Archive for April, 2017

Vitamins are defined as organic substances occurring in many foods in small amounts that are necessary for the normal metabolic functioning of the body. Vitamin D is one of the fat-soluble vitamins. Our bodies can't manufacture vitamins but can manufacture hormones, and some experts feel that vitamin D should be classified as a hormone rather than a vitamin because our bodies manufacture it when exposed to the sun.

"Current Medical Diagnosis and Treatment 2016" notes that vitamin D deficiency "is increasing throughout the world as a result of diminished exposure to sunlight caused by urbanization, automobile and public transportation, modest clothing and sunscreen use."

Defining vitamin D deficiency can be confusing due to two ways of measuring level: nmol/liter and ng/ml (e.g. 112 nmol/liter is the same as 50 ng/ml). The latter is the way levels are usually reported and is what will be used in today's column. Significant vitamin D deficiency is defined as a level less than 20 ng/ml and this occurs in 29 percent of postmenopausal American women and 25 percent of American men older than 65. Severe deficiency is defined as a level less than 10 ng/ml and is present in 3.5 percent of Americans.

Almost all cells, organs and tissues in our bodies have vitamin D receptors, and vitamin D can also turn on hundreds of genes. Over the years, vitamin D supplementation has been touted as a panacea for all sorts of health problems, but according to Dr. Michael Greger's website nutritionfacts.org, better studies done in the last few years have discounted many of these claims. Here's what the current science tells us about vitamin D deficiency:

Vitamin D promotes calcium absorption by the intestines, and also stimulates the activity of bone-forming cells called osteoblasts. Deficiency can cause osteoporosis and osteomalacia, which like osteoporosis causes brittle bones and fractures, but is not exactly the same as osteoporosis. In children with developing bones, osteomalacia is called rickets, which can result in permanent skeletal deformities.

There are vitamin D receptors in our muscles and nervous systems including our brains, but as we age the number of receptors decreases. Elderly people with low vitamin D levels are more apt to suffer falls due to muscle weakness and balance problems.

Vitamin D boosts our immune system, and people with low D levels have an increased incidence of respiratory infections.

Low vitamin D levels are associated with increased all-cause mortality (i.e. you live longer if you maintain normal vitamin D levels).

Vitamin D helps fight inflammation. Asthma, ulcerative colitis and Crohn's disease all inflammatory diseases improve and in some cases even go into remission once D levels have normalized.

According to Dr. Joel Fuhrman, author of "Eat to Live" and other books, "Vitamin D regulates several genes and cellular processes related to cancer progression." People with low levels of D are more apt to get several cancers including breast and colon, and once they get cancer it is more likely to progress.

What are normal levels? Greger points out that the cradle of civilization was in equatorial Africa, "when people were running around outside naked." Vitamin D levels in African tribes living traditional lifestyles are around 50. Breast milk lacks vitamin D, and therefore breast-fed babies are given D supplements, which doesn't make sense from an evolutionary point of view. But if a breast-feeding mother's D level is 50 or greater, her breast milk does contain vitamin D. So while some guidelines say we should shoot for levels of D greater than 30, most of the science points to levels of 50 or more as ideal.

How much D should people take to achieve levels of 50 or above? For most people, 2,000 units a day achieves optimal D levels, with some caveats:

Vitamin D is stored in fat, so obese people need to take 4,000 units a day a day to achieve optimal levels.

Absorption is hampered in the elderly, so the American Geriatrics Society recommends 4,000 units in people 65 and older.

The type of vitamin D you should take is D3, which is what your body makes when exposed to sunlight; versus D2 present in yeast and mushrooms, which isn't as effective.

D is absorbed better if taken with a meal that contains some fat, such as nuts and seeds.

The practice of taking very high doses (e.g. 50,000 units) intermittently is now frowned upon, because the very high levels that result can cause problems.

How about just getting sun exposure rather than taking a supplement? The problem is that sun ages your skin and causes skin cancer. When outside you should cover up and apply sunscreen to exposed areas of your skin such as your face, but this interferes with vitamin D production. Tanning booths have the same problems as sun exposure, and aren't very effective in vitamin D production anyway.

Should everyone have their vitamin D levels tested? Most guidelines don't recommend this because:

Almost all Americans are lower than optimal in vitamin D.

Most insurance companies and Medicare won't cover the test when coded as a screen.

The test for vitamin D is done on a blood sample, and is not a very accurate test in that a lot of variation can occur between labs and even on the same sample tested repeatedly in the same lab.

Vitamin D is inexpensive and has no side effects except in very high doses, such as 10,000 units a day, which can result in dangerously high blood levels.

Dr. Feinsinger, who retired from Glenwood Medical Associates after 42 years as a family physician, now has a nonprofit Center For Prevention and Treatment of Disease Through Nutrition. He is available for free consultations about heart attack prevention and any other medical concerns. Call 970-379-5718 for an appointment. For questions about his columns, email him at gfeinsinger@comcast.net.

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Doctor's Tip: The low-down on vitamin D - Glenwood Springs Post Independent

Once a pioneer, Johns Hopkins Hospital is returning to the field of transgender healthcare. But the controversial beliefs of Dr. Paul McHugh still pose complications.

Paula Neira still lives by a motto she learned in the navy: semper porro, ever forward.

Thats why Neira, the clinical director of the new Johns Hopkins Center for Transgender Health and a transgender woman herself, isnt letting herself get bogged down by the institutions rocky past around transgender issues.

Its real easy to change course on a little patrol boat; its real difficult to change course on a dime on an aircraft carrier, she told The Daily Beast. These institutions are big institutions. But when you have the commitment from the leadership on down to change course, youll change course.

Once the first medical institution in the U.S. to offer sex reassignment surgery in the 1960s, it has taken nearly forty years for Johns Hopkins Medicine to return to the field of transgender health care after the original clinic was shut down in 1979 at the urging of Dr. Paul McHugh, then chair of the psychiatry department.

McHugh, now 85, has remained vocally opposed to transgender medical care ever since. And his continuing affiliation with the university has left some in the LGBT community concerned that the new centerscheduled to open in the summer of 2017, offering hormone therapy, surgeries, and other services to transgender patientswont do enough to repair Hopkins reputation.

For instance, after the Washington Post published a front-page feature last week focusing largely on McHughs reaction to the opening of the Center for Transgender Health, Human Rights Campaign national press secretary and transgender advocate Sarah McBride responded with an opinion piece saying that Hopkins has a long way to go before transgender people feel safe.

While the opening of the clinic is important, that alone cannot heal the wounds inflicted by Hopkins against transgender people nor alleviate the ongoing harm caused by the continued invocation of its credibility to support Mr. McHughs essays attacking LGBTQ people, McBride concluded. The only path toward inclusive care requires Hopkins to clearly speak out.

McHughs name, as The Daily Beast has previously reported, has become a staple in anti-LGBT circles. His 2014 Wall Street Journal op-ed Transgender Surgery Isnt the Solution, in particular, has been widely-cited. But his views on transgender health care are dramatically out of step with major medical associations, which support and affirm transgender medical care.

According to the American Medical Association, for example, there is an established body of medical research that shows the effectiveness and medical necessity of mental health care, hormone therapy and sex reassignment surgery as forms of therapeutic treatment for many transgender people.

And as the disjuncture between McHughs views and medical consensus has become more pronounced, Johns Hopkins Medicine has taken public steps to reassure the LGBT community that no single individual represents the institution as a whole.

Last October, two months after McHugh co-authored a paper questioning the concepts of sexual orientation and gender identity, Johns Hopkins Medicine leadership published a Dear colleagues letter stating that the institutions commitment to the LGBT community is strong and unambiguous, acknowledging that some have questioned our position because of the varied individual opinions expressed publicly by members of the Johns Hopkins Medicine community.

And in a new statement to The Daily Beast issued in response to questions about concerns like McBrides that exist in the transgender community, a Johns Hopkins Medicine spokesperson stated: Johns Hopkins Medicine has and is taking steps toward becoming an employer and provider of choice for all, including transgender individuals. And statements or actions to the contrary by current or former affiliates of Johns Hopkins do not reflect our institutions current views. We are committed to being a caring, inclusive place for all patients, families and employees.

Despite the October 2016 statement, the Human Rights Campaign this March deducted 25 points from Johns Hopkins Hospitals Healthcare Equality Index score, attributing it to Johns Hopkins Medicines failure to address HRCs concerns regarding deeply disturbing anti-LGBTQ misinformation written by McHugh.

Neirawho transitioned after leaving the navy, going on to become a registered nurse and an attorney advocating for the repeal of Dont Ask, Dont Tellfully realizes that McHughs presence will have some impact on the transgender communitys perception of the new center.

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Were aware that theres going to be skepticism in some quarters because of the history, because of McHughs writings, she told The Daily Beast. But his writings dont reflect the institutions values and where were going.

Neira believes that transgender people are smart consumers who tend to be much more informed about their care than averageoften out of necessity, given how frequently they have to educate their own physiciansand she hopes that the new center will be judged by its actions rather than the words of one doctor.

The folks in the community will make up their mind about what we do, she said. And as the clinical program director for the center, I wouldnt be in this role if [Hopkins] wasnt committed to doing this right.

The World Professional Association of Transgender Health, the leading organization for transgender health care professionals, is cautiously optimistic about the new Hopkins clinic. In written statements to The Daily Beast, treasurer Dr. Walter Bouman said that WPATH welcomes new transgender healthcare services to increase access of care for trans people and immediate past president Dr. Jamison Green said that it was a positive move.

We will have to see what policies the clinic uses, and what principles inform their procedures, added Green. I think it is a positive sign, though, that Johns Hopkins is willing to add to the knowledge base concerning genderand I expect the new crop of clinicians will have benefited from all the good work that has been done in the field since their original clinic was closed nearly 40 years ago. (Neira indicated to The Daily Beast that the new center would indeed use WPATHs Standards of Care.)

In a follow-up interview with The Daily Beast, Green disagreed with the idea that Hopkins Medicine should specifically call out McHughs writings: If they give him that much attention, that is simply going to elevate his profile.

