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Did Gene Therapy Cure Sickle Cell Disease? – American Council on Science and Health

A number of recent headlines imply a case study just published in the New England Journal of Medicine proves that gene therapy has cured sickle cell diseasea genetic disorder that incurs tremendous pain, suffering and diminished life expectancy. Here, we will unpack the significance of the researchers findings.

First, lets address why this news could be so groundbreaking to those afflicted and their loved ones.

Sickle Cell Disease is an inherited condition that causes a mutated hemoglobinthe protein within red blood cells (RBCs) that carries oxygen for delivery to vital tissues. Oxygen feeds our organs so they can stay healthy and perform their respective jobs. This Hemoglobin S (aka Sickle Hemoglobin) polymerizes on deoxygenation and rids the RBCs of their malleability. As a result, these malformed sickled cells are stiff and clump together thereby occluding vessels which in turn prompts organ damage.

Roughly 90,000 Americans have Sickle Cell Disease. (1) The natural course of the illness involves a complex cascade of events intermingled with crises often triggered by infections. Anemia is commonplace (and often profound) given these faulty cells get readily destroyed, over consumed and dont last as long as healthy RBCs. Vasoocclusive Crises result from infarction and ischemiain infants the hands and feet swell, in particular. Basically, adequate blood flow is halted wherever the obstruction takes place. Aggressive pain management and rehydration is essential.

Prophylactic antibiotics are a mainstay in an effort to stave off infection which can routinely catapult patients into a life-threatening crisis. By early childhood, they develop a functional asplenia or ineffective spleen. So, they become especially susceptible to overwhelming infection by encapsulatedbacteriahence, why vaccination for pneumococcus and the like is so important. Sepsis can result. Parvovirus can cause an aplastic crisis.

Strokes. Pulmonary infarcts with subsequent hypoxia. Acute Chest Syndrome. Gallstones. Blood transfusions are frequent. Though the blood supply is well-tested for safety, recurrent transfusion can lead to issues like iron overload, for instance. This too must be treated. The list goes on of the challenges, battles and treatment complexities these patients endure. Because fetal hemoglobin has a higher oxygen carrying capacity, a disease-modifying drug like Hydroxyurea that increases its presence is used.

Allogeneic hematopoietic stem-cell transplantation represents the only cure, but less than 18% of those with severe disease have sibling donors who are a match. (2) This is also not without great risk, though those need to be weighed against how advanced the disease. Due to such limited progress in management of this condition, this team of researchers sought to examine whether therapeutic ex vivo gene transfer into autologous hematopoietic stem cells referred to as gene therapy, may provide a long-term and potentially curative treatment for sickle cell disease. (3)

What does this mean? They took samples from the bone marrow of a patient with severe disease. The cells here provide the origins of our blood components which includes our red blood cells. This is where the problem begins in generating the sickling. A cancer drug, busulfan, was used to condition the body expected adverse effects from this occurred which resolved with standard care (e.g. anemia, low platelets, neutropenia and so on). Using a lentiviral vector, they transferred an anti-sickling gene into the patients stem cells (retrieved from the bone marrow) which get put back into the patient in the hope they will multiply and replace the cells made with the defective gene.

In a study funded in part by Bluebird Bio whose product is LentiGlobin BB305 (the antisickling gene therapy subject of this publication), the team concludes their patient had complete clinical remission with correction of hemolysis and biologic hallmarks of the disease. Furthermore, after fifteen months the antisickling protein remained high at approximately 50% and the patient had no crises or hospitalizations. Before, the patient required regular transfusions. After, all medications were stopped, no pain ones were needed, and the patient returned to full activities at school. (4)

Ongoing research is underway in a U.S. multi center, phase 1/2 clinical study. The intention is to use this gene therapy to treat those with severe sickle cell disease and another condition called beta-thalessemia. So far, in the few patients who have participated, their results seemingly support this work. Clearly, longer term follow-up and larger populations are crucial to understanding the significance of this report. Additionally, stem cell transplantation is no minor feat.

That said, for a disease that disables at such a young age, this option could be quite an extraordinary one if the success persists. ACSHs Senior Fellow in Molecular Biology, Dr. Julianna LeMieux, puts the promise of gene therapy into even greater context for this and other disease entities:”This is an incredibly promising result, even with the obvious caveat that it is only one person. Sickle Cell is a disease that is ripe for genetic advances for a few reasons. First,the gene that is affected is known andcan be replaced by the healthy variant. Also, the cells that are needed to be alteredare easily accessible inthe bone marrow. In many diseases, this is not the case. But, this one success story is incredibly encouraging for the sickle cell community and for moving the field of curing diseases using genetic editing forward.”

The team proved their concept. To know if “cure” is in this gene therapy’s future, much more data needs to be acquired and study be implemented. Promising with cautious optimism might be the most apt description.


(1) (2) (3) (4) Jean-Antoine Ribeil, M.D., Ph.D. et al. Gene Therapy in a Patient with Sickle Cell Disease. N Engl J Med. 376;848-855. March 2, 2017.


To learn more about “Orphan Diseases” or rare ones that afflict less than 200,000 (but in total impact 25 million Americans) and drug discovery challenges, review: Did Pompe Disease Geta New Champion in President Trump? and Pompe Disease, Newborn Screening and Inborn Errors of Metabolism.

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Did Gene Therapy Cure Sickle Cell Disease? – American Council on Science and Health

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Skin cells of schizophrenia patients reveal faulty genetic pathway that began in womb – Genetic Literacy Project

The skin cells of four adults with schizophrenia have provided an unprecedented window into how the disease began while they were still in the womb, according to a recent paper in Schizophrenia Research.

The paper was publishedby researchers at the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo in collaboration with the Icahn School of Medicine at Mount Sinai. It provides what the authors call the first proof of concept for their hypothesis that a common genomic pathway lies at the root of schizophrenia.

The researchers say the work is a first step toward the design of treatments that could be administered to pregnant mothers at high risk for bearing a child with schizophrenia, potentially preventing the disease before it begins.

The authors gained insight into the early brain pathology of schizophrenia by using skin cells from four adults with schizophrenia and four adults without the disease that were reprogrammed back into induced pluripotent stem cells and then into neuronal progenitor cells.

The next step in the research is to use these induced pluripotent stem cells to further study how the genome becomes dysregulated, allowing the disease to develop.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: Faulty genomic pathway linked to schizophrenia developing in utero, study finds

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Skin cells of schizophrenia patients reveal faulty genetic pathway that began in womb – Genetic Literacy Project

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Murray Ballard shoots cryonics in The Prospect of Immortality – British Journal of Photography

Patient Care Bay (Bigfoot dewar being filled with liquid nitrogen), Alcor Life Extension Foundation, Scottsdale, Arizona, USA. October 2006. From The Prospect of Immortality Murray Ballard

As his project goes on show at Newcastle’s Side Gallery, we republish an article on Ballard’s eye-opening series first printed in BJP’s July 2011 Ones to Watch issue

As debut projects go, Murray Ballard could scarcely have chosen a more intriguing subject than cryonics. The practice of preserving dead bodies at very low temperatures, in the hope of bringing them back to life far in the future, is commonly thought to exist only in science fiction, where it is generally known by its technically inaccurate name of cryogenic freezing.

Yet as Ballard (no relationto his namesake, the sci-fi author JG) discovered during his five- year investigation, hundreds of people around the world have alreadyinvested in what he has calls The Prospect of Immortality.

The 27-year-old began documenting cryonicists while studying photography at the University of Brighton, after he discovered there was a group of British believers based just along the Sussex coast in Peacehaven. He was soon making much longer excursions, his work taking him to the Alcor Life Extension Foundation in Arizona three times, the rival Cryonics Institute in Michigan twice, and the burgeoning Kriorus facility just outside Moscow on a further two occasions.

Portable perfusion kit. Home of Alan and Silvia Sinclair. Peacehaven, East Sussex, UK. May 2007. From The Prospect of Immortality Murray Ballard

Having worked as an assistant to Magnum photographer Mark Power for four years, Ballard is now looking at biotechnology for his first commission, which will be shown as part of the British Science Festival. He revels in the honesty that working with a large format camera allows.

Youre not saying, Look at this bit of the picture, youre saying all of it is equally as important, and all of the details are there to piece together meaning and narrative, he explains.

Power was on hand last month to formerly open Ballards first major solo exhibition at Impressions in Bradford, featuring Ballards images of the people involved in this pursuit of real-life resurrection, and the equipment to which they are entrusting their dreams of everlasting life.

