Stem cell scientists had what first appeared to be an easy win for regenerative medicine when they discovered mesenchymal stem cells several decades ago. These cells, found in the bone marrow, can give rise to bone, fat, and muscle tissue, and have been used in hundreds of clinical trials for tissue repair. Unfortunately, the results of these trials have been underwhelming. One problem is that these stem cells don't stick around in the body long enough to benefit the patient.

But Harvard Stem Cell Institute (HSCI) scientists at Boston Children's Hospital aren't ready to give up. A research team led by Juan Melero-Martin, PhD, recently found that transplanting mesenchymal stem cells along with blood vessel-forming cells naturally found in circulation improves results. This co-transplantation keeps the mesenchymal stem cells alive longer in mice after engraftment, up to a few weeks compared to hours without co-transplantation. This improved survival gives the mesenchymal stem cells sufficient time to display their full regenerative potential, generating new bone or fat tissue in the recipient mouse body. The finding was published in the Proceedings of the National Academy of Sciences (PNAS).

"We are losing mesenchymal stem cells very rapidly when we transplant them into the body, in part, because we are not giving them what they need," said Melero-Martin, an HSCI affiliated faculty member and an assistant professor of surgery at Boston Children's Hospital, Harvard Medical School.

"In the body, these cells sit very close to the capillaries, constantly receiving signals from them, and even though this communication is broken when we isolate mesenchymal stem cells in a laboratory dish, they seem to be ok because we have learned how to feed them," he said. "But when you put the mesenchymal stem cells back into the body, there is a period of time when they will not have this proximity to capillary cells and they start to die; so including these blood vessel-forming cells from the very beginning of a transplantation made a major difference."

Melero-Martin's research has immediate translational implications, as current mesenchymal clinical trials don't follow a co-transplantation procedure. He is already collaborating with surgical colleagues at Boston Children's Hospital to see if his discovery can help improve fat and bone grafts. However, giving patients two different types of cells, as opposed to just one, would require more time and experiments to determine safety and efficacy. Melero-Martin is seeking to identify the specific signals mesenchymal stem cells receive from the blood vessel-forming cells in order to be able to mimic the signals without the cells themselves.

"Even though mesenchymal stem cells have been around for a while, I think there is still a lack of fundamental knowledge about communication between them and other cells in the body," he said. "My lab is interested in going even beyond what we found to try to understand whether these cell-cell signals are different in each tissue of the body, and to learn how to educate both blood vessel-forming and mesenchymal stem cells to co-ordinate tissue specific regenerative responses."

Other Harvard Stem Cell Institute researchers are studying mesenchymal stem cells as bioengineering tools to deliver therapeutics, which is possible because of the cell type's unique ability to not trigger an immune response. Jeffrey Karp, PhD, at Brigham and Women's Hospital has developed several methods to turn these cells into drug-delivery vehicles, so that after transplantation they can, for example, hone in on swollen tissue and secrete anti-inflammatory compounds. And Khalid Shah, PhD, at Massachusetts General Hospital has designed a gel that holds mesenchymal stem cells in place so that they can expose brain tumors to cancer-killing herpes viruses.

"A lot of these applications have no real direct link with mesenchymal stem cells' supposed progenitor cell function," Melero-Martin said. "In our study, we went back to the collective ambition to use these cells as a way to regenerate tissues and we are not in a position to say how that affects other uses that people are proposing."

Story Source:

The above story is based on materials provided by Harvard University. Note: Materials may be edited for content and length.

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Research team pursues techniques to improve elusive stem cell therapy

Apollo Health City team did autologous stem cell procedure to save both the hip joints

Hyderabad, June 30:

A team of doctors from a city hospital have harvested stem cells of a person using bone morrow from the pelvis area to replace some dead tissues in the hip. In this process, they saved the patient from undergoing a hip replacement.

The Apollo Health City team, headed by orthopaedic specialist Paripati Sharat Kumar, diagnosed a 39-year-old woman to be suffering from Avascular Necrosis, making her writhe with pain in her two hip joints. Her condition would require undergoing a replacement of hips.

After assessing her condition, the team has decided to go for autologous stem cell procedure (where donor and the receiver is the same person) to save both the hip joints.

The minimally invasive procedure involved taking bone marrow aspirate from the patients pelvis. Stem cells were harvested from the aspirate, through a process that takes about 15 minutes. Stems cells were planted in the area of damage under fluoroscopy control following core decompression, Sharat Kumar said here in a statementon Monday.

He felt that autologous stem cell treatments could edge out joint replacement procedures to a large extent in days to come. The scope of this procedure in orthopaedics and sports medicine is enormous. This could be extended to indications include osteoarthritis of knee, shoulder, hip, elbows, ankle and spine, he said.

