeling.Comprehensive analysis of Predictive Genetic Testing & Consumer/Wellness Genomics Market Based oncurrent analysis & future analysis, which is based on historic data also featured in this Reports.Presenting the Predictive Genetic Testing & Consumer/Wellness Genomics Market Factor Analysis-Porters Five Forces, Supply/Value Chain, PESTEL analysis, CAGR value, Market Entropy, Patent/Trademark Analysis, andPost COVID Impact Analysis

Get FREE PDF Sample Report Copy For Your Research:https://www.syndicatemarketresearch.com/sample/predictive-genetic-testing-consumer-wellness-genomics-market

Our Research Analyst Delivered Free PDF Sample Report copy as per your Research Requirement, also including impact analysis of COVID-19 on Predictive Genetic Testing & Consumer/Wellness Genomics market Industries

Dont miss out on business opportunities in Predictive Genetic Testing & Consumer/Wellness Genomics Market. Speak to our analyst and gain crucial industry insights that will help your business growth while filling Free PDF Sample Reports

Advantage of requesting FREE Sample PDF Report Before purchase

The above-mentioned Global Predictive Genetic Testing & Consumer/Wellness Genomics market report presentation has been estimated at length and according to expert analysis, is anticipated to entail an impressive growth of xx million USD in 2020 and is projected to further reach a total growth estimation of xx million USD through the forecast till 2026, growing at a CAGR of xx%,and you get accurate CAGR according to Predictive Genetic Testing & Consumer/Wellness Genomics market size which actual exist

This global study of the Predictive Genetic Testing & Consumer/Wellness Genomics Systems market offers an overview of the existing market trends, metrics, drivers, and restrictions and also offers a point of view for important segments. The report also tracks product and services demand growth forecasts for the market. There is also to the study approach a detailed segmental review. A regional study of the global Predictive Genetic Testing & Consumer/Wellness Genomics Systems industry is also carried out in North America, Latin America, Asia-Pacific, Europe, and the Near East & Africa. The report mentions growth parameters in the regional markets along with major players dominating the regional growth.

Key Research Techniques of Predictive Genetic Testing & Consumer/Wellness Genomics Market Report include:

The qualities of this Predictive Genetic Testing & Consumer/Wellness Genomics study in the industry experts industry, such as CEO::Marketing Director::Technology and Innovation Director::Vice President::Founder and Key Executives of key core companies and institutions in major Predictive Genetic Testing & Consumer/Wellness Genomics around the world in the extensive primary research conducted for this study we interviewed to acquire and verify both sides and quantitative aspects.

The main sources of Predictive Genetic Testing & Consumer/Wellness Genomics are industry experts from the Predictive Genetic Testing & Consumer/Wellness Genomics industry, including management organizations::processing organizations::and analytical services providers that address the value chain of industry organizations. We interviewed all major sources to collect and certify qualitative and quantitative information and to determine prospects.

Predictive Genetic Testing & Consumer/Wellness Genomics Industrial Analysis: By Applications

Breast & Ovarian Cancer, Cardiovascular Screening, Diabetic Screening & Monitoring, Colon Cancer, Parkinsonism/Alzheimers Disease, Urologic Screening/Prostate Cancer Screening, Orthopedic & Musculoskeletal, Other Cancer Screening, Other diseases

Predictive Genetic Testing & Consumer/Wellness Genomics Business Market Trends: By Product

Predictive Testing, Consumer Genomics, Wellness Genomics

Get Full Research Report On Global Predictive Genetic Testing & Consumer/Wellness Genomics Market By Syndicate Market Research:https://www.syndicatemarketresearch.com/market-analysis/predictive-genetic-testing-consumer-wellness-genomics-market.html

Predictive Genetic Testing & Consumer/Wellness Genomics Market Segment : By Region

Asia Pacific

North America

Europe

Latin America

The Middle East and Africa

TOC of 15 Key Chapters Covered in the Global Predictive Genetic Testing & Consumer/Wellness Genomics Market:

Key Highlights & Touch Points of the Predictive Genetic Testing & Consumer/Wellness Genomics Market Worldwide for the Forecast Years 2020-2026:

Read Our Other Exclusive Blogs:https://www.linkedin.com/posts/varsharani-lavate-951970102_homehealthcare-healthcaremonitoring-healthcare-activity-6728986246502326272-RyRe/

If you want Special Requirement or any other report Requirement, let us know about it, we will give you data as per your RESEARCH needsales@syndicatemarketresearch.com

About Syndicate Market Research:

At Syndicate Market Research, we provide reports about a range of industries such as healthcare & pharma, automotive, IT, insurance, security, packaging, electronics & semiconductors, medical devices, food & beverage, software & services, manufacturing & construction, defense aerospace, agriculture, consumer goods & retailing, and so on. Every aspect of the market is covered in the report along with its regional data. Syndicate Market Research committed to the requirements of our clients, offering tailored solutions best suitable for strategy development and execution to get substantial results. Above this, we will be available for our clients 247.

Contact US:

Syndicate Market Research244 Fifth Avenue, Suite N202New York, 10001, United StatesWebsite:https://www.syndicatemarketresearch.com/Blog:Syndicate Market Research Blog

Read more:
Global Predictive Genetic Testing & Consumer/Wellness Genomics Market Comprehensive Analysis on types and application 2020-2026 - The Courier

Army Veteran Keisha Bellamy begins each day with a cup of coffee, opens her laptop and logs in to Zoom.

She facilitates a six-week course on healthy living for Veterans, and on one particular morning, one woman in the group was struggling. The Veteran lives with post-traumatic stress disorder, and was feeling defensive, disrupting the group conversation.

Bellamy has seen these actions before in others. As a registered nurse with a background in mental health nursing, she redirects the conversation with skill and compassion.

You see, at VA we offer a safe haven to Veterans like this woman who others might dismiss or find difficult to work with in a group setting, Bellamy says. Thats because many employees at VA are Veterans. We see ourselves in our patients and they see themselves in us.

After six weeks, the Veterans graduate with new skills and goals for managing chronic illness. They also finish the course as comrades in healthier living, offering each other friendship and support as they manage their illness together.