Margaret Kiseleva, holding a photograph of her mother, Ludmila, KrioRus facility, Alabushevo, Moscow. September 2010. From The Prospect of Immortality Murray Ballard

The Prospect of Immortality by Murray Ballard is on show at Side Gallery from 0 March 30 April. The images in the exhibition are taken from a larger touring show, which was originally commissioned by Impressions Gallery and curated by director Anne McNeill. http://www.amber-online.commurrayballard.comBallard also published a book of the project last year with GOST Books

This textwas originally published as part of the Ones to Watch series of articles on emerging photographers in July 2011. This issue is now sold out, but other back issues can be bought

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Murray Ballard shoots cryonics in The Prospect of Immortality – British Journal of Photography

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Sickle cell anemia patient ‘cured’ by gene therapy, doctors say – FOX31 Denver

FOX31 Denver
Sickle cell anemia patient 'cured' by gene therapy, doctors say
FOX31 Denver
Gene therapy holds promise because a patient serves as his own donor, and the risks are much reduced since there's no possibility of a mismatch, said Thompson, who was not involved in this research but is an investigator on a related gene therapy
Gene therapy shows early promise against sickle cellChicago Tribune
Doctors Claim They've Cured a Boy of a Painful Blood Disorder Using Gene TherapyFuturism
Teenager's sickle cell reversed with world-first therapyBBC News
BioNews –Fox News –New England Journal of Medicine
all 79 news articles »

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Sickle cell anemia patient ‘cured’ by gene therapy, doctors say – FOX31 Denver

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USMLE Step 1: Postpartum amenorrhea and hormone levels – American Medical Association (blog)

If youre preparing for the United States Medical Licensing Examination (USMLE) Step 1 exam, you might want to know which questions are most often missed by test takers. Check out this example from Kaplan Medical, and view an expert video explanation of the answer. Also check out all posts in this series.

A 32-year-old woman comes to the physician because of amenorrhea for the past 15 months after delivering a baby. She says that she has also had fatigue, facial swelling, cold intolerance and has gained an additional 4.5 kg (10 pounds) since her baby was born. A review of her records shows that the delivery was complicated by severe hemorrhage. Laboratory studies of serum show:

Injection of 500 g of thyrotropin-releasing hormone fails to produce an increase in either serum TSH or prolactin. Assay of other hormones is most likely to show normal levels of which of the following hormones?

A. Aldosterone

B. Cortisol

C. Follicle-stimulating hormone (FSH)

D. Gonadotropin-releasing hormone (GnRH)

E. Growth hormone

The correct answer is A.

Sheehan syndrome is hypopituitarism caused by ischemic damage to the pituitary resulting from excessive hemorrhage during parturition. The pituitary is enlarged during pregnancy; it is more metabolically active and more susceptible to hypoxemia. The blood vessels in the pituitary may be more susceptible to vasospasm because of high estrogen levels.

In about 30 percent of women who have excessive hemorrhage during parturition, some degree of hypopituitarism eventually manifests. The symptoms depend on how much of the pituitary is damaged and what cell types are destroyed. Although some pituitary hormones may be unaffected even in severe hypopituitarism, pituitary hormones and the hormones controlled by them are more likely to be reduced than hormones that are not primarily controlled by anterior pituitary function.

Our patient has amenorrhea (decreased LH) and symptoms of hypothryoidism (decreased TSH). Aldosterone secretion is relatively independent of adrenocorticotropic hormone (ACTH); it is controlled mainly by angiotensin II and plasma potassium concentration. Aldosterone is least likely to be reduced by hypopituitarism. Treatment is replacement of thyroid hormone and cortisol.

Read these explanations to understand the important rationale for why each answer is incorrect.

Choice B: Cortisol is controlled by pituitary production of ACTH; because ACTH is often impaired in Sheehan syndrome, reduced secretion of cortisol is likely.

Choice C: The pituitary necrosis that is the root cause of Sheehan syndrome is highly likely to reduce secretion of FSH. The observation of reduced estradiol in this patient strongly suggests that FSH is low because estradiol increases as follicular development occurs.

Choice D: The presence of the depressed levels of estradiol and LH in this patient releases hypothalamic secretion of GnRH from its normal feedback control. GnRH levels are likely to increase above normal.

Choice E: Growth hormone is very likely to be reduced by the pituitary necrosis.

For more prep questions on USMLE Steps 1 and 2, view other posts in this series.

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USMLE Step 1: Postpartum amenorrhea and hormone levels – American Medical Association (blog)

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Doc: Pill’s hormone regulation benefits women with PCOS – The Detroit News

Keith Roach, To Your Health 5:40 p.m. ET March 2, 2017

Dear Dr. Roach: My daughter has just been diagnosed with polycystic ovary syndrome. She is in her mid-20s, exercises regularly and watches what she eats, but still puts on weight. She does not want to go on birth control. Where do we start? I know there is no cure.


Dear B.M.: Polycystic ovary syndrome is very prevalent, affecting 6 to 8 percent of women, but it is variable in terms of both the types of symptoms and their severity. The most common symptoms are menstrual irregularities and consequences of high male hormones (such as excess body hair and acne). Being overweight or experiencing weight gain is important, as is the metabolic risk from diabetes and abnormal cholesterol levels. Lesser-known symptoms that are seen regularly in women with PCOS include depression, anxiety and eating disorders. The polycystic appearance of the ovaries themselves is demonstrated in women with PCOS, but also can be seen in normal women and therefore is not needed for diagnosis. Diagnosis is made after a careful history and physical exam, and by laboratory testing ( also necessary to exclude some other causes.)

Treatment of PCOS is intended to reduce symptoms, to reduce the risk of heart disease and diabetes, to manage fertility (including contraception, if needed, and helping women get pregnant, if desired) and to reduce risk of abnormal growth of the lining of the uterus (the endometrium) associated with abnormal hormone levels. The primary treatment is with lifestyle changes, so I agree with getting regular exercise and a prudent diet. If these are inadequate, it is reasonable to consider medication treatment.

For women with symptoms of high androgen (male hormone) levels, birth-control pills are the most prescribed treatment, and your daughters doctors should discuss your daughters concerns about taking them. They provide many benefits in women with PCOS, especially by reducing male hormone effects (acne, excess body hair) and reducing risk of abnormal endometrial growth (and possibly cancer). Of course, they have side effects, including risk of blood clots.

For women who cannot take birth control pills, metformin, which reduces insulin levels, has some benefits. It helps with weight gain and menstrual irregularities, and it probably reduces diabetes risk. It does not help with the body hair.

PCOS is a large topic that I cant cover fully. I recommend starting with the information on the American Congress of Obstetricians and Gynecologists FAQ page at

Dear Dr. Roach: My primary-care physician and gynecologist are recommending I take Fosamax. What is the alternative to taking Fosamax?


Dear K.J.: If there are no other treatable causes, such as low vitamin D or high parathyroid hormone levels, then I think Fosamax or another bisphosphonate is a good choice. There are others, but Fosamax has been well-studied.

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Doc: Pill’s hormone regulation benefits women with PCOS – The Detroit News

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Area doctor to launch book on women’s health during menopause – Southgate News Herald

For decades, doctors have assumed that symptoms of menopause, such as hot flashes, sweating and reduced sex drive, are caused by a reduction in the amount of estrogen that the body is producing. Dr. Charles Mok, a former emergency room physician who is board certified in Anti-Aging Medicine and the founder of Allure Medical Spa and Allure Vein Center of Southgate, has evidence that doctors have focused too much on estrogen for menopause and science has found low testosterone levels are also responsible for many symptoms. In his new book Testosterone: Strong Enough for a Man, Made for a Woman (Forbes Hardcover $25.99, ISBN 978-0998365503), Dr. Mok provides peer-reviewed studies which support this view, translating scientific literature into an easy to understand manual for hormone replacement therapy. The book launch is scheduled for March 2 at a Macomb County Allure Medical Spa location.

The evidence for testosterone therapy is overwhelming, and we want to get the message to doctors and, importantly, to their patients, says Dr. Mok. Clinical research shows that testosterone reduces the risk of breast cancer by 50 to 75 percent, which has enormous health implications, and natural estrogen cuts the risk of heart attacks by over 70 percent if used long term. For women suffering from debilitating symptoms such as mood swings, anxiety, hot flashes and reduced libido, the treatment can really improve overall quality of life.

Dr. Moks book takes readers on a journey that chronicles the start of hormone replacement therapy in the 1940s through to the flawed 2002 Womens Health Initiative which suggested women taking a combination of synthetic estrogen drugs and synthetic progesterone had an increased risk of heart disease and breast cancer, which ultimately dissuaded millions of women from seeking hormonal help. It is important to note that further research into the Womens Health Initiative data showed that when women took even the synthetic hormones around the time of menopause and stayed on them, there was a 40 percent reduction in their risk heart attacks and premature mortality.