Green called it a big deal that a health care leader like Hopkins would open a transgender health center as it reinforces the idea that this is meaningful.

As for McHugh himself, he reiterated in a phone interview with The Daily Beast that he disagrees with the idea that his writings pose any threat to transgender people.

I certainly am not wounding anybody, he said. I am saying that the treatments that are being offered do not help them.

McHugh has known for months that Hopkins would re-enter the field of transgender medicine. As the Washington Post reported in last weeks feature, McHugh received a visit from the head of plastic and reconstructive surgery last fall, who informed him that Johns Hopkins Hospital would once again start offering sex reassignment surgery. The Post reported, paraphrasing McHugh, that he bears no animus for transgender health providers at Hopkins.

He told The Daily Beast that although he has no hard feelings for his colleagues at Hopkins who made the call, he has voiced his disagreement.

When asked by The Daily Beast if he thought it was a mistake for Johns Hopkins Medicine to open the new transgender health center, he said, Yes and I think they will regret it and Ive told them so.

Asked what they said back to him, McHugh laughed gently.

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Can Trans People Trust Johns Hopkinss New Clinic? - Daily Beast

Many children and adolescents are "trying out" identifying as transgender because they think it will make them "different," an Australian psychiatristwho runs a gender clinic in Brisbane told the tabloidThe Courier-Mail. He said many more teens and young adults come into his clinic seeking transgender treatments than are actually suffering with long-term gender dysphoria.

"One said to me, 'Dr Steve ... I want to be transgender, it's the new black'," Dr. Stephen Stathis, a pediatric psychiatrist and medical director of child and youth mental health services at Children's Health Queensland Hospital and Health Service, toldThe Courier-Mail. He said he had seen a lot of adolescents "trying out being transgender" to stand out.

Stathis estimated that his gender service would see about 180 children with gender issues, but argued that only a minority would be diagnosed with gender dysphoria. By the time these kids reach puberty, most will identify as their birth gender, he predicted.

Rather than a cultural fad like transgenderism among teens seems to have become, gender dysphoria is a strong, persistent feeling of identification with the opposite gender and discomfort with one's own assigned gender.

One of the trends Stathis has seen is truly heartbreaking. Many young girls have come into his clinic after having been sexually abused, and wanted to change their gender to avoid more abuse.

"The girls say, 'If only I had been a male I wouldn't have been abused'," Stathis said. Situations like this are tragic, but not a justification for long-term surgeries and hormone treatments which may end up damaging girls who have already been abused. Rather than a solution, it seems this would only worsen their trauma.

The Courier-Mail reported that Stathis "has also seen transgender children so desperate to start puberty blockers then progress to irreversible hormone treatment they harm themselves." Warning: this description will be very disturbing.

"I've seen genital mutilation, some who try to cut off their penis," the psychiatrist said. "The thought of touching their genitals is so abhorrent they don't wash them and get infections."

At the end of last year, Stathis reported there was a two-year waiting list of 100 children wanting to be assessed at the hospital. With state funding, the wait is now down to three or four months, and the service has seen more than 60 patients since December. While it is a good thing more adolescents will actually be checked out by professionals who acknowledge that not all kids who identify as transgender are truly suffering from gender dysphoria, some would argue that even the minority who do are ill-served by suggestions they should irreversibly alter their bodies to match their inner identities.

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Kids 'Trying Out' Transgenderism Say 'It's the New Black' - PJ Media

Ottawa Sun

Jonathan Pitre 'anxious' as he readies for his second transplant Thursday Ottawa Sun Boileau goes into surgery at the University of Minnesota Masonic Children's Hospital at 5:30 a.m. Thursday to have bone marrow drawn from her hip. Surgeons will bore two holes into her pelvis and withdraw the bone marrow, a material rich in stem cells; ... and more »

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Jonathan Pitre 'anxious' as he readies for his second transplant Thursday - Ottawa Sun

It will be several weeks before Jonathan Pitre finds out if his second stem-cell transplant was successful. Tina Boileau / -

The perilous wait now begins for Jonathan Pitre.

Pitre, 16, was transfused with blood and marrow drawn from his mothers hip late Thursday afternoon. The stem-cell rich material holds the power to alter the course of Pitres aggressive skin disease, epidermolysis bullosa (EB), and change his life.

So far, so good, saidPitres mother, Tina Boileau.

It will be several weeks before Pitre finds out whether the transplant has worked its magic.

While waiting for that answer, theRussell teenager will have to travel the most difficult part of his medical journey: a time when his immune system is at its lowest ebb, and when he feels the full effects of high-dose chemotherapy and radiation.

His physician, Dr. Jakub Tolar, has warned that the period represents the highest risk for complications, the most common of which are infections and graft-versus-host disease (GVHD). It is a potentially life-threatening situation in which the implanted stem cells produce T-cells that attack normal cells.

In about two weeks time, doctors will start to look for signs that Boileaus stem cells have successfully established themselves in Pitres bone marrow.The presence of white blood cells is one of the earliest signs of stem-cell growth; an improvement in the condition of Pitres skin could also signal that the stem cells have started to work.

Last year, after his first stem-cell transplant, Pitre and his mother were thrilled when doctors discovered new white cells in his bloodstream. But their hopes were crushed when tests showed Pitres own stem cells had recolonized his bone marrow, and were producing the cells.

This time, Boileau said, they will wait to see more lab results before getting their hopes too high.

I think we will have that uncertainly until we know for sure through skin and bone marrow biopsies that the engraftment worked, she said.

Boileau went into surgery early Thursday morning to have blood and bone marrow drawn from her hip. She was at her sons bedside later in the afternoon to watch as the stem cells dripped through an intravenous tube connected to the right atrium of her sons heart.

If the transplant works, Boileaus stem cells will establish themselves in her sons bone marrow, grow, divide and make new blood cells equipped with the power to provide Pitre with the key protein he needs to rebuild his damaged skin.

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His stem-cell transplant complete, the wait begins for Jonathan Pitre - Ottawa Citizen

The most complex organ in humans is the brain. Due to its complexity and, of course, for ethical reasons, it is extremely difficult to do scientific experiments on it -- ones that could help us to understand neurodegenerative diseases like Parkinson's, for example. Scientists at the Luxembourg Centre for Systems Biomedicine (LCSB) of the University of Luxembourg have now succeeded in turning human stem cells derived from skin samples into tiny, three-dimensional, brain-like cultures that behave very similarly to cells in the human midbrain. In the researchers' petri dishes, different cell types develop, connect into a network, exchange signals and produce metabolic products typical of the active brain. "Our cell cultures open new doors to brain research," says Prof. Dr. Jens Schwamborn, in whose LCSB research group Developmental & Cellular Biology the research work was done. "We can now use them to study the causes of Parkinson's disease and how it could possibly be effectively treated." The team publishes its results today in the scientific journal Stem Cell Reports.

The human midbrain is of particular interest to Parkinson's researchers: it is the seat of the tissue structure known medically as the substantia nigra. Here, nerve cells -- specifically dopaminergic neurons -- produce the messenger dopamine. Dopamine is needed to maintain smooth body movements. If the dopaminergic neurons die off, then the person affected develops tremors and muscle rigidity, the distinctive symptoms of Parkinson's disease. For ethical reasons, researchers cannot take cells from the substantia nigra to study them. Research groups around the world are therefore working on cultivating three-dimensional structures of the midbrain in petri dishes. The LCSB team led by stem cell researcher Jens Schwamborn is one such group.

The LCSB scientists worked with so-called induced pluripotent stem cells -- stem cells that cannot produce a complete organism, but which can be transformed into all cell types of the human body. The procedures required for converting the stem cells into brain cells were developed by Anna Monzel as part of her doctoral thesis, which she is doing in Schwamborn's group. "I had to develop a special, precisely defined cocktail of growth factors and a certain treatment method for the stem cells, so that they would differentiate in the desired direction," Monzel describes her approach. To do this, she was able to draw on extensive preparatory work that had been done in Schwamborn's team the years before. The pluripotent stem cells in the petri dishes multiplied and spread out into a three-dimensional supporting structure -- producing tissue-like cell cultures.

"Our subsequent examination of these artificial tissue samples revealed that various cell types characteristic of the midbrain had developed," says Jens Schwamborn. "The cells can transmit and process signals. We were even able to detect dopaminergic cells -- just like in the midbrain." This fact makes the LCSB scientists' results of extraordinary interest to Parkinson's researchers worldwide, as Schwamborn stresses: "On our new cell cultures, we can study the mechanisms that lead to Parkinson's much better than was ever the case before. We can test what effects environmental impacts such as pollutants have on the onset of the disease, whether there are new active agents that could possibly relieve the symptoms of Parkinson's -- or whether the disease could even be cured from its very cause. We will be performing such investigations next."

The development of the brain-like tissue cultures not only opens doors to new research approaches. It can also help to reduce the amount of animal testing in brain research. The cell cultures in the petri dishes are of human origin, and in some aspects resemble human brains more than the brains of lab animals such as rats or mice do. Therefore, the structures of human brains and its modes of function can be modelled in different ways than it is possible in animals. "There are also attractive economic opportunities in our approach," Jens Schwamborn explains: "The production of tissue cultures is highly elaborate. In the scope of our spin-off Braingineering Technologies Sarl, we will be developing technologies by which we can provide the cultures for a fee to other labs or the pharmaceutical industry for their research."

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Materials provided by University of Luxembourg. Note: Content may be edited for style and length.

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Brain tissue from a petri dish: Stem cell research -- ScienceDaily - Science Daily

April 10, 2017 06:00 ET | Source: Mesoblast Limited

NEW YORK and MELBOURNE, Australia, April 10, 2017 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today announced that thePhase 3 trial ofits allogeneic mesenchymal precursor cell (MPC) product candidate MPC-150-IM in patients with moderate to advanced chronic heart failure (CHF)was successful in thepre-specified interim futility analysisof the efficacy endpoint in the trial's first 270 patients. It is expected that the trial will enroll in total approximately 600 patients. After notifying the Company of the interim analysis results, thetrials Independent Data Monitoring Committee (IDMC) additionally stated that they had no safety concerns relating to MPC-150-IM and formally recommended that the trial should continue as planned.

