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Gladstone scientists use stem cell technology to tackle Huntington's disease

Public release date: 28-Jun-2012 [ | E-mail | Share ]

Contact: Diane Schrick 415-734-2538 Gladstone Institutes

SAN FRANCISCO, CAJune 28, 2012Scientists at the Gladstone Institutes and an international team of researchers have generated a human model of Huntington's diseasedirectly from the skin cells of patients with the disease.

For years, scientists have studied Huntington's disease primarily in post-mortem brain tissue or laboratory animals modified to mimic the disease. Today, in Cell Stem Cell, the international team shows how they developed a human model of Huntington's disease, which causes a diverse range of neurological impairments. The new model should help scientists better understand the development of Huntington'sand provide better ways to identify and screen potential therapeutics for this devastating disease.

This new model comes at a time of concentrated federal efforts to accelerate solutions for diseasesincluding a number of debilitating conditions that touch only small percentages of the population. Last year, the National Institutes of Health consolidated its efforts to attack rare diseases under the new National Center for Translational Sciences.

Huntington's is such a rare disease, although it is the most common inherited neurodegenerative disorder. It afflicts approximately 30,000 people in the United Stateswith another 75,000 people carrying the gene that will eventually lead to it. Caused by a mutation in the gene for a protein called huntingtin, the disease damages brain cells so that people with Huntington's progressively lose their ability to walk, talk, think and reason.

"An advantage of this human model is that we now have the ability to identify changes in brain cells over timeduring the degeneration process and at specific stages of brain-cell development," said Gladstone Senior Investigator Steve Finkbeiner, MD, PhD. "We hope this model will help us more readily uncover relevant factors that contribute to Huntington's disease and especially to find successful therapeutic approaches."

In this research, Dr. Finkbeiner and others took advantage of advanced "reprogramming" techniques pioneered by Gladstone Senior Investigator Shinya Yamanaka, MD, PhD. They reprogrammed skin cells from Huntington's disease patients into stem cells known as induced pluripotent stem cells, or iPS cellswhich can become virtually any cell type in the body. The researchers then instructed the iPS cells to develop into neurons, a key type of brain cell. Importantly, each cell line contained a complete set of the genes from each Huntington's disease patient. Because each patient has a different pattern of disease onset and duration, this model may replicate Huntington's more faithfully than animal models do. The model is likely to prove more useful in understanding the disease's progression.

"The iPS cells will provide insights into Huntington's disease, helping us to develop new therapies and test drug candidates," said Dr. Finkbeiner, who is also a professor of neurology and physiology at the University of California, San Francisco, with which Gladstone is affiliated. "We hope that drugs developed with this new human model will have greater success in clinical trials. The track record of animal models for predicting therapies that will work in people has been poor, making drug discovery for neurodegenerative diseases very costlyand therefore less attractive to drug companies. We hope to change that."


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Osiris Bolsters its Stem Cell Intellectual Property Estate


Osiris Therapeutics, Inc. (OSIR), announced today the expansion of its intellectual property protection around Prochymal (remestemcel-L). The United States Patent and Trademark Office recently granted Osiris two patents that cover multiple mechanisms of action related to cardiac tissue repair. Additionally, Osiris has enhanced its mesenchymal stem cell (MSC) patent estate with the issuance of patents across Europe and Australia covering stem cells expressing all therapeutically useful levels of cell surface receptors for TNF-alpha, a receptor essential to the cell's ability to counteract inflammation. These patents further support Osiris' considerable intellectual property position, which includes 48 issued U.S. patents around the production, composition, testing and use of the mesenchymal stem cell from both allogeneic and autologous sources.

"These recent additions to Osiris patent estate, combined with the existing broad coverage of our pioneering MSC platform technology, reinforce our industry leading IP portfolio and bolster our dominant position regarding the manufacture and use of mesenchymal stem cells for the treatment of a broad range of diseases, said Chris Alder, Chief Intellectual Property Counsel of Osiris. We have invested significant time and resources building our intellectual property estate, and with the commercialization of Prochymal, we are preparing to take the necessary action to enforce our considerable rights.

