Fate Therapeutics Presents Preclinical Efficacy Data for WNT7A-Analog Program at Muscular Dystrophy Association 2013 …

SAN DIEGO, April 22, 2013 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc., a biopharmaceutical company engaged in the discovery and development of adult stem cell modulators, announced today the presentation of preclinical data from its WNT7a protein analog program for the treatment of muscular dystrophy at the Muscular Dystrophy Association (MDA) 2013 Scientific Conference, April 21-24, in Washington DC. The presentations describe the engineering of pharmaceutically optimized WNT7a protein analogs, as well as their mechanism of action and efficacy profile in preclinical pharmacology studies.

"The data presented today provide strong preclinical support for the therapeutic potential of WNT7a analogs in muscular dystrophy, a complex group of disorders with a large unmet need for novel, differentiated and potentially complementary treatment approaches," commented Christian Weyer, M.D., M.A.S., President and Chief Executive Officer of Fate Therapeutics. "We are working towards the nomination of an investigational new drug (IND) candidate later this year, and are excited about the potential to advance this new mechanism toward clinical studies."

In the MDX mouse model of muscular dystrophy, intramuscular injection of a novel WNT7a analog resulted in significant dose dependent muscle hypertrophy and several-fold expansion of the satellite stem cell population. Moreover, three weeks after a single intramuscular injection, functional assessment revealed a significant increase in strength of the targeted tibialis anterior muscle (+18%, p

The findings obtained with Fate's optimized WNT7a analogs expand upon those previously reported with non-modified WNT7a protein. In November 2012, muscle biology expert and Fate Therapeutics scientific founder Dr. Michael Rudnicki published data demonstrating the potential of WNT7a to ameliorate muscle degeneration in the MDX mouse model of muscular dystrophy (Von Maltzahn et. al., PNAS 2012). In previous studies, Dr. Rudnicki elucidated the unique biology of WNT7a and its dual mechanism of action of driving the expansion of the muscle satellite stem cell population and muscle hypertrophy.

About Muscular Dystrophy

Muscular dystrophies encompass a group of disorders with diverse pathophysiological manifestations resulting from genetic aberrations which include mutations or deletions to over 30 distinct genes. The most prevalent and well characterized is Duchenne muscular dystrophy (DMD), an X-linked form of muscular dystrophy which is seen in 1/3500 live male births. DMD typically manifests in early childhood and progresses to an advanced stage of severe muscular degeneration resulting in impairment of ambulation and premature mortality. A core pathophysiologic phenomenon seen in muscular dystrophy is a cycle of muscle degeneration leading to continuous compensatory satellite cell activation and differentiation to affect a regenerative response, but resulting in the eventual exhaustion of the regenerative capacity and significant loss of muscle function. Enhancing the underlying molecular and cellular mechanisms to restore the regenerative capacity of muscle satellite stem cells thus represents a promising and unique approach for therapeutically intervention in various forms of muscular dystrophy as well as other neuromuscular diseases.

About Fate Therapeutics, Inc.

Uniquely positioned at the intersection of stem cell science and orphan disease, Fate Therapeutics is pioneering the discovery and development of innovative adult stem cell modulator therapeutics with the potential to cure or transform the lives of patients with rare life-threatening disorders. The Company's lead program, ProHema, an innovative cord blood-derived cell therapy containing ex-vivo pharmacologically-modulated hematopoietic stem cells (HSCs), is currently in Phase 2 testing in patients with leukemia undergoing hematopoietic transplantation. The Company plans to pursue clinical evaluation of pharmacologically modulated HSCs in patients with rare genetic disorders, an area of tremendous unmet medical need in which the curative potential of cord blood transplantation is well recognized. In addition, Fate Therapeutics is developing proprietary WNT7a-based protein therapeutics that have shown efficacy in preclinical models of muscular dystrophy. To advance its discovery efforts, the Company applies its award-winning, proprietary, induced pluripotent stem cell (iPSC) technology to generate rare cell populations and model disease. Fate Therapeutics is headquartered in San Diego, CA, with a subsidiary in Ottawa, Canada. For more information, please visit http://www.fatetherapeutics.com.