Dr. Emerson C. Perin,Director, Research in Cardiovascular Medicine and Medical Director, Stem Cell Center at the Texas Heart Institute, and a lead investigator on the ongoing Phase 3 trial said: "It is very pleasingto see that thislarge and rigorously conducted Phase 3 trialof Mesoblast's cell therapy was successful in the pre-specified interim futility analysis for the trial's efficacy endpoint in the first 270 patients. Advancedheart failure is a very serious and life-threatening disease, and there is an urgent need to develop a safe and effective new therapy for these patients that may halt or reverse disease progression and prevent the high associated mortality.

Mesoblast Chief Executive Silviu Itescucommented: Passing this interim futility analysis for MPC-150-IM is an important milestone for Mesoblast and our cardiovascular disease program. This validates our strategy and our prioritization of this valuable program.

This ongoing double-blinded randomized (1:1) trial is currently being conducted across multiple study sites in the United States and Canada.It is evaluating MPC-150-IM in adult patients with moderate to advanced New York Heart Association (NYHA) Class II/III chronic heart failure with left ventricular systolic dysfunction. The trials primary efficacy endpoint is a comparison of recurrent non-fatal heart failure-related major adverse cardiac events (HF-MACE) in moderate to advanced CHF patients receiving either MPC-150-IM by catheter injection into the damaged left ventricular heart muscle or sham control. A Joint Frailty Model is the statistical method that evaluates multiplenon-fatal heart failure-relatedevents per patient (such as repeated hospitalizations for decompensated heart failure) while accounting for increased likelihood of a terminal cardiac event (such as death, implantation of a mechanical heart assist device or a heart transplant) for patients with multiple non-fatal heart failure events. In line with best practice for blinded Phase 3 clinical trials, the interim analysis data are only reviewed by the IDMC. Mesoblast, the United States Food and Drug Administration (FDA), and trial investigators are blinded to grouped safety and efficacy data for the ongoing trial as well as the numerical results of this interim analysis.

About Mesoblasts MPC-150-IM Cardiovascular Program MPC-150-IM is Mesoblast's lead allogeneic, cell-based product candidate for the treatment of moderate to advanced chronic heart failure (CHF) due to left ventricular systolic dysfunction.

In Phase 2 results, a single injection of MPC-150-IM into the myocardium of patients with moderate to advanced chronic heart failure prevented any HF related hospitalizations or cardiac deaths over three years of follow-up.1 Nonclinical studies showed that intramyocardial administration of MPCs in animal models of heart failure improved cardiac function and attenuated pathological ventricular remodelling. These effects were attributable, at least in part, to MPC secretion of biomolecules that stimulate reparative processes in the failing heart including new blood vessel formation, cardiac muscle cell survival, and reduction in tissue fibrosis.

MPC-150-IM is also being studied in a Phase 2b trial in 159 patients with NYHA Class IV end-stage heart failure patients in conjunction with implantation of a left ventricular assist device (LVAD).A major objective of this trial, which is being sponsored by the United States National Institutes of Health (NIH), is to assess the ability of MPC-150-IM to help wean patients from a LVAD dependent existence for survival (so-called bridge to recovery).

Additionally, the FDA recently cleared the commencement of a 24-patient trial which is being sponsored by Bostons Childrens Hospital. This study combines Mesoblast's proprietary allogeneic MPC-150-IM product with corrective heart surgery in children under the age of 5 with hypoplastic left heart syndrome.

About Chronic Heart Failure In 2016, more than 15 million patients in the seven major global pharmaceutical markets are estimated to have been diagnosed with CHF.2 Prevalence is expected to grow 46% by 2030 in the United States alone, affecting more than 8 million Americans.3 CHF is a progressive disease and is classified in relation to the severity of the symptoms experienced by the patient. The most commonly used classification system was established by the NYHA and ranges from Class I (mild) to Class IV or end stage (severe). Approximately half of people who develop heart failure die within 5 years of diagnosis.4 Patients with late NYHA Class II or Class III CHF continue to represent a significant unmet medical need despite recent advances in new therapies. CHF causes severe economic, social, and personal costs. In the United States, it is estimated that CHF results in direct costs of $60.2 billion annually when identified as a primary diagnosis and $115 billion as part of a disease milieu.5

1.Perin EC, Borow KM, Silva GV, et al. A phase II dose-escalation study of allogeneic mesenchymal precursor cells in patients with ischemic or nonischemic heart failure. Circ Res. 2015; 117:576-84

2.GlobalData-PharmaPoint (2016): Heart Failure-Global Drug Forecast and Market Analysis to 2025

3.AHA Statistical Update Heart Disease and Stroke Statistics-(2017). Circulation. 2017;131:00-00. DOI: 10.1161/CIR.0000000000000485

4.Mozzafarian D, Benjamin EJ, Go AS, et al. on behalf of the American Heart Association Statistics Committee and Stroke Statistics Subcommittee. Heart disease and stroke statistics2016 update: a report from the American Heart Association. Circulation. 2016;133:e38-e360

5.A Re-Evaluation of the Costs of Heart Failure and its Implications for Allocation of Health Resources in the United States. Voigt J. Clinl.Cardiol. 37, 5, 312-321 (2014)

About Mesoblast Mesoblast Limited (Nasdaq:MESO) (ASX:MSB)is a global leader in developing innovative cell-based medicines. The Company has leveraged its proprietary technology platform, which is based on specialized cells known as mesenchymal lineage adult stem cells, to establish a broad portfolio of late-stage product candidates. Mesoblasts allogeneic, off-the-shelf cell product candidates target advanced stages of diseases with high, unmet medical needs including cardiovascular conditions, orthopedic disorders, immunologic and inflammatory disorders and oncologic/hematologic conditions.

Forward-Looking Statements This press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast's actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

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Successful Interim Analysis of Efficacy Endpoint in Mesoblast's Phase 3 Trial for Chronic Heart Failure - GlobeNewswire (press release)

Inkjet printers and lasers are parts of a new wayto produce cells important to research on nerve regeneration.

Schwann cells, for example, form sheaths around axons, the tail-like parts of nerve cells that carry electrical impulses. They promote regeneration of those axonsand secrete substances that promote the health of nerve cells. But theyre hard to come by in useful numbers.

This technology could lead to a better way to differentiate stem cells.

So researchers have been taking readily available mesenchymal stem cells (also called bone marrow stromal stem cells that can form bone, cartilage, and fat cells) and using a chemical process to differentiate them into Schwann cells. But its an arduous and expensive process.

Researchers at Iowa State University have developed a nanotechnology that uses inkjet printers to print multi-layer graphene circuits and also uses lasers to treat and improve the surface structure and conductivity of those circuits.

It turns out mesenchymal stem cells adhere and grow well on the treated circuits raised, rough, and 3D nanostructures. Add small doses of electricity100 millivolts for 10 minutes per day over 15 daysand the stem cells become Schwann-like cells.

This technology could lead to a better way to differentiate stem cells, says co-first author Metin Uz, a postdoctoral research associate in chemical and biological engineering. There is huge potential here.

The electrical stimulation is very effective, differentiating 85 percent of the stem cells into Schwann-like cells compared to 75 percent by the standard chemical process, according to the paper. The electrically differentiated cells also produced 80 nanograms per milliliter of nerve growth factor compared to 55 nanograms per milliliter for the chemically treated cells.

The researchers report the results could lead to changes in how nerve injuries are treated inside the body.

These results help pave the way for in vivo peripheral nerve regeneration where the flexible graphene electrodes could conform to the injury site and provide intimate electrical stimulation for nerve cell regrowth, the researchers write in a summary of their findings.

The paper reports several advantages to using electrical stimulation to differentiate stem cells into Schwann-like cells:

A key to making it all work is a graphene inkjet printing process that takes advantages of graphenes wonder-material propertiesits a great conductor of electricity and heat, its strong, stable, and biocompatibleto produce low-cost, flexible, and even wearable electronics.

But there was a problem: once graphene electronic circuits were printed, they had to be treated to improve electrical conductivity. That usually meant high temperatures or chemicals. Either could damage flexible printing surfaces including plastic films or paper.

The research group of lead author Jonathan Claussen, assistant professor of mechanical engineering and an associate of the US Department of Energys Ames Laboratory, solved the problem by developing computer-controlled laser technology that selectively irradiates inkjet-printed graphene oxide.

The treatment removes ink binders and reduces graphene oxide to graphenephysically stitching together millions of tiny graphene flakes. The process makes electrical conductivity more than a thousand times better.

That led to experimental attempts to grow stem cells on printed graphene and then to electrical stimulation experiments.

We knew this would be a really good platform for electrical stimulation, says Suprem Das, a postdoctoral research associate in mechanical engineering and an associate of the Ames Laboratory. But we didnt know it would differentiate these cells.

But now that it has, the researchers say there are new possibilities to think about. The technology, for example, could one day be used to create dissolvable or absorbable nerve regeneration materials that could be surgically placed in a persons body and wouldnt require a second surgery to remove.

The findings appear in Advanced Healthcare Materials. Funding came from the Roy J. Carver Charitable Trust, the US Army Medical Research and Materiel Command, and Iowa State.

Source: Iowa State University

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How inkjet printers help transform stem cells - Futurity: Research News

University of Minnesota researcher Perry Hackett, calls his breakthrough in using DNA to fight cancer one of the grandest Minnesota fishing stories ever.

Hackett, a professor of cell biology and genetics at the University, was given the Impact Award last month for inventing the Sleeping Beauty Transposon system a basis for many cancer-fighting immunotherapies.

Though Hacketts scientific journey began nearly 40 years ago when he was tasked with genetically engineering larger fish, his more recent work can reprogram a persons immune system to fight cancer by introducing a gene into a cell that will recognize foreign cells in the body.