Prochymal is now approved in Canada and New Zealand, and is currently available in seven other countries including the United States under an Expanded Access Program. With Prochymal (remestemcel-L) entering commerce, Osiris has initiated the process of identifying entities that may be infringing upon its intellectual property rights and will take appropriate action as necessary.

About Prochymal (remestemcel-L)

Prochymal is the worlds first approved drug with a stem cell as its active ingredient. Developed by Osiris Therapeutics, Prochymal is an intravenous formulation of MSCs, which are derived from the bone marrow of healthy adult donors between the ages of 18 and 30 years. The MSCs are selected from the bone marrow and grown in culture so that up to 10,000 doses of Prochymal can be produced from a single donor. Prochymal is truly an off-the-shelf stem cell product that is stored frozen at the point-of-care and infused through a simple intravenous line without the need to type or immunosuppress the recipient. Prochymal is approved in Canada and New Zealand for the management of acute graft-versus-host disease (GvHD) in children and is available for adults and children in eight countries including the United States, under an Expanded Access Program. Prochymal is currently in a Phase 3 trial for refractory Crohns disease and is also being evaluated in clinical trials for the treatment of myocardial infarction (heart attack) and type 1 diabetes.

About Osiris Therapeutics

Osiris Therapeutics, Inc. is the leading stem cell company, having developed the worlds first approved stem cell drug, Prochymal. The company is focused on developing and marketing products to treat medical conditions in inflammatory, cardiovascular, orthopedic and wound healing markets. In Biosurgery, Osiris currently markets Grafix for burns and chronic wounds, and Ovation for orthopedic applications. Osiris is a fully integrated company with capabilities in research, development, manufacturing and distribution of stem cell products. Osiris has developed an extensive intellectual property portfolio to protect the company's technology, including 48 U.S. and 144 foreign patents.

Osiris, Prochymal, Grafix and Ovation are registered trademarks of Osiris Therapeutics, Inc. More information can be found on the company's website, (OSIRG)

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UMass stem cell lab to close

The laboratory grew and stored human stem cells, which are capable of becoming any cell in the body, and made them available to scientists nationwide for use in experiments to study diseases such as diabetes and spinal cord injuries. When it is dismantled, several thousand vials of stem cellswill be sent back to the research centers where they originated, and the equipment will be given to other UMass labs.

Susan Windham-Bannister, president of the Massachusetts Life Sciences Center, a quasi-public agency that oversees the $1 billion life sciences initiative, defended the decision to initially fund the stem cell bank. She said there are many examples of technology that in hindsight are unnecessary, but at the time it was conceived, when the investment was made, it was absolutely state of the art. The center, she said, was one of them.

Originally, the bank was seen as a repository for embryonic stem cell lines that were being created but were not eligible for federal funding under Bush-era restrictions. The field has evolved significantly since then, with President Obamas loosening of restrictions on federal funding and the development of new technologies for making stem cells.

Still, stem cell banks are seen as useful by some. The California Institute for Regenerative Medicine, for example, is preparing to invest $10 million in its own stem cell banking initiative, and another $20 million to underwrite the creation of stem cells from patients with specific diseases.

Massachusetts Senate minority leader Bruce Tarr, Republican of Gloucester, said he was concerned that lawmakers had not been told the bank would close.

Given the fact that this is a resource that was created by an act of the Legislature, I would hope anyone seeking to change its status would consult with the Legislature, he said. The notion has always been we have been working hard to make Massachusetts a leader in stem cell research, and I dont know how ceasing the operations of the stem cell bank advances that goal.

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Medistem Receives Notice of Patent Allowance Covering Fat Stem Cell Therapy of Autoimmune Diseases

SAN DIEGO CA--(Marketwire -06/29/12)- Medistem Inc. (MEDS) announced today notice of allowance from the United States Patent and Trademark Office (USPTO) for a patent covering the use of fat stem cells, and cells associated with fat stem cells for treatment of diseases related to a dysfunctional immune system. Such diseases include multiple sclerosis, Type 1 diabetes, rheumatoid arthritis and lupus. The allowed patent, entitled "Stem Cell Mediated Treg Activation/Expansion for Therapeutic Immune Modulation" has the earliest priority date of December 2006.