(This article was published on June 30, 2014)

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Her own stem cells saved her from hip replacement

Geordie Shore star Charlotte Crosby has become the latest person to sign up to the Anthony Nolan bone marrow register

Geordie Shore star Charlotte Crosby has spat out her support for a baby in need of a life-saving operation.

Charlotte has signed up with the Anthony Nolan Trust after reading about the plight of nine-month-old Joey Ziadi, who is suffering from a rare blood disorder that affects one in nine million people.

The tot from Northampton needs a lifesaving transplant but has not yet found a matching donor so Charlotte has enlisted her 1.89m twitter followers to join the cause.

After hearing about Joeys plight, Charlotte tweeted a selfie with her Anthony Nolan spit kit - the simple piece of equipment which allows people to leave a DNA sample and go on the bone marrow donor register.

She said: I saw the gorgeous Joey Ziadi in the news and I couldnt believe it when I heard how ill he was and that only one in nine million people have his condition I felt like crying. I knew I had to do something, but I didnt know how to help.

When I found out how simple it was to sign up to the Anthony Nolan register, I didnt have to think about it. I just thought Its so easy, why doesnt everyone do this?

Anthony Nolan saves lives by matching people willing to donate their bone marrow or blood stem cells to patients in need of a transplant.

The charity also needs more young men to sign up, as they are most likely to be chosen to donate but make up just 14% of the register. Charlotte said: I was quite shocked that young lads are so underrepresented on the register though. Come on lads, just sign up online and spit into a tube! Im doing it, and I just hope one day I have the chance to save a life.

Joey was diagnosed with an extremely rare blood disorder Diamond Blackfan Anaemia in February. His family have been campaigning to recruit more potential donors to the Anthony Nolan donor register after being told that his best hope of a cure is a bone marrow transplant from a stranger.

Link:
Charlotte Crosby helps young boy in need of bone marrow transplant

PUBLIC RELEASE DATE:

19-Jun-2014

Contact: David McKeon DMcKeon@nyscf.org 212-365-7440 New York Stem Cell Foundation

NEW YORK, NY (June 18, 2014) Induced Pluripotent Stem Cells (iPS) hold enormous potential to unravel the mechanisms of human illness and to develop new therapeutics. Until now, there has been no easily searchable database for investigators to find and share these important resources. This has been a major obstacle to the implementation of iPS technology.

Recognizing the research potential of shared iPS cell lines, the New York Stem Cell Foundation (NYSCF) Research Institute and the eagle-i Network will make NYSCF iPS cell lines and related information available to the public on a user-friendly, web-based, searchable database. The database (called the Induced Pluripotent Stem Cell database) will help scientists find valuable resources, enabling collaboration, preventing duplicative work, and ultimately accelerating research.

NYSCF and eagle-i will establish an open access repository of information on large numbers of iPS cell lines. eagle-i will display information as linked open data, enabling discovery by any third party search engine. NYSCF derives hundreds of iPS cell lines from skin samples of patients with a wide variety of diseases using the NYSCF Global Stem Cell ArrayTM technology, an automated platform for high-throughput iPS cell production and differentiation. Scientists will be able to search for NYSCF iPS cell lines under several categories including disease, how the cells were reprogrammed, and patient age at the time the sample was collected.

"This important tool should have significant impact on the science community," said Lee Nadler, principal investigator of Harvard Catalyst and eagle-i. "I'm thrilled that we will contribute to this partnership by creating a user-friendly, searchable database for the iPS cell lines that NYSCF has produced, enabling researchers to search for available lines on an open access platform. The opportunities this will create are tremendous."

"We were very excited to develop this resource for stem cell scientists," said Susan L. Solomon, NYSCF Chief Executive Officer. "It is important to have open access to available resources and this collaboration with eagle-i is a prime example of interdisciplinary teams working together to provide this for the scientific community."

The alpha version of the website will be presented during the International Society for Stem Cell Research (ISSCR) Annual Conference in Vancouver, Canada in June 2014. Future versions of the database will include genomic and other clinical and cellular phenotype information, including a mechanism that will allow scientists to order lines directly from the website. Soon, NYSCF and eagle-i will invite other institutions from around the world to join this collaboration and contribute their iPS cell lines to the Induced Pluripotent Stem Cell database, creating an even more robust research tool.

At the ISSCR Conference this week, Richard V. Pearse, PhD, from eagle-i will be at poster F-2245 during poster session III and NYSCF will be at booth 918 with information pertaining to this new initiative.

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NYSCF and eagle-i Network co-develop iPS cell database

Published on June 25, 2014

Scientists develop designer T cells to guard against infection after bone marrow transplants

WASHINGTON - Bone marrow transplants save thousands of lives but patients are vulnerable to severe viral infections in the months afterward, until their new immune system kicks in. Now scientists are developing protection for that risky period injections of cells specially designed to fend off up to five different viruses at once.