For Bellamy, this is the best part of her job.

Bellamy has 15 years of service at VA with the last two years at the San Francisco VA Healthcare System. There, she oversees programs in health promotion and disease prevention.

To encourage Veterans to sign-up, its important to be a good role model, Bellamy says. You have to build relationships and practice what you preach.

So, every week before the pandemic, Bellamy would unroll her yoga mat and go into downward-facing dog with fellow Veterans at a community yoga studio. Afterward, she would chat with classmates and tell them about other health and wellness programs at San Francisco VA.

And when VAs Million Veteran Program launched in 2011, Bellamy was one of the first to enroll.

The program collects a blood sample from participating Veterans for genetic testing along with information on their health, lifestyle and military exposures to better understand disease in Veteran populations.

With this data, the program supports groundbreaking research in precision medicine that will one day offer Veterans the personalized care they deserve.

Being part of MVP allows me to explain what its like to enroll and be part of the program, says Bellamy, who is now joined by 830,000 fellow Veterans in what has become one of the largest genetics research programs in the world.

Bellamy has a connection with Veterans that other colleagues do not. As a Black female health care provider, Black Veterans in the Bay Area feel heard and understood by her.

Its rare for patients of color to see someone on the clinical side who looks like them, especially on the West Coast, Bellamy says. And theres still some concern as it relates to research, she adds.

While MVP has the largest Black cohort of any genetic research study approximately 19% of Veterans in the program are African American the program aims to increase diversity so it can support groundbreaking discoveries for Veterans of all genders and races.

She hopes her participation in MVP will show Veterans of color, especially women Veterans like herself, that this research is safe and valuable.

Its important that women and people of color are represented if we also want tomorrows medicine, Bellamy says. Our community and the future of their health care depends on our participation.

To learn more about VAs Million Veteran Program, visit mvp.va.gov or call 866-441-6075.

Sandra Glover is the chief communications officer forVISN 9.

Excerpt from:
Army Veteran a champion for health and research - VAntage Point - VAntage Point Blog

But new scientific evidence suggests that's not what happened.

Genomic testing shows at least two of the Australian's babies likely died from a previously undiscovered genetic mutation that led to heart complications -- meaning she may have been wrongfully imprisoned for almost two decades.

The ramifications don't end there.

Early years

In the late 1980s, she married Craig Folbigg, who she had met at a disco in the Australian city of Newcastle. They had their first child when she was 21, a boy named Caleb.

Folbigg soon fell pregnant again, and in 1990 she had another son, Patrick. Tests showed he was normal and healthy. But at four months, he had an unexplained ALTE, an apparent life-threatening event, that left him with brain damage and seizures. Four months later, he died as a consequence of seizures.

Her third child, Sarah, died age 10 months -- her cause of death was listed as SIDS. When her fourth daughter, Laura, died age 18 months on March 1, 1999, police started investigating.

The couple's marriage broke down. After Folbigg left, her husband found her diary and read an entry that he said made him want to vomit. He took the diary to police on May 19, 1999, according to the inquiry.

On April 19, 2001, Folbigg was arrested and charged with four counts of murder.

Her childhood best friend Tracy Chapman describes Folbigg as a caring animal lover who was a "really good mom." But at trial in 2003, the prosecution argued Folbigg had smothered her children. There was no conclusive forensic proof -- instead, the prosecution relied on a maxim credited to British pediatrician Roy Meadow: "One sudden infant death is a tragedy, two is suspicious and three is murder, until proven otherwise."

The prosecutor compared the chance of the children dying of natural causes to pigs flying.

"I can't disprove that one day some piglets might be born with wings and that they might fly. Is that some reasonable doubt? No," the prosecutor told the jury during the 2003 trial. "There has never ever been before in the history of medicine that our experts have been able to find any case like this. It is preposterous. It is not a reasonable doubt. It is a fantasy and, of course, the Crown does not have to disprove a fanciful idea."

The prosecution pointed to Folbigg's journals, which they said contained virtual admissions of guilt.

"I feel like the worst mother on this Earth, scared that (Laura) will leave me now, like Sarah did. I knew I was short tempered and cruel sometimes to her and she left, with a bit of help," Folbigg wrote in one. "It can't happen again. I'm ashamed of myself. I can't tell (my husband) about it because he'll worry about leaving her with me."

Folbigg didn't confess, there was no obvious motive, and no one claimed to have seen her murder her children. But the jury found her guilty of the murder of three children and the manslaughter of one.

Folbigg was eventually sentenced on appeal to 30 years in prison with a non-parole period of 25 years. By the time she is eligible for parole, Folbigg will be 60 years old.

The fight to get her out

In 2015, with her appeals exhausted, Folbigg's lawyers submitted a petition to the governor of New South Wales, asking him to direct that an inquiry be held into her convictions. The lawyers argued new evidence had come to light since her unsuccessful appeals -- including a growing understanding of SIDS -- that lead to a "feeling of disquiet" over her convictions. If the former NSW District Court chief judge Reginald Blanch, who headed the inquiry, agreed, he could refer the case back to the Court of Criminal Appeal.

As part of that inquiry, Folbigg's legal team approached Prof. Carola Vinuesa, co-director of the Centre for Personalised Immunology at Australian National University, to ask her to sequence the children's genomes to see if there was a genetic mutation that could have caused SIDS.

"There was a chance -- even though it might be a long shot -- that (Folbigg) was carrying something that might be passed on to the children," Vinuesa said. "To my knowledge, this is the first case in which a court (anywhere in the world) has used whole genome sequencing to find evidence of a cause of death."

When they sequenced the genomes of all four children, they found both daughters carried the same CALM2 mutation as their mother.

After the inquiry had closed, more evidence came to light, prompting Vinuesa and her team to write to the judge telling him it was likely the daughters died as a result of the variant. Despite the new finding, Judge Blanch opted not to reopen the inquiry. After taking all the evidence -- including the diaries -- into account, Blanch said he remained of the view Folbigg had smothered Sarah and Laura.

New developments

Last November, scientists published even more compelling evidence.

Led by Danish professor Michael Toft Overgaard, a team of experts across six countries found the CALM2 variant in Folbigg and her two girls could cause disease -- just like other CALM2 variants.