"We have previously published that giving multiple sclerosis patients cells extracted from their own fat tissue, which contains stem cells, appears to confer clinical benefit in a pilot study," said Thomas Ichim, CEO of Medistem. "The current patent that has been allowed, in the broadest interpretation of the claims, gives us exclusive rights to the use of specific types of fat stem cell therapy for autoimmune diseases such as multiple sclerosis."

Subsequent to the filing of the patent application, Medistem together with collaborators at the Lawson Health Sciences Research Institute, Canada, reported data that fat tissue contains high numbers of T regulatory cells, a type of immune cell that is capable of controlling autoimmunity.

This finding was independently confirmed by Dr. Diane Mathis' laboratory at Harvard University, who published a paper in the prestigious journal, Nature Medicine, in which detailed experimental evidence was provided supporting the initial finding that adipose tissue contains high numbers of T regulatory cells. A video describing the paper can be accessed at

The current patent discloses the use of T regulatory cells from fat, combinations with stem cells, and use of fat-derived mononuclear cells. Given that there are currently several groups utilizing this technology in the USA in treating patients, Medistem believes revenue can be generated through enforcement of patent rights.

"Our corporate philosophy has been to remain highly focused on our ongoing clinical stage programs using Medistem's universal donor stem cell, the Endometrial Regenerative Cell (ERC), in the treatment of critical limb ischemia and congestive heart failure," said Dr. Vladimir Bogin, Chairman and President of Medistem. "However, due to the ease of implementation of our fat stem cell technology, combined with the major burden that autoimmune diseases have on our health care system, we are highly incentivized to explore partnering, co-development and licensing opportunities."

Autoimmune conditions occur as a result of the body's immune system "turning on itself" and attacking its own organs or cells. Current treatments for autoimmune conditions are based on "globally" suppressing the immune system by administration of immunosuppressive drugs. This is associated with an increased predisposition to infections and significant side effects. The utilization of stem cells and T regulatory cells offers the potential to selectively suppress pathological immunity while preserving the ability of the body to fight bacteria and viruses. According to the NIH there are approximately 23 million victims of autoimmune conditions.

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Stem cell therapy in Hawaii going to the dogs

HAWAII KAI (HawaiiNewsNow) -

Cutting-edge technology is helping Hawaii's pets live better lives for months, even years. We were there as a beloved dog named Kumba received one of the first-ever, in-clinic stem cell therapy surgeries in the islands.

13 year old Kumba doesn't know he's a guinea pig. The Rottweiler-Lab mix is one of the first in Hawaii to undergo the stem cell procedure at Surf Paws in Hawaii Kai.

Kumba suffers severe arthritis in his hips and knees, doesn't eat much, and is even a bit depressed. "It's an effort for him to get up off the floor, and when he gets up and crosses the room, you can see the stiffness," says his owner, Rumi Hospodar.

Kumba's kids learn some of details of his surgery. Then, he's moved to a table and nods off from anesthesia. Once he's prepped, the procedure begins. The vet removes about two tablespoons of fat tissue from Kumba's shoulder. From there, the stem cells are separated from the fat and activated. Then, they're injected back into the affected areas.

The entire process takes four hours, but the dog is actually only under for about 20 minutes. Surf Paws used to send the tissue to the mainland for processing, but with technology from Medi-Vet America, they can do it all here.

"The patient had to be, you know, go home and come back a few days later and the timing was a little bit difficult. Now, everything is same day," says Surf Paws veterinarian Dr. Cristina Miliaresis.

Cost depends on the size of animal but can run up to $2,800. It's mainly done on dogs, cats, and horses who suffer osteoarthritis, hip dysplasia, ligament and cartilage damage, and other degenerative diseases. Their quality of life can improve within a couple of weeks.

Dr. Miliaresis says, "Some people might say, 'Oh, the dog's 13. Why are you doing this for a 13 year old dog? But even 6 months, pain-free, after a very, it's not simple, but it's a pretty straightforward procedure, to me (would be) just amazing."