Your immune system has memory, and it can target specific things that are bad, he said. It does so by targeting virus-infected cells and things like that.

Transposons are DNA that are not uniform throughout an organism a concept easily seen in Indian corn, where the kernels are multicolored because a DNA element is hopping around the corn genome.

Its named Sleeping Beauty because it was a gene that was active 13 million years ago but went extinct, Hackett said.

Because this system does not use viruses like other cancer treatments, Hackett said he and his team of three other University faculty members were awarded a grant to research using the system for human gene therapy.

The problem with viruses is theyre expensive to make, they take a long time to get and theyre very costly to purify, he said.

But the Sleeping Beauty Transposon is simple enough for an undergraduate student to make. Thats how trivial this technology is, he said.

The transposons history starts when Hackett accepted a job at the University in 1980 to study retroviruses. He said there were few restrictions on researchers and what they could do in the lab.

He and his colleagues were making mutations in cancer viruses and were not paid much attention.

A couple of my friends came to me and said, You know how to genetically engineer stuff. We want to make big fish, he said, adding that the governor at the time, Rudy Perpich, had asked someone from the medical school how it could help the fishing industry.

Hackett received money from the state and other organizations and successfully created faster-growing fish, but he said many environmentalists at the time were concerned about modified fish being in nature.

Though fish in Minnesota years ago were naturally larger and the fish population worldwide is decreasing rapidly, Hackett said the engineered fish never made it out into the wild.

All the lakes here in Minnesota are feeling pressure, he said. I would say that there now is a global fisheries crisis due to people called environmentalists and people called conservationists. The result is that the natural animal population cant keep up.

While Hackett and his teams engineered fish project came to a halt, he said they made the procedure used on the fish more efficient and eventually came up with a new transposon system.

In the early 2000s, the team merged with the Genetic Cell Biology and Development Department, which was new at the time, and started investing in transposons and gene therapy.

He said viruses have been used in the past to improve the immune system, but a few years ago immunotherapy became the focus.

Fundamentally, its a fishing story, Hackett said of creating the transposon system. "It is one of the grandest Minnesota fishing stories ever. You start to find a way to improve the lives of fishermen and you wind up with a cutting-edge tool to treat cancer.

Dan Voytas, a genetics, cell biology and development professor and the director of the Center for Genome Engineering, said he took a job at the University in 2008 partly because of Hacketts work in genetics.

Voytas said he first met Hackett shortly after the transposon discovery was made.

Part of the motivation for the move was certainly my excitement about working with professors at the University of Minnesota who are interested in editing [and] modifying DNA in cells, he said.

Voytas sees the Sleeping Beauty Transposon System as Hacketts greatest contribution to the University.

It has many applications, he said. Its been helpful in understanding how cancer progresses. Its been important to correct genetic diseases that people inherit. More recently its been important in turning our immune systems against cancer.

Voytas said the recent immune system discovery was commercially licensed in the last year and a half. Since Hacketts discovery, he said there have been other developments that allow DNA to be more precisely edited.

The Center for Genome Engineering implements other peoples systems into editing human, animal and plant genes, Voytas said.

He added that in the future, therapies based on Hacketts transposon system could eliminate or correct the symptoms of inherited diseases.

Its the therapeutic outcomes of that technology that people will appreciate and recognize, he said.

Allen Levine, the Universitys interim vice president for research and a member of the committee that awarded Hackett, said he was given the award in March because of his discovery and use of the Sleeping Beauty Transposon system.

Ive heard a lot about [Hackett] over the years, Levine said. The work that he has done has had a major impact in terms of cancer therapies.

Levine said most cancer therapies, which are relatively new, look to change the immune system to attack only cancer cells. He said 80 percent of people who use this technology have complete recovery or remission of cancer.

The University will keep on working in these arenas, he said. We want to reward that innovative thinking. I always say that genius is the recognition of the accident."

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UMN research reprograms immune system to fight cancer - Minnesota Daily

Q. Im in doubt regarding myelodysplasia is it multipotent or pluripotent?

A. Thats a great question because it lets us talk about hematopathology (yay!) and also stem cells (which can be confusing unless someone explains some simple stuff).

What is a stem cell? First, lets talk about stem cells. The thing that makes a stem cell a stem cell, at least in my mind, is the ability to self-renew. This means that the stem cell can either divide into two daughter cells which will mature into grown up cells, or (and more commonly) it can give rise to two cells: one that will become a mature cell, and another which retains the capacity to divide again. Its called asymmetric division: instead of giving rise to two of the same cells, you get one regular cell and another stem cell (which can continue this cycle of replication for a long long time).

(Virtually) limitless replication Most cells have a limited number of times that they can divide. This is because the telomeres (little protective DNA sequences) on the end of the chromosomes get a little shorter every time the DNA replicates and eventually they are so short that they cant protect the DNA and the cell is unable to divide. Stem cells and cancer cells have an enzyme called telomerase that replenishes the telomeres, keeping them nice and long so the cell can keep on dividing. Stem cells do eventually die so technically, there are a limited number of cell divisionsbut its a really, really big number. Cancer cells, on the other hand, are often totally immortal they can just keep on dividing and dividing.

Totipotent Another cool thing about stem cells is that they can give rise to many different kinds of cells. Heres where things can get murky. There are stem cells in an embryo which are able to give rise to any of the cell types in the body: hepatocytes, epithelial cells, neurons, cardiac muscle cellseverything. This makes sense: if youre going to grow into a human, you have to have cells that give rise to all the necessary cell types. These stem cells are called totipotent or pluripotent stem cells. Theres a slight difference between the two words: totipotent means that the stem cell can give rise to any and all human tissue cells and it can even give rise to an entire functional human. The only totipotent cells in human development are the fertilized egg and the cells in the next few cell divisions.

Pluripotent After those few cell divisions, the cells become pluripotent. Pluripotent cells are similar to totipotent cells in that they can give rise to any and all human tissue cells. Theyre different, though, because they are not capable of giving rise to an entire organism. On day four of development, the tiny little embryo forms two layers: one that will become the placenta and the other that will become the baby. The cells that will become the baby can give rise to any human tissue type (obviously) but those cells alone cant give rise to the entire organism (because you cant form the baby without the placenta). Slight difference but enough to make a separate term.

Multipotent Another term you should know is multipotent. Multipotent stem cells cannot give rise to any old cell in the body they are restricted to a limited range of cell types. For example, there are multipotent stem cells in the bone marrow that can give rise to red cells, white cells and platelets. They cant give rise to hepatocytes, or any other cell type, though so they are not totipotent or pluripotent.

There are lots of multipotent stem cells in the adult human body. They reside in the bone marrow, skin, muscle, GI tract, endothelium, and mesenchymal tissues. This means that there is a nice source for replacing cells that have died or been sloughed away.

What about myelodsyplasia? So back to your question. Myelodysplasia is a hematopoietic disorder in which cells in the bone marrow grow funny (dysplasia) they might be binucleate, or not have the normal number of granules, or whatever. In addition, some cases have an increase in blasts in the bone marrow but not over 20%, or youd call it an acute leukemia. Some cases transform, eventually, into an acute myeloid leukemia; others just stay the way they are and dont become nasty.

Check out the image above, from a case of myelodysplasia. There is a bizarre, multinucleated erythroblast at 11 oclock (this is called dyserythropoiesis, or disordered red cell growth). There are also two messed-up neutrophils (dysgranulopoiesis) at 4 oclock and 10 oclock the one at 4 oclock has only two nuclear lobes, and both are hypogranular (not enough specific granulation). Theres also an increase in blasts, if this field is representative: theres one in the middle and (probably) one at 5 oclock.

This disorder (actually, its a group of disorders) involves stem cells in the bone marrow. Sometimes only one cell line is involved (red cells, say); other times all three cell lines are involved (red cells, white cells and platelets). Either way, the disorder involves a stem cell, and since the stem cells in the bone marrow are multipotent, it would be correct to say that myelodysplasia is a disorder of multipotent stem cells in the bone marrow. Its kind of redundant, though, because as far as we know, there arent any other kind of stem cells in the bone marrow! But at least you know the answer to your question now.

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Multipotent vs. pluripotent stem cells - Pathology Student

RANCHO CORDOVA, Calif., April 11, 2017 (GLOBE NEWSWIRE) -- Cesca Therapeutics Inc. (KOOL), a market leader in automated cell processing and point-of-care, autologous cell-based therapies, today announced that Dr. Xiaochun (Chris) Xu, Chairman and Interim Chief Executive Officer and Chairman of Boyalife Group, will present an overview of the Companys cardiovascular clinical research program at the 2017 International Symposium of Translational Medicine in Stem Cell Myocardial Repair, being held April 10-12, 2017 at the Hope Hotel in Shanghai, China.

Details of the presentation are as follows:

Despite recent therapeutic and surgical advances, the effects of peripheral arterial disease, including heart attack and critical limb ischemia (CLI), remain among the worlds leading causes of morbidity and mortality and represent a rapidly escalating public health crisis, noted Dr. Xu. I look forward to presenting a review of our latest findings, including key feasibility study results and an overview of our Phase 3 Critical Limb Ischemia Rapid Stemcell Treatment (CLIRST) trial, which we believe highlight the potential of Cesca Therapeutics proprietary AutoXpress point-of-care platform to deliver autologous cell-based therapies that may represent a new paradigm in patient treatment going forward.

About the Symposium of Translational Medicine in Stem Cell Myocardial Repair

The 2017 International Symposium of Translational Medicine in Stem Cell Myocardial Repair brings together more than 650 of the worlds cardiac disease thought leaders to discuss the potential of translational and regenerative medicine in treating myocardial infarction (MI) and cardiac failure. The symposium is co-sponsored by the Shanghai Society for Cell Biology, the Institute of Health Sciences, the Shanghai Cardiovascular Disease Institute, the Guangzhou Institutes of Biomedicine and Health, and the Key Laboratory of Stem Cell Biology, Shanghai.

About Cesca Therapeutics Inc.