They concluded the variant altered the girls' heart rhythm, making them susceptible to heart conditions -- particularly given the medication they were given. Sarah was on antibiotics for a cough, while Laura was being treated with paracetamol and pseudoephedrine for a respiratory infection shortly before she died. Laura had inflammation of the heart when she died to the extent that three professors said they would have listed it as her cause of death.

"To my knowledge, this is the first case in which a court (anywhere in the world) has used whole genome sequencing to find evidence of a cause of death."Professor Carola Vineusa

In both of the boys, scientists found other variations in their BSN, also known as Bassoon, genes -- one variant had been inherited from their mother, and the other likely from their father, although he refused to provide a sample to the researchers. When both copies of the BSN gene are defective in mice it can cause them to die young during epileptic fits. Scientists are still investigating whether this variant could have caused the two boys' deaths. Patrick experienced seizures before he died.

Only 75 people in the world are known to carry mutations in their CALM1, CALM2 or CALM3 genes that have been shown to be lethal in children. But while genetic mutations that cause SIDS may be rare in the general population, once a parent has a genetic mutation there is a high chance of them passing it on, Vinuesa says.

"In the end it's not about these variations being very rare in the world, it's about the chances of Kathleen meeting someone like Craig and having this combination of mutations between both of them. Once genetics come into play, statistics go out the window," Vinuesa added.

Vinuesa said the case shows that contrary to what was suggested at trial, there doesn't need to be one explanation for all four of the deaths.

"The pathology already told us there were different causes," Vinuesa said.

The research hasn't yet freed Folbigg, but it has already had an impact. Folbigg's lawyers launched a case in the New South Wales Court of Appeal, arguing that the commissioner of the 2019 inquiry incorrectly applied the law to his decisions. The genome findings also prompted a petition with more than 90 signatures to the New South Wales governor earlier this month.

"It is deeply concerning that medical and scientific evidence has been ignored in preference of circumstantial evidence. We now have an alternative explanation for the death of the Folbigg children," Prof. Fiona Stanley, who has been recognized for her work on child health, said in a statement at the time of the petition.

"The reality is, Kath's lost four children. And she hasn't been allowed to grieve as a mother should."Tracy Chapman

A spokesperson for Gov. Margaret Beazley said the state's attorney general is considering the petition and will advise her. According to New South Wales' Department of Communities and Justice, few people have ever received a pardon by the state.

Even if Folbigg is freed, her legal fight may not be over. She will need to go to the Court of Criminal Appeal to get her conviction overturned if she wants to clear her name -- and it will be another legal matter again if she wants to get compensation for the years she's spent in prison.

For Folbigg, the research offers some hope -- but it was also emotional for her to hear, says Chapman, who talks to Folbigg every day.

"You're being told potentially the thing that you carried has been passed onto the children. So that's emotionally quite heart-wrenching," Chapman said. "The reality is, Kath's lost four children. And she hasn't been allowed to grieve as a mother should."

Science in the court room

Folbigg's case is part of a bigger picture -- a growing understanding of SIDS, a changing view about what multiple deaths in a family means, and a wider criticism of how science is presented in the courtroom.

Much of Folbigg's conviction was based on a maxim credited to Meadows that three infant deaths are murder, unless proven otherwise -- a maxim that had already started to draw skepticism.

"In our community, and in any civilized community, that is abhorrent."

In a similar case to Folbigg's, Australian woman Carol Matthey was accused of murdering her four children between 1998 and 2003, but the case against her was dropped due to a lack of evidence -- even though the same experts that testified against Folbigg were set to do so against Matthey.

Gary Edmond, a law professor at the University of New South Wales who is an expert in trial evidence and forensic science, said it was unfortunate Folbigg went to trial when she did. If she was tried a few years later, when the doubt over Meadow's maxim was better established, the courts might have been more cautious about admitting the expert evidence used to convict her, he said.

But even now, the way Australian courts handle evidence is outdated -- and lagging behind United States, Canada, New Zealand and the United Kingdom, Edmond said. While other jurisdictions assess whether science is reliable before it comes to court, Australia's system leaves it up to the juries to decide what's valid. That's a problem because juries don't have enough knowledge to make complex scientific judgments, Edmond said.

Folbigg's lawyer Rhanee Rego -- who has been working on her case for almost five years unpaid -- agrees courts need to be careful about which experts are allowed to give evidence. "I think that one of the biggest lessons we can take from this case is that we need to listen more carefully in the legal system to peer-reviewed and evidence-based science and medicine," she said.

Genetic answers

The advances in genetic testing -- including the findings in Folbigg's case -- could also help give answers for others dealing with the unexplained deaths of their children.

Vinuesa says it's likely that in the next few years, other families who have experienced SIDS will find a genetic mutation is to blame.

"We need to listen more carefully in the legal system to peer reviewed and evidence-based science and medicine."Rhanee Rego

"In most families where there have been SIDS deaths, nobody has yet gone back and sequenced the genomes of the children," she said. As molecular autopsies become more common, she thinks there will be more genetic explanations for otherwise unexplained deaths.

That could help families looking for answers -- and also help those worried about being targeted by the law.

"Many families live in fear, because they've had two or more children dying and they're worried that one day someone will be knocking at their door with some type of police investigation," she said. "We know now that when you have a genetic condition ... it's not rare."

Chapman says her childhood friend hopes her case helps other parents explain otherwise unexplainable deaths.

"It's not just about having Kath free," Chapman says. "The most important thing after Kath is freed is that this never happens to anybody else ever again."

View original post here:
Genetics may free a woman convicted of killing her 4 babies and help other parents explain the unexplainable - CNN

The results showed that in half of patients with cancers of unknown origin, next-generation sequencing located the primary site of origin and, in those who were matched to a therapy, half achieved a clinical benefit.

New study results support the use of next-generation sequencing (NGS) as standard-of-care (SoC) in patients with advanced solid tumors, particularly for certain groups.

Appearing in JAMA Oncology,1 results showed that in half of patients with cancers of unknown origin, NGS located the primary site of origin and in those who were matched to a therapy, half achieved a clinical benefit.