"These viruses are a huge problem, and there's a huge need for these products," said Dr. Ann Leen, who leads a team at Baylor College of Medicine and Texas Children's Hospital that found an easier way to produce these long-desired designer T cells.

Healthy people have an army of T cells that roams the body, primed to recognize and fight viruses. People with suppressed immune systems such as those undergoing a bone marrow transplant to treat leukemia or other diseases lack that protection. It can take anywhere from four months to more than a year for marrow stem cells from a healthy donor to take root and start producing new immune cells for the recipient. When patients get sick before then, today's antiviral medications don't always work and cause lots of side effects.

The proposed solution: Take certain virus-fighting T cells from that same bone marrow donor, and freeze them to use if the recipient gets sick. Years of experiments show it can work. But turning the idea into an easy-to-use treatment has been difficult. A dose had to be customized to each donor-recipient pair and protected against only one or two viruses. And it took as long as three months to make.

Wednesday, Leen reported a novel technique to rapidly manufacture so-called virus-specific T cells that can target up to five of the viruses that cause the most trouble for transplant patients: Epstein-Barr virus, adenovirus, cytomegalovirus, BK virus, and human herpesvirus 6.

Essentially, Leen came up with a recipe to stimulate donated T cells in the laboratory so that they better recognize those particular viruses, and then grow large quantities of the cells. It took just 10 days to create and freeze the designer T cells.

To see if they worked, Leen's team treated 11 transplant recipients. Eight had active infections, most with multiple viruses. The cell therapy proved more than 90 per cent effective, nearly eliminating all the viruses from the blood of all the patients, Leen reported in the journal Science Translational Medicine.

The other three patients weren't sick but were deemed at high risk. They were given early doses of the T cells protectively and remained infection-free, Leen said.

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Scientists develop designer T cells to guard against infection after bone marrow transplants

Dr. Lox, 8 WFLA News, Stem Cell Therapy
Dr. Lox | http://www.drlox.com | 727-462-5582 "It was like a miracle" - Watch as Judy Loar describes her experience with Dr. Dennis Lox to WFLA #39;s Gayle Guyardo.

By: Dr. Lox

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Dr. Lox, 8 WFLA News, Stem Cell Therapy - Video

Dr Lox Stem Cell Therapy WFLA News 8
Dr. Lox | http://www.drlox.com | 727-462-5582 (WFLA) When Judy Loar, 68, could not bear to walk any longer due to excruciating pain in both of her knees from degenerative joint disease, she did what...

By: Dr. Lox

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Dr Lox Stem Cell Therapy WFLA News 8 - Video

Durham, NC (PRWEB) June 27, 2014

A new study released today in STEM CELLS Translational Medicine suggests a new way to produce endothelial progenitor cells in quantities large enough to be feasible for use in developing new cancer treatments.

Endothelial progenitor cells (EPCs) are rare stem cells that circulate in the blood with the ability to differentiate into the cells that make up the lining of blood vessels. With an intrinsic ability to home to tumors, researchers have focused on them as a way to deliver gene therapy straight to the cancer. However, the challenge has been to collect enough EPCs for this use.

This new study, by researchers at the Institute of Bioengineering and Nanotechnology, National University of Singapore and Zhejiang University led by Shu Wang, Ph.D., explored whether human induced pluripotent stem cells (iPSCs) could provide the answer. iPSCs, generated from adult cells, can propagate indefinitely and give rise to every other cell type in the body, much like human embryonic stem cells, which are considered the gold standard for stem cell therapy.

However, human iPS cells can be generated relatively easily through reprogramming, a procedure that circumvents the bioethical controversies associated with deriving embryonic stem cells from human embryos, Dr. Wang said.

After inducing human iPS cells to differentiate into the EPCs, the research team compared the stability and reliability of the induced EPCs with regular EPCs by injecting them into mice with breast cancer that had metastasized (traveled) to the lungs. The results showed that their induced EPCs retained the intrinsic ability to home to tumors, just as regular EPCs do. They also did not promote tumor growth or metastasis.

We next tested the induced EPCs therapeutic potential by infusing them with an anticancer gene and injecting them into the mice, Dr. Wang said. The results indicated that the tumors were reduced and the animals survival rates increased.

Since this approach may use patient's own cells to prepare cellular therapeutics and is based on non-toxic immunotherapy, it holds potential for translation to clinical application and may be particularly valuable as a new type of anti-metastatic cancer therapy.

With the increasing potential of using EPCs as cancer therapeutics, it is important to have a reliable and stable supply of human EPCs, said Anthony Atala, M.D., editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. This study demonstrates the feasibility of generating EPs from early-passage human iPS cells.

###

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New Stem Cell Production Method Could Clear Way for Anticancer Gene Therapy

TOKYO: Japanese stem cell scientists have succeeded in slowing the deterioration of mice with motor neuron disease, possibly paving the way for eventual human treatment, according to a new paper.