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Voices Against Brain Cancer Comments on New Study Claiming Fat Tissue Stem Cells can Fight Brain Cancer

Medical Daily reports on a new study where stem cells from fat tissue were used to help track difficult regions in the brain prone to remission. Voices Against Brain Cancer releases a statement encouraging more stem cell research.

New York, NY (PRWEB) April 18, 2013

According to the Medical Daily article by Ansa Varughese, the study showed that stem cells from fat tissue can be used to track difficult regions in the brain prone to remission. Scientists cannot yet determine why or how these stem cells target the prone areas, but the cells seem naturally drawn to the damaged areas. Researchers are using the stem cells as transporters to help deliver drugs for treatment in the cancer spreading regions of the brain.

Alfredo Quinones-Hinojosa, the lead researcher and professor of neurosurgery, oncology, and neuroscience at Johns Hopkins, was quoted in the article as saying, The biggest challenge in brain cancer is the migration of cancer cells. Even when we remove the tumor, some of the cells have already slipped away and are causing damage somewhere else. Building off our findings, we may be able to find a way to arm a patient's own healthy cells with the treatment needed to chase down those cancer cells and destroy them. It's truly personalized medicine.

The Medical Daily article goes on to mention that harvesting the mesenchymal stem cells from the fat tissue is safer than getting the cells from bone marrow. While it will still take years before a clinical trial happens in the U.S., the new stem cell treatment could play a major part in battling brain cancer.

Michael Klipper, Chairman of VABC, offers his comments on the new study. This is a great step toward battling brain cancer. While its still a new study, it seems to be having positive results, and can hopefully become something used in brain cancer treatment in the future. Dealing with brain tumors and the treatment after can be a major, painful ordeal. Hopefully this new study will lead to a new way patients can be treated after theyve had a tumor removed.

VABC has a wide variety of initiatives in place for brain cancer research, awareness and support. The organizations research grants fund cutting-edge research programs that will have a monumental impact on the diagnosis and treatment of brain cancer. VABC currently funds research at several esteemed institutions such as Brookhaven National Laboratory, Cleveland Clinic, Columbia, Cornell, Duke, Harvard, John Hopkins, Memorial Sloan-Kettering and Yale, to name a few.

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Alicia McAllister 5W Public Relations 646-430-5155 Email Information

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Voices Against Brain Cancer Comments on New Study Claiming Fat Tissue Stem Cells can Fight Brain Cancer

Stem cell donor ditches Stanford University professor

MUMBAI: Healthy stem cells from a person of the same genetic make can mean life for Stanford University professor Nalini Ambady, or death if she doesn't find a donor in a month's time. A matching donor, recently found in Mumbai after an almost six-month-long search across India, backed out after initially consenting to stem-cell donation.

When her eight-year-old leukemia made a comeback last year, doctors in the US suggested she look for an Indian donor for a greater match probability, given ethnic similarities. After failing to find a match in the US' National Marrow Donor Program (NMDP) that boasts of a database of 10 million registered donors, Ambady's family finally directed their search towards India last November. Yet, two prominent stem cell donor registries in India with a combined pool of 50,000 donors could not find a match until recently.

Beating the odds of one in over 20,000, the unexpected happened last week when city-based Marrow Donor Registry of India (MDRI) found the Human Leukocyte Antigens (HLAs) match in a software engineer. HLA match refers to immunological compatibility, and doctors look for a match in at least 10 counts of crucial antigens to go ahead with a transplant. Coincidentally, not only did the donor hail from Ambady's hometown in Kerala, six out of 10 HLA parameters also matched. But Ambady's hopes crashed when after initially giving consent and registering as a donor, the engineer refused to go ahead with the donation citing health and later family problems.