Based on the data presented by Cobain et al and others, it is evident that such precision medicine strategies are especially fruitful in cancer types without clear standard-of-care options, such as carcinoma of unknown primary and other rare tumors, said researchers in an accompanying editorial.2 Such efforts will allow us to deliver personalized therapies with potential therapeutic benefit to patients and to further refine the development of precision medicine efforts in oncology.

In total, there were 1000 patients included in the study, which analyzed approximately 7 years worth of data.

The findings also have important implications for inheritable cancer risk, as results showed that potentially inheritable cancer risk was detected in 16% of patients. The researchers of the study said this suggests directed germline testing for inherited cancer predisposition in all patients with advanced cancer is warranted.

Any family members who have also inherited those same mutations may be at increased risk for cancer, explained study author Erin Cobain, MD, an oncologist at Michigan Medicine in a press release. So, a lot of this testing prompted downstream genetic testing and counseling across families. Thats how sequencing can have even more far-reaching impact than just looking for therapies to directly help a current patient.

Overall, potentially actionable genomic alterations were identified in approximately 80% of patients. There were 132 patients who underwent sequencing-directed therapy, 20% of whom had exceptional responses and around 40% of whom exhibited any clinical benefit.

However, study authors acknowledged challenges associated with determining the clinical utility of NGS testing in the cancer space, including:

References:

1. Cobain EF, Wu Y, Vats P, et al. Assessment of clinical benefit of integrative genomic profiling in advanced solid tumors. JAMA Oncol. Published online February 25, 2021. doi:10.1001/jamaoncol.2020.7987

2. Yap TA, Johnson A, and Meric-Bernstam F. Precision medicine in oncologytoward the integrated targeting of somatic and germline genomic aberrations. JAMA Oncol. Published online February 25, 2021. doi:10.1001/jamaoncol.2020.7988

More here:
Study Makes the Case for NGS Testing as SoC in Certain Patients With Advanced Cancer - AJMC.com Managed Markets Network

The global cell and gene therapy drug delivery devices market was valued at $55.75 thousand in 2019, and is expected to reach $375.13 thousand by 2030, registering a CAGR of 16.61% during the forecast.

The global cell and gene therapy drug delivery devices marketis projected to reach $375.13 thousand by 2030, reveals the premium market intelligence study by BIS Research. The study also highlights that the market is set to witness a CAGR of 16.61% during the period between 2020 and 2030.

The comprehensive study of global cell and gene therapy drug delivery devices market BIS Research extensively covers the following:

The detailed study is a compilation of 19 Market Data Tables and 184 Figures spread through 315 Pages and in-depth TOC bisresearch.com/industrarket.html

Besides these parameters, the study also encompasses the market growth drivers, opportunities, market restraining factors, competition mapping, and segmental analysis.

BIS Research study indicates that the increasing global geriatric population, prevalence of genetic disorders, the increased demand for gene therapeutics that not only cure the chronic conditions completely but also improve the quality of life of the patients are the major factors anticipated to contribute to the growth of the global cell and gene therapy drug delivery devices market.

The study highlights the various emerging opportunities, such as strong pipeline and drug approvals of cell and gene therapies, introduction of cell and gene therapy drug delivery devices, potential technologies in cell and gene therapy drug delivery devices market, original equipment manufacturers, clinical trial scenario, and approved cell and gene therapy drug delivery devices. Scope of cell and gene therapy drug delivery devices, the clinical trial landscape of cell and gene therapies in China, the U.S, and across the world, challenges in cell and gene therapy drug delivery devices, and massive scope for adoption of cell and gene therapy drug delivery devices in emerging nations that can be leveraged by players operating in the market.

Data from different segments of the market has been analyzed minutely to gain a holistic view of the market. These segments include market by product type, commercialized drugs, and regions.

Each of these segments is further categorized into sub-segments and micro-segments to compile an in-depth study.

To emphasize the dominance of the intravenous catheter segment of cell and gene therapy drug delivery devices market by product segment over other segments under the product category of cell and gene therapy drug delivery devices market in 2020 and 2030, Raviteja Palakurthy, Senior Research Analyst BIS Research, states, "The reason for market growth and the dominance of intravenous catheter segment can be attributed to the increasing global usage of intravenous catheters to deliver drugs that are dosed frequently for fairly long periods of time and for specific disease conditions. For most of currently approved cell and gene therapies such as Kymriah, Yescarta, Zolgensma, Provenge, Strimvelis, Zynteglo, and Tecartus intravenous catheter are used as its drug delivery device

Request for a Sample: bisresearch.com/requeste=download

Key insights are drawn from in-depth interviews with the key opinion leaders of more than 20 leading companies, market participants, and vendors. The key players profiled in the report include Amgen, Inc., Bausch & Lomb Incorporated, Becton, Dickinson and Company, Bluebird bio, Inc., Castle Creek Biosciences, Inc (Fibrocell Technologies, Inc.), Dendreon Pharmaceuticals LLC., Helixmith Co., Ltd (ViroMed Co., Ltd), Human Stem Cell Institute, Kite Pharma, Inc., Kolon Tissue Gene, inc., Novartis AG, Orchard Therapeutics plc., Pfizer, Inc., Renova Therapeutics, Spark Therapeutics, Inc., uniQure N.V., and Vericel Corporation.

The study also offers strategic recommendations that can help organizations in tracking various products, trends, and technologies that are changing the dynamics of the market. The recommendations by BIS Research also offer bespoke research services to help organizations meet their objectives.

Who should buy this report?

How can market intelligence on Cell and Gene Therapy Drug Delivery Devices add value to an organizations decision-making process?

Insightful Questions Covered to Enable Companies Take Strategic Decisions

About BIS Research:

BIS Researchis a global B2B market intelligence and advisory firm focusing on deep technology and related emerging trends which can disrupt the market dynamics in the near future. We publish more than 200 market intelligence studies annually that focus on several deep technology verticals.

Our strategic market analysis emphasizes on market estimations, technology analysis, emerging high-growth applications, deeply segmented granular country-level market data, and other important market parameters useful in the strategic decision-making for senior management.