Dr. Moks book provides compelling evidence about the important role of testosterone in shaping womens health. The hormone is 10-20 times more abundant than estrogen in young women, but then falls as women age. Dr. Mok provides myriad long term studies showing that modern testosterone therapy (normally delivered in the form of a tiny pellet, about the size of a grain of rice, inserted underneath the skin) keeps women healthier, with no adverse side-effects.

Benefits can include weight loss, better sex life, improved mood, fewer sleep problems, reduced hot flashes and night sweats, thicker hair, lower risk of breast cancer and lower risk of heart disease.

Dr. Mok became interested in preventive medicine while working as an emergency room doctor, recognizing that many of his patients conditions could have been avoided. His medical career has now taken him on a path to help patients live their best lives, and for many this approach has been life-changing.

Dr. Mok will launch his book with a lecture, followed by a party, at 6 p.m. March 2 at Allure Medical Spa, 8180 26 Mile Road, Shelby Township.

The event is free to the public and all attendees will receive a copy of the book. RSVP by calling 586-992-8300 or emailing

Source: CKC Agency

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Area doctor to launch book on women’s health during menopause – Southgate News Herald

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Obesity May Raise Girls’ Risk of Asthma, Allergies – WebMD

By Alan Mozes

HealthDay Reporter

MONDAY, March 6, 2017 (HealthDay News) — Obese girls may face a significantly higher risk for developing allergies, a new study suggests.

But the researchers found the opposite was true for obese boys: They may actually face a slightly diminished risk for asthma, food allergies and eczema when compared to normal-weight boys.

“We found a direct increase in the number of atopic [allergic] diseases associated with obesity in urban female children and teenagers, but not in males,” said study co-author Dr. Sairaman Nagarajan. He’s a resident physician in the department of pediatrics at SUNY Downstate Medical Center in New York City.

“These results were highly significant, even after adjusting for the effects of age and race,” he said.

Nagarajan and his colleagues were scheduled to present their findings Monday at the annual meeting of the American Academy of Allergy, Asthma & Immunology (AAAAI), in Atlanta.

Their investigation focused on 113 children (45 percent girls, 55 percent boys), about a quarter of whom were obese.

All the children lived in Brooklyn, N.Y., and were on average between the ages of 8 and 9. Allergies aside, all were deemed to be relatively healthy.

Medical histories were taken to assess for a range of allergic conditions, including asthma, food allergies, hay fever and/or eczema. The children were then given allergy scores, with those struggling with more allergic conditions getting higher scores.

The researchers found that obese girls had allergy scores higher than normal-weight girls: 4 vs. 2.6.

In contrast, obese boys were found to have slightly lower allergy scores than normal-weight boys: 3 vs. 3.4.

The upshot, said Nagarajan, is the possibility “that lifestyle modification therapies and exercise and diet programs may be specifically beneficial to urban obese girls.”

But why?

“We hypothesize that there are hormonal differences causing girls to have higher atopy [allergies],” said Nagarajan.

For example, he pointed to the possibility that higher adrenal sex hormone levels found among girls may predispose them to a higher risk for both becoming obese and also for having a stronger overall inflammatory response.

This, Nagarajan said, may “[cause] them to react to things non-obese females wouldn’t have.”

Still, the study wasn’t designed to prove that obesity caused the allergies, and the research team acknowledged that more study is needed.

That point was echoed by Dr. James Baker Jr., CEO and chief medical officer Food Allergy Research & Education (FARE), an allergy information organization.

“These appear to be preliminary findings,” Baker said, “and really need to be validated in a larger, prospective study to understand their significance.”

Dr. Jay Lieberman, an assistant professor of pediatrics with the University of Tennessee Health Science Center, and LeBonheur Children’s Hospital in Memphis, agreed.

“Overall, I am not too surprised by these findings,” he said, adding that many studies have suggested “that the obesity effect on allergic diseases may be more pronounced in females rather than males.”

“There are many theories as to why,” Lieberman added. “The main theory is the role that hormones — estrogen, estradiol, progesterone, etcetera — may play [a role] in driving allergies, and that hormonal levels may be imbalanced in obese patients. And, thus, females with obesity are more prone to allergies than obese males, who do not produce these hormones at levels that females do.”

Still, Lieberman cautioned that “one must take into account that this was a retrospective study on a relatively small sample of children.” Thus, the results must be taken with a grain of salt, meaning the findings could very well be due to chance, he said.

Research presented at meetings should be considered preliminary until published in a peer-reviewed journal.

WebMD News from HealthDay

SOURCES: Sairaman Nagarajan, M.D., MPH, resident physician, department of pediatrics, SUNY Downstate Medical Center, New York City; Jay Lieberman, M.D., assistant professor, pediatrics, University of Tennessee Health Science Center and LeBonheur Children’s Hospital, Memphis; James Baker Jr., M.D., CEO and chief medical officer, Food Allergy Research & Education; March 6, 2017, presentation, American Academy of Allergy, Asthma & Immunology meeting, Atlanta

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Introducing Bio-Identical Hormone Replacement Therapy at Young Medical Spa in Center Valley and Lansdale … – PR Web (press release)

Montgomery County and the Lehigh Valley, PA (PRWEB) February 28, 2017

Dr. Thomas E. Young, Owner and Medical Director of Young Medical Spa, is excited to announce the addition of a new service: Bio-Identical Hormone Replacement Therapy for restoring hormonal balance and improving overall health and vitality.

Suitable for both men and women, Bio-Identical Hormone Replacement Therapy may help correct hormonal imbalances that can result from aging, relieving such symptoms as depression, fatigue, migraines, low libido, urinary incontinence, lack of mental clarity, trouble sleeping, and more.

A customizable treatment that is tailored to each patients exact needs, Bio-Identical Hormone Replacement Therapy at Young Medical Spa involves inserting tiny pellets beneath the skin that gradually release a steady stream of natural hormones over time. Patients can receive therapy for low testosterone, estrogen, or a combination of both hormones, as needed.

In concert with Bio-Identical Hormone Replacement Therapy, Young Medical Spa clients have access to a complete line of nutraceuticals that promote thyroid, heart, liver, and brain health. The combination of nutraceuticals and Bio-Identical Hormone Replacement Therapy provides a natural approach for long-lasting results without the potential negative side effects of prescription medications and traditional synthetic hormone replacement therapy methods.

Young Medical Spa offers Bio-Identical Hormone Replacement Therapy at both of its locations in Lansdale and Center Valley, convenient to patients in the greater Philadelphia and Lehigh Valley areas. Please visit to learn more about these treatments at Young Medical Spa. Center Valley patients can also call 610-816-0077; Lansdale residents can learn more about this new service by calling 215-660-4121.

About Young Medical Spa in Center Valley and Lansdale Pennsylvania

Dr. Thomas E. Young founded Young Medical Spa with the mission to provide the highest quality aesthetic treatments and services within the comfort of a relaxing, spa-like environment. At Young Medical Spa in the Lehigh Valley region, all patients are treated under the supervision of medical aesthetic experts to ensure that results match the teams passion for aesthetic medicine.

Dr. Young is a leader within the medical aesthetic industry, and shares his expertise by training other physicians with his advanced techniques and wealth of experience. At Young Medical Spa, Dr. Young works diligently to ensure that each patient is able to achieve their desired results within a state-of-the-art location and in the company of a friendly and welcoming staff.

Services offered include Bio-identical hormone replacement therapy (BHRT), hand rejuvenation, intimate rejuvenation with ThermiVa, P-Shot, and O-Shot. Patients can undergo facelifts, neck lifts, eyelid surgery, thread lifts, Kybella, and PrecisionTX for facial contouring. Body sculpting and shaping treatments include SmartLipo Triplex, CoolSculpting, SculpSure, Brazilian butt lift, natural breast augmentation, cellulite reduction, and the Young Diet Program. Botox, Dysport, and dermal fillers (Juvderm, Restylane, Radiesse, Belotero Balance, and Voluma are administered with clinical and aesthetic expertise. Young Medical Spa is a Brilliant Distinctions participant and a Cynosure Center of Excellence. Additional services include Laser Hair Removal and skin rejuvenation (IPL photofacials, microneedling, skin tightening, laser skin resurfacing, stretch marks & scar revision, laser leg vein therapy, chemical peels, microdermabrasion, and clinical facials. Clients can also purchase from a suite of professional medical-grade skin care products.

Young Medical Spa has two locations serving the Lehigh Valley and greater Philadelphia areas. Their Center Valley medical spa is convenient to Allentown, Bethlehem, and Easton. The Lansdale medical spa is located in Montgomery County outside of Philadelphia, serving King of Prussia and Lower Bucks County. Every patient is given the time and attention necessary to develop a unique treatment plan and foster a valuable relationship with the staff. Young Medical Spa is dedicated to patient care and comfort, maximizing results while ensuring the utmost safety.