A team of researchers from the Kyoto University and Keio University transplanted specially created cells into mice with amyotrophic lateral sclerosis (ALS), also called Lou Gehrig's, or motor neuron disease.

The progress of the creatures' neurological degeneration was slowed by almost eight per cent, according to the paper, which was published on Thursday in the scholarly journal Stem Cell Reports.

ALS is a disorder of motor neurons -- nerves that control movement -- leading to the loss of the ability to control muscles and their eventual atrophy.

While it frequently has no effect on cognitive function, it progresses to affect most of the muscles in the body, including those used to eat and breathe.

British theoretical physicist Stephen Hawking has been almost completely paralysed by the condition.

In their study, the Japanese team used human "iPS" -- induced pluripotent stem cells, building-block cells akin to those found in embryos, which have the potential to turn into any cell in the body.

From the iPS cells they created special progenitor cells and transplanted them into the lumbar spinal cord of ALS mice.

Animals that had been implanted lived 7.8 per cent longer than the control group without the procedure, the paper said.

"The results demonstrated the efficacy of cell therapy for ALS by the use of human iPSCs (human induced pluripotent stem cells) as cell source," the team said in the paper.

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Cell scientists slow degeneration in motor neuron mice

Friday 27 June 2014 22.31

Japanese stem cell scientists have succeeded in slowing the deterioration of mice with motor neurone disease, possibly paving the way for eventual human treatment.

A team of researchers from the Kyoto University and Keio University transplanted specially created cells into mice with amyotrophic lateral sclerosis (ALS), also called Lou Gehrig's, or motor neurone disease.

The progress of the creatures' neurological degeneration was slowed by almost eight percent, according to the paper, which was published Thursday in the scholarly journal Stem Cell Reports.

ALS is a disorder of motor neurones -- nerves that control movement -- leading to the loss of the ability to control muscles and their eventual atrophy.

While it frequently has no effect on cognitive function, it progresses to affect most of the muscles in the body, including those used to eat and breathe.

British theoretical physicist Stephen Hawking has been almost completely paralysed by the condition.

In their study, the Japanese team used human "iPS" -- induced pluripotent stem cells, building-block cells akin to those found in embryos, which have the potential to turn into any cell in the body.

From the iPS cells they created special progenitor cells and transplanted them into the lumbar spinal cord of ALS mice.

Animals that had been implanted lived 7.8% longer than the control group without the procedure, the paper said.

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Scientists slow degeneration in motor neurone mice

Poway, CA (PRWEB) June 27, 2014

South Reno Veterinary Hospital and Mathew Schmitt, VMD have recently discovered the secret to prolonging a pets quality of life with the use of stem cell therapy, and the ability to bank stem cells for the future care of a pet. Dr. Schmitt and South Reno Veterinary Hospital offer stem cell therapy and stem cell banking through Vet-Stem, Inc. for small animal osteoarthritis and partial ligament tears.

As many as 65% of dogs between the ages of 7 and 11 years old will be inflicted with some degree of arthritis. For certain specific breeds the percentage is as high as 70, such as Labrador Retrievers. Barley, a Labrador mix, was treated using cells from a sample of his own fat, and some stem cells are also stored (or banked) with Vet-Stem just in case he needs future treatment with Dr. Schmitt. Those banked stem cells do not have to be used for the same use as they were originally used for either. For example, if a pet has stem cell therapy initially for osteoarthritis pain and inflammation, the banked stem cells can be used years later for an acute injury.

After rupturing the canine cruciate ligaments in both of his stifles, or hind knees, Barleys pain was managed by medication but then medication was finally not enough and he was facing the possibility of surgery. Dr. Schmitt reported shifting lameness in Barleys hind end, which was a sign of severe discomfort. Barleys owner did not want to put him through surgery on both knees. Instead, Barleys owner elected for stem cell therapy.

I fully believe stem cell therapy has significantly prolonged Barleys quality of life and I am so glad I found out about the therapy when he was injured at six years old. He just turned 13 and his legs are still doing well. It truly is a miracle of science and I tell all my friends about it, said Barleys mom.

Vet-Stem, along with countless research and academic institutions, is working to support additional uses for stem cells which may include treatment for liver disease, kidney disease, auto-immune disorders, and inflammatory bowel disease in animals. These uses for stem cells are in the early stages of development and may provide additional value to the ability to bank stem cells to ensure a pets quality of life into the future.

