Stem-cell research: $37 million

Two teams including UC Irvine scientists will receive $37 million to push stem-cell treatments toward human testing one for a condition that leads to blindness, another for Alzheimers disease.

The awards, made Wednesday by the states stem-cell funding agency, include $17.3 million for a team that will cultivate retinal progenitor stem-cells to treat a disease known as retinitis pigmentosa.

Human neural stem cell.

COURTESY STEMCELLS, INC.

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The team includes Henry Klassen, a UC Irvine associate opthalmology professor, as well as researchers from UC Santa Barbara and Cedars-Sinai Medical Center. The UC Irvine share of that award is about $6 million.

The disease is often diagnosed when patients are in their teens or young adulthood, and progresses into middle age.

First theres night blindness, Klassen said. Then tunnel vision, and eventually, complete blindness.

The treatment hes developing relies on retinal stem cells that have matured enough to be specific to eye function. In previous testing, it has restored vision in rats.

The funding will allow more preliminary work in preparation for human testing. Food and Drug Administration approval, Klassen said, could come as soon as the end of next year.

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Stem-cell research: $37 million

Stem-cell patients said to recover body sensation

Two patients in one of the worlds first tests of a stem-cell treatment for spinal-cord injury have reported feeling sensation in parts of their bodies below the injury where they felt no sensation before.

The two patients, whose identity and gender were kept confidential, are part of a trial in Switzerland of a treatment developed by a team that included UC Irvine researchers.

UC Irvine husband-wife researchers Brian Cummings and Aileen Anderson pose with their daughter, Camryn, and their dogs.

FILE PHOTO: MARK RIGHTMIRE, THE ORANGE COUNTY REGISTER

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A third patient reported no improvement in sensation, but, like the others, has suffered no ill effects from the treatment.

All three patients had suffered spinal-cord injuries at the chest level that left them with no feeling below the site of the injury.

Two of them, however, reported feeling touch and warmth down to their belly-button region after receiving injections of 20 million human neural stem cells at the injury site.

Scientists involved in the trial did not say how long it took for the patients to feel sensation after their injections, though they did say the patients improved over a six-month period.

The stem-cell treatment was developed by the UCI husband-wife research team, Brian Cummings and Aileen Anderson, using neural stem cells grown in the laboratory by StemCells Inc. of Newark, in the Bay Area.

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Stem-cell patients said to recover body sensation

Stem Cell Research and Your Dog

By Kim Ribbink, Studio One Networks

Stem cell research often conjures images of political firestorms and futuristic science, yet it's a field that's already offering hope for humans and dogs alike. While the ideal of fixing spinal injuries and curing disease may be a long way off, dogs treated with stem cells are enjoying a new lease on life.

Pepper, a 10-year-old standard poodle, is a case in point. Crippled with arthritis in both his hips, Pepper came to James Gaynor, DVM, M.S., medical director of Animal Anesthesia & Pain Management Center in Colorado Springs, Colo., with his owners, who fully expected to have to put their pet to sleep. Conventional treatments hadn't worked, or had made their dog even sicker. In fact, Pepper's owners were so certain nothing could be done that they bought another puppy. "At our 60 day recheck, the owner was hugging me and crying out of happiness because, in her words, we gave her back her dog," Dr. Gaynor says. "The only problem was she now had Pepper and a puppy."

Healthy Bones It wasn't long ago that dogs like Pepper with arthritis had few options beyond conventional anti-inflammatory treatments -- including a variety of non-steroid anti-inflammatory drugs (NSAIDs) such as Metacam, Previcox, Rimadyl and phenylbutazone; steroid medications such as Prednisone; and disease-modifying osteoarthritis drugs (DMOADs) such as Adequan Canine -- that sometimes don't work. Now stem cells are providing an alternative. One California-based company, Vet-Stem, uses stem cells from dogs' own fat to treat animals in pain.

According to Julie Ryan Johnson, DVM, vice president of sales and marketing, studies have shown that fat is very rich with stem cells, making it an ideal source, and one that is nearly free from controversy, given that most of us don't mind having a bit of fat removed. "The way we do this is a veterinarian will send us a sample of the dog's fat," Dr. Ryan Johnson says. "We isolate the stem cells from that and then send the stem cells back to the veterinarian who injects them back into the dog -- for example, into an arthritic hip or elbow."

Once in the dog, the stem cells communicate with other cells in their environment. While it's not known exactly how they work, they do decrease the dog's pain level. "It's provided the veterinarian with another solution for helping these animals that have pain or difficulty moving," Dr. Ryan Johnson says. "Most importantly, for the dog and the dog owner, it offers quality of life."

The Possibilities Richard Vulliet, Ph.D., DVM, professor and director of the Laboratory of Veterinary Cytotherapeutics at UC Davis, says stem cells haven't cured any diseases yet, but researchers are working hard to change that. "I think that stem cells in general will rewrite the medical textbooks in the next 10 to 20 years," Dr. Vulliet says. "They will have an impact on human, canine, feline and equine health and will allow us to treat diseases that we can only dream about at this time."

Tony Kremer, DVM, an Illinois-based veterinary surgeon, says that as research progresses into the origin of diseases, there is hope that stem cell therapy might one day be used to treat diabetes and muscular dystrophy in dogs. "It is hoped that this research can repair or replace diseased organs, severed spinal cords, or brain cells destroyed by Alzheimer's disease in humans and dogs," he says.