Young Medical Spa is conveniently located at 4025 West Hopewell Road in Center Valley, PA, and at 635 North Broad Street, Lansdale, PA.

About Dr. Thomas Young Thomas E. Young, M.D. is the owner and medical director of Young Medical Spa located in the Lehigh Valley and Lansdale, PA. Double board-certified by the American Board of Internal Medicine, he is a native of the Lehigh Valley, and has been practicing medicine in the Lehigh Valley and surrounding areas since completing his residency at the Harrisburg Hospital. He is double-board certified by the American Board of Internal Medicine in Internal Medicine and Geriatrics.

Dr. Young is an experienced cosmetic injector and specializes in multiple awake tumescent procedures and techniques, and began performing SmartLipo Laser Body Sculpting soon after approval by the FDA. Dr. Young has performed well over 6,000 procedures to date, identifying him as the most experienced awake tumescent liposuction physician in the region.

Dr. Young is consistently the first physician to introduce many advanced aesthetic procedures and technologies to the region including awake tumescent laser liposuction, natural breast augmentation, Brazilian butt lift, Cellulaze cellulite reduction, CoolSculpting, SculpSure and stem cell procedures. He also trains other physicians across the country in awake tumescent liposuction & liposculpture procedures.

Dr. Young received an International Award in the category of Best Overall Body Make-Over (a combination of procedures) at the inaugural THE Aesthetic Awards at THE Aesthetic Show in Las Vegas, NV. He also has been voted Leading Aesthetic Physician Botox, Laser & Cosmetic Enhancements in the 2011, 2012, 2013, 2014, 2015, and 2017 Whos Who in Business for the Lehigh Valley, and was twice voted Best Aesthetic Physician by Lehigh Valley Magazines Best of the Valley Readers Poll. Additionally, Young Medical Spa was voted Best Medical Spa in The Morning Calls Readers Choice Awards in 2011, 2012, 2013, 2014 and 2015.

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Introducing Bio-Identical Hormone Replacement Therapy at Young Medical Spa in Center Valley and Lansdale … – PR Web (press release)

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Is testosterone-replacement therapy good or bad? One man suffering from low levels searches for answers. – Men’s Fitness

Men’s Fitness
Is testosterone-replacement therapy good or bad? One man suffering from low levels searches for answers.
Men’s Fitness
But, leading experts agree, first you need to visit a urologist or an endocrinologistspecialists more adept at diagnosing and treating hormone deficiencies than the average family doctor or low-T clinicto have your blood-testosterone level checked.

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Is testosterone-replacement therapy good or bad? One man suffering from low levels searches for answers. – Men’s Fitness

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Hormone Therapy Clinic | Human Growth Hormone …

Hormone Replacement Therapy

Hormones are a key part of the way your body functions.They are the chemical messengers secreted by the many glands of the endocrine system. Some of the most important hormones are: testosterone, estrogen, progesterone, human growth hormone, estrogen, and the thyroid hormones. The production and secretion of these crucial hormones all decrease over time, leading to the difficulties and conditions we normally relate to ageing. Hormone Replacement Therapies are all about renewing vitality by giving you back what age takes away. Read More

Testosterone is one of the most important hormones for men. Many of the conditions that you think are just normal signs of aging slower metabolism, gaining weight, feeling tired, feeling moody, and memory issues may actually be due to low testosterone. In addition, while many factors other than age from diet to stress, to sleep can all affect your sex drive or libido, the drop in testosterone that occurs as men age, is often the cause of sexual wellness issues. Read More

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Doctor Allie International Hormone Success Doctor Joins the … – Satellite PR News (press release)

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Phoenix, AZ (SBWIRE) 03/06/2017 Many women find their lives deeply affected by symptoms of stress and hormonal imbalance, such as tiredness, mood swings, depression, lack of focus, and weight gain. Their productivity at work and relationships at home also suffer. Doctor Allie suffered adrenal fatigue, underactive thyroid, and of course perimenopause herself so she knows where many women are coming from! She can help women of all ages to break free from their hormone prison & live life with vitality again through her functional medicine & bio-identical hormone clinic in Hove, United Kingdom or online.

Dr. Allison Grimston graduated as a Medical Doctor from Charing Cross and Westminster Medical School (now Imperial College School of Medicine) in 1994. She was awarded honors in Gynecology. She obtained a BSc in Pharmacology as Applied to Medicine and an MPhil at St Marys Hospital in Paddington. She joined a General Practice in Hailsham in 1999 as a full-time GP where she worked until June 2016. For a year, she was also a Governing Body member of the Eastbourne, Hailsham and Seaford Clinical Commissioning Group which is a GP-led membership organization responsible for commissioning most of the health services for local people. Doctor Allie empowers women through her functional medicine & bio-identical hormone clinic in Hove, UK and online.

In 2014, Doctor Allie Grimston launched her coaching and mentoring programs to help patients and other coaches achieve Hormone Success through a unique, combined approach that considers medical application of Bio Identical Hormone Replacement Therapy and effective lifestyle management. She is an accomplished professional speaker and her online workshops reach people in the UK and countries as far apart as New Zealand, Australia, USA and Canada. Doctor Allies popular workshops focus on adrenal fatigue, stress and burnout, thyroid balance, and menopause.

Doctor Allie will join The Womb Happy Hour on VoiceAmerica Health and Wellness channel on March 8, 2017 at 3PM Pacific / 6 PM EST. Archives of The Womb Happy Hour can be found on Lorraine Giordano is the founder of Inspired To Health She is dedicated to helping women live healthful lives.

For more information about Dr. Alison Grimston and to access her valuable hormone resources or have her speak at an event contact Dr. Alison at

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Planned Parenthood New York Now Offers Transgender Hormone Replacement Therapy – The Mary Sue

Planned Parenthood, ever the bastion of healthcare for many people throughout the United States, has improved their care with the integration of transgender hormone replacement therapy services. According to the programs new website, theyre offering,hormone therapy for transgender and gender nonconforming patients. They go on to also say, We also offer the full range of sexual and reproductive health care to people of all genders and identities.

The introduction of the program in New York actually began back in November, according to an e-mail from Planned Parenthood New Yorks Director of Digital and Media Relations, Carrie Mumah. With the addition of Planned Parenthood New York, hormone replacement therapy is now being offered in 16 different states in the U.S..

The move comes at a pretty heated time when transgender equality and rights are under severe fire from the federal government. Just last week, the Justice and Education departments moved to rescind an Obama-era guideline protecting transgender students from discrimination based on their gender identity. Although this was an expected move, it nonetheless causedwaves throughout those communities fighting for equality for all LGBTQIA identities.

As well, Planned Parenthood has seen better days, as theyve consistently faced legislation that threatens to defund and otherwise bankrupt the healthcare organization. As part of the bill pushed forward to gut the Affordable Care Act (aka Obamacare), the funding Planned Parenthood receives from the federal government will also be pulled. But through various charity drives and organized donations since the election, Planned Parenthood hasseen an unprecedented40-fold increase in donations(as of December, at least).

Of course, that can only last for so long. The misinformation campaign that exists around Planned Parenthoods very existence is still going strong, and thus, the misconception that they only exist to offer abortions continues to run rampant. Its unfortunate, and bears a resemblance to the ACA/Obamacare mix-up debacle that has many Trump supporters reconsidering just what the hell theyve gotten us all into.

The move to ensure trans people get the hormones and healthcare they need is a sorely needed one, and its yet another reason as to why organizations like Planned Parenthood deserve our support.

(via Uproxx, image via Shutterstock)

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Isoflavones in food associated with reduced mortality for women with some breast cancers – Tufts Now

BOSTON (March 6, 2017)An epidemiological analysis of data from more than 6,000 American and Canadian women with breast cancer finds that post-diagnosis consumption of foods containing isoflavonesestrogen-like compounds primarily found in soy foodis associated with a 21 percent decrease in all-cause mortality. This decrease was seen only in women with hormone-receptor-negative tumors, and in women who were not treated with endocrine therapy such as tamoxifen.

The study, led by nutrition and cancer epidemiologist Fang Fang Zhang, M.D., Ph.D., from the Friedman School of Nutrition Science and Policy at Tufts University, was published March 6 in Cancer.

At the population level, we see an association between isoflavone consumption and reduced risk of death in certain groups of women with breast cancer. Our results suggest, in specific circumstances, there may be a potential benefit to eating more soy foods as part of an overall healthy diet and lifestyle, said Zhang, who is also the 2016-2017 Miriam E. Nelson Tisch Faculty Fellow at the Jonathan M. Tisch College of Civic Life and an adjunct scientist in nutritional epidemiology at the Jean Mayer USDA Human Nutrition Research Center on Aging at Tufts.