Several rounds of counselling by members of MDRI, including founder head Sunil Parekh, did not cut much ice. Parekh said this was the sixth instance where the registry failed to convert a match into a transplant. Ambady's family and students have started a massive campaign across social media websites to hunt for a donor in time.

"Since we started the registry in 2010, there have been 55 requests for a match and we have managed to find six matches so far. But barring one instance, the donors backed out in all cases," he said, adding donor attrition has emerged as the biggest problem in the way of unrelated stem-cell transplants in the country.

While over 500 related stem cell transplants are carried out to cure leukemia, lymphoma and several other life-threatening cancers in the country annually, the numbers drop to almost one-tenth when it comes to transplants through unrelated donors. Also, in most of the unrelated stem cell transplants, the cells have to be imported from the US. "Just because the cells are imported, the cost of transplant could go up to around Rs 50 lakh and therefore remains out of bounds for the majority," said Dr Navin Khattry of Tata Memorial Hospital's Advanced Centre for Treatment, Research and Education in Cancer, which is the only one to carry out unrelated transplants in Mumbai. "The solution to this is obviously having a larger database of Indian donors," he said.

In global registries too, ethnic groups, particularly Asians, are not too well represented, which majorly reduces their chances of finding a match. According to Parekh, the problem of donor attrition only aggravates it and adds to the higher mortality rate. Donor attrition is usually due to apprehensions about the process and its long-term outcome. However, Parekh said stem cell donation, like blood donation, has no long-term effects on the donor and cells are replenished in six to eight weeks.

However, Raghu Rajagopal, the co-founder and CEO of Datri, which has the biggest database of 36,000 registered stem cell donors, said even global registries face an attrition of about 50%. "But in their case, if one donor backs out, there would be three more willing to come forward," he said.

Datri has also been holding camps for Ambady in Kochi. Ambady's family and friends, however, have appealed that only those who are serious about donating should come forward.

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Stem Cell Treatment in India for Diabetes : Testimonial – Video

Stem Cell Treatment in India for Diabetes : Testimonial
Stem cell treatment of diabetes is indicated at all stages of the disease. It is, however, the most effective in the cases of: New-onset insulin-dependent di...

By: StemRx BioScience

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Stem Cell Treatment in India for Diabetes : Testimonial - Video

Sen. Carrell to receive stem cell transplant soon

OLYMPIA, Wash.

Republican state Sen. Mike Carrell, who has a blood condition and has been hospitalized since last month, will receive a stem cell transplant from his brother next week.

In an email sent to constituents this week, Carrell says he started initial treatment on Wednesday at the Seattle Cancer Care Alliance. He said that the 100-day process begins with low doses of chemotherapy to lower his body's defenses and help increase his chances that next Tuesday's procedure will be successful.

Earlier this year, Carrell was diagnosed with myelodysplastic syndrome, also known as pre-leukemia. The chronic disease affects blood-forming stem cells.

Carrell was hospitalized last month after contracting a complicating illness while serving in Olympia, and he has not been back to the Capitol since. Republican leaders have said he would only be called back to the Legislature if his presence is needed for a crucial vote. Lawmakers are nearing the end of the 105-day session, which is set to end on April 28.

Carrell said he'll first undergo a low dose of full-body radiation, and then within a few hours will be injected with his brother's stem cells.

"Doctors are hopeful that my brother's healthy stem cells will march in and take over for the weakened marrow cells in my body," he wrote.

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Sen. Carrell to receive stem cell transplant soon

Japanese Firm Luring Investors With Nobel-Winning Technology

Retina Institute Japan K.K., which is employing Nobel Prize-winning stem-cell technology to treat eye diseases, plans to sell a stake in itself to a group of Japanese companies next month ahead of a possible initial public offering in five years.