About Vet-Stem, Inc. Vet-Stem, Inc. was formed in 2002 to bring regenerative medicine to the veterinary profession. The privately held company is working to develop therapies in veterinary medicine that apply regenerative technologies while utilizing the natural healing properties inherent in all animals. As the first company in the United States to provide an adipose-derived stem cell service to veterinarians for their patients, Vet-Stem, Inc. pioneered the use of regenerative stem cells in veterinary medicine. The company holds exclusive licenses to over 50 patents including world-wide veterinary rights for use of adipose derived stem cells. In the last decade over 10,000 animals have been treated using Vet-Stem, Inc.s services, and Vet-Stem is actively investigating stem cell therapy for immune-mediated and inflammatory disease, as well as organ disease and failure. For more on Vet-Stem, Inc. and Veterinary Regenerative Medicine, visit http://www.vet-stem.com/ or call 858-748-2004.

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South Reno Veterinary Hospital is Prolonging Pet Quality of Life with Stem Cell Therapy and the Ability to Bank Cells ...

Introduction

Despite advances in early recognition and treatment, spinal cord injuries continue to produce devastating and long lasting disabilities. Patients suffer paralysis that can vary from a partial leg to almost the entire body. In addition, the medical cost of a spinal cord injury patient over a lifetime ranges from $500,000-2,000,000.

The purpose of this paper is to help readers gain a greater understanding of the use of adult stem cells for spinal cord injuries. We also want to offer a framework for evaluating if stem cell treatment should be considered an option for you or your loved one. The paper covers the following:

Feel free to skip to sections that provide information that is helpful to you. For more information including definitions and descriptions of spinal cord injuries visit:

Skip to next section

Spinal cord injuries occur at a rate of 40 persons per million per year in the United States. That translates to over 12,000 new cases a year. Motor vehicle accidents account for almost half of the cases. Falls, violence such as gunshot wounds and sports injuries make up most of the rest. Up to 80% of victims are male. This is a disorder that affects the young; the average age of victims in the US is 38.

The most obvious symptom of spinal cord injury is the paralysis in affected areas. The amount and severity of paralysis depends on several factors including the location and type of injury. Patients can experience anything from a weakness in one extremity to complete paralysis of everything below the neck.

Red areas indicate loss of sensation and motion for injuries at that level

Spinal cord injury patients also experience a number of other complications that include:

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Adult Stem Cells for Spinal Cord Injuries | Innovations ...

PUBLIC RELEASE DATE:

25-Jun-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, June 25, 2014Mary Ann Liebert, Inc., publishers website and Genetic Engineering & Biotechnology News (GEN) are proud to announce that they will again serve as joint platinum media sponsors of the Genetics Policy Institute 2014 World Stem Cell Summit that will take place at the Marriott River Center, December 4-6, 2014 in San Antonio, Texas.

In a new collaborative effort in 2014, Mary Ann Liebert, Inc. will also organize a World Stem Cell Summit panel, comprised of leading editors from their peer-reviewed journals intersecting the field to predict the most innovative translational research that will impact regenerative medicine in the next five years.

Mary Ann Liebert, Inc. will also publish the 2014 World Stem Cell Report as a special supplement to the peer-reviewed journal Stem Cells and Development. Dr. Graham Parker, Editor-in-Chief of Stem Cells and Development, and Bernard Siegel, Executive Director of Genetics Policy Institute (GPI), will serve as Co-Editors-in-Chief of the Report, joined by Rosario Isasi (McGill University) as Managing Editor. The World Stem Cell Report will be made available to all subscribers of Stem Cells and Development and attendees of the World Stem Cell Summit. It will also be available free online in 106 developing countries, courtesy of the Publisher, to facilitate global stem cell research.

"We are very pleased to expand our collaboration with Mary Ann Liebert, Inc., and GEN," says Bernard Siegel, Founder and Co-chair of the Summit. "The commitment by those prestigious publishers to journalistic integrity and scientific knowledge and education matches our enthusiasm to advance the field of stem cells and regenerative medicine for the betterment of humanity. We look forward to working with Graham Parker and the skilled editorial team at Stem Cells and Development to publish our annual Report. We are especially excited to have the expertise of the Liebert editors engaged on the program at the World Stem Cell Summit."

"The World Stem Cell Summit is unequivocally a paramount meeting that brings together the leaders in the field from academia, industry, and business, thereby ensuring the advancement of collaborative opportunities," says Mary Ann Liebert, publisher & CEO of both Stem Cells and Development and GEN. "Bernie Siegel and GPI also recognize the importance of public advocacy at this most important international conference. Mary Ann Liebert, Inc. is delighted to expand our own collaboration with Bernie Siegel and GPI and to publish the 2014 Report.

GEN Editor-in-Chief John Sterling stated, "The World Stem Cell Summit is the critical global meeting, providing the best opportunity for the GEN community to participate in the world of regenerative medicine. Our platinum media sponsorship allows GEN readers and advertisers to have a front row seat to listen and learn from the top experts on the very dynamic and expertly conceptualized Summit platform."

The Summit program delivers on the "big picture," featuring over 200 prominent scientists, business leaders, regulators, policy-makers, advocates, economic development officers, experts in law and ethics, and visionary gurus who will discuss the latest scientific discoveries, business models, legal and regulatory solutions, and best practices. The Summit is expected to attract attendees from more than 40 nations.