Since we only examined naturally occurring dietary isoflavone, we do not know the effect of isoflavone from supplements. We recommend that readers keep in mind that soy foods can potentially have an impact, but only as a component of an overall healthy diet, she adds.

Isoflavones have been shown to slow the growth of breast cancer cells in laboratory studies, and epidemiological analyses in East Asian women with breast cancer found links between higher isoflavone intake and reduced mortality. However, other research has suggested that the estrogen-like effects of isoflavones may reduce the effectiveness of endocrine therapies used to treat breast cancer. Because of this double effect, it remains unknown whether isoflavone consumption should be encouraged or avoided by breast cancer patients.

In the current study, Zhang and her colleagues, including Esther John, Ph.D., senior cancer epidemiologist at the Cancer Prevention Institute of California, analyzed data on 6,235 American and Canadian breast cancer patients from the Breast Cancer Family Registry, a National Cancer Institute-funded program that has collected clinical and questionnaire data on enrolled participants and their families since 1995. Women were sorted into four quartile groups based on the amount of isoflavone they were estimated to have consumed, calculated from self-reported food frequency questionnaires. Mortality was examined after a median follow-up of 9.4 years.

The team found a 21 percent decrease in all-cause mortality among women in the highest quartile of intake, when compared to those in the lowest quartile. The association between isoflavone intake and reduced mortality was strongest in women with tumors that lacked estrogen and progesterone receptors. Women who did not receive endocrine therapy as a treatment for their breast cancer had a weaker, but still significant association. No associations were found for women with hormone-receptor-positive tumors and for women who received endocrine therapy.

While the study categorized women in the highest quartile as those who consumed 1.5 milligrams or more of isoflavone per dayequivalent to a few dried soybeansthe authors caution that individuals tend to underestimate their food intake when filling out questionnaires.

The comparisons between high and low consumption in our study are valid, but our findings should not be interpreted as a prescription, Zhang said. However, based on our results, we do not see a detrimental effect of soy intake among women who were treated with endocrine therapy, which has been hypothesized to be a concern. Especially for women with hormone-receptor-negative breast cancer, soy food products may potentially have a beneficial effect and increase survival.

The large size and diverse racial/ethnic makeup of the Breast Cancer Family Registry allowed the researchers to evaluate mortality risk across different subtypes of breast cancer and subgroups of patients, and adjust for confounding factors. However, the authors note that dietary isoflavone intake was correlated with socioeconomic and lifestyle factors, which may also play a role in lowering mortality. In particular, women who consumed higher levels of dietary isoflavone were more likely to be Asian Americans, young, physically active, more educated, not overweight, never smokers, and drink no alcohol. Although the team controlled for these factors in the analyses, the possibility of a partial confounding effect on the associations identified in the study cannot be ruled out.

Whether lifestyle factors can improve survival after diagnosis is an important question for women diagnosed with hormone-receptor negative breast cancer, a more aggressive type of breast cancer. Our findings suggest that survival may be better in patients with a higher consumption of isoflavones from soy food, John said.

Additional authors on this study are Danielle E. Haslam, nutritional epidemiology doctoral candidate at the Friedman School of Nutrition Science and Policy at Tufts University, Mary Beth Terry, Ph.D., professor of epidemiology at Mailman School of Public Health at Columbia University, Julia A. Knight, Ph.D., professor of epidemiology at the Dalla Lana School of Public Health at the University of Toronto and senior investigator at the Lunenfeld-Tanenbaum Research Institute, Sinai Health System in Toronto, Irene L. Andrulis, Ph.D., professor of molecular genetics at the Dalla Lana School of Public Health at the University of Toronto and senior investigator at the Lunenfeld-Tanenbaum Research Institute, Sinai Health System in Toronto, Mary Daly, M.D., Ph.D., chair and professor in the department of clinical genetics and director of risk assessment program at the Fox Chase Cancer Center in Philadelphia, Saundra S. Buys, M.D., medical director of Huntsman Cancer Institute’s high risk breast cancer clinic and a professor in the department of medicine at the University of Utah School of Medicine.

This work was supported by an award from National Cancer Institute of the National Institutes of Health (CA164920).

Zhang, F. F., Haslam, D. E., Terry, M. B., Knight, J.A., Andrulis, I. L., Daly, M., Buys, S.S., and John, E. M. (2017). Dietary isoflavone intake and all-cause mortality in breast cancer survivors: the Breast Cancer Family Registry. Cancer. Published online: March 6, 2017. DOI: 10.1002/cncr.30615.

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About the Friedman School of Nutrition Science and Policy at Tufts University

The Gerald J. and Dorothy R. Friedman School of Nutrition Science and Policy at Tufts University is the only independent school of nutrition in the United States. The schools eight degree programs which focus on questions relating to nutrition and chronic diseases, molecular nutrition, agriculture and sustainability, food security, humanitarian assistance, public health nutrition, and food policy and economics are renowned for the application of scientific research to national and international policy.


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Using Stem Cells to Predict Toxicity of Chemotherapy Drugs … – (blog)

A team of scientists has developed a new safety index for a common group of chemotherapy drugs, by using a stem cell model to screen such therapies for their potential to damage patients hearts.

The study, published in Science Translational Medicine, was co-authored by Paul Burridge, PhD, assistant professor of Pharmacology.

Tyrosine kinase inhibitors (TKIs), a class of chemotherapy drugs, have become increasingly important in treating many types of cancer. But almost all TKIs are also associated with cardiovascular side effects ranging from arrhythmias to heart failure and there has not yet been an effective tool to predict this cardiotoxicity.

In the current study, the scientists demonstrated that human-induced pluripotent stem cells can be used to model how TKIs might affect the hearts of patients receiving chemotherapy.

To do so, the scientists took stem cells from both a control group and patients with cancer and reprogrammed them to become cardiomyocytes, or heart muscle cells. Using high-throughput screening, they then evaluated how the heart cells responded to treatment with 21 different FDA-approved TKIs, looking at factors like cell survival, signaling and alterations in their ability to beat properly.

With the stem-cell data, the scientists were able to create a cardiac safety index, which ranks the TKIs on their likelihood of inflicting heart damage. That index correlates with the toxicity that has been observed in patients clinically a validation that suggests the screening system might be a powerful tool in predicting toxicity before therapies are ever administered to patients.

Future research could establish even more specific predictions, by comparing the genomes of patients who might experience a certain drug side effect, such as atherosclerosis, with those who dont. Long-term, what my lab is interested in is taking a patients whole genome and, based on the work weve done in the past, being able to predict whether a patient will have an adverse drug event, said Burridge, also a member of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University. This is the whole idea of pharmacogenomics, or precision medicine: Everyone is going to have a different response to a drug, and that response good or bad is already encoded in all of us.

In the study, the scientists also discovered that administering insulin or insulin-like growth factor 1 alongside TKIs seemed to protect against some of the heart damage associated with the drugs. While its still early, this is the first step toward opening up a whole new field of identifying cardioprotectants to reduce the toxicity of these drugs, Burridge said.

The research was supported by the National Institutes of Health (NIH) grants K99/R00 HL121177, 14BGIA20480329, R01 HL132875, R01 HL130020, R01 HL128170, R01 HL123968, and R24 HL117756; the NIH Directors Pioneer Award; the American Heart Association Predoctoral Fellowship; the American Heart Association Beginning Grant-in-Aid; American Heart Association Grant-in-Aid; the American Heart Association Established Investigator Award; the National Science Foundation Graduate Research Fellowship; the Endowed Faculty Scholar Award of the Lucile Packard Foundation for Children and Child Health Research Institute at Stanford and Burroughs Wellcome Foundation Innovation in Regulatory Science.

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Student profile: Keegan Mendez – Harvard School of Engineering and Applied Sciences

This sky-diving, squash-playing, thrill-seeking student is gearing up for her next great adventurein biomedical engineering research and discovery.

Why did you decide to concentrate in biomedical engineering?

As a child, I always had a love of math and science, and I liked to use my hands to create thingsI shunned Barbie dolls for building blocks. I was already a math and science nerd, but what appealed to me about bioengineering specifically is the breadth and diversity of research options, from organs on a chip to medical device design, and everything in between. The research that is happening right nowlike trying to grow a human heart outside the bodyis so cutting-edge and exciting.

Tell us about some of the bioengineering research youve had the opportunity to conduct at SEAS.

During my sophomore year, I began working in David Mooneys lab on developing the TheraCardium, which is a cardiac device for stem cell delivery to the heart for patients who have suffered a heart attack. The device supports regrowth of the damaged tissue and helps to prevent scarring of the dead heart muscle, in an effort to help prevent future cardiac events.

Mendez works on a biomedical research project in the Mooney lab. (Photo by Eliza Grinnell/SEAS Communications.)