Dr. Vulliet works with adult bone marrow stem cells to investigate potential cures for diseases that cause misery for many dogs. Your dog may soon be able to get breakthrough treatment in the following areas:

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Stem Cell Research and Your Dog

UCI researchers' work could help paralyzed patients

Two UC Irvine associate professors have laid the foundation for a treatment that could help people who are paralyzed by spinal cord injuries.

Aileen Anderson and Brian Cummings developed a stem cell treatment in collaboration with Northern California-based StemCells Inc. that is being used in clinical trials in Switzerland.

The treatment has resulted in the world's first case of patients regaining some feeling, according to UCI.

"We think this really bodes well for the next stage of the trial," Anderson said.

The initial clinical trial at the Balgrist University Hospital in Zurich injected adult human neural stem cells into the spinal cords of three paralyzed patients.

Two have regained some feeling of touch and heat, according to StemCells Inc., which developed the cell.

The treatment has the potential to not only restore feeling, but also help restore control over bowel movements, said UCI spokesman Tom Vasich.

The trial shows strong evidence that the cell is doing what it was created to do, but the results are not yet concrete, Anderson said. The trial only used three patients, an initial group to determine if the approach is safe, and it didn't have a control group.

"Despite all of these caveats, it's much more than we hoped for," she said. "We're all ecstatic."

The clinical trials are the newest phase of a nearly 10-year research project.

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UCI researchers' work could help paralyzed patients

Stem-cell-protecting drug could prevent the harmful side effects of radiation therapy

Public release date: 6-Sep-2012 [ | E-mail | Share ]

Contact: Elisabeth Lyons elyons@cell.com 617-386-2121 Cell Press

Radiation therapy is one of the most widely used cancer treatments, but it often damages normal tissue and can lead to debilitating conditions. A class of drugs known as mammalian target of rapamycin (mTOR) inhibitors can prevent radiation-induced tissue damage in mice by protecting normal stem cells that are crucial for tissue repair, according to a preclinical study published by Cell Press in the September issue of the journal Cell Stem Cell.

"We can exploit the emerging findings for the development of new preventive strategies and more effective treatment options for patients suffering this devastating disease," says senior study author J. Silvio Gutkind of the National Institute of Dental and Craniofacial Research.

In response to radiation therapy, cancer patients often develop a painful condition called mucositistissue swelling in the mouth that can leave these patients unable to eat or drink and force them to rely on opioid-strength pain killers. Radiation therapy may cause this debilitating condition by depleting normal stem cells capable of repairing damaged tissue.

In the new study, Gutkind and his team found that the mTOR inhibitor rapamycin protects stem cells taken from the mouths of healthy individuals (but not cancer cells) from radiation-induced death and DNA damage, dramatically extending the lifespan of these normal stem cells and allowing them to grow. Rapamycin exerted these protective effects by preventing the accumulation of harmful molecules called reactive oxygen species. Moreover, mice that received rapamycin during radiation treatment did not develop mucositis.

Because rapamycin is approved by the Food and Drug Administration and is currently being tested in clinical trials for the prevention and treatment of various types of cancer, the new findings could have immediate and important implications for a large proportion of cancer patients. "Mucositis prevention would have a remarkable impact on the quality of life and recovery of cancer patients and at the same time would reduce the cost of treatment," Gutkind says. "Our study provides the basis for further testing in humans, and we hope that these findings can be translated rapidly into the clinic."

###

Iglesias-Bartolome et al.: "mTOR inhibition prevents epithelial stem cell senescence and protects from radiation-induced mucositis."

Finkel et al.: "Relief with Rapamycin: mTOR Inhibition Protects against Radiation-Induced Mucositis"(In Translation Article)

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Stem-cell-protecting drug could prevent the harmful side effects of radiation therapy

Progenitor Cell Therapy, a NeoStem Company, Invited to Present at Two Conferences in September

NEW YORK, Sept. 5, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NBS) ("NeoStem" or the "Company"), a cell therapy company, today announced that Company management of a NeoStem company, Progenitor Cell Therapy ("PCT"), an internationally recognized contract development and manufacturing organization (CDMO), has been invited to present on its core expertise in development of commercial manufacturing processes for cell therapy at two cell therapy conferences in September. At each, PCT will offer its unique perspective as an industry leader in contract development and manufacturing of cell therapy products, with over 12 years of exclusive cell-therapy focused experience.

Timothy Fong, Ph.D, M.B.A, PCT's Vice President, Technology and Product Development, will be sharing PCT's expertise in cell therapy manufacturing with a focus on commercialization. At IBC Life Sciences' Cell Therapy Bioprocessing Conference, he will chair a panel on quality assurance and controls and will give a presentation entitled "From Concept to Product: Considerations for Developing a Robust Commercial Manufacturing Process", which will include considerations for developing a robust commercial manufacturing process. He will also speak at the Stem Cells USA and Regenerative Medicine Congress on "Cell manufacturing considerations for first-in-world stem cell therapeutics".

Dr. Fong stated, "As a cell therapeutic progresses from concept to product, the development of a commercial manufacturing process may contain unexpected technical and quality issues. The development path should follow several defined steps. My presentations will discuss the key steps in the process and highlight critical areas that need to be addressed to develop a successful commercial manufacturing process. PCT helps clients bridge the gap between discovery and patient care through efficient transfer of cell-based therapies from laboratory into clinical practice."

NeoStem and PCT invite you to attend the conference(s), see Dr. Fong's talks, and connect with the PCT team at PCT's booths. If you are a colleague of PCT or NeoStem, PCT can offer you a registration discount. Please contact PCT at bdm@pctcelltherapy.com for more