The rest is here:
2014 World Stem Cell Summit presented by GPI, Mary Ann Liebert, Inc, and GEN

June 25, 2014 - Stem Cell Therapy / David Steenblock

By: Dennis Courtney

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June 25, 2014 - Stem Cell Therapy / David Steenblock - Video

Animal Cell Therapies - Tucker #39;s Story
Tucker shows notable improvement in gait and movement after receiving stem cell therapy from Animal Cell Therapies.

By: Animal Cell Therapies, Inc.

Excerpt from:
Animal Cell Therapies - Tucker's Story - Video

By Jim Kasuba The News-Herald Twitter: @JKasuba

Elizabeth Disney (left) turned out to be a suitable blood stem donor for her sister, Brittany, who is afflicted with Burkitt lymphoma, considered to be an extremely rare disease for a young woman of only 23. Photo courtesy of Donna Smith

RIVERVIEW Battling a rare cancer has been a rough road for Brittany Disney, but the worst may be behind her.

The good news is that a suitable stem cell donor has been found and the transplant surgery went well. The not-so-good news is that theres a long recovery period and shes still in a lot of pain.

Donna Smith, a close friend of the family, said the young woman underwent stem cell replacement on June 6.

There was a one in four chance that a sibling would be a match, Smith said. (Her sister) Elizabeth had five out of six markers to be a stem cell donor for her.

Disney, 23, was diagnosed late last year with stage four Burkitt lymphoma, a form of non-Hodgkins lymphoma in which cancer starts in immune cells called B-cells. Recognized as the fastest growing human tumor, Burkitt lymphoma is associated with impaired immunity and is rapidly fatal if left untreated.

Burkitt lymphoma is so rare in young adult women that the Henry Ford Health System wrote about the case in a medical journal, said one of Disneys college friends.

In November, friends and family sponsored a spaghetti dinner fundraiser to assist the family with medical bills and expenses, but things continued to look bleak, as a stem cell transplant appeared to be the only answer to treating the condition.

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Riverview woman recovers from stem cell transplant; family seeking notes of encouragement

By LAURAN NEERGAARD AP Medical Writer

WASHINGTON (AP) - Bone marrow transplants save thousands of lives but patients are vulnerable to severe viral infections in the months afterward, until their new immune system kicks in. Now scientists are developing protection for that risky period - injections of cells specially designed to fend off up to five different viruses at once.

"These viruses are a huge problem, and there's a huge need for these products," said Dr. Ann Leen, who leads a team at Baylor College of Medicine and Texas Children's Hospital that found an easier way to produce these long-desired designer T cells.

Healthy people have an army of T cells that roams the body, primed to recognize and fight viruses. People with suppressed immune systems - such as those undergoing a bone marrow transplant to treat leukemia or other diseases - lack that protection. It can take anywhere from four months to more than a year for marrow stem cells from a healthy donor to take root and start producing new immune cells for the recipient. When patients get sick before then, today's antiviral medications don't always work and cause lots of side effects.

The proposed solution: Take certain virus-fighting T cells from that same bone marrow donor, and freeze them to use if the recipient gets sick. Years of experiments show it can work. But turning the idea into an easy-to-use treatment has been difficult. A dose had to be customized to each donor-recipient pair and protected against only one or two viruses. And it took as long as three months to make.

Wednesday, Leen reported a novel technique to rapidly manufacture so-called virus-specific T cells that can target up to five of the viruses that cause the most trouble for transplant patients: Epstein-Barr virus, adenovirus, cytomegalovirus, BK virus, and human herpesvirus 6.

Essentially, Leen came up with a recipe to stimulate donated T cells in the laboratory so that they better recognize those particular viruses, and then grow large quantities of the cells. It took just 10 days to create and freeze the designer T cells.

To see if they worked, Leen's team treated 11 transplant recipients. Eight had active infections, most with multiple viruses. The cell therapy proved more than 90 percent effective, nearly eliminating all the viruses from the blood of all the patients, Leen reported in the journal Science Translational Medicine.

The other three patients weren't sick but were deemed at high risk. They were given early doses of the T cells protectively and remained infection-free, Leen said.

Next, her team is beginning a bigger step - to try creating a bank of those cells from a variety of healthy donors that any patient could use, without having to custom-brew each dose.

Original post:
Designer T cells fight viruses after transplants - Quincy Herald-Whig | Illinois & Missouri News, Sports

PUBLIC RELEASE DATE:

24-Jun-2014

Contact: Jacob M. van Laar j.m.vanlaar@umcutrecht.nl The JAMA Network Journals

Among patients with a severe, life-threatening type of sclerosis, treatment with hematopoietic stem cell transplantation (HSCT), compared to intravenous infusion of the chemotherapeutic drug cyclophosphamide, was associated with an increased treatment-related risk of death in the first year, but better long-term survival, according to a study in the June 25 issue of JAMA.