Why was that research experience beneficial for you?

By working on that project, I experienced many different types of research, from preclinical studies in animals, to tissue engineering, to the materials science involved in building the device, to various soft robotic manufacturing techniques. I had the opportunity to work with many new technologies that I hadnt been exposed to in the classroom.

What is the topic of your senior thesis project?

Drawing on my work on the TheraCardium, I am designing a soft robotic drug delivery system. The device involves a hydrogel adhered to a soft robotic balloon that could be placed on the surface of the heart to directly delivery therapy to the muscle. Inflation of the balloon stretches the mesh size of the hydrogel, enabling delivery of the drug encapsulated within the hydrogel. By controlling the balloon inflation, we can achieve radio control, or the ability for on-and-off delivery. The device could also incorporate multiple balloons, delivering different drugs to separate areas of the heart.

In addition to your academic and research success, youve also served as co-captain of the Harvard Womens Squash team. How did you get involved with that sport?

I started playing squash competitively when I was 8 years old. The neighborhood where I grew up had one of the best junior squash programs in the country. I was inspired by my older sister, Haley, who is a great squash player. She was recruited to play squash at Harvard. When it came time for me to apply to college, the coach told me he had used all his recruiting spots, but if I could get into Harvard, I could play, too. It all worked out, and Ive been on the team for the past four years. There is a big mental aspect to squash. Your tactics and shot selection become critically important at the college level, since all the players are very technically proficient.

Are you and your older sister squash rivals?

Were a very competitive family. When we play board games, it gets so competitive it is almost scary. Haley has always been better than I was on the squash court, but we still play all the time. We are definitely competitive academically, as well, and while I love squash, I feel like my true passion lies in academics.

When playing squash at the college level, tactics and shot selection become incredibly important, Mendez said. (Photo by Eliza Grinnell/SEAS Communications.)

Do you think academics will play a role in your future plans?

Definitely. I am planning to apply for Ph.D. programs in bioengineering, and Harvard is my first choice. Ive been really excited about the research Ive been able to do as an undergraduate, and I want to continue contributing to science and advancing the field. The projects Ive been working on are just so cool, and I want to keep my research momentum going.

How do you feel that SEAS has prepared you for your future?

Beyond learning the technical skillslike how to code and use machinesbeing able to work closely with my peers on teams has given me a lot of confidence. As an engineer, you need to be able to communicate ideas effectively to people who may not be engineers. Collaboration is key within engineering, with each team member contributing an important piece to the puzzle. I have also been humbled, and learned when to ask for help, when to seek out peers, and when to work collaboratively in groups as opposed to attempting to do everything myself.

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Researchers report positive bone marrow transplant case | The Herald – The Herald

People with a certain gene have an adverse reaction to the antiretroviral efavirenz

Liz HighleymanCorrespondent An HIV-positive bone marrow transplant recipient at the Mayo Clinic experienced prolonged viral remission lasting nearly 10 months longer than the so-called Boston patients after interrupting antiretroviral therapy (ART), according to a report at the Conference on Retroviruses and Opportunistic Infections (CROI 2017) last month in Seattle.

Although his viral load eventually rebounded, his HIV reservoirs appeared to be reduced.

The only person known to be cured of HIV Timothy Ray Brown, known as the Berlin Patient stopped ART when he received a bone marrow transplant to treat leukaemia and has not had detectable virus for 10 years. Brown received a transplant from a donor with a double CCR5-delta-32 mutation, meaning they lack the CCR5 co-receptors most types of HIV use to enter T-cells.

It is unclear whether his sustained remission is attributable to the donors CCR5 mutation, the strong chemotherapy conditioning regimen used to kill off cancerous blood cells, a graft-versus-host reaction or multiple factors.

Bone marrow transplantation is apparently not sufficient to eradicate HIV.

A few years ago, Timothy Henrich reported on two HIV-positive bone marrow transplant patients in Boston who got stem cells from wild-type donors without the CCR5-delta-32 mutation, received a milder conditioning regimen and experienced acute graft-versus-host disease (GVHD). Both men maintained undetectable viral load longer than expected after interrupting ART, but eventually they experienced viral rebound at three and eight months after stopping HIV treatment.

The latest case, presented by Nathan Cummins of the Mayo Clinic in Rochester, Minnesota, and colleagues, involved a 55-year-old man who was diagnosed with HIV in 1990 and started combination ART in 1999 with a CD4 T-cell count of 300 cells/mm3. He stopped treatment between 2004 and 2009 for unexplained reasons, then restarted ART consisting of ritonavir-boosted atazanavir (Prezista) plus tenofovir disoproxil fumarate (DF) and emtricitabine (the drugs in Truvada).

In April 2013 the man was diagnosed with B-cell acute lymphoblastic leukaemia.

In anticipation of chemotherapy, his ART regimen was switched to raltegravir (Isentress), etravirine (Intelence), and tenofovir DF/emtricitabine. In October 2013 he underwent reduced intensity conditioning followed by an allogeneic stem cell transplant from a CCR5 wild-type donor.

At the time of transplantation the man had an HIV viral load of 25 copies/ml and a CD4 count of 288 cells/mm3, and he stayed on ART without interruption. After the transplant he developed opportunistic infections (E. coli septicaemia and pneumocystis pneumonia) and experienced GVHD at four months post-transplant.

The man continued on ART for more than two years after transplantation, mostly with detectable plasma viral load levels. HIV RNA was also undetectable in gut biopsy samples. HIV DNA in his peripheral blood cells became undetectable by day 56, and repeated leukapheresis procedures showed significant reductions in HIV RNA and DNA reservoir size.

In addition, that mans HIV antibody levels decreased, as indicated by weaker Western blot bands. However, single genome sequencing and phylogenetic analysis identified identical HIV clones at day 142, possibly due to homoeostatic proliferation, or replication of latently infected cells, while he had GVHD.

After having such low HIV levels for a prolonged period, the man underwent an analytic treatment interruption, or carefully monitored discontinuation of ART. His plasma HIV RNA levels were tested every two weeks for the first 12 weeks of ART interruption, then every four weeks.

At day 288 9,6 months after stopping ART he was found to have low-level viral rebound to 60 copies/ml. This rose to 1640 copies/ml by day 293, requiring that he restart HIV treatment.

The man had no evidence of drug resistance and his viral load was re-suppressed within a month.

Allogeneic peripheral blood stem cell transplantation in the setting of HIV is associated with significant reductions in HIV reservoir size by multiple measures, including prolonged combination ART-free remission, the researchers concluded.

They added that stem cell transplantation in the setting of suppressed viral replication may be associated with loss of HIV-specific immunity, and hypothesised that immune activation in the setting of GVHD without anti-HIV specific immunity may cause homoeostatic proliferation of latently infected cells, decreasing the chance of HIV eradication.

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Could you save his life? Edmonton boy needs to find stem cell match –


Brady and his dad Terry Mishio.

Another stem cell swab event is taking place on Thursday with the hopes of finding a match for an eight-year-old Edmonton boy with leukemia.

In November, Brady Mishios dad took him to the hospital because he thought he had the flu.

Quickly they did some tests and then they did some more tests and X-rays and things and I knew at that point you know, a parent realizes something is awry there, Terry Mishio said.

WATCH:Edmonton boy with leukemia needs bone marrow transplant

That night, Bradywas diagnosed with acute myeloid leukemia, which is cancer of the blood.

I didnt know what the oncology unit meant, now I certainly do, Mishiosaid. Then, I think it was a nurse or someone who said, They think your son has cancer It turned out to be trueHe was diagnosed with cancer and he had leukemia.

Youre hoping its not true, Mishio said. Youre hoping they made a mistake.

Brady has already gone through three rounds of chemotherapy. Doctors say he needs a bone marrow transplant.

Now, the family is desperately searching for a stem cell match.

The world needs to know that this is what we need and this can save kids lives, Mishio said. Not just Bradys life theres other families in the Stollery Theyre waiting for their matches.

Its going to be a matter of minutes, he said of the testing process. Just to go in there, fill out the screen, the paper work, and then give a mouth swab and thats it. And then youre in the bank and you could save a life.

READ MORE:Alberta mother battling leukemia desperately searching for stem cell match

A massive swabbing event is takingplace on March 9 from 4 p.m. to 9 p.m. at the Holy Cross Ukrainian Catholic Parish at 9003 153 Avenue.

Organizers are expecting a big turnout, including Edmonton police officers and recruits, who will be getting swabs and registered as potential stem cell donors.

Basically, were looking for somebodys genetic twin, explained Robyn Henwood, with Canadian Blood Services.

In Canada alone we have about 1,000people at any given time looking for a match. Based on our international database, well find a match for about 50 per cent of them.