Cesca is engaged in the research, development, and commercialization of cellular therapies and delivery systems for use in regenerative medicine. The Company is a leader in the development and manufacture of automated blood and bone marrow processing systems that enable the separation, processing and preservation of cell and tissue therapeutics. Cesca is an affiliate of the Boyalife Group (http://www.boyalifegroup.com), a China-based industrial-research alliance among top research institutes for stem cell and regenerative medicine.

Forward-Looking Statement

The statements contained herein may include statements of future expectations and other forward-looking statements that are based on managements current views and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. A more complete description of risks that could cause actual events to differ from the outcomes predicted by Cesca Therapeutics' forward-looking statements is set forth under the caption "Risk Factors" in Cesca Therapeutics annual report on Form 10-K and other reports it files with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements.

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CESCA Therapeutics to Present at the 2017 International Symposium of Translational Medicine in Stem Cell ... - Yahoo Finance

Virginia Western Community College will host a student-led Be the Match donor drive on April 19 from 10 a.m. to 2 p.m. in the courtyward between the Fralin Center and Business Science Building and the Pedestrian bridge. Through the drive, potential donors will learn if they could provide life-saving bone marrow or peripheral blood stem cell (PBSC) transplants.

At the drive, potential donors will complete a registration form with contact information, health information and a signed agreement to join the Be The Match Registry. To help you complete the form, bring along:

Personal identification (such as a driver's license or passport)

Contact information for two family members or friends who would know how to reach you in the future if your contact information changes

You will provide a swab of cheek cells to be tissue-typed. We will use the results to match you to patients

During the drive, an individual who has battled leukemia and received a stem cell transplant will speak to perspective donors on the importance of donation. Please join us to learn how you could help those in need.

For more information on Be the Match, visit http://www.bethematch.org.

Submitted by Josh Meyer

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VWCC to host bone marrow donor drive April 19 - Roanoke Times

Sumitomo Dainippon Pharma Co Ltd has ordered cell culture technologies from Hitachi as part of its effort to develop a treatment for Parkinsons disease.

The order financial terms of which were not provided will see Hitachi supply automated cell culturing technologies designed for the manufacture of induced pluripotent stem cells (iPS).

Dainippon is developing a cell therapy for Parkinsons-related dopamine neuron loss and neurodegeneration in collaboration with both Hitachi and Center for iPS Cell Research and Application, Kyoto University (CiRA).

Part of the project which is funded by the Japanese Agency of Medical Research and Development (AMED) - involves the development of processing methods and technologies for the production of stem cells for regenerative therapies.

The Japanese drug firm has announced several regenerative medicine-based research projects in recent years, beginning in 2015 when it partnered with Sanbio to develop SB623, an allogenic cell therapy for ischemic stroke to improve motor abilities.

Regenerative meds

Regenerative medicine which engineers or replaces damaged cells within human patients has become a popular area of research in Japan sinceShinya Yamanaka won the 2012 Noel Prize for medicine for the discovery that mature cells can be reprogrammed to become pluripotent.

Regenerative medicine is also a big focus for the Japanese Government.

Laws introduced in November 2014 therevised pharmaceutical affairs law and newregenerative medicines legislation mean such products could be reviewed and approved in just two years, if deemed to be effective.

Japans Government further underlined its commitment to regenerative medicine in its budget in January 2015, allocating Y2.5bn ($20.8bn) to the industrialisation of regenerative medicine evaluation fundamental technology development business.

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Sumitomo Dainippon buys cell therapy processing tech from Hitachi - In-PharmaTechnologist.com

Are you a deathist? A deathist is someone who accepts the fact of death, who thinks the ongoing massacre of us all by ageing is not a scandal. A deathist even insists that death is valuable: that the only thing that gives life meaning is the fact that it ends an idea not necessarily embraced by someone about to be murdered on video by an Isis fanatic.

But what is the alternative? There has never been one, which is why until recently no one needed to coin the term deathist. But now many tech entrepreneurs and scientists take a different view: death, they say, is simply an engineering challenge. Biotechnology should, in principle, be able to reverse the wear-and-tear on cellular machinery in our bodies and keep us in our prime indefinitely, barring violent accident. Consider how many lives this would save. If you think such research should not be pursued, then you are a throwback, a deathist, a morose Luddite thanatophile.

Anti-deathism is one of the main strands of a set of sci-fi dreams that come under the umbrella term transhumanism, the subject of the Irish literary critic Mark OConnells engaging tour. He visits a cryonics facility in the desert outside Phoenix, where customers have paid to have their whole bodies or just their heads (called, Greekly, cephalons in the facilitys distancing jargon) preserved by freezing, in the hope that science will one day figure out how to revive them. He goes to a robotics fair where the audience gasps at humanoid robots that can operate door handles or egress successfully from a car. He hangs out with a gang of grinder cyborgs, that like to implant boxes of electronics under their skin in order to, say, be able to sense the presence of an electromagnetic field. He interviews people working on the idea of uploading human minds to computers, and those like the philosopher Nick Bostrom who fear that one day soon they, and we, might be killed by an omnipotent artificial intelligence of our own creation.

This is all related in a sort of wryly melancholy version of gonzo narrative non-fiction, structured in the simple What I Did Next For My Research style. Think a more overtly erudite version of Jon Ronson. As with that writer, you do occasionally feel that OConnell is expending energy on a less interesting figure simply because they provide so much freakish colour. Some of his transhumanist subjects are pitiful (the virginal man who looks forward to sexbots) but others for instance, the American scientist Laura Deming, who focuses on life extension research are extremely intelligent and persuasive. Overall, the book is thoughtful, modestly unsure of its own opinion, and often disarmingly funny. (Cryogenically frozen brains are left in their skulls, OConnell explains, because technically, it is kind of a hassle to remove the thing entirely.)

The author is especially alert to the assumptions encoded within tech-utopian rhetoric for example, the habit of saying that we should solve death:

The word solve seemed to me to encapsulate the Silicon Valley ideology whereby all of life could neatly be divided into problems and solutions solutions that always took the form of some or other application of technology.

And the very prefix trans- in the word transhumanism expresses, for some, a forlorn desire for spiritual transcendence of mere meat. As one cyborg tinkerer explains to the author:

Ask anyone whos transgender. Theyll tell you theyre trapped in the wrong body. But me, Im trapped in the wrong body because Im trapped in a body. All bodies are the wrong body.

The apparent paradox, then, is that so many transhumanists, while bent on defeating or solving death, also seem rather, well, misanthropic. To be transhumanist is on some level also to be anti-humanist: people tell OConnell what contemptible monkeys current humans are, how disgusting it is that they are doing all this breeding, and how theyd rather be machine-based consciousnesses exploring the vastness of space. But when it comes down to it, you might think there is not all that much to distinguish this, as a consummation devoutly to be wished, from good old-fashioned death.

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Who on earth wants to live forever with the people who want to live forever? - Spectator.co.uk

Gene therapy carries the promise of cures for many diseases and for types of medical treatment that didn't seem possible until recently. With its potential to eliminate and prevent hereditary diseases such as cystic fibrosis and hemophilia and its use as a possible cure for heart disease, AIDS, and cancer, gene therapy is a potential medical miracle-worker.

But what about gene therapy for children? There's a fair amount of risk involved, so thus far only seriously ill kids or those with illnesses that can't be cured by standard medical treatments have been involved in clinical trials using gene therapy.

As those studies continue, gene therapy may soon offer hope for children with serious illnesses that don't respond to conventional therapies.

Our genes help make us unique. Inherited from our parents, they go far in determining our physical traits like eye color and the color and texture of our hair. They also determine things like whether babies will be male or female, the amount of oxygen blood can carry, and the likelihood of getting certain diseases.

Genes are composed of strands of a molecule called DNA and are located in single file within the chromosomes. The genetic message is encoded by the building blocks of the DNA, which are called nucleotides. Approximately 3 billion pairs of nucleotides are in the chromosomes of a human cell, and each person's genetic makeup has a unique sequence of nucleotides. This is mainly what makes us different from one another.

Scientists believe that every human has about 25,000 genes per cell. A mutation, or change, in any one of these genes can result in a disease, physical disability, or shortened life span. These mutations can be passed from one generation to another, inherited just like a mother's curly hair or a father's brown eyes. Mutations also can occur spontaneously in some cases, without having been passed on by a parent. With gene therapy, the treatment or elimination of inherited diseases or physical conditions due to these mutations could become a reality.

Gene therapy involves the manipulation of genes to fight or prevent diseases. Put simply, it introduces a "good" gene into a person who has a disease caused by a "bad" gene.

The two forms of gene therapy are:

Currently, gene therapy is done only through clinical trials, which often take years to complete. After new drugs or procedures are tested in laboratories, clinical trials are conducted with human patients under strictly controlled circumstances. Such trials usually last 2 to 4 years and go through several phases of research. In the United States, the U.S. Food and Drug Administration (FDA) must then approve the new therapy for the marketplace, which can take another 2 years.

The most active research being done in gene therapy for kids has been for genetic disorders (like cystic fibrosis). Other gene therapy trials involve children with severe immunodeficiencies, such as adenosine deaminase (ADA) deficiency (a rare genetic disease that makes kids prone to serious infection), sickle cell anemia, thalassemia, hemophilia, and those with familial hypercholesterolemia (extremely high levels of serum cholesterol).

Gene therapy does have risks and limitations. The viruses and other agents used to deliver the "good" genes can affect more than the cells for which they're intended. If a gene is added to DNA, it could be put in the wrong place, which could potentially cause cancer or other damage.

Genes also can be "overexpressed," meaning they can drive the production of so much of a protein that they can be harmful. Another risk is that a virus introduced into one person could be transmitted to others or into the environment.

Gene therapy trials in children present an ethical dilemma, according to some gene therapy experts. Kids with an altered gene may have mild or severe effects and the severity often can't be determined in infants. So just because some kids appear to have a genetic problem doesn't mean they'll be substantially affected by it, but they'll have to live with the knowledge of that problem.