BIS Research offers syndicate as well as, custom studies, and expert consultations to firms, providing them specific and actionable insights on novel technology markets, business models, and competitive landscape.

BIS Healthcare vertical offers intelligence in the healthcare technology market for Medical Devices, Digital Health, Life Sciences, Robotics and Imaging, Information Technology, Precision Medicine, and other emerging healthcare technologies, covering the entire industry spectrum. In the past 5 years, BIS Healthcare has published more than 50 reports under the precision medicine banner.

Additionally, BIS Research has been nominating Top 25 Voices in Precision Medicine on its Insight Monk platform for the past two years successfully.

This email address is being protected from spambots. You need JavaScript enabled to view it.

See the rest here:
BIS Research Study Highlights the Global Cell and Gene Therapy Drug Delivery Devices Market to Reach $10.82 Billion by 2030 investigated in the latest...

BioCentriq, the New Jersey Innovation Institutes cell and gene therapy development and manufacturing center, on March 23 announced it partnered with Kytopen, a Cambridge-based startup spun out of the Massachusetts Institute of Technology (MIT).

Our mission at BioCentriq is to work with innovative industry partners like Kytopen to advance the production and manufacturing of cell and gene therapies, making them accessible and affordable for the patients who so desperately need them, said Haro Hartounian, and SVP, general manager, BioCentriq. This partnership aligns perfectly with that mission.

Kytopen leadership team KYTOPEN

Kytopens proprietary Flowfect technology is a flexible, complete technology solution for non-viral cell engineering that integrates the discovery, development, and manufacturing of cell and gene therapeutics. The platform speeds therapies from the clinic to commercial use by enabling cell engineering without compromising functionality or viability. Kytopens technology reduces risk and provides maximum control and flexibility to drive higher yields, faster approvals, and better outcomes for curative cellular disease treatment.

The Flowfect platform is a transformative solution that eliminates the complexity of gene delivery for cell engineering and links discovery, development and manufacturing in one flexible scalable solution, stated Paulo Garcia, CEO and co-founder of Kytopen. Our goal is to enable simple and efficient non-viral manufacturing of cell therapies in days versus weeks to help patients; our partnership with BioCentriq accelerates that goal.

Our Flowfect technology utilizes a novel combination of electrical energy and continuous fluid flow to engineer cells, said Bethany Grant, head of research and development at Kytopen. Our ability to engineer billions of cells in minutes with minimal disruption unlocks new opportunities to enable curative therapies in autologous or allogeneic therapeutic applications.

In the initial phase of the collaboration, the Kytopen and BioCentriq teams will demonstrate the impact to both autologous and allogeneic cell therapies by integrating this novel transfection technology with other steps in the manufacturing process.

BioCentriq has a manufacturing facility in Newark and a pilot plant in South Brunswick.

Here is the original post:
BioCentriq partners with Kytopen to advance production and manufacturing of cell and gene therapies - NJBIZ

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Maze Therapeutics, a company translating genetic insights into new precision medicines, today revealed its first three lead therapeutic candidates in the companys wholly owned pipeline. The candidates include:

Each of the three lead candidates was enabled by Mazes COMPASS platform, which uncovered important new findings for the genetic target, discerning which specific signals may be critical for the treatment of patients, and which are likely non-actionable. The Maze pipeline will have the potential to serve as precision medicines for rare diseases and mechanistically defined subsets of common diseases based on certain genetic drivers.

In addition, Maze is concurrently leveraging COMPASS to advance additional discovery-stage research programs across three main therapeutic areas of focus: metabolic, cardio/renal and neurological diseases. These programs will constitute a broad, diverse pipeline for Maze and will be a combination of wholly owned and partnership-led collaborations.

Maze was built by co-founders, including Charles Homcy and other preeminent thinkers in the field of genetics, on a bold vision to leverage growing knowledge of genetic drivers of disease in order to create precision medicines for the treatment of both rare and more common diseases, said Jason Coloma, Ph.D., president and chief executive officer of Maze. Since our founding, we have been leveraging insights from leading geneticists, combined with the growing availability of paired human genetic and clinical data, the evolution of functional genomic technologies and advances in computational power, to build our COMPASS platform in order to bring unique insights into efficient, genetics-based drug development. We are excited by the significant progress we have made with our platform and pipeline, bringing us an important step closer to our goal of delivering the right drug to the right patient at the right time.

Mazes therapeutic candidates are designed to: 1) target genes whose activity affects the phenotype associated with another, often distant, gene, referred to as genetic modifiers; 2) mimic the activity of protective genetic variants; 3) correct the effects of toxic genetic variants; or 4) leverage new genetic insights to address otherwise challenging drug targets.

COMPASS is a proprietary, purpose-built platform that combines human genetic data, functional genomic tools and data science technology to map novel connections between known genes and their influence on susceptibility, timing of onset and rate of disease progression. In addition, Maze is exploring applications of COMPASS in diseases of haploinsufficiency by identifying genetic mechanisms that increase levels of a deficient protein and translating them into therapeutics.

New findings using COMPASS helped fill in fundamental data gaps, turning known but challenging targets into exciting, differentiated approaches to the genetic drivers of disease for our first three programs, said Sarah Noonberg, M.D., Ph.D., chief medical officer of Maze. While it has been shown that targets with human genetic evidence are more likely to yield efficacious treatments, very few groups have had the capabilities to then turn genetic insights into viable drug programs. We believe our COMPASS platform, integrated with our extensive drug discovery capabilities, will allow us to accelerate the pace of therapeutic development, as well as increase the likelihood of producing therapies that provide meaningful clinical benefit for patients. We are excited to advance these initial programs and look forward to continued progress toward the clinic as efficiently as possible.

About Mazes Wholly Owned Programs

GYS1 Program for Pompe DiseasePompe disease is a rare, inherited autosomal recessive disorder with an incidence of approximately 1 in 40,000 live births in the U.S., and is estimated to affect 5,000 to 10,000 patients worldwide. It is caused by mutations in the GAA gene, which codes for an enzyme responsible for breaking down lysosomal glycogen into glucose. As a result of this mutation, glycogen accumulates in various tissues, particularly skeletal and cardiac muscle tissues, causing progressive weakness and respiratory insufficiency.