READ MORE:Young Edmonton boy searching for rare stem cell donor

If you cant make Thursdays event, visit Canadian Blood Services for more information or to set up an appointment. You need to be between 17 and 35 years old to register.

Its a little awkward to scrub the inside of your cheeks with Q-tips, Henwood said, but thats all it takes to register and potentially be the person that families are waiting for.

In 2013, 1,255 Canadians were diagnosed with acute myelogenous leukemia.

Eight-year-old Brady Mishio.

Eight-year-old Brady Mishio.

Eight-year-old Brady Mishio.

Brady and his dad Terry Mishio.

2017Global News, a division of Corus Entertainment Inc

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Stem Cell Therapy: Modern Solution to Joint Pain Relief – PR Newswire (press release)

PLAINVIEW, N.Y., March 6, 2017 /PRNewswire/ — Board certified surgeon Dr. Andrew J. Rochman promotes the specialized treatment of Stem Cell Therapy in his private practice in Plainview, NY. The recent wave of positive response from its ability to target a growing number of health conditions has brought increased attention and demand from pain sufferers everywhere- including the Long Island area.

As of the fall of 2016, Dr. Rochman officially opened the Cell Therapy Center of NY (CTCNY) where he focuses on the treatment of osteoarthritis & rheumatoid arthritis, eroded cartilage and joint issues like tennis elbow, jumpers knee and golf elbow. He dedicated his practice to the large population suffering from musculoskeletal damage arising from sports injuries or the wear and tear from aging. “We are always seeking a safer and more effective alternative to surgery to battle physiological symptoms with the hopes of giving patients a pain-free life.”

Dr. Rochman underwent extensive training from U.S. Stem Cell, Inc. (Sunrise, FLA) – a center for the development of effective cell technologies recognized for treating a variety of diseases and injuries. U.S. Stem Cell’s discoveries include multiple cell therapies in various stages of development that repair damaged tissue due to injury or disease. Chief Science Officer Kristin C. Comella, expert in regenerative medicine with a focus on adipose derived stem cells, pioneered stem cell therapies derived from various sources including cord blood, bone marrow and muscle. “By harnessing the body’s own healing potential, we may be able to reverse damaged tissue to normal function…. stem cells have the ability to form many types of tissues like bone, cartilage, muscle and even help to reverse some effects that have been caused by damaged tissue,” states Ms. Comella.

Dr. Rochman’s treatment center has recently seen an influx of patients from Long Island’s large athletic and fitness community. President of the Wildwood Warriors triathlon team, John Graziano is one of the many joint and back pain sufferers from sports injuries seeking this alternative pain treatment. “In the world of triathlon, I train- I race- and I live with pain and lots of it!”

“The potential here is limitless,” states Dr. Rochman. “It’s actually a simple yet unique form of therapy with the possibilities of doing miraculous things. We found out within the past several years that human beings have stem cells in every tissue of their body and they actually live around the blood vessels.” Today’s stem cell therapy has been shown to manage and target a wide span of healing possibilities from blood cell diseases to cardiac disorders to autoimmune diseases. “So what we can do now is to extract fat cells from the belly or bone marrow cells and isolate the cells from those damaged tissues… perhaps in the future we can utilize this process to target neurological diseases, heart diseases… and we don’t even know where it ends,” says Dr. Rochman.

Dr. Andrew J. Rochman is a native New Yorker and a board-certified surgeon. He is a graduate of Colgate University and received his formal medical training from Nordestana University. He is an active member of the American Medical Association, the Medical Society of the State of New York, Nassau County Medical Society and the American College of Phlebology. Dr. Rochman manages several practices in specialized studies such as advanced vein therapy and gallbladder surgery. His current certification is with U.S. Stem Cell, Inc. specializing in cardiovascular treatment through stem cell technology. The Cell Therapy Center of NY is located at 700 Old Country Road, Suite 205 Plainview, NY. For more information, visit: or call 516-280-1333.

Media contact: Lennard Gettz 631-553-8748

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SOURCE Cell Therapy Center of NY

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Stem Cell Therapy An Option For ENC Patients – Public Radio East

Stem cell therapy is a quickly advancing treatment being used across the country. Now, its becoming more prevalent in eastern North Carolina to those living with chronic pain an alternative to surgery. The minimally invasive procedure is showing results in alleviating back, knee, hip and shoulder pain. Though stem cell therapy is classified by the Food and Drug Administration as experimental, patients say theyre finding relief. Meet New Bern resident and a local endodontist Dr. Donnie Luper. He was skeptical of the procedure at first.

How did you know what those stem cells were going to differentiate into? I mean was I going to grow a foot out of my shoulder or something like that?

Luper tore his rotator cuff 25 years ago during a tubing incident on the Trent River. A subsequent fall during a golf trip in 2015 sent him to a specialist.

I went to see a shoulder surgeon in Richmond. He told me that he didnt think it was a complete tear of my rotator cuff, that I could probably have a minor surgical procedure done and I asked him about stem cell.

After talking with a friend who opted for stem cell treatment for her knee pain, Luper decided to find out more.

My option was if I would have had that shoulder surgery and they had do that bicep tendon repair, I mean I would have been in a sling for six weeks and probably not working for three months.

According to the Food and Drug Administration, stem cells sometimes called the bodys master cells – have the ability to divide and develop into many different cell types. Each new cell has the potential to remain a stem cell or become another type of cell, such as a nerve cell, a skin cell, or a red blood cell. They may also help repair the body by dividing to replenish cells that are damaged by disease, injury or normal wear. Parkinsons disease, spinal cord injuries, damaged organs and cancer could all be possibly treated with the use of stem cells, but more research is needed. Dr. Angelo Tellis is the owner/physician of Aegean Medical, which provides stem cell therapy to patients in Cary, Jacksonville, Morehead City and New Bern.

The adult stem cells we call multipotent stem cells so they can only differentiate into very specific or certain kinds of tissue. Whereas the embryonic stem cells we call pluripotent and can become a variety, almost any tissue. But I only deal with adult stem cells, theyre found to be more useful in clinical applications.

Dr. Tellis says adult stem cells are more responsive to growing tissue in very specific locations. When patients go into Dr. Tellis office for the two hour procedure, he starts by numbing an area of the abdomen and performing liposuction to collect one or two syringes of body fat.

Stem cells can be found in a lot of different tissues throughout the body, but theyre actually in one of the highest concentrations in your own body fat.

The stem cell sample is combined with platelet rich plasma or PRP collected through a blood draw.

That has a lot of the chemical signals and messengers that activate stem cells. So Ill typically combine that with some of the stem cells collected from the body fat and then go under x-ray guidance and put it exactly in the targeted location where we want to create that healing process.

Soreness and stiffness can be expected immediately following the procedure and for about a week after. Dr. Tellis says the results tend to improve with time, taking about three to six months for full recovery. This was Lupers experience in 2016.

Really didnt have to take any pain medications. The joint was really sore over the weekend just because of the injection of the fluid there and after that, I had a small amount of discomfort, but nothing I really had to take medication for.

After three months, Luper says he felt 90 percent better. But he decided to get a second opinion from a shoulder surgeon.

And he told me he thought the stem cells had done a lot but that I still had one little bone spur that was rubbing against the muscle and constantly tearing the little bit of the muscle.

After surgery, Luper says his left shoulder started feeling significantly better in about a month. He was also able to return to one of his favorite pastimes golf. While surgery helped eliminate all of his pain, Luper believes stem cells helped regenerate tissue that was damaged years ago.

He said my rotator cuff muscle didnt even look like it had been torn. I actually tore that, Im sixty now, and I actually tore that when I was 34, 35 tubing on the river and I had to do physical therapy for about three months, but he said he saw absolutely no evidence that Id ever had a rotator cuff tear.

Even though some have found relief and possibly a cure through stem cell therapy, the Food and Drug Administration has not approved any stem cell-based products for use, other than HEMACORD (HE-muh-cord). According to their website, the use of stem cells raises safety concerns such as excessive cell growth, the development of tumors as well as cells migrating from the site of administration and differentiating into inappropriate cell types. And then, theres the cost of the procedure, which is not covered by insurance. The price for the treatment ranges from $2,500 to $5,000. But for those who want to avoid major surgery and the downtime associated with recovery, the risk and cost may be worth it.

If Id have surgery, my deductible would have been that because I have an out-of-pocket max. And I would want to do anything to avoid surgery, especially something that would keep me out of work for three months.

The FDA recommends that consumers interested in stem cell therapy should start a conversation with their doctor about the potential risk to benefit ratio. In addition to Aegean Medical, Advanced Health and Physical Medicine in Greenville and Regenerative Medicine Clinic of Wilmington also provide stem cell therapy in eastern North Carolina.