Kids could be tested for disorders if there is a medical treatment or a lifestyle change that could be beneficial or if knowing they don't carry the gene reduces the medical surveillance needed. For example, finding out a child doesn't carry the gene for a disorder that runs in the family might mean that he or she doesn't have to undergo yearly screenings or other regular exams.

To cure genetic diseases, scientists must first determine which gene or set of genes causes each disease. The Human Genome Project and other international efforts have completed the initial work of sequencing and mapping virtually all of the 25,000 genes in the human cell. This research will provide new strategies to diagnose, treat, cure, and possibly prevent human diseases.

Although this information will help scientists determine the genetic basis of many diseases, it will be a long time before diseases actually can be treated through gene therapy.

Gene therapy's potential to revolutionize medicine in the future is exciting, and hopes are high for its role in ;curing and preventing childhood diseases. One day it may be possible to treat an unborn child for a genetic disease even before symptoms appear.

Scientists hope that the human genome mapping will help lead to cures for many diseases and that successful clinical trials will create new opportunities. For now, however, it's a wait-and-see situation, calling for cautious optimism.

Date reviewed: April 2014

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Gene Therapy and Children (For Parents) - KidsHealth

Science Daily

Success of sensory cell regeneration raises hope for hearing restoration Science Daily In an apparent first, St. Jude Children's Research Hospital investigators have used genetic manipulation to regenerate auditory hair cells in adult mice. The research marks a possible advance in treatment of hearing loss in humans. The study appears in ...

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Success of sensory cell regeneration raises hope for hearing restoration - Science Daily

First bone marrow stem cell transplantations performed in Armenia

YEREVAN, APRIL 10, ARMENPRESS. The first two stem cell transplantations of bone marrow in Armenia were performed in the Yolyan Hematology Center by Professor Dr. NicolausKrger, head of the transplantation department of Hamburgs Eppendorf Clinic and the Yolyan Hematology Clinics team.

Professor Smbat Daghbashyan, head of the Armenian transplantation doctors team, told reporters the transplantation passed successfully.

The patients, who trusted her health to the doctors, is a woman from Artsakh, who had to travel abroad for undergoing the same surgery. The second patient is a man, who had a repetition of the disease after chemotherapy, he said, adding that 60 patients annually need stem cell transplantation in Armenia.

We will continue cooperation with our colleagues from Hamburg. The patient who had to receive the transplantation in Hamburg, can get it here the same way. We will perform transplantations in 7-10 patients during this year, since this a gradual process, he said.

Dr. NicolausKrger congratulated the Armenian doctors in introducing the new treatment method in Armenia.

This method is considered to be innovative in the world and is used for treating cancerous diseases, he said.

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First bone marrow stem cell transplantations performed in Armenia - Armenpress.am

Credit: St. Jude Children's Research Hospital

Like private investigators on a stake out, St. Jude Childrens Research Hospital scientists used patience and video surveillance-like tools to identify cells that trigger blood cell development. The findings offer clues for making blood-forming stem cells in the laboratory that may ultimately help improve access to bone marrow transplantation.

The research will likely open new avenues of investigation in stem cell biology and blood development and provide insight to aid efforts to make transplantable hematopoietic stem cells in the lab, said corresponding author Wilson Clements, Ph.D., an assistant member of the St. Jude Department of Hematology.

Blood-forming stem cells are capable of making any type of blood cell in the body. They are also used in transplant therapies for cancers like leukemia or other blood diseases like sickle cell. They are starting to be used to deliver gene therapy. However, a shortage of suitable donors limits access to treatment, and efforts to produce blood from pluripotent stem cells in the laboratory have been unsuccessful. Pluripotent stem cells are the master cells capable of making any cell in the body.

All blood-forming stem cells normally arise before birth from certain endothelial cells found in the interior blood vessel lining of the developing aorta. This processincluding how endothelial cells are set on the path to becoming blood stem cellsis not completely understood.

Clements and first author Erich Damm, Ph.D., a St. Jude postdoctoral fellow, have identified trunk neural crest cells as key orchestrators of the conversion of endothelial cells to blood stem cells. Trunk neural crest cells are made in the developing spinal cord and migrate throughout the embryo. They eventually give rise to a variety of adult cells, including neurons and glial cells in the sympathetic and parasympathetic nervous system, which control feeding, fighting, fleeing and procreating.

Using time-lapse video, the researchers tracked the migration of neural crest cells in the transparent embryos of zebrafish. Zebrafish and humans share nearly identical blood systems, as well as the programming that makes them during development. After about 20 hours, the neural crest cells had reached the developing aorta. After hour 24, the migrating cells had cozied up to the endothelial cells in the aorta, which then turned on genes, such as runx1, indicating their conversion to blood stem cells.

The investigators used a variety of methods to show that disrupting the normal migration of neural crest cells or otherwise blocking their contact with the aorta endothelial cells prevented the birth of blood stem cells. Meanwhile, other aspects of zebrafish development were unaffected.

Researchers have speculated that the endothelial cells that give rise to blood-forming stem cells are surrounded by a support niche of other cells whose identity and origins were unknown, Damm said. Our results support the existence of a niche, and identify trunk neural crest cells as an occupant.

Adult bone marrow includes niches that support normal function and notably feature cells derived from trunk neural crest cells.

The findings also suggest that trunk neural crest cells use a signal or signals to launch blood stem cell production during development. The researchers have eliminated adrenaline and noradrenaline as the signaling molecules, but work continues to identify the signaling proteins or small molecules involved.

The research was supported in part by a grant (R00HL097) from the National Heart, Lung and Blood Institute of the National Institutes of Health; the March of Dimes; and ALSAC, the fundraising arm of St. Jude.

Reference:

Damm, E. W., & Clements, W. K. (2017). Pdgf signalling guides neural crest contribution to the haematopoietic stem cell specification niche. Nature Cell Biology. doi:10.1038/ncb3508

This article has been republished frommaterialsprovided by St. Jude Children's Research Hospital. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Cells Essential for 'Birth' of Blood Stem Cells Revealed - Technology Networks

Written by AZoNanoApr 11 2017

Iowa State University researchers, left to right, Metin Uz, Suprem Das, Surya Mallapragada and Jonathan Claussen are developing technologies to promote nerve regrowth. The monitor shows mesenchymal stem cells (the white) aligned along graphene circuits (the black). CREDIT: Photo by Christopher Gannon.

Scientists searching for the means to regenerate nerves might find it difficult to acquire the important tools needed for research. One such example is Schwann cells that form sheaths enclosing axons, which are tail-like portions of nerve cells that convey electrical impulses. In addition to promoting regeneration of the axons, the Schwann cells discharge substances, boosting the health of nerve cells.

To put it differently, the Schwann cells prove to be helpful to researchers working towards the regeneration of nerve cells, particularly peripheral nerve cells located outside the spinal cord and brain. However, the count of Schwann cells is too low to be of any use.

Scientists have been using noncontroversial, readily available mesenchymal stem cells that is, bone marrow stromal stem cells with the ability to form cartilage, bone, and fat cells by differentiating them into Schwann cells by means of a chemical process. Unfortunately, this process is costly and laborious.

The Iowa State University research team have been looking for a better way to transform the stem cells into Schwann-like cells, and have created a nanotechnology that employs inkjet printers for printing multi-layer graphene circuits. It also employs lasers to treat and enhance conductivity and the surface structure of the circuits.

The mesenchymal stem cells have been found to adhere and grow in a better manner on the rough, raised, and 3-D nanostructures of the treated circuit. When small doses of electricity of about 100 mV were applied for 10 minutes per day, for a time period of 15 days, the stem cells transformed into Schwann-like cells.

This discovery has made it to the front cover of Advanced Healthcare Materials, a scientific journal. The lead author of the study is Jonathan Claussen, assistant professor of mechanical engineering at Iowa State University and an associate of the U.S. Department of Energys Ames Laboratory. The first authors of the study are Suprem Das, a postdoctoral research associate in mechanical engineering and an associate of the Ames Laboratory, and Metin Uz, a postdoctoral research associate in chemical and biological engineering.

The research has been funded by the Roy J. Carver Charitable Trust, the U.S. Army Medical Research and Materiel Command, and Iowa States College of Engineering, including the Department of Mechanical Engineering. The research has also been supported by The Carol Vohs Johnson Chair in Chemical and Biological Engineering, Surya Mallapragada. She is a co-author of the study, an Anson Marston Distinguished Professor in Engineering, as well as an associate of the Ames Laboratory.

This technology could lead to a better way to differentiate stem cells. There is huge potential here.

Metin Uz

When compared to the standard chemical process with the ability of differentiating only 75% of the stem cells into Schwann-like cells, the highly effective electrical stimulation carried out in the new technique can differentiate 85%. In addition, the electrically differentiated cells generated a nerve growth factor of 80 ng/mm when compared with 55 ng/mm in the case of the chemically treated cells.

The research team believes the outcome might result in changes in the ways nerve injuries are cured inside the body.

These results help pave the way for in vivo peripheral nerve regeneration, where the flexible graphene electrodes could conform to the injury site and provide intimate electrical stimulation for nerve cell regrowth.

The research team

Various benefits of using electrical stimulation for transforming stem cells into Schwann-like cells are reported in the paper:

A graphene inkjet printing process, created in Claussens research lab, is an important part of making the process work. Flexible, inexpensive, and wearable electronics can be produced through the process by making appropriate use of the benefits of wonder-material graphene, namely high stability, high strength, biocompatibility, and higher electrical and heat conductivity.

The research team confronted one major challenge after printing the graphene electronic circuits, the circuits mandated further treatment to enhance the electrical conductivity, normally done using chemicals or high temperatures. Both of these methods can damage the flexible printing surfaces which include paper or plastic films.

Claussen and his colleagues overcame the challenge by developing a computer-controlled laser technology with the ability to selectively irradiate inkjet-printed graphene oxide. This step eliminates ink binders and converts the graphene oxide to graphene by physically connecting millions of tiny graphene flakes together. This improves the electrical conductivity by over a thousand times.