Maze is developing a novel, oral approach to treating Pompe disease by inhibiting the protein muscle glycogen synthase, which is encoded by the gene GYS1. Targeting this protein leads to reduction in the synthesis of glycogen, which is expected to restore glycogen balance through a mechanism called substrate reduction. While GYS1 has been a therapeutic target of interest, its attractiveness as a therapeutic target has been limited due to its structural complexity and uncertainties related to the tolerability of a long-term reduction in muscle glycogen levels. Critical insights derived from COMPASS have enabled Maze to overcome these challenges. Maze has interrogated the structurally complex protein to develop an oral inhibitor of muscle glycogen synthase, a target not previously addressable by small molecule therapies. Maze is rapidly progressing its GYS1 program toward an Investigational New Drug application and expects to initiate clinical trials in the first half of 2022.

APOL1 Program for Chronic Kidney DiseaseCKD affects approximately 37 million people in the U.S., including more than 700,000 patients who suffer from end-stage renal disease (ESRD), many of whom require chronic dialysis. Individuals of African ancestry are at an approximately 3.5-fold greater risk of developing ESRD than individuals of European ancestry. Previous studies have shown that two coding variants of the apolipoprotein L1 (APOL1) encoded by the gene APOL1 cause toxic gain-of-function variants and are important genetic drivers of kidney disease that are responsible for much of the increased risk for CKD and ESRD in individuals of African ancestry. There are currently no approved therapies that address the underlying causes of APOL1-associated CKD, and efficacious treatment options for individuals with APOL1 risk variants and CKD represent a significant unmet medical need.

Maze employed COMPASS to functionalize human genetic variants to uncover the underlying biology of the target and has designed a small molecule that corrects the effects of toxic gain-of-function variants to potentially enable a therapeutic solution. Maze plans to name the development candidate in early 2022.

ATXN2 Program for Amyotrophic Lateral SclerosisALS is a progressive and fatal neurodegenerative disease with a prevalence of approximately 16,000 patients in the U.S. Current available treatments for ALS primarily focus on providing symptomatic relief and have limited impact on disease progression. A high variability in disease phenotype and life expectancy is observed and believed to be related to the presence of genetic modifiers.

One of Mazes founders, Aaron Gitler, identified a potent genetic modifier, ATXN2, whose inhibition has been shown to limit the toxicity of a certain protein, TDP-43, which is involved in pathologic aggregates seen in up to 97% of all ALS cases. Maze is translating these important insights by developing a novel microRNA gene therapy that targets ATXN2 and has used the proprietary application of its functional genomics tools to optimize its properties. Maze plans to name the development candidate in early 2022.

About Maze TherapeuticsMaze Therapeutics is focused on translating genetic insights into new precision medicines for rare diseases and mechanistically defined subsets of common diseases. Maze has developed the COMPASS platform, a proprietary, purpose-built platform that combines human genetic data, functional genomic tools and data science technology to map novel connections between known genes and their influence on susceptibility, timing of onset and rate of disease progression. Using COMPASS, Maze is building a broad portfolio, including wholly owned programs targeting Pompe disease, chronic kidney disease and amyotrophic lateral sclerosis, as well as partnered programs in cardiovascular and ophthalmic diseases. Maze is based in South San Francisco. For more information, please visit mazetx.com, or follow us on LinkedIn.

Excerpt from:
Maze Therapeutics Reveals Its Initial Three Lead Programs Targeting Underlying Genetic Drivers of Life-Threatening Diseases - Business Wire

- Initial data from Cohort 1 of the PEARL-1 study shows safety and tolerability of repeat KB301 injections

- Dr. Bhushan Hardas M.D., MBA appointed President, Jeune, Inc.

PITTSBURGH, March 24, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (Krystal) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced the launch of Jeune, Inc., a wholly owned subsidiary of Krystal Biotech, and initial safety data from the ongoing Phase 1 trial of Jeunes lead product candidate, KB301 for treatment of aesthetic skin conditions.

Jeune was formed to advance innovative aesthetic medicines and has an exclusive license to a portfolio of candidates derived from Krystals proprietary technology platform. Jeunes products are designed to directly address biological changes in the skin associated with intrinsic and extrinsic aging. The lead product candidate, KB301, delivers the human COL3A1 gene to increase production of normal type III collagen at the site of administration.

My initial clinical experience with KB301 injections has been highly encouraging, said Dr. Mark Nestor, director of the Center for Clinical and Cosmetic Research and the Center for Cosmetic Enhancement. Not only were the injections well-tolerated, but we see clear signs of new collagen generation which underscores the potential of this treatment to directly address the declining levels of collagen that lead to wrinkles and other skin changes.

Initial data from Cohort 1 in the PEARL-1 studyThe Phase 1, open-label, dose-ranging study is being conducted in adult subjects aged 18-75 (NCT04540900). The primary outcome measure in this first-in-human study was to assess the safety profile of KB301. Secondary outcome measures include COL3A1 transgene expression. In Cohort 1, three different dose levels of KB301 were evaluated in seven (7) healthy subjects who received two intradermal injections into healthy buttock tissue spaced 30 days apart (day 0, day 30). KB301 injected areas were compared to uninjected or saline injected control tissue within the same subject. Treatment and control sites were biopsied at day 2 or day 32. Initial results are as follows:

More detailed data from Cohort 1 will be presented as an e-Poster talk at the Society for Investigative Dermatology (SID) Annual Meeting, held virtually May 3-8.

The presentation will be available on-demand for those registered for the SID conference from May 3, 2021 May 31, 2021. The poster will also be available on the companys website at http://www.jeuneinc.com

The company plans to begin enrollment in the efficacy cohorts of the Phase 1 study in the second half of 2021.

Jeune, Inc. LeadershipJeune has assembled a veteran team of leaders and advisors, comprised of pharmaceutical and biotechnology executives who together have decades of experience developing products in the aesthetic medicine space. Dr. Bhushan Hardas M.D., MBA will join the company on March 29th, 2021 as President of Jeune. Before joining Jeune, Dr. Hardas served as Chief Scientific Officer, Executive Vice President, Global Head of Licensing at Almirall and previously served as Chief Medical Officer of Allergan's Dermatology and Medical Aesthetics business.