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Stem Cell Therapy An Option For ENC Patients – Public Radio East

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Applied StemCell Announces the Appointment of Dr. Michele Calos … – Business Wire (press release)

MILPITAS, Calif.–(BUSINESS WIRE)–Applied StemCell (ASC), a leading stem cell and genome-editing company with a goal to advance genome editing and stem cell technologies for biomedical research and clinical applications, welcomes Dr. Michele Calos as a member of the companys Scientific Advisory Board (SAB).

Dr. Michele Calos is a Professor of Genetics at the Stanford University School of Medicine, Vice President of the American Society of Gene and Cell Therapy, and has served as an Advisory Committee member for the US FDA, grant review panels for the NIH and NSF, and on numerous editorial review committees of scientific journals. She is a leader in the field of molecular genetics and has developed several novel vector systems for genetic manipulation of mammalian cells. In particular, she developed novel methods for sequence-specific integration in mammalian cells using the C31 phage integrase system. A similar integrase system was also successfully used in site-specific integration in human ES and iPS cells. For this work, Dr. Calos holds a joint patent application with Applied StemCells Chief Scientific Officer, Dr. Ruby Yanru Chen-Tsai and several other Stanford researchers. Dr. Calos pioneering work with C31 integrase also set the scientific stage for ASCs TARGATT integrase technology, which was co-developed by Dr. Chen-Tsai and Dr. Liqun Luo of Stanford University for gene modification in mouse models.

We are extremely pleased to have Dr. Calos join as a member of our scientific advisory board. With her impressive background in integrase gene modification technology and gene therapy, Dr. Calos will be an invaluable guide in furthering expansion of our genome editing platforms and our gene/cell therapy pipeline, said Ruby Yanru Chen-Tsai, Ph.D., Co-founder and Chief Scientific Officer of Applied StemCell.

Dr. Calos and her research team are currently focused on gene therapy and genome engineering for the treatment of Duchenne and Limb Girdle Muscular Dystrophies and developing further novel strategies for gene and cell therapy.

About Applied StemCell, Inc.

Applied StemCell, Inc. is a leading stem cell and gene-editing company focused on the development of products and therapeutics that are enabled by its proprietary gene editing platform technologies TARGATT and CRISPR/Cas9. For more information, please visit

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Applied StemCell Announces the Appointment of Dr. Michele Calos … – Business Wire (press release)

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Researchers develop controllable gene therapy, make rats glow … – The Stanford Daily

Researchers at Stanford have made mice glow using a new gene therapy technique, showing that the process can work on living animals.

(Courtesy of Linda Cicero).

Named charge-altering releasable transporters (CARTs), the new technique allows researchers to control how much of a desired protein is expressed inside a cell, and how long the gene therapy lasts. It has a variety of applications to many central problems in biology and medicine, including immunology and cancer research.

Previous gene therapy techniques have relied on permanently changing the DNA within a cell. Colin McKinlay, a third-year Ph.D. student in chemistry and co-lead author on the paper, explains that CARTs take advantage of messenger RNA (mRNA) rather than DNA to give researchers greater control over the process.

By introducing mRNA into the cells, you can basically tell those cells to produce any given protein, McKinlay said. Its more of a temporary effect and you have a lot more control over doing that.

However, mRNA molecules are too large to enter the cell on their own. CARTs are able to latch onto the mRNA, cross the cell membrane, release the mRNA into the cell and quickly degrade into small molecules called metabolites naturally recognizable by the cell. After that, the cell takes over, translating the mRNA into the desired proteins.

Its kind of like the cell already has all of the ingredients, McKinlay said. Were just providing the recipe, and the cell then puts all the pieces together.

One possible application of the new gene therapy technique is creating new types of vaccinations. Typical vaccination techniques involve introducing a dead or weakened antigen, bacteria and foreign substances such as viruses into the cell, which the body then uses to create antibodies. CARTs could allow researchers to temporarily introduce specific proteins from the antigens into cells in order to specify targets for the immune system that are less sensitive to antigen mutation.

CARTs also have the potential to be used as a research tool. As transient polycations, CARTs allow proteins to be introduced and manufactured by the cell in controlled quantities and for a controlled amount of time, making them a valuable resource for studying signaling cascades and other biological phenomena.

The team behind CARTs primarily consists ofWender and Waymouth Group researchers, andalso drawson collaborators across Stanford. As the team begins to test the potential applications of CARTs, more researchers are expected to come on board.

In their recent paper on bioluminescent proteins in mice, researchers worked with Christopher Contag, a professor of pediatrics at Stanford, to show that the technique can work in vivo in animal models,bringing the team a step closer to using it in humans.

We couldnt have done it if we were stuck just within the confines of the chemistry department, said Jessica Vargas 16, a formerPh.D. student in the Wender Lab and a co-lead author on the paper. The work in general is a true testament to Stanfords collaborative spirit.

Contact Aulden Foltz at afoltz at

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Researchers develop controllable gene therapy, make rats glow … – The Stanford Daily

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Sickle cell anemia patient ‘cured’ by gene therapy, doctors say – FOX 61

FOX 61
Sickle cell anemia patient 'cured' by gene therapy, doctors say
FOX 61
In a world first, a teenager with sickle cell disease achieved complete remission after an experimental gene therapy at Necker Children's Hospital in Paris, researchers say. People with sickle-cell disease, a group of inherited blood disorders, have
Gene therapy shows early promise against sickle cellChicago Tribune
Gene therapy lets teen dodge sickle cell diseaseWDEF News 12
Doctors Claim They've Cured a Boy of a Painful Blood Disorder Using Gene TherapyFuturism
The Durango Herald –BioNews –BBC News –New England Journal of Medicine
all 78 news articles »

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Sickle cell anemia patient ‘cured’ by gene therapy, doctors say – FOX 61

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Adverum Biotechnologies Reports Fourth Quarter and Full Year 2016 Financial Results and Provides Update – P&T Community

Adverum Biotechnologies Reports Fourth Quarter and Full Year 2016 Financial Results and Provides Update
P&T Community
The companies' research collaboration and license agreement, entered into in May 2014 for an initial period of three years, was created to discover, develop and commercialize novel gene therapy products for the treatment of ophthalmologic diseases.

and more »

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Adverum Biotechnologies Reports Fourth Quarter and Full Year 2016 Financial Results and Provides Update – P&T Community

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Opinion/Commentary: Global stem cell therapy market to showcase … – The Daily Progress

LONDON Technavio analysts forecast the global stem cell therapy market to grow at a compound annual growth rate of close to 37 percent during the forecast period, according to their latest report.

The research study covers the present scenario and growth prospects of the global stem cell therapy market for 2017-2021. To determine the market size, the study considers revenue generated from allogenic and autogenic stem cell therapies.

The Americas are the largest regional segment of the global stem cell therapy market, responsible for generating over 56 percent of the total revenue (2016 figures). The region is expected to continue market dominance through the forecast period, driven by increasing demand for stem cell therapy products and investments into R&D.

Technavio analysts highlight the following factors as contributing to the growth of the global stem cell therapy market:

Increase in federal funding in stem cell therapy.

Sapna Jha, one of the lead research analysts at Technavio for medical imaging research, says, Many stem cell research institutes and small companies are involved in cutting-edge R&D and are yielding encouraging results. These institutions are witnessing an increased flow of investments from federal organizations, due to the realization of the importance of regenerative medicine.

The U.S. National Institutes of Health, a major funding government organization invested approximately USD 1.5 billion in stem cell research projects in 2016. Similarly, several state-level organizations such as California Institute for Regenerative Medicine has contributed USD 3 billion to stem cell research in 2014. Such funding will help various research institutes to discover and develop regenerative medicines, which will boost the global regenerative medicine market enormously.

Growing demand for personalized medicine.

The health care sector is creating a high demand for personalized medicine, which could offer game-changing opportunities for the vendors. These medicines offer treatments based on the individual characteristics, needs, and preferences, which will vastly improve the quality of health care. Individuals are increasingly banking their stem cells for future treatments. Research organizations are also extensively exploring ways to develop personalized treatments with stem cells, which could eventually erase the conventional medicine system and help in the effective treatment of various diseases such as diabetes and cancer.

Demand for development of effective drugs for cardiology and degenerative disorders.

There has been an increased demand to develop effective drugs for cardiology and degenerative disorders, for which there were no effective treatment plans before the advent of stem therapies. The discovery of possible cardiac stem cells uncovered new arenas to repair hearts injured due to acute myocardial infarction or coronary artery disease, says Sapna.

Researchers are studying and developing approximately 19 product candidates for the treatment of cardiac disorders, with eight of them in Phase III, and six in Phase II.

Technavio is a global technology research and advisory company. This report was made available through The Associated Press.

Opinion/Commentary: Global stem cell therapy market to showcase … – The Daily Progress

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