Systemic sclerosis is an autoimmune connective tissue disease characterized by vasculopathy (a disorder of the blood vessels), low-grade inflammation, and fibrosis (development of excess fibrous connective tissue) in skin and internal organs. Previously, small studies have shown that systemic sclerosis is responsive to treatment with autologous HSCT, although it has been unclear whether HSCT improves survival, according to background information in the article. For this study, autologous HSCT involved a multistep process beginning with infusion of high doses of cyclophosphamide and an antibody against immune cells, followed by reinfusion of the patient's own stem cells that had been previously collected from blood and purified.

Jacob M. van Laar, M.D., Ph.D., of the University Medical Center Utrecht, Utrecht, the Netherlands and Dominique Farge M.D., Ph.D, of the Assistance Publique - Hopitaux de Paris, Paris 7 Diderot University, France, and colleagues randomly assigned 156 patients with early diffuse cutaneous (widespread skin involvement) systemic sclerosis to receive HSCT (n = 79) or cyclophosphamide (n = 77; 12 monthly infusions). The phase 3 clinical trial was conducted in 10 countries at 29 centers; patients were recruited from March 2001 to October 2009 and followed up until October 2013.

During a median follow-up of 5.8 years, 53 adverse events occurred: 22 in the HSCT group (19 deaths and 3 irreversible organ failures) and 31 in the control group (23 deaths and 8 irreversible organ failures). Patients treated with HSCT experienced more adverse events (including death) in the first year but had better long-term event-free survival than those treated with cyclophosphamide.

Patients in the HCST group experienced higher mortality in the first year but had better long-term overall survival than those treated with cyclophosphamide. During year 1 there were 11 deaths (13.9 percent, including 8 treatment-related deaths) in the HSCT group vs 7 (9.1 percent, no treatment-related deaths) in the control group. After year 2 of follow-up, there were 12 deaths (15.2 percent) in the HSCT group vs 13 (16.9 percent) in the control group. After 4 years of follow-up, there were 13 deaths (16.5 percent) in the HSCT group vs 20 (26.0 percent) in the control group.

The authors add that HSCT was also more effective than intravenous cyclophosphamide on measures evaluating skin, functional ability, quality of life, and lung function, consistent with previous studies.

"Among patients with early diffuse cutaneous systemic sclerosis, HSCT was associated with increased treatment-related mortality in the first year after treatment. However, HCST conferred a significant long-term event-free survival benefit," the authors conclude.

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Stem cell transplantation for severe sclerosis associated with improved long-term survival

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Newswise Among patients with a severe, life-threatening type of sclerosis, treatment with hematopoietic stem cell transplantation (HSCT), compared to intravenous infusion of the chemotherapeutic drug cyclophosphamide, was associated with an increased treatment-related risk of death in the first year, but better long-term survival, according to a study in the June 25 issue of JAMA.

Systemic sclerosis is an autoimmune connective tissue disease characterized by vasculopathy (a disorder of the blood vessels), low-grade inflammation, and fibrosis (development of excess fibrous connective tissue) in skin and internal organs. Previously, small studies have shown that systemic sclerosis is responsive to treatment with autologous HSCT, although it has been unclear whether HSCT improves survival, according to background information in the article. For this study, autologous HSCT involved a multistep process beginning with infusion of high doses of cyclophosphamide and an antibody against immune cells, followed by reinfusion of the patient's own stem cells that had been previously collected from blood and purified.

Jacob M. van Laar, M.D., Ph.D., of the University Medical Center Utrecht, Utrecht, the Netherlands and Dominique Farge M.D., Ph.D, of the Assistance Publique - Hopitaux de Paris, Paris 7 Diderot University, France, and colleagues randomly assigned 156 patients with early diffuse cutaneous (widespread skin involvement) systemic sclerosis to receive HSCT (n = 79) or cyclophosphamide (n = 77; 12 monthly infusions). The phase 3 clinical trial was conducted in 10 countries at 29 centers; patients were recruited from March 2001 to October 2009 and followed up until October 2013.

During a median follow-up of 5.8 years, 53 adverse events occurred: 22 in the HSCT group (19 deaths and 3 irreversible organ failures) and 31 in the control group (23 deaths and 8 irreversible organ failures). Patients treated with HSCT experienced more adverse events (including death) in the first year but had better long-term event-free survival than those treated with cyclophosphamide.

Patients in the HCST group experienced higher mortality in the first year but had better long-term overall survival than those treated with cyclophosphamide. During year 1 there were 11 deaths (13.9 percent, including 8 treatment-related deaths) in the HSCT group vs 7 (9.1 percent, no treatment-related deaths) in the control group. After year 2 of follow-up, there were 12 deaths (15.2 percent) in the HSCT group vs 13 (16.9 percent) in the control group. After 4 years of follow-up, there were 13 deaths (16.5 percent) in the HSCT group vs 20 (26.0 percent) in the control group.