The cooperation between Claussens team of nanoengineers (who developed printed graphene technologies), and Mallapragadas team of chemical engineers (who investigated nerve regeneration), started as a consequence of informal conversations on campus.

This resulted in experimental efforts to grow stem cells on printed graphene and then to perform electrical stimulation experiments.

We knew this would be a really good platform for electrical stimulation. But we didnt know it would differentiate these cells.

Suprem Das

Since the process has been successful in differentiating the stem cells, the scientists believe that there may be further prospective applications to consider. For instance, in future, the technology could be applied to develop absorbable or dissolvable nerve regeneration materials. These could be surgically positioned inside a patients body without the need for subsequent surgery to remove the materials.

Excerpt from:
Innovative Process for Differentiating Stem Cells into Schwann-Like Cells - AZoNano

Pennsylvania state trooper Matt Uram was talking with his wife at a July Fourth party in 2009 when a misjudged spray of gasoline burst through a nearby bonfire and set him alight. Flames covered the entire right side of his body, and after he fell to the ground to smother them, his wife beat his head with her bare hands to put out his burning hair. It was only on the way to the ER, as the shock and adrenaline began to wear off, that the pain set in. It was intense, he says. If you can imagine what pins and needles feel like, then replace those needles with matches.

From the hospital, Uram was transferred to the Mercy Burn Center in Pittsburgh, where doctors removed all of the burned skin and dressed his wounds. It was on the border between a second- and third-degree burn, and he was told to prepare for months of pain and permanent disfigurement. Not long after this assessment, however, a doctor asked Uram if he would be willing to take part in an experimental trial of a new device.

The treatment, developed by German researcher Dr. Jrg Gerlach, was the worlds first to use a patients stem cells to directly heal the skin. If successful, the device would mend Urams wounds using his bodys ability to regenerate fully functioning skin. Uram agreed to the procedure without hesitation.

Five days after the accident, surgeons removed a small section of undamaged skin from Urams right thighabout the size of a postage stampand used it to create a liquid suspension of his stem cells that was sprayed in a fine mist onto the damaged skin. Three days later, when it was time to remove the bandages and re-dress the wounds, his doctor was amazed by what he saw. The burns were almost completely healed, and any risk of infection or scarring was gone.

Pennsylvania State Trooper Matt Uram's arm eventually healed without scarring. RenovaCare

A study subsequently published in the scientific journal Burns described how the spray was able to regrow the skin across the burn by spreading thousands of tiny regenerative islands, rather than forcing the wound to heal from its edge to the inside. The technique meant reducing the healing time and minimizing complications, with aesthetically and functionally satisfying outcomes, the paper stated.

Dozens more burn victims in Germany and the U.S. were successfully treated with the spray following Urams procedure, and in 2014 Gerlach sold the technology to RenovaCare. The medical technology startup has now transformed the proof-of-concept device from a complicated prototype into a user-friendly product called a SkinGun, which it hopes clinicians will be able to use outside of an experimental setting. For that to happen, RenovaCare is preparing clinical studies for later this year, with the aim of Food and Drug Administration approval for the SkinGun.

Once these obstacles are overcome, RenovaCare CEO Thomas Bold believes, the SkinGun can compete with, or even replace, todays standard of care.

Current treatment of severe burns involves transplanting healthy skin from one area of the body and stitching it to another in a process called skin grafting or mesh skin grafting. It is a painful procedure that creates an additional wound at the donor site and can cause restricted joint movement because the transplanted skin is unable to grow with the patient. It is able to cover an area only two to three times as large as the harvested patch. The current standard of care is just horrible, says Bold. We are part of regenerative medicineit is the medicine of the future and will be life-changing for patients.

RenovaCare's SkinGun sprays a liquid suspension of a patient's stem cells onto a burn or wound in order to regrow the skin without scars. RenovaCare

Beyond regulatory matters, there are also limitations to the technology that make it unsuitable for competing with treatments of third-degree burns, which involve damage to muscle and other tissue below the skin. Still, stem cell researcher Sarthak Sinha believes that while the SkinGun may not be that advanced yet, it shows the vast potential of this form of regenerative medicine. What I see as the future of burn treatment is not skin repair but rather functional regeneration of skin and its appendagessuch as hair follicles, glands and fat, says Sinha. This could be achieved by engaging deeper layers of skin and its resident stem cells to partake in tissue regeneration.

Research is already underway at RenovaCare to enable treatment of third-degree burns, which Bold describes as definitely within the range of possibility. Bold claims the adaptations to the SkinGun would allow it to treat other damaged organs using a patients stem cells, but for now the company is focusing solely on burns and wounds to skinthe largest organ of the human body.

Urams burns are now completely unnoticeable. There is no scar tissue or even pigment discoloration, and the regenerated skin even tans. If I show someone where I was burnt, I bet $100,000 they couldnt tell, he says. Theres no scars, no residual pain; its like the burn never happened. Its a miracle.

Uram is frustrated that the treatment is not available to other burn victims, particularly children. I want to see the FDA get off their butts and approve this, he says. A grown man like me to be scarred is OK, but think about the kids that have to live the rest of their lives with pain and scarring. Thats not OK.

Read more here:
Spray-On Skin: 'Miracle' Stem Cell Treatment Heals Burns Without ... - Newsweek

Over recent years, I have seen a growing interest in stem cells and a particular preparation called Platelet Rich Plasma (PRP). Many famous athletes including Tiger Woods have received PRP for various musculoskeletal problems and some have credited it with their accelerated healing and more rapid return to play.

PRP is plasma, the liquid part of blood, concentrated with many more platelets than typically found there. Platelets are known for their importance in clotting blood, but they also contain hundreds of proteins called growth factors. These are responsible for the cascade of events naturally involved in tissue repair. Your own innate stem cells are attracted to the site of injury and play a critical role in the healing process.

Typically, PRP is isolated from your own blood, drawn in the office while you wait. The highly concentrated growth factors are then delivered back into the body at the site of interest. PRP injections have been used for musculoskeletal problems such as sprained knees, osteoarthritis, and chronic tendon injuries. Previously, these types of conditions were treated with medications, physical therapy, and surgery, but PRP recipients commonly report less pain and stronger, more stable joints. It may even promote new cartilage formation in aging joints enabling you to put off joint replacement surgery.

PRP can also be very effective in treating chronic tendon injuries, especially tennis elbow, a common injury of the tendons on the outside of the elbow. Previously, cortisone injections were commonly used, but we know steroids will ultimately weaken tendons and promote rupture. In contrast, now PRP treatments lead to stronger tendons.

Promoting healing after tendon surgery is another use for PRP. For example, an athlete with a completely torn heel cord may require surgery to repair the tendon. Healing of the torn tendon can potentially be improved by treating the injured area with PRP during surgery. With a shorter recovery time, less chronic pain and stronger tissue, you can see why athletes love PRP!

More recently, PRP is being used extensively in aesthetic medicine to keep us looking younger and to promote hair growth. In the same ways the growth factors in PRP facilitate tissue repair from injury or surgery, they also regenerate aging skin. PRP injected into the facial skin has been called the vampire facial made famous by some Hollywood stars.

Today we use a more advanced technique called micro-needling. The PRP is layered across your face and delivered to the skin using a handheld device called a Micropen. This device has 12 tiny micro-needles that drive the PRP in, calling in the tissue repair team to get to work! The result is accelerated collagen production with new, thicker, stronger collagen. The procedure is well tolerated and done in the office while you are awake. It takes less than a couple of hours to complete and usually two to three treatments are recommended spaced four to six weeks apart. The collagen repair process can take four to six weeks, we expect to see the full results blossom over the course of months and continue to improve over a year.

The best thing about PRP Micropen Facelift is that theres not serious downtime like you get with laser resurfacing or surgery. Plus, unlike dermal fillers, which will fade in months, these results will continue to improve over the year. Most commonly we treat faces, but the procedure is safe to use all over the body including necks, chests, hands, and even eye lids. It is also quite helpful for minimizing and fading stretch marks.

If you have any questions or want to learn more about PRP for musculoskeletal or skin rejuvenation, please plan to attend our free interactive community lecture on this topic at the Coronado Library Winn Room from 6-7PM on Thursday 04/06/2017!

Lauren Mathewson, ND is Board certified in naturopathic medicine and Patrick Yassini, MD is board certified in family medicine, integrative and holistic medicine. They practice at Peak Health Group, 131 Orange Avenue, #100, Coronado, Calif.; the office number is (619) 522-4005.

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Now You Can Harness Your Own Stem Cells - Coronado Eagle ... - Coronado Eagle and Journal

By Paulina De Alva, Prosper Press

The fifth annual Dancing With Dominic fundraiser, which benefits the research to find a cure for Hunter Syndrome, was hosted on Saturday, April 1, 2017 at Hughes Elementary School by the Henriquez family, whose 7-year-old son Dominic was diagnosed with the disease in October of 2011, when he was 22 months old.

The event included a dance in the gymnasium, performances by Prosper ISDs dance teams, face painting by the art students, a catered dinner, a silent auction, a raffle, kids activities and crafts, a photo booth and other activities for all the families.

Dominics mother, Jeanette Espinola, said she is incredibly thankful for the amount of community support shes seen during the planning process, and that the event was made possible all because of the help and support of the community of Prosper. She added that about 15 or 16 families from different parts of the country who have been affected by the disease attended the event.

We had a family gathering in conjunction with the event, she said. Because theres so few of us, were a very close community.

Hunter Syndrome, or Mucopolysaccharidosis Type II (MPS II), is a rare genetic disorder affecting 1 in 150,000 males that slowly destroys the bodys cells due to a missing enzyme, which results in the accumulation of cellular waste throughout the body. It is a progressive and life-limiting disease that mainly affects young boys, and the prognosis is that the children wont live past their teenage years. Espinola said it was a devastating diagnosis for her family.

All of a sudden you lose basically all your dreams that you had for your child, Espinola said. You were going to see him grow up, see him become an adult. But he may not make it past his teenage years.