I am thrilled to be joining Jeune at such an exciting time. With the ability to deliver genes directly to skin cells, this platform has tremendous potential to address underlying biological changes in aging or photo-damaged skin, noted Dr. Hardas. We are starting with KB301 and type III collagen which I look forward to advancing through the clinic, and the team is already working on pipeline programs that will address additional proteins of interest.

Prior to joining Allergan, Dr. Hardas served as Senior Vice President, Global Head of Dermatology and Aesthetics R&D and Chief Scientific Officer of the North American Business at Merz Pharmaceuticals. Dr. Hardas received advanced training in clinical immunology and molecular biology at King's College at the University of London, in London, England. He also completed a research fellowship in the Department of Dermatology at the University of Michigan, and received his Master of Business Administration degree in healthcare management from the University of California - Irvine.

We are thrilled to welcome Bhushan to Jeune, said Krish S. Krishnan, chairman and chief executive officer of Krystal Biotech. His expertise and development experience in aesthetics is an important asset presently and will help guide next steps for both the pipeline and Jeune overall.

Jeune, Inc. Board Krish Krishnan, Chairman and CEO at Krystal Biotech will serve as the Chairman of the Jeune Board. Joining Mr. Krishnan on the Board are Marc Forth, President and CEO of Aeon BioPharma and Suma Krishnan, Founder and COO of Krystal Biotech.

AboutJeune Inc. Jeune Inc., a subsidiary of Krystal Biotech, is a biotechnology company leveraging a clinically validated gene-delivery platform to fundamentally address and reverse the biology of aging and/or damaged skin. For more information, please visithttp://www.jeuneinc.com

AboutKrystal BiotechKrystal Biotech, Inc.(NASDAQ:KRYS) is a pivotal-stage gene therapy company leveraging its novel, redosable gene therapy platform and in-house manufacturing capabilities to develop therapies to treat serious rare diseases. For more information, please visit http://www.krystalbio.com.

Forward-Looking StatementsAny statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc., or its subsidiary Jeune, Inc., including but not limited to statements about the development of Krystals and Jeunes product candidates, such as plans for the design, conduct and timelines of ongoing clinical trials of KB301 the clinical utility of KB301, the ability of these candidates to fundamentally address and potentially reverse the biology of aging or damaged skin, plans to pursue research and development of other product candidates; and other statements containing the words anticipate, believe, estimate, expect, intend, may, plan, predict, project, target, potential, likely, will, would, could, should, continue, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and conduct of clinical trials, availability and timing of data from clinical trials, whether results of early clinical trials or trials will be indicative of the results of ongoing or future trials, uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including KB301 and such other important factors as are set forth under the caption Risk Factors in Krystals annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Krystals views as of the date of this release. Krystal anticipates that subsequent events and developments will cause its views to change. However, while Krystal may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Krystals views as of any date subsequent to the date of this release.

CONTACTS:

Investors:Whitney Ijemwijem@krystalbio.com

Media:Mary CoyleTellMed Strategiesmary.coyle@tmstrat.com

Source: Krystal Biotech, Inc.; Jeune, Inc.

More here:
Krystal Biotech Announces Launch of Jeune, a Gene-Based Aesthetics Company, and Initial Phase 1 Safety Data for KB301 in Aesthetic Indications -...

Company to use its novel gene editing technology to explore a potential in vivo gene therapy treatment for cystic fibrosis

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)-- Life Edit Therapeutics Inc, a next generation gene-editing company, today announced that it has received an award from the Cystic Fibrosis Foundationto identify potential gene editing approaches to treat certain patients with cystic fibrosis (CF). The award will enable Life Edit to screen its library of proprietary base editors for a potential treatment targeting people with CF that are not able to be treated by existing small molecule treatments due to what are known as nonsense genetic mutations (also known as stop mutations). This award is part of the Cystic Fibrosis Foundations Path to a Cure initiative that was launched in October 2019 to address and treat the underlying cause of CF.

Due in large part to the efforts of the CF Foundations support for the development of new medicines, there are now effective therapies available to most people living with the disease, but there remain as many as 7% of patients with CF for whom recent medical advances are not effective, said Mitchell Finer, Ph.D., Chief Executive Officer, Life Edit Therapeutics. Were looking forward to working with the CF Foundation to leverage the unique benefits that our platform offers to develop a highly targeted gene editing approach for these individuals. We believe our science and this approach can be applied across a range of diseases, which will be our focus as we work to build a pipeline of life-changing therapies for severe genetic diseases like CF.

Dr. Allie Crawley, Principal Investigator for the project and member of the Life Edit team, continued by saying, We are thankful to be a part of the Path to a Cure initiative from the CF Foundation which is focused on curing cystic fibrosis by addressing the underlying cause of the disease. We believe our base editor technology has potential to make a great impact in the lives of cystic fibrosis patients with nonsense mutations and are excited about the opportunity to begin early research in this development.

Despite tremendous progress in advancing therapeutics to help people with CF live longer and healthier lives, there remain unmet needs to help all those living with this disease. Approximately 13% of people living with CF have nonsense mutations. These mutations cause the cells to stop the production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein midway through the process, resulting in shortened, non-functional protein.

As part of the $400,000 award from the Foundation, Life Edit will explore its large collection of adenine base editors, or A-base editors, that can potentially be used to correct the six most common, Class I, cystic fibrosis nonsense mutations to restore CFTR function in vivo. A unique feature of the base editors under development by Life Edit is their small size which will allow in vivo delivery with Adeno-associated viruses (AAV) vectors to specific tissue types in the lungs. As part of the agreement, Life Edit will benefit from materials, resources, and expertise from the Cystic Fibrosis Foundation.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare genetic disease found in more than 30,000 people in the U.S. CF is a hereditary disease that affects the lungs and digestive system that is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that affect the production of the CFTR protein. When the CFTR protein is not made correctly, it affects the balance of salt and fluids inside and outside of the cell. This imbalance leads to thick, sticky mucus in the lungs, pancreas, and other organs. In the lungs, the mucus clogs the airways and traps germs, like bacteria, leading to infections, inflammation, respiratory failure, and other complications. CF is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.