The company, based in Fukuoka City, Japan, will raise 1 billion yen ($10.2 million) from the sale to fund development of a treatment for age-related macular degeneration -- a leading cause of blindness in the elderly -- using technology developed by Riken, Japans state-controlled research institute, Chief Executive Officer Hardy Kagimoto said in an interview.

After raising about 32 billion yen so far from investors, Retina is developing technology from a discovery that won Shinya Yamanaka, a professor at Kyoto University, the Nobel Prize for medicine in October. Yamanaka discovered a way to turn ordinary skin cells into what are called induced pluripotent stem, or iPS, cells.

The development of retina treatment with iPS cells can lead to development of the cell-utilized therapies for a wide range of diseases, said Akitsu Hotta, an assistant professor at Kyoto University who studies stem cells. Retinas commercialization of the technology will be a big milestone.

Retina last month estimated the potential market for its treatments at $21 billion.

The sale of a 3 percent stake values Retina at 33 billion yen. Dainippon Sumitomo Pharma Co. (4506), which paid 1.5 billion yen for 5 percent of Retina, jumped as much as 8 percent in Tokyo trading the day after that deal was announced in March.

Shin Nippon Biomedical Laboratories Ltd. (2395), which said April 9 that it plans invest 300 million yen in Retina, jumped as much as 12 percent today. Tella Inc. (2191), which is buying 100 million yen of shares in the company, was up 7.4 percent. Both stocks have more than quadrupled over the past six months.

Investors are rushing to buy shares of companies that are involved in stem-cell therapy and treatment technologies, said Tsutomu Yamada, a Tokyo-based analyst at Kabu.com Securities Co.

Kagimoto, 36, declined to name the companies participating in the current round of fundraising and said he has no plans to sell shares to local or foreign private equity firms.

Retina is targeting an IPO in five years in Japan and the U.S. to finance commercialization of the treatment, said Kagimoto, whos also a medical doctor. The company will conduct a clinical trial in Japan for treatment for age-related macular degeneration, and similar testing in the U.S., he said.

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Japanese Firm Luring Investors With Nobel-Winning Technology

Stem cell senescence drives aging, study suggests

Apr. 18, 2013 Declining levels of the protein BubR1 occur when both people and animals age, and contribute to cell senescence or deterioration, weight loss, muscle wasting and cataracts. Mayo Clinic researchers have shown that adult progenitor or stem cells -- important for repair and regeneration of skeletal muscle and maintenance of healthy fat tissue -- are subject to cellular senescence, and that clearance of these cells limits age-related deterioration of these tissues.

The findings appear today online in the journal Cell Reports.

BubR1 is an essential part of the mitotic checkpoint, the mechanism controlling proper cell division or mitosis. Without sufficient levels of BubR1, chromosomal imbalance will occur, leading to premature aging and cancer. Using mutant mice that expressed low levels of BubR1, the researchers found development of dysfunctional tissue with impaired cell regeneration. In analyzing the progenitor populations in skeletal muscle and fat, they found that a subset of progenitors was senescent and that the tumor suppressor p53 was acting to prevent this from happening through activation of p21.

"Earlier we discovered that senescent cells accumulate in tissues with aging and that removal of these cells delays age-related functional decline in these tissues," says Jan van Deursen, Ph.D., a Mayo Clinic molecular biologist and senior author of the study. "The key advance of the current study is that the progenitor cell populations are most sensitive for senescence, thereby interfering with the innate capacity of the tissue to counteract degeneration."

Not only do the findings contribute to knowledge on cell senescence as it relates to aging and related diseases, but understanding the mechanisms may lead to future therapies, say the researchers.

Co-authors include Darren Baker, Ph.D., and Robbyn Weaver both of Mayo Clinic.

The research was supported by the Ellison Medical Foundation, the Noaber Foundation, the National Institutes of Health (CA96985 and AG41122), and the Mayo Clinic Robert and Arlene Kogod Center on Aging.

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