The techs move all 97 pounds of Kumba to post-op - while his anxious owner looks on.

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Rensselaer and New York State Launch New Stem Cell Research Center

Newswise Troy, N.Y. Ground-breaking research to advance the application of stem cells to address critical injuries and diseases will be taking place at Rensselaer Polytechnic Institute, in a new center funded by New York state and opened today (June 22, 2012).

The Rensselaer Center for Stem Cell Research was launched officially by Rensselaer President Shirley Ann Jackson, New York State Department of Health Commissioner Nirav Shah, and Jonathan Dordick, director of the Rensselaer Center for Biotechnology and Interdisciplinary Studies (CBIS) and the Howard P. Isermann 42 Professor of Chemical and Biological Engineering. They were joined at the ribbon cutting by Glenn Monastersky, CBIS operations director and biomedical engineering professor of practice. Monastersky is also principal investigator under the $2.45 million grant awarded to fund the new center, from the New York State Stem Cell Science Program (NYSTEM).

The opening of the Rensselaer Center for Stem Cell Research marks a milestone on the path toward this important area of exploration, which promises so much in terms of alleviating disease and improving health, said Jackson. At the center we will work at the frontiers of this promising discipline in collaboration with New York state and investigators from across the region.

This research complements stem cell research, sponsored by New York and the National Institutes of Health, that is already in progress at Rensselaer, Jackson added. Due to our commitment to biotechnology that began over 10 years ago under The Rensselaer Plan, we are able to link engineering with the life and physical sciences in ways that allow us to explore new possibilities. We are grateful to Dr. Shah and the state of New York for their leadership in this important research area, and for providing the funding to launch this center.

Governor Cuomo recognizes that stem cell research is a vital and growing industry that helps create jobs here in the Capital Region and around the state, said Nirav R. Shah, M.D., M.P.H., New York State Commissioner of Health. Targeting our investment in results-oriented research enterprises like this center will lead to medical advances as well as expand our economy and make New York the place to be for 21st century health and science research.

According to Dordick, the new center continues to place CBIS and the research conducted there on the leading edge of efforts to harness advances in biotechnology to address 21st century health challenges.

Ranging from our work on the blood anti-coagulant drug heparin to solutions to fighting some of todays super bugs to important advances in understanding Alzheimers disease, we are focusing our efforts on scientific advances that will ultimately open the doors to new cures for traumatic injuries or treatments for long-term conditions and diseases, Dordick said. Now, working with our partners at New York state and other researchers in the region, we will expand our work on stem cells to help the medical and scientific research communities advance efforts to better understand those cells and how they can be used in medicine.

Research on stem cells offers promise in an array of health areas, ranging from trying to regenerate damaged nerve cells following spinal cord injuries to offering potential cures for autoimmune diseases such as multiple sclerosis, lupus, rheumatoid arthritis, and Type 1 diabetes. According to the National Institutes of Health (NIH), stem cells are important because unlike other cells in the human body they are capable of dividing and renewing themselves for long periods. In addition, because stem cells are unspecialized meaning that they are not associated with any tissue-specific functions early research has shown that under the right circumstances these cells can give rise to cells associated with specific functions, under a process called differentiation. Today, according to NIH, scientists are just beginning to understand the so-called triggers that can start stem cell differentiation into, for example, nerve, muscle, or bone cells.

The new state-of-the-art center is housed on the ground floor of the Rensselaer biotechnology center. In addition to advanced cell biology research equipment, new lab equipment acquired with funding from New York state includes an Olympus VivaView microscopy/incubation system and a Thermo Fisher Arrayscan cell-imaging system that utilizes advanced optics and analytical software to guide the analysis of stem cell development.

The Rensselaer Center for Stem Cell Research and its associated scientific staff, will enable collaborations with several New York partners including the New York Neural Stem Cell Institute, Albany Medical College, the University at Albany, the University of Rochester Medical School, and the Trudeau Institute. The main focus of the center is the basic science critical to development of stem cell-based therapies for human diseases and traumatic injuries.

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