About Genome Editing and Life Edit Therapeutics Platform

Genome editing technologies have revolutionized the way cell and gene therapies and regenerative medicines are discovered and developed by allowing genetic material to be removed, added, or altered at specific locations in the genome. While these technologies are in widespread use experimentally, enzymes that offer broader coverage and greater specificity are needed for creating novel cell and gene therapies.

To meet the need for better genome editing approaches, Life Edit Therapeutics has built one of the worlds largest and most diverse arrays of novel RNA-guided nucleases (RGNs) and base editors that are active in mammalian cells. These RGNs were developed using AgBiomes proprietary collection of more than 90,000 microbes and their complete genomes. Life Edit Therapeutics is investigating these proprietary RGNs, which are sourced exclusively from non-pathogenic organisms, to develop new gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. Life Edit Therapeutics nuclease collection also has a broad range of Protospacer Adjacent Motifs (PAMs) short sequences that must follow the targeted DNA sequence in order for the enzyme to make cuts that offer unprecedented access to genomic loci of interest. The Life Edit Therapeutics RGNs offer flexible editing options which encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with its proprietary deaminases.

Life Edit Therapeutics next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of gene therapies, Life Edit Therapeutics will continue to build its platform of novel nucleases, provide gene editing expertise to strategic partners and ElevateBios portfolio companies, and form other third-party partnerships to discover and develop new therapies.

About Life Edit Therapeutics Inc.

Life Edit Therapeutics is a next-generation gene editing company that has built a highly innovative genome editing platform with one of the worlds largest and most diverse collections of novel RNA-guided nucleases (RGNs) and base editors. Life Edit Therapeutics next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. The company is continuing to strengthen the platform, developing a pipeline of in vivo gene therapies to address severe genetic disease, and sharing its expertise through strategic partnership. Life Edit is an ElevateBio portfolio company. For more information visit lifeeditinc.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210323005136/en/

See the original post here:
Life Edit Therapeutics Announces Award from Cystic Fibrosis Foundation - BioSpace

-- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measured by western blot ---- Mean NSAD score improvement of 5.7 points from baseline was sustained through 24 months in low-dose cohort, and mean NSAD score improvement of 4.0 points from baseline at one year in high-dose cohort ---- Results in both cohorts continue to reinforce the safety and tolerability profile of SRP-9003 --

CAMBRIDGE, Mass., March 18, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Companys investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). In the first look at expression data from biopsies taken two years after a single administration of SRP-9003, results found sustained protein expression in muscle tissue. In functional outcomes assessments taken two years following treatment in Cohort 1 (low-dose cohort) and one year after treatment in Cohort 2 (high-dose cohort), patients continued to demonstrate stability in their NSAD (North Star Assessment for Dysferlinopathies) total score and improvements on timed function tests. Results are being presented today at the 2021 Muscular Dystrophy Association (MDA) Annual Clinical and Scientific Conference.

SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins. SRP-9003 is a gene therapy construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-SG protein, the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease.

This data is the first look at longer-term expression data with any gene therapy for muscular dystrophy. The meaningful and sustained levels of beta-sarcoglycan protein expression at two years and continued strength of the functional outcomes measured are tremendously positive and support continued advancement of this investigational treatment for patients, said Louise Rodino-Klapac, Ph.D., executive vice president and chief scientific officer, Sarepta Therapeutics. In Cohort 2, we also saw strong expression of delta-sarcoglycan and gamma-sarcoglycan proteins in addition to beta-sarcoglycan, which suggests that SRP-9003 is working to restore the dystrophin associated protein complex, or DAPC, which provides biological support for the sustained functional benefits observed in both cohorts. LGMD2E is one of the most severe forms of LGMD and causes significant disability in children while frequently leading to early mortality and the data continue to suggest this treatment could bring much needed hope to these patients.

Efficient transduction in skeletal muscle and robust beta-sarcoglycan protein expression were seen in both dose cohorts following infusion with SRP-9003, and significant creatine kinase (CK) reductions were observed.

Cohort 1 (Dosed at 1.851013 vg/kg), 24 months following treatment:

Cohort 2 (Dosed at 7.411013 vg/kg), 12 months following treatment:

In an exploratory evaluation of all SRP-9003 treated patients compared to a natural history cohort; patients treated with SRP-9003 demonstrated significant improvements in functional outcomes after 24 months. The mean decline in total NSAD score for patients in the natural history cohort was 4.6 points while SRP-9003 treated patients demonstrated a mean improvement of 4.6 points for a clinically meaningful difference of 9.2 points.

Since the last update from this study in October 2020, there have been no new drug-related safety signals observed, and no decreases in platelet counts outside of the normal range and no evidence of clinical complement activation observed in either dose cohort.

About SRP-9003 and the StudySRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. AAVrh74 has lower immunogenicity rates than reported with other human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.

This open label, first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of 4 and 15 years with significant symptoms of disease. The SRP-9003 study has two cohorts, each studying a different dose-per-kilogram based on the weight of the patient. Three participants in the low-dose cohort (Cohort 1) were treated with a one-time infusion of SRP-9003 dosed at 1.851013 vg/kg and an additional three participants in the high-dose cohort (Cohort 2) received a one-time infusion dosed at 7.411013 vg/kg based on linear standard qPCR titer method. The six participants were between the ages of 4 and 13. Post-treatment biopsies were taken at 60 days.

Sarepta has exclusive rights to the LGMD2E gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About Limb-girdle Muscular DystrophyLimb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.

Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding, SRP-9003 being the ideal candidate to treat peripheral neuromuscular diseases; the potential benefits of SRP-9003, including its potential to restore the dystrophin associated protein complex (DAPC); the potential benefits of MHCK7 and the AAVrh74 vector, including its potential to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle; and potential market opportunities.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; if the actual number of patients suffering from LGMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2020 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

InternetPosting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com.Weencourageinvestorsandpotentialinvestorsto consult our website regularly for important information about us.

Source:Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

Here is the original post:
Sarepta Therapeutics' Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and...