The authors add that HSCT was also more effective than intravenous cyclophosphamide on measures evaluating skin, functional ability, quality of life, and lung function, consistent with previous studies.

Among patients with early diffuse cutaneous systemic sclerosis, HSCT was associated with increased treatment-related mortality in the first year after treatment. However, HCST conferred a significant long-term event-free survival benefit, the authors conclude. (doi:10.1001/jama.2014.6368; Available pre-embargo to the media at http://media.jamanetwork.com)

Editors Note: Please see the article for additional information, including other authors, author contributions and affiliations, financial disclosures, funding and support, etc.

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Stem Cell Transplantation For Severe Sclerosis Linked With Improved Long-term Survival

EXTON, Pa.--(BUSINESS WIRE)--Fibrocell Science, Inc., (NYSE MKT:FCSC), an autologous cell therapy company primarily focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet medical needs, announced today that it has entered into an exclusive license agreement with The Regents of the University of California. Under the agreement, Fibrocell acquired the rights to commercially apply patented discoveries and technologies resulting from the ongoing scientific collaboration between the University of California at Los Angeles (UCLA) and Fibrocell Science.

The technologies from the UCLA collaboration and exclusive license agreements enable Fibrocell to expand its proprietary Personalized Biologics platform which uses human fibroblasts and stem cells from skin to create localized therapies that are compatible with the unique biology of each patient. Specifically, the newly licensed patents and technologies relate to two advancements in the therapeutic application of cell therapies:

The technologies from these licenses further strengthen Fibrocells rich development platform, said David Pernock, chairman and chief executive officer of Fibrocell. These potentially transformational technologies offer partnering opportunities for Fibrocell.

The license agreements build upon an existing research collaboration between Fibrocell and UCLA that has already yielded discoveries and technologies related to stem cells and regenerative cells in human skin. Such research is led by James A. Byrne, Ph.D., an Assistant Professor in UCLAs Department of Molecular and Medical Pharmacology at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research.

About Fibrocell Science, Inc.

Fibrocell Science, Inc. (NYSE MKT:FCSC) is an autologous cell therapy company primarily focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet medical needs. Based on its proprietary autologous fibroblast technology, Fibrocell is pursuing breakthrough medical applications of azficel-T for restrictive burn scarring and vocal cord scarring. The companys collaboration with Intrexon Corporation (NYSE:XON), a leader in synthetic biology, includes using genetically-modified fibroblasts for treating rare and serious skin and connective tissue diseases for which there are no currently approved products. For additional information, visit http://www.fibrocellscience.com.

About UCLA

UCLAis Californias largest university, with an enrollment of more than 40,000 undergraduate and graduate students. The UCLA College of Letters and Science and the universitys 11 professional schools feature renowned faculty and offer 337 degree programs and majors. UCLA is a national and international leader in the breadth and quality of its academic, research, health care, cultural, continuing education and athletic programs. Seven alumni and six faculty have been awarded the Nobel Prize.

Forward-Looking Statements

This press release contains, and our officers and representatives may from time to time make, statements that are forward-looking statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Examples of forward-looking statements include, among others, statements we make regarding (i) our ability to develop breakthrough therapies for the treatment of skin and connective tissues diseases and (ii) our ability to successfully leverage our relationship with UCLA to expand our proprietary Personalized Biologics platform. These forward-looking statements rely on a number of assumptions concerning future events and are subject to a number of risks, uncertainties, and other factors, many of which are outside of Fibrocell Sciences control. Important factors that could cause our actual results and financial condition to differ materially from those indicated in the forward-looking statements include, among others, the following: (i) uncertainties relating to the initiation and completion of clinical trials; (ii) whether clinical trial results will validate and support the safety and efficacy of azficel-T; and (iii) our ability to establish additional strategic partnerships, as well as those set forth under the caption Item 1A. Risk Factors in Fibrocell Sciences most recent Form 10-K filing, as updated in Item 1A. Risk Factors in Fibrocell Sciences most recent Form 10-Q filing. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. In addition, Fibrocell Science operates in a highly competitive and rapidly changing environment, and new risks may arise. Accordingly, you should not place any reliance on forward-looking statements as a prediction of actual results. Fibrocell Science disclaims any intention to, and undertakes no obligation to, update or revise any forward-looking statement. You are also urged to carefully review and consider the various disclosures in Fibrocell Sciences most recent annual report on Form 10-K, our most recent Form 10-Q as well as other public filings with the SEC since the filing of Fibrocell Sciences most recent annual report.

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FIbrocell Science Announces Exclusive Technology License Agreements with UCLA to Advance the Development of ...