She said the way she dealt with it was to do what she loves to do and plan to host a big fundraiser party along with her husband, who is a DJ, and with the help of the community in Virginia, where they lived before they moved to Prosper. Dominic loves to dance, so that party in 2012 became the first Dancing With Dominic event.

It was our way of contributing and helping find a cure, and bringing awareness, Espinola said. I think thats the other huge piece of it, is bringing awareness that there are these disorders, there are these kids, and that there is this potential right now to really help them, and the research is pretty much there, we just need the funds right now.

Dominics parents, Jeanette Espinola and Freddy Henriquez, founded the Hunter Syndrome Foundation in 2013, after having hosted already two Dancing With Dominic events, for the specific purpose of funding potential treatments and research and ultimately finding a cure for the disease.

There is one approved treatment for Hunter Syndrome. It consists of an infusion of the man-made version of the enzyme Dominic is missing, which is administered through a four-hour weekly IV treatment that prevents the disease from progressing fast. The medicine he gets, called Elaprase, costs about $12,000 per week, amounting to around half a million dollars per year, and is the second most expensive medicine in the country.

Hes been getting that for five years now since he was diagnosed, Espinola said. But the issue with that is that it doesnt cross into his brain. So he could still lose his cognitive skills, he could still begin regressing.

Its not a complete treatment, so for the past two years hes been in a clinical trial in Chicago where hes getting the enzyme to his brain. It helps in slowing down the progression of the disease in his brain.

Researchers have been searching for a permanent cure, so gene therapy is the next step they are working toward. The gene therapy research for Hunter Syndrome is led by two doctors, Douglas McCarty, Ph.D., and Haiyan Fu, Ph.D. at Nationwide Childrens Hospital in Columbus, Ohio. All of the Hunter Syndrome Foundation funds have benefited their work to find a cure. Dr. McCarty said the gene therapy for MPS II is the result of more than a decade of collaborative research efforts with support from MPS II patient family foundations.

This gene therapy approach targets the root cause of MPS II by delivering the correct gene using a vector that can cross the blood-brain-barrier, McCarty said. Our preclinical data have shown great promise with lifelong benefits. We believe that we are well positioned to move forward towards a phase 1/2 clinical trial in patients with MPS II.

The vector for the gene therapy will cost $1.4 million to produce, and it will cost another million dollars to begin the clinical trials. The family donates the money raised from the yearly Dancing with Dominic event to the Hunter Syndrome Foundation, and through it, 100 percent of the funding goes toward the doctors research at Nationwide Childrens Hospital so they can find a cure.

Im not doing this by myself, there are families throughout the country who are also raising funds, Espinola said. So all of the families efforts put together throughout the country have raised over $500,000 so far to help the doctors in their research.

Espinola said she hopes the family-led efforts are able to fully fund the clinical trials for gene therapy and for some normalcy for her son, Dominic, in the future.

I hope that Dominic continues to do well and better treatments are found, she said. Maybe one day he can be an adult and lead somewhat of an independent life.

See the article here:
Prosper nonprofit holds fundraiser to help research cure for Hunter Syndrome - Nueces County Record Star

Researchers may have brought us one step closer to gene therapy for the treatment of hearing loss, after discovering a way to regenerate auditory hair cells in mice.

It is estimated that about 15 percent of adults in the United States have some form of hearing loss, with men being twice as likely to develop the condition than women.

Damage to the auditory hair cells is one of the leading causes of hearing loss.

Aging is a common risk factor for such damage, although the ailment can also arise through prolonged exposure to loud noise, injury (such as head trauma), ear infections, and other illnesses and diseases.

Auditory hair cells are the tiny sensory cells of the cochlea the inner part of the ear that enable us to hear.

These cells consist of hair-like projections, called stereocilia, that are responsible for transforming sound vibrations into electrical signals that are sent to the brain.

In humans, auditory hair cells are unable to regenerate in order to replace damaged ones. In fish and birds, however, these cells can regenerate.

The process involves down-regulating expression of the protein p27 and up-regulating the expression of the protein ATOH1, notes study co-author Jian Zuo, Ph.D., of the Department of Developmental Neurobiology at St. Jude Childrens Research Hospital in San Francisco.

For their study published today in the journal Cell Reports Zuo and team set out to determine whether they could trigger the same process in mice.

Read More: Get the facts on age-related hearing loss

Using genetic manipulation, the researchers deleted the p27 protein and increased ATOH1 expression in mice.

When the mice experienced auditory hair cell damage as a result of exposure to loud noise, the researchers found that the cells supporting the auditory hair cells began to transform into auditory hair cells themselves.

Further investigation revealed that a number of proteins work together in order to regenerate auditory hair cells.

The researchers found that the deletion of p27 increased levels of a protein called GATA3 and boosted the expression of the POU4F3 protein. This increased ATOH1 expression, leading to auditory hair cell regeneration in the rodents.

The researchers explain that ATOH1 is a transcription factor required for the development of auditory hair cells. In humans, the production of ATOH1 stops in the womb.

According to Zuo and colleagues, however, their findings suggest that it may be possible to reactivate ATOH1 production in humans by genetically manipulating the p27, GATA3, and POU4F3 proteins.

Work in other organs has shown that reprogramming cells is rarely accomplished by manipulating a single factor," said Zuo. "This study suggests that supporting cells in the cochlea are no exception and may benefit from therapies that target the proteins identified in this study."

The researchers now plan to conduct a phase I clinical trial that will involve using gene therapy to reinstate ATOH1 production in humans.

The aim is to determine whether such a strategy can trigger auditory hair cell regeneration in humans, and whether this might be an effective treatment for hearing loss.

"Work continues to identify the other factors, including small molecules, necessary to not only promote the maturation and survival of the newly generated hair cells, but also increase their number," said Zuo.

Read More: What? Hearing loss expected to rise dramatically

Read the rest here:
Gene Therapy May Help Those With Hearing Loss - Healthline

NEW YORK, April 10, 2017 /PRNewswire-iReach/ -- Physician and business leader Dr. Charles Mok today announced the publication of Testosterone: Strong Enough for a Man, Made for a Woman (available now). The book is published with ForbesBooks, the exclusive business book publishing imprint of Forbes Media.

In the book, Dr. Mok, who has persistently followed the research on hormone replacement therapy (HRT) and its health benefits for 30 years, confronts medical professionals for their slow integration of testosterone into HRT. With support from a wealth of peer-reviewed studies, he shows how testosterone therapy can help women facing menopause maintain their weight, increase their sexuality, and reduce the health risks associated with aging.

In his new book, Dr. Mok describes the dramatic transformation HRT has undergone in the past several decades. For years, synthetic hormones were widely prescribed to treat women in the United States experiencing menopause. HRT clinical trials in the early 2000s called the practice into question after observing an increase in health risks in women taking synthetic hormones. Those safety concerns, Dr. Mok argues, were valid, but misleading. Today's treatment protocolsusing natural hormones such as testosteroneare entirely different.

"Clinical studies show that testosterone therapy reduces the risk of breast cancer by 50 to 75 percent and relieves virtually all symptoms of menopause with no adverse effects," said Dr. Mok. "The benefits of implementing testosterone into hormone replacement therapy are virtually unprecedented, and yet the larger medical community continues to ignore the facts."

The book Testosterone: Strong Enough for a Man, Made for a Woman is now available for purchase on Amazon.com.

About ForbesBooks

Launched in 2016 in partnership with Advantage Media Group, ForbesBooks is the exclusive business book publishing imprint of Forbes Media, the 99-year-old global media, branding and technology company. ForbesBooks offers business and thought leaders an innovative, speed-to-market publishing model and a suite of services designed to strategically and tactically support authors and promote their expertise. For more information, visitwww.forbesbooks.com.

About Dr. Charles Mok

A speaker, author and authority in his field,Dr. Charles Mok has dedicated his life to helping patients gain confidence, feel younger and live a healthier lifestyle. After receiving his medical degree, Dr. Mok began a career in emergency medicine, eventually working as the vice chairman of the emergency department at Mt. Clemens General Hospital, now known as McLaren Macomb. During these years, he saw countless patients with health emergencies that were fully preventable. In 2003, Dr. Mok founded Allure Medical Spa, one of the largest and most successful medical practices in the state of Michigan, to improve the lives of patients with treatments including varicose veins, hair loss and fat reduction, cosmetic surgery, stem cell therapy, and more. His mission is to reveal the clinical research supporting natural hormone therapy's safety and effectiveness to educate and change the lives of many women for the better. For more information, visit http://www.drcharlesmok.com.

Media Contacts

Mary Scott, Allure Medical Spa, mscott@alluremedicalspa.com, (313) 3784651

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Dr. Charles Mok Releases Innovative New Book on Women's Health - Yahoo Finance

Travis Rathbone

Helpful solutions to issues with sleeping well.

Hot flashes may be keeping you up and insomnia can actually get worse after menopause. The reason: Various age-related ailments can lead to sleep issues, and so can the medicines we take for them, says internist and sleep-medicine specialist Raj Dasgupta, spokesperson for the American Academy of Sleep Medicine. "Depression is linked to insomnia, for example, but the drugs we prescribe for it selective serotonin reuptake inhibitors can also cause insomnia." Try this...

Sleep in a cave. Get blackout shades for your bedroom, cover power lights on electronics and dim your clock's display. Keep your room at a cool 60 to 65 degrees, which can help fend off hot flashes.

Ban gadgets in bed. The blue light from your smartphone or tablet screen suppresses melatonin, the sleep-inducing hormone. A study in the journal PNAS showed that people who read print books right before bed slept better than those who read e-books on a tablet.

Don't just lie there. When you're counting sheep, get up, go to another room and do something low key, like knitting or reading (no TV!), until you feel sleepy again.

Act now if: Bad sleep is affecting your ability to function during the day. Insomnia can be a symptom of other health issues, such as arthritis, hyperthyroidism and acid reflux. It also increases your risk of serious accidents. Tell your doctor about it.

Read the original here:
Mysteries of the Female Body - AARP News