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

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

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

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

About Applied StemCell, Inc.

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

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

(Courtesy of Linda Cicero).

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

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

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

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

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

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

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

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

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

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

Contact Aulden Foltz at afoltz at stanford.edu.

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Researchers develop controllable gene therapy, make rats glow ... - The Stanford Daily

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FOX 61

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

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Sickle cell anemia patient 'cured' by gene therapy, doctors say - FOX 61

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

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

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

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

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

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

Increase in federal funding in stem cell therapy.

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

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

Growing demand for personalized medicine.

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

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

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

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

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

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Opinion/Commentary: Global stem cell therapy market to showcase ... - The Daily Progress

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Science Daily

Scientists wage fight against aging bone marrow stem cell niche Science Daily Red staining reveals the abundant presence of the protein osteopontin (OPN) in bone and endosteum of the marrow cavity, which is important to maintaining a healthy environment for blood-forming hematopoietic stem cells. Shown in blue are cell nuclei. and more »

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Scientists wage fight against aging bone marrow stem cell niche - Science Daily

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Almost 30 years ago, the federal government helped make it easier for patients with leukemia and lymphoma to receive lifesaving stem cell transplants. Now, we need the federal governments help again to ensure that Medicare patients with these cancers and other serious blood disorders can access the care they need.

In 1987, Congress approved funding for a national database of patients willing to donate bone marrow or peripheral blood stem cells. That database is now known as the Be The Match Registry, operated by the National Marrow Donor Program/Be The Match. According to the NMDP/Be The Match, patients searching the registry have access to 27 million potential volunteer bone marrow and peripheral blood stem cell donors worldwide, along with more than 680,000 units of cord blood donated by mothers after giving birth.

Having access to such a large registry has made it easier for patients to find a match if they dont have a fully matched sibling donor, which is the case for about 70 percent of patients who receive a stem cell transplant. The registry has helped 80,000 patients receive bone marrow transplants, peripheral blood stem cell transplants, or cord blood transplants from an unrelated donor.

While the federal governments foresight and financial support have helped make adult stem cell and cord blood transplants the only cure available for these diseases possible for thousands of patients, Medicare coverage policies have not kept pace with this breakthrough treatment.

Medicare is more restrictive than private insurance companies in deciding for what indications stem cell transplants and cord blood transplants will be covered. With private insurance companies, we have the opportunity to talk with a medical director about the indication and provide literature to support the decision for a transplant. This opportunity is not available for our Medicare patients.

In most cases, Medicare doesnt decide whether to cover a stem cell or cord blood transplant until after the procedure is completed. This leaves most Medicare patients an impossible choice: Turn down their only chance at a cure or potentially face paying the significant cost of a transplant themselves. Even when Medicare does decide to reimburse for these transplants, according to the NMDP/Be The Match, it covers less than half the cost of the transplant.

Addressing this issue is especially important because seniors make up a large portion of the patients with the cancers and blood diseases that can be cured by a stem cell or cord blood transplant. For example, 24 of the 65 patients who received stem cell or cord blood transplants at University of Virginia Health System in 2016 had Medicare coverage.

So I am asking the Centers for Medicare & Medicaid Services to expand Medicare coverage for stem cell and cord blood transplants, along with paying for the search and procurement costs as they already do for solid organ transplants.

The federal government has helped save the lives of tens of thousands of patients through better access to stem cell and cord blood transplants. I hope now they will act to make sure all Medicare patients who need one of these transplants can receive it.

Tamila L. Kindwall-Keller, DO, MS is the associate clinical director of the Stem Cell Transplant Program at the University of Virginia Health System.

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Opinion/Commentary: Seniors put at risk by outdated Medicare policies - The Daily Progress

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By Jessica Williams / Times-News

Shawna Hamlett will be born again June 8.

The 40-year-old Alamance native and single mother was diagnosed with relapsing remitting multiple sclerosis 13 years ago, and has dealt with episodes of numbness, sudden blindness, deafness, difficulty walking, pain and extreme fatigue.

But May 1, Hamlett will travel to Chicagos Northwestern Hospital to undergo a month-and-a-half-long experimental procedure called hematopoietic stemcell transplant, which uses stemcells from the patients own bone marrow, along with chemotherapy, essentially toreset the immune system.

By the time she receives the stemcell transplant June 8, she will have lost her hair and nearly all of the antibodies built up from vaccines that shes received since birth, but will no longer have multiple sclerosis.

This is mind-boggling to me, Hamlett said. My body wont remember that I had MS at all because my stemcells are going to be frozen [for] two weeks, and when they give them back, they dont know they had MS ever. The chemo completely reboots your immune system so much so that the vaccines you got when you were an infant your body no longer has.

She applied for the procedure in November and was, to her surprise, approved for it in late January. It couldnt come a moment too soon, Hamlett said, as the neurologist in Chicago told her during a consultation that shes one legion away from a catastrophic event maybe even paralysis, an even greater concern considering that she has two sons, 13 and 17.

I just wish I had done this years ago, when the doctor first started doing it, because I could have had their whole childhood without having it, and Ive had to struggle at ballgames. Ive been on the sidelines at soccer games with an IV in my hand because Ive had an IV at home and Im not going to miss their games, Hamlett said. But theyre going to grow up, and theyre going to get married and have kids, and I dont want to be not able to be around.

AS WITH ANY MEDICAL procedure, a heavy cost is involved. Along with spending the better part of nearly two months away from her kids, the longest shes ever been away from them, Hamlett will have to pay $25,000to $35,000 after insurance.

Shes enlisted help from community businesses, friends and family to help cover that expense, and has plans for seven fundraisers.

Hamlett says community support is essential for people with MS, and she would advise anyone just diagnosed to seek out others.

Talk to people who are in the same boat as you, she said. Theres a huge community of people on both Instagram and Facebook that suddenly become your family because nobody, we always say, nobody gets it until they get it. People who understand exactly what youre going through are out there, so youre not ever alone.

REGARDLESS OF HOW much money is raised, Hamletts life is about to change for the better. Shes nervous, but eager, and says its going to be a huge relief when shes fully-recovered.

The thing about MS is you never know from day to day how its going to be the next day, which is good and bad because, if its bad, then tomorrow could be a good day, but if its good, then tomorrow could be a bad day, she said. Id like to be on an even keel and just know that after the procedure there wont be any more unknowns.

Hamlet has set up a donation page through HelpHOPELive for donors to contribute to the cost of the procedure and hotel expenses for her time in Chicago. Visit https://helphopelive.org/campaign/12529 or search Shawna Hamlett HelpHopeLive to find out more.

All donations are tax-deductible, held by HelpHOPELive in the South Atlantic Stem Cell Transplant Fund, and administered by HelpHOPELive for transplant-related expenses only.

Reporter Jessica Williams can be reached at jessica.williams@thetimesnews.com or at 336-506-3046. Follow her on Twitter at @jessicawtn.

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'Reset' for the immune system - Burlington Times News

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International Stem Cell Corporation Announces Third Patient with Parkinson's Disease in Phase I Clinical Trial P&T Community 28, 2017 (GLOBE NEWSWIRE) -- International Stem Cell Corporation (OTCQB:ISCO), a California-based clinical stage biotechnology company developing stem cell-based therapies and biomedical products, today announced the third patient in the clinical trial ... and more »

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The company proposing Australia's first cryonics lab has gained approval to build in Holbrook, southern NSW, and plan to begin freezing and stories bodies next year.

Approval has been granted for the world's second cryonics facility outside the United States to be built in Holbrook.

Building is set to start now the plans have been given the tick by Greater Hume Shire Council and by next year Southern Cryonics plans to begin storing and freezing dead bodies in the expectation that in the future science will be able to bring them back to life.

Company secretary Matt Fisher and his team of four had hoped to unveil a facility in 2014 under the company name Stasis Systems, but ran into difficulties.

In the intervening years, despite there still being no scientific guarantee of revival, Australians had warmed to the idea of cryonics.

"We have had quite a lot of people express interest, perhaps a dozen at this stage, that want to sign up as clients once we are up and running," he said.

A price has not been set for the service but Mr Fisher said whole body preservation would cost $A80,000-$90,000.

The facility will have the capacity to store 40 bodies in 10 specialised stainless steel vessels.

It is hard to get a clear picture of how many people have been cryopreserved to date as there is no system of recording this information. However, there are estimated to be several hundred in the US and Russia where facilities exist.

It has been a long road, but Mr Fisher said it was essential to find an appropriately zoned site for cemetery and mortuary use, in a location with low risk of disaster and bushfire.

Safeguarding the facility was a priority, as was developing a corporate structure to survive as long as the built one.

Greater Hume Council general manager Steven Pinnuck said there were no objections to the development but to satisfy the terms of the approval, Southern Cryonics needed to seek licenses from NSW Health to hold and store remains on site.

"It is certainly a different type of activity. We are quite comfortable with it," he said.

"It's going to be in an industrial area and as it turns out, it will be almost adjacent to the local cemetery so we don't see it as being out of character with the area."

"The patient has to be declared legally dead for any cryopreservation procedures to begin," Mr Fisher said.

"The patient is put in an ice bath and medications are administered to prevent blood clotting."

Bodies are brought down to dry ice temperature (-78.5 Celsius) as a temporary phase.

"Once they get to the facility, Southern Cryonics would take over and bring that down further to liquid nitrogen temperature which is -196 Celsius."

The rule of thumb with cryonics was the faster the better and the colder the better.

The focus of cryonics is to preserve the brain to the highest fidelity so deaths with trauma to the brain or head or degenerative conditions such as dementia were problematic.

Mr Fisher said while there were known concerns which would limit the success of a possible future revival, clients would not be medically assessed by Southern Cryonics.

The elderly and others with illnesses had made inquiries but Mr Fisher said a growing number of young people were keen to know more, particularly as it was soon to be a real third end-of-life option.

Mr Fisher, a software engineer, had his father's brain frozen - or what's called neurally coded - at a facility in Sydney.

His passion for cryonics stems from the assumption that medical technology will improve to the point where people can live "in a healthy physical state in perpetuity", meaning theoretically that life expectancy would become open-ended.

"Anyone who has died in the years leading up to that point is going to miss out on the amazing opportunity of experiencing being fit and healthy for however long that they want to," he said.

"I would like to be on the other side of that transition and want everyone I know and care about to be on the other side of that transition as well."

The story Building set to start on Australia's first cryonics lab first appeared on The Sydney Morning Herald.

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Gene therapy is the use of DNA as a potential therapy to treat a disease.In many disorders, particularly genetic diseases caused by a single genetic defect, gene therapy aims to treat a disease by delivering the correct copy of DNA into a patients cells.The healthy, functional copy of the therapeutic gene then helps the cell function correctly.

In gene therapy, DNA that encodes a therapeutic protein is packaged within a vector, often a naked virus, which is used to transfer the DNA to the inside of cells within the body. Gene therapy can be delivered by a direct injection, either intravenously (IV) or directly into a specific tissue in the body, where it is taken up by individual cells. Once inside cells, the correct DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patients disease and can provide long-term benefit.

Abeona is developing next generation adeno-associated virus (AAV) gene therapies. Viruses such as AAV are utilized because they have evolved a way of encapsulating and delivering one or more genes of the size needed for clinical application, and can be purified in large quantities at high concentration. Unlike AAV vectors found in nature, the AAV vectors used by Abeona have been genetically-modified such that they do not replicate. Although the preclinical studies in animal models of disease demonstrate the promising impact of AAV-mediated gene expression to affected tissues such as the heart, liver and muscle, our programs use a specific virus that is capable of delivering therapeutic DNA across the blood brain barrier and into the central nervous system (CNS), making them attractive for addressing lysosomal storage diseases which have severe CNS manifestations of the disease.

Lysosomal storage diseases (LSD) are a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. These diseases are characterized by progressive accumulation of storage material within the lysosomes of affected cells, ultimately leading to cellular dysfunction. Multiple tissues ranging from musculoskeletal and visceral to tissues of the central nervous system are typically involved in disease pathology.

Since the advent of enzyme replacement therapy (ERT) to manage some LSDs, general clinical outcomes have significantly improved; however, treatment with infused protein is lifelong and continued disease progression is still evident in patients. Thus, viral gene therapy may provide a viable alternative or adjunctive therapy to current management strategies for LSDs.

Our initial programs are focused on LSDs such as Mucopolysaccharidosis (MPS) IIIA and IIIB, also known as Sanfilippo syndromes type A and type B. MPS III is a progressive neuromuscular disease with profound CNS involvement. Our lead product candidates, ABO-101 and ABO-102, have been developed to replace the damaged, malfunctioning enzymes within target cells with the normal, functioning version.

Delivered via a single injection, the drug is only given once.

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Gene Therapy - Abeona Therapeutics

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RALEIGH The North Carolina Biotechnology Center revealed Tuesday that pharmaceutical giant Pfizer Inc. has committed $4 million to the center to establish multi-year academic fellowships to aid the gene-therapy sector in the state.

The new program, which will be managed by N.C. Biotech, will support distinguished postdoctoral fellowships in North Carolina university research laboratories focused on gene therapy research.

The $4 million grant was announced during the CED Life Science Convention being held in Raleigh this week.

Pfizers contribution represents another step in its relationship with the state. The company operates a pharmaceutical manufacturing facility in Sanford, and last August, it purchased the Chapel Hill-based gene-therapy company Bamboo Therapeutics Inc. for $645 million.

Jude Samulski, director of the Gene Therapy Center at the University of North Carolina at Chapel Hill co-founded Bamboo. Samulski was recruited to UNC in 1993 as part of a $430,000 N.C. Biotech grant and Bamboos former parent company received more than $700,000 in Biotech Center grants and loans.

The announcement of the fellowship is an indicator that Bamboo is still committed to the area after the Pfizer acquisition.

The funding provided by Pfizer will enable N.C. Biotech to create two-year fellowships for postdoctoral scientists. The funding will cover salaries, benefits, materials, professional development and travel for the scientists. Information about the fellowships can be found at http://www.ncbiotech.org/pfizer-fellowships.

Pfizer embraced the opportunity to work with us given weve proven for more than 30 years that we have the expertise and success metrics to maximize impact, Biotech Center CEO Doug Edgeton said in a statement. We not only have outstanding research institutions across our state, but we also have a well-respected culture of partnering and collaboration that allows us to be nimble and responsive.

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Recent advancements in stem cells research have given hope for successfully treating diabetic heart disease (DHD), renowned New Zealand-based researcher in cardiovascular diseases Dr Rajesh Katare said on Tuesday.

DHD affected the muscular tissues of the heart leading to complications and it had been demonstrated that resident stem cells of myocardium can be stimulated to repair and replace e degenerated cardiac myocytes resulting in a novel therapeutic effect and ultimately cardiac regeneration, he said.

Katare, Director of Cardiovascular Research Division in the University of Otago, New Zealand, was delivering the keynote address at the continuing medical education programme on "Role of Micro-RNAs and stem cells in cardiac regeneration in diabetic heart disease" at the Karaikal campus of premier health institute JIPMER.

Presenting clinical evidences, Katare said stem cell therapy certainly presented a new hope for successfully treating DHD.

Jawaharlal Institute of Post Graduate Medical Education (JIPMER) Director Dr Subash Chandra Parija pointed out that it was the first such programme on the role of stem cells in cardiac regeneration in the whole of the country.

He said as diabetes was highly prevalent in the country, providing treatment for DHD had become a big challenge. Patients suffering from the condition have to undergo lifelong treatment and medications. "In this backdrop, advancements in stem cell therapy assume significance," he said. (REOPENS MES10)

Parija also said the government general hospital in

Karaikal being currently used by JIPMER for clinical teaching of students would have upgraded facilities.

He said a new building for the college would be constructed at a cost of Rs 497.10 crore soon.

The proposed up-gradation of the GH having 506 beds would help in imparting advanced clinical teaching and effective exposure of the medicos to various nuances of the diagnosis.

The Director also said JIPMER (Karaikal) had drawn up special post-graduate and fellowship programmes including on family medicine, tropical medicine, trauma care and cancer management.

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Stem cell therapy can help in treating diabetic heart disease - Business Standard

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In a world first, a teenager with sickle cell disease achieved complete remission after an experimental gene therapy at Necker Childrens Hospital in Paris, researchers say.

People with sickle-cell disease, a group of inherited blood disorders, have abnormal hemoglobin in their red blood cells, causing blood to clog in the tiny vessels and organs of the body.

After 15 months since treatment, the patient who began therapy at age 13 no longer needs medication, and his blood cells show no further sign of the disease, according to a case report published Thursday in The New England Journal of Medicine.

Since therapy was applied, he hasnt had any pain, any complications. He is free of any transfusions. He plays sports and goes to school, said Dr. Philippe Leboulch, an author of the new research and a professor of medicine at the University of Paris. So we are quite pleased with the results.

This success provides proof of concept for human patients, Leboulch said.

According to Dr. Marina Cavazzana, senior author of the study and head of the biotherapy department at Necker, all the biological tests we perform lead us to think he is cured. Yet, she added, the answer to the question of whether he is truly cured can be provided only by the longer follow-up.

Still, hopes are running high that patients with this very devastating disease can receive this therapy in the next five years, Cavazzana said. This is our hope, and we work very hard to attain it.

A global burden

Worldwide, more than 275,000 infants are born with sickle cell disease each year. In the United States, approximately 100,000 people, most of African ancestry or identifying as black, currently have it. About one in every 365 black children in the US is born with sickle cell disease, for which the life expectancy is now about 40 to 60 years.

Sickle cell disease is one of the most common gene disorders in the world, explained Leboulch. A genetic mutation causes hemoglobin, the main constituent of red blood cells, to distort the shape of the cell, and this causes the blood to aggregate or clog.

This leads to tremendous pain, anemia and also lesions of organs that ultimately result in shortness of life expectancy, Leboulch said. So what we did here was, we tried to inhibit the process of aggregation.

Essentially, researchers extracted bone marrow from the patient, harvested the stem cells and altered the genetic instructions so that they would make normal hemoglobin. Next, they treated the patient with chemotherapy for four days to eliminate his diseased stem cells. Finally, they returned the treated stem cells via an IV into his bloodstream.

At that point, the new cells that were modified outside the body started to make new blood cells, and we hope this will be stable for the life of the patient, Leboulch said.

Before receiving treatment, the teen had terrible pain and needed blood transfusions, which required twice-yearly hospitalizations, Leboulch explained. His many complications included necrosis of the hip, which necessitated hip replacement surgery.

Hope for all patients

Going forward, the plan is to proceed through clinical trials and, if results are promising, make the treatment available to patients. Leboulch and his colleagues are using the same genetic therapy to treat a similar disease called thalassemia, another inherited blood disorder in which patients have less hemoglobin and fewer red blood cells than normal. Severe forms require regular blood transfusions.

Leboulch and his colleagues have global phase 2 and phase 3 trials for the thalassemia treatment underway in France, the US, Australia and Thailand.

For sickle cell disease, a companion trial in the US is underway. I understand that seven (sickle cell) patients have been treated already. Of course, the outcome is much shorter, and we dont have the results just yet, but its coming along, Leboulch said.

To apply this to a baby or a very young child should be at least as effective or more, he said. Doing it with older patients, who have had years of complications, could be more challenging.

Leboulch also noted that gene therapy is easier on patients than procedures requiring outside donors. Previously, hematopoietic stem cell transplant, which replaces a patients bone marrow with that of a donor, has proved an effective cure for some patients.

According to Dr. Alexis Thompson, president-elect of the American Society of Hematology, the majority of sickle cell disease patients do not have a sibling who would be an appropriate match for bone marrow donation.

Gene therapy holds promise because a patient serves as his own donor, and the risks are much reduced since theres no possibility of a mismatch, said Thompson, who was not involved in this research but is an investigator on a related gene therapy study.

I think this is a really very exciting advancement, she said, adding that if the results seen in France can be duplicated, this would provide for a new direction for patients who need a curative option.

According to Dr. Trish Wong of Oregon Health and Science University, the new study is truly amazing work proof of principle that a cure for this chronic, devastating disease is in sight. Wong was not involved in the new research.

Gene therapy offers hope for all patients with sickle cell disease, regardless of whether they have a bone marrow match or not, Wong wrote in an email.

Time is still needed to assess the success of this treatment and the possibility of later side effects, said Wong. But any patient with severe sickle cell disease will tell you that being able to live a life for even a year without medications or fear of pain or hospitalization is substantial.

Finally, Dr. Grace Onimoe of the American Sickle Cell Anemia Association noted that the life expectancy of a patients with sickle cell disease remains decades lower than that of the general population while children throughout the world continue to suffer. Onimoe, who was not involved in the new research, said, As more work continues in the area of gene therapy to enhance safety and reduce potential complications, we remain optimistic of the promise it holds.

Leboulch also feels very hopeful.

Now, we want to be cautious, of course, and we dont want to say that this is the cure for tomorrow or the next day for everybody, he said. At the same time, what weve observed is really convincing, and we just hope that we can move this along to make it available to patients.

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Sickle cell anemia patient 'cured' by gene therapy, doctors say - wtkr.com

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WEDNESDAY, March 1, 2017 (HealthDay News) -- Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow -- causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant.

In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells -- which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible -- and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team, led by Dr. Marina Cavazzana, of Necker Children's Hospital's biotherapy department, focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait -- that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin -- to replace the abnormal "adult" hemoglobin seen in sickle cell.

The hope, Williams said, is that gene therapy will ultimately offer a one-time treatment that cures sickle cell. But no one knows yet whether that will happen.

According to Williams, two key questions are: What's the long-term safety? And will the altered stem cells last for a patient's lifetime?

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published March 1 in the New England Journal of Medicine.

See original here:
Gene Therapy: A Breakthrough for Sickle Cell Anemia? - Lincoln Journal Star

Recommendation and review posted by Bethany Smith

Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

RELATED: TRENDING LIFE & STYLE NEWS THIS HOUR

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the U.S., about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant. In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team led by Dr. Marina Cavazzana focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin to replace the abnormal "adult" hemoglobin seen in sickle cell.

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published in March in the New England Journal of Medicine.

Visit link:
Gene therapy shows early promise against sickle cell - Chicago Tribune

Recommendation and review posted by sam

An Edmonton police officer is counting on the city he serves to help save his sons life.

Brady Mishio, 8, has an aggressive form of leukemia and needs a bone marrow transplant. His dad Terry desperately hopes someone who is a match will hear his plea.

Bradys family received the news of his illness in November after they took him for treatment of a persistent fever and flu.

You become that family that's searching for hope and have all these dreams and things for your children, and then one day, one day it's taken away from you so quickly, said Mishio.

The diagnosis was a form of cancer that starts in blood stem cells called acute myelogenous leukemia (AML). It is the most common leukemia found in adults but is less common in children. Brady began chemotherapy right away.

Four days into chemo he had a reaction to some of the drugs that they were giving him and he quit breathing and had a seizure, said Mishio, a 20-year veteran of the Edmonton Police Service who was once forced off work for a year with a brain injury after being kicked while making an arrest.

Bradys doctors found a drug combination that worked and the boy just finished his third round of treatment. He is in an isolation unit in hospital and the next step is a bone marrow transplant.

It gives Brady a second chance at life, at cleaning out his bone marrow and hopefully eliminating the cancer, said his father, while fighting tears.

Bradys family is not a match and Mishio is reaching out publicly, hoping he can help his child by finding a suitable donor. The test is a simple mouth swab. Donors must be between 17 and 35 and be in relatively good health, says Robyn Henwood, a stem cell territory manager for Canadian Blood Services. Younger people have fewer antibodies in their blood, lowering the risk of rejection.

Once registered, potential donors stay in the agencys registry until they are 60 years old.

Less than 25 per cent of patients find a bone marrow match in their own family, says Henwood.

So every single person we add to our database is giving hope to those who are looking for a match or who are likely going to die if we don't find them one.

In most cases, the process to donate bone marrow is much like giving blood and takes four to six hours to complete.

A clinic is being held Thursday, March 9 from 4 p.m. to 8 p.m. at Holy Cross Ukrainian Catholic Church. Interested donors who cant make that clinic can be tested at the Canadian Blood Services clinic next to University Hospital or register at blood.ca to be mailed a swab kit.

Mishio is amazed by the bravery of his son through this battle.

He's a fighter and there's many days where he'll be rubbing my back and kind of telling me it's going to be OK, and that's when I'm like, 'I got to be strong for him.'

Read more from the original source:
Edmonton police officer appeals for bone marrow donor to save 8-year-old son - CTV News

Recommendation and review posted by sam

Scientists have created an easy way to identify the state and fate of stem cells earlier than previously possible.

Understanding a stem cells fatethe type of cell it will eventually becomeand how far along it is in the process of development can help scientists better manipulate cells for stem cell therapy.

Having the ability to visualize a stem cells future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases.

The beauty of the method is its simplicity and versatility, says Prabhas V. Moghe, a professor of biomedical engineering and chemical and biochemical engineering at Rutgers and senior author of a study published recently in the journal Scientific Reports. It will usher in the next wave of studies and findings.

Existing methods look at the overall population of cells but arent specific enough to identify individual cells fates. But when implanting stem cells (during a bone marrow transplant following cancer treatment, for example), knowing that each cell will become the desired cell type is essential.

Also, many protein markers used to distinguish cell types dont show up until after the cell has transitioned, which can be too late for some applications.

To identify earlier signals of a stem cells fate, scientists used super-resolution microscopy to analyze epigenetic modifications. Epigenetic modifications change how DNA is wrapped up within the nucleus, allowing different genes to be expressed.

Some modifications signal that a stem cell is transitioning into a particular type of cell, such as a blood, bone or fat cell. Using the new method, the team of scientists was able to determine a cells fate days before other techniques.

Having the ability to visualize a stem cells future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases, says Rosemarie Hunziker, program director for tissue engineering and regenerative medicine at the National Institute of Biomedical Imaging and Bioengineering. Its a relatively simple way to get a jump on determining the right cells to use.

The approach, called EDICTS (Epi-mark Descriptor Imaging of Cell Transitional States), involves labeling epigenetic modifications and then imaging the cells with super resolution to see the precise location of the marks.

Were able to demarcate and catch changes in these cells that are actually not distinguished by established techniques such as mass spectrometry, Moghe says.

He described the method as fingerprinting the guts of the cell, and the results are quantifiable descriptors of each cells organization (for example, how particular modifications are distributed throughout the nuclei).

The team demonstrated the methods capabilities by measuring two types of epigenetic modifications in the nuclei of human stem cells cultured in a dish. They added chemicals that coaxed some of the cells to become fat cells and others to become bone, while another set served as control.

Within three days, the localization of the modifications varied in cells destined for different fates, two to four days before traditional methods could identify such differences between the cells. The technique had the specificity to look at regional changes within individual cells, while existing techniques can only measure total levels of modifications among the entire population of cells.

The levels are not significantly different, but how theyre organized is different and that seems to correlate with the fact that these cells are actually exhibiting different fates, Moghe says. It allows us to take out a single cell from a population of dissimilar cells, which can help researchers select particular cells for different stem cell applications.

The method is as easy as labeling, staining, and imaging cellstechniques already familiar to many researchers, he says. As the microscopes capable of super resolution imaging become more widely available, scientists can use it to sort and screen different types of cells, understand how a particular drug may disrupt epigenetic signaling, or ensure that stem cells to be implanted wont transform into the wrong cell type.

Collaborators are from Stanford University School of Medicine, Case Western Reserve University, Seoul National University, Princeton University, the University of Akron, the University of Pennsylvania, and MIT.

Source: Teal Burrell for the National Institute of Biomedical Imaging and Bioengineering via Rutgers University

Originally posted here:
This simple method can predict a stem cell's fate - Futurity - Futurity: Research News

Recommendation and review posted by simmons

WEDNESDAY, March 1, 2017 (HealthDay News) -- Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow -- causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant.

In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells -- which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible -- and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team, led by Dr. Marina Cavazzana, of Necker Children's Hospital's biotherapy department, focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait -- that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin -- to replace the abnormal "adult" hemoglobin seen in sickle cell.

The hope, Williams said, is that gene therapy will ultimately offer a one-time treatment that cures sickle cell. But no one knows yet whether that will happen.

According to Williams, two key questions are: What's the long-term safety? And will the altered stem cells last for a patient's lifetime?

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published March 1 in the New England Journal of Medicine.

View original post here:
Gene Therapy: A Breakthrough for Sickle Cell Anemia? - Twin Falls Times-News

Recommendation and review posted by Bethany Smith

THE family of a man urgently in need of a bonemarrow transplant has appealed to the Asian community to donate their stem cells in the hope of finding a suitable match to save his life.

Father of two, Yevi Ilangakoon, was diagnosed with myelofibrosis in 2009. It is a rare condition where scar tissue builds up inside the bone marrow, affecting its ability to create healthy blood cells, which affects one person in every 100,000.

The 63-year-old, who is originally from Sri Lanka and now lives in Whitstable, has seen his health deteriorate rapidly and his illness could now progress into leukemia if he is not treated.

His only option is to have a bone-marrow transplant using stem cells. However, specialists have been unable to find a 100 per cent match despite searching worldwide registers.

From the entire register, only four per cent are from a south Asian background.

Yevis son Yovaan told Eastern Eye: Its a lifethreatening disease and has been managed with medication for the past eight years, but the condition has got more and more aggressive, especially over the last few months. If he doesnt have a stem-cell transplant, it will be a few months to a year that he will have to live.

So it is quite crucial that we get as close to 100 per cent match as we can. He gets very, very tired because his hemoglobin levels are low. If he has an injury, it takes ages to heal. We are praying and being positive and trying to raise awareness.

Yovaan highlighted the issue on social media, which attracted the attention of Sri Lankan cricketer Mahela Jayawardena, but the family are still urging members of the public to get on the bone marrow register to find a match for Yevi.

The 29-year-old added: It could be your family member your mum or your dad, you dont know what position you are going to be in in a few years time.

If you are on the register, you have the chance of saving someones life. Its a really easy process.

Signing up online takes two minutes and participants simply need to swab the inside of their cheek with a cotton bud they are sent, and send it back in a pre-paid envelope.

Sarah Rogers of the Anthony Nolan charity said: We urgently need more people from Indian and South Asian backgrounds to register as stem cell donors to make sure that everyone, regardless of background, can receive a second chance at life.

At the moment we find a perfect match for about 60 per cent of northern European patients who need a transplant, but that drops to around 20 per cent for any patient of ethnic minority.

If you are above 30, go to: http://www.dkms.org.uk/en/ register-now. Under 30, register at http://www.anthonynolan.org/apply-join-bone-marrow-register.

The rest is here:
Family appeals for bone marrow donor to save father - Easterneye (press release) (subscription)

Recommendation and review posted by Bethany Smith

"Since therapy was applied, he hasn't had any pain, any complications. He is free of any transfusions. He plays sports and goes to school," said Dr. Philippe Leboulch, an author of the new research and a professor of medicine at the University of Paris. "So we are quite pleased with the results."

This success provides proof of concept for human patients, Leboulch said.

According to Dr. Marina Cavazzana, senior author of the study and head of the biotherapy department at Necker, "all the biological tests we perform lead us to think he is cured." Yet, she added, the answer to the question of whether he is truly cured "can be provided only by the longer follow-up."

Still, hopes are running high that patients with this very devastating disease can receive this therapy "in the next five years," Cavazzana said. "This is our hope, and we work very hard to attain it."

Worldwide, more than 275,000 infants are born with sickle cell disease each year. In the United States, approximately 100,000 people, most of African ancestry or identifying as black, currently have it. About one in every 365 black children in the US is born with sickle cell disease, for which the life expectancy is now about 40 to 60 years.

Sickle cell disease is one of the most common gene disorders in the world, explained Leboulch. A genetic mutation causes hemoglobin, the main constituent of red blood cells, to distort the shape of the cell, and this causes the blood to aggregate or clog.

This leads to "tremendous pain, anemia and also lesions of organs that ultimately result in shortness of life expectancy," Leboulch said. "So what we did here was, we tried to inhibit the process of aggregation."

Essentially, researchers extracted bone marrow from the patient, harvested the stem cells and altered the genetic instructions so that they would make normal hemoglobin. Next, they treated the patient with chemotherapy for four days to eliminate his diseased stem cells. Finally, they returned the treated stem cells via an IV into his bloodstream.

"At that point, the new cells that were modified outside the body started to make new blood cells, and we hope this will be stable for the life of the patient," Leboulch said.

Before receiving treatment, the teen had terrible pain and needed blood transfusions, which required twice-yearly hospitalizations, Leboulch explained. His many complications included necrosis of the hip, which necessitated hip replacement surgery.

Going forward, the plan is to proceed through clinical trials and, if results are promising, make the treatment available to patients. Leboulch and his colleagues are using the same genetic therapy to treat a similar disease called thalassemia, another inherited blood disorder in which patients have less hemoglobin and fewer red blood cells than normal. Severe forms require regular blood transfusions.

Leboulch and his colleagues have global phase 2 and phase 3 trials for the thalassemia treatment underway in France, the US, Australia and Thailand.

For sickle cell disease, a companion trial in the US is underway. "I understand that seven (sickle cell) patients have been treated already. Of course, the outcome is much shorter, and we don't have the results just yet, but it's coming along," Leboulch said.

"To apply this to a baby or a very young child should be at least as effective or more," he said. "Doing it with older patients, who have had years of complications, could be more challenging."

According to Dr. Alexis Thompson, president-elect of the American Society of Hematology, the majority of sickle cell disease patients do not have a sibling who would be an appropriate match for bone marrow donation.

"Gene therapy holds promise because a patient serves as his own donor," and the "risks are much reduced" since there's no possibility of a mismatch, said Thompson, who was not involved in this research but is an investigator on a related gene therapy study.

"I think this is a really very exciting advancement," she said, adding that if the results seen in France can be duplicated, this would provide "for a new direction for patients who need a curative option."

"Gene therapy offers hope for all patients with sickle cell disease, regardless of whether they have a bone marrow match or not," Wong wrote in an email.

"Time is still needed" to assess the success of this treatment and the possibility of later side effects, said Wong. "But any patient with severe sickle cell disease will tell you that being able to live a life for even a year without medications or fear of pain or hospitalization is substantial."

Leboulch also feels very hopeful.

"Now, we want to be cautious, of course, and we don't want to say that this is the cure for tomorrow or the next day for everybody," he said. "At the same time, what we've observed is really convincing, and we just hope that we can move this along to make it available to patients."

Read more here:
Sickle cell anemia patient 'cured' by gene therapy, doctors say - CNN

Recommendation and review posted by simmons

Debra Pugh 2015

Debra Pugh 2015

Walking has many Health Benefit

There are many different forms of exercise for beginners to experts, with various health benefits too. One basic form of exercise is walking. It is a gentle, low impact exercise that has many benefits to improve a persons health. It is safe, simple, does not require a certain skill, or practice, and does not cost any money.

Hippocrates said that walking is mans best medicine. Thousands of years later, experts are still discovering the many benefits of a daily walking program which is explained below by the Mayo Clinic and the Arthritis Foundation.

Heart Health

Walking can reduce the risk of heart attack or strokes. It lowers the low-density lipoprotein (LDL) cholesterol (the bad cholesterol) and at the same time it raises the high-density lipoprotein (HDL) cholesterol (the good cholesterol). It also lowers peoples blood pressure.

Diabetes

Walking can decrease the chance of getting Type 2 diabetes. If a person already has this diagnosis, it can help to manage the symptoms. It can also lower peoples insulin level.

Weight Loss

Walking can help people to lose weight. A pound of fat is the same as 3,500 calories. The faster a person walks then the more calories they burn. Walking tones the muscles throughout the body, but especially tones the legs and the core muscles of the back, waist and abdomen. The more muscle a person has, then the higher the metabolism will be which means that they burn more calories. If a person has a healthy weight, it takes pressure off of the hips, knees, ankles and feet which can reduce pain. Walking also reduces the stress hormone cortisol which is known to cause weight gain.

Arthritis

Because walking is a weight bearing activity, it strengthens the bones which can help to prevent osteoporosis (a form of arthritis where bones become more fragile and likely to fracture). The movement increases the production of synovial fluid (lubricates the knees) which can decrease pain and stiffness. Walking can also lower the risk of blood clots by helping the calf muscles squeeze deep veins and keep blood pumping back to the heart.

Brain Health

Studies show that walking can decrease the risk of Alzheimers disease and age related dementia. It can improve a persons mood by helping them to feel less anxious or sad. Because of the increase of blood flow and oxygen to the brain, it can improve peoples memory. Ginkgo Sense is a supplement made from the ginkgo plant and it has been known to help peoples memory for over 3,000 years.

For people who are looking for a simple way to improve their health, take the advice of Hippocrates when he said walking is mans best medicine and reap the many health benefits. For help with energy in starting a walking program, try Peak Endurance. It is a natural berry flavored drink that is packed with many B vitamins, minerals, electrolytes, and ATP (adenosine triphosphate that decreases as we age) that improves energy, metabolism, stress, aging, and thinking clearer. It only has 30 calories, 0 fat, and 1 gram of natural sugar from the stevia plant.

(Debra Pugh, author of the book, Fibromyalgia and Fatigue: A Recipe for Feeling Fit and Healthy, has a bachelors degree, is a certified personal trainer, and a certified sports nutritionist. She hasover 25 years of experience in counseling people in Health and Wellness such as weight loss, exercise, nutrition, increased energy, balance, fibromyalgia, chronic fatigue, auto-immune disorders, arthritis, hormones, toxins, cleansers, and supplements. For more information or to buy a book, call 806-298-5504, email DebraPugh@windstream.net, or website at http://www.FibroAndFit4Life.com.)

Go here to see the original:
Walking has many Health Benefit - Plainview Daily Herald

Recommendation and review posted by Bethany Smith

Home Men's Health Declining libido? This may be the cause

Contrary to popular belief, menopause is not just a female problem. Male menopause the nickname for late-onset hypogonadism that occurs as a mans testosterone levels wane with age can mimic its female counterpart and include symptoms such as hot flashes, disrupted sleep, fatigue, and flagging libido, as well as moodiness.

Low testosterone levels affect as much as 25 percent of middle-aged men, making the condition more common than it may seem. However, unlike the rapid changes women experience, the decline in testosterone happens more gradually which can make symptoms difficult to spot and attribute to the correct cause. Being more tired and not sleeping well may lead you to think that you need a new mattress or should cut back on the caffeine. Because of hot flashes, you may think the room is warmer or youre coming down with a fewer. And your flagging libido could potentially be related to that fatigue or increased stress. (Siberian secret refuels energy from within.)

As these symptoms are all so general and easy to overlook, hypogonadism (insufficient production of testosterone) can be overlooked and leave its sufferers confused and agitated. Luckily, a simple blood test performed by your doctor will tell you if your testosterone levels are lower than average, and there are treatments available to get your levels back to where they should be to relieve symptoms.

However, these treatments have proven most effective in men who have been diagnosed with conditions like testicular cancer and the merit of treating men with low testosterone due to aging is still up for debate. In the guidelines released by the Endocrine Society, it is recommended that physicians only prescribe medical testosterone therapy for men who are experiencing consistently low levels, and significant sexual and physical complications. (Boost your testosterone naturally with this one thing.)

Related: 5 sure-fire ways to boost your libido

Related Reading:

Low libido? It could be something more serious

Spark your libido with these 6 fixes

http://www.health.com/health/condition-article/0,,20408367,00.html

Read more here:
Declining libido? This may be the cause - Bel Marra Health

Recommendation and review posted by Bethany Smith

Many rely on blood transfusions

Joe Amon/The Denver Post via Getty

By Andy Coghlan

A TEENAGE boy with an inherited disease that affects millions worldwide seems to have been cured using gene therapy. The treatment appears to have stopped the painful symptoms of sickle cell disease, demonstrating the potential for gene therapy to treat common genetic diseases.

All the blood tests we performed show that the teenager has been cured of sickle cell disease

The idea of gene therapy using strands of DNA to compensate for a persons malfunctioning genes is almost three decades old. However, the approach has so far mostly been used to treat very rare diseases (see Long road to success). In contrast, sickle cell disease affects 100,000 people in the US alone. If the treatment proves successful in larger trials, it could bring gene therapy into widespread use.

It could be a game changer, says Deborah Gill at the University of Oxford. The fact the team has a patient with real clinical benefit, and biological markers to prove it, is a very big deal.

People with sickle cell disease make abnormal versions of haemoglobin, the blood protein that carries oxygen around the body. This can be caused by mutations in the gene that makes a subunit of haemoglobin, called beta-globin. The mutations cause haemoglobin to clump together, distorting red blood cells into a sickle-shape that can get stuck in blood vessels around the body.

People with the disorder are given blood transfusions to clear these painful blockages and prevent new ones. Bone marrow transplants can treat the disease, but matching donors can only be found for around 10 per cent of people with the condition.

Now a team in France seems to have developed a treatment that would work for everyone with the disorder. First, the team took bone marrow stem cells from the boy when he was 13, and gave them extra, mutated versions of the gene that codes for beta-globin. These were designed to make beta-globin that would interfere with the boys faulty proteins, stopping them from clumping together.

The researchers then put these stem cells back into the boys body. After around three months, he began producing large quantities of haemoglobin that behaves normally (New England Journal of Medicine, DOI: 10.1056/NEJMoa1609677). The patient is now 15 years old and free of all previous medication, says Marina Cavazzana at the Necker Childrens Hospital in Paris, who led the team. He has been free of pain from blood vessel blockages, and has given up taking opioid painkillers.

Cavazzana is confident these benefits will last. All the tests we performed on his blood show that hes been cured, but more certainty can only come from long-term follow-up. She says her team has treated seven other patients, who are showing promising progress.

We are all very excited by the work, and this success provides support for this and other genetic strategies targeting this horrible disease, says John Tisdale at the US National Heart, Lung, and Blood Institute in Maryland.

David Williams, at Boston Childrens Hospital in Massachusetts, suggests that the boy may still occasionally experience blockages, because his own original genes are still able to produce faulty haemoglobin. Its important to see what happens over time, and how many other patients see similar benefits.

However, should the gene therapy prove to be effective in larger trials, its expense may limit its use to richer nations. We should be realistic in remembering that there are hundreds of thousands of sickle cell patients in less developed countries, and that the therapy is not easily exportable or adaptable to countries with less well-developed health systems, says Stuart Orkin at Harvard Medical School.

Twenty years ago, gene therapy was touted as a cure for everything from cancer to cystic fibrosis. Now it is finally starting to fulfil its promise.

In 2012, Glybera became the first gene therapy to be approved, for people with a rare disorder that makes them unable to process dietary fat. Last year, the first commercial gene therapy that alters a persons DNA was approved for children with a severe immune disorder. Gene therapies for rare forms of blindness are also showing promise.

But these conditions all affect very small numbers of people. Research into sickle cell disease (see main story), beta thalassaemia, haemophilia and cystic fibrosis mean gene therapy may not be too far from becoming mainstream medicine for the most common genetic diseases.

This article appeared in print under the headline Gene therapy breakthrough

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As the Trump Administration backs away from certain legal protections for transgender Americans the city-run hospital system has opened its first center for transgender patients looking to undergo gender-reassignment surgery. Health Reporter Erin Billups has the details.

Brandi Concepcion says a 40-year journey to find her place in society brought her to Metropolitan Hospital in Harlem, part of the the city's municipally run hospital system.

"I went through the year of therapy that was required," Concepcion said. "Just to make sure that this was the right choice for me. I started with hormones after I was given my initial letter. It took me like a year to start dressing more feminine like. Wearing makeup, letting my hair grow. And here we are, three years later."

Conception will be one of the first transgender patients to undergo "top surgery" at the city-run hospital, also known as breast augmentation or chest masculinization surgery, depending on the gender change. It's part of Metropolitan's newly expanded LGBTQ services.

"It was just something that was part of our journey as a hospital of becoming a place where we listen and respond to the needs of our patients was a no brainer in terms of what we needed to add to our program," said Sarah Bender, the hospital's director of LGBTQ services.

Patients will soon see the difference, including a clinic within the hospital with its own unique signage.

Concepcion says after growing up male in the deep South, and becoming accustomed to others' discomfort with her, it's nice to have a place to go to for medical care centered on her comfort.

She says acceptance of who she is has led to a healthier life.

"I lived off anti-anxiety pills," she said. "I lived off a lot of medications, which surely was not good for me. I wasa chain smoker. I would drink about a twelve-pack of soda a day. I was unproductive. I lived off SSI. Today I am employed. I went back to school. I live on my own."

Medicaid now covers medically necessary hormone therapy and gender reassignment surgeries, but there are questions whether the Trump administration will continue those policies. The city-run hospital system says it intends to maintain its services for the transgender community.

"It's our mission as the public health system of New York City to provide care to anybody regardless of a whole host of identifying factors, gender identity included," Bender said. "So, you know, we're going to continue to push forward I think regardless."

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Municipal Hospital System Opens NYC's First Public Clinic Offering Gender-Reassignment Surgery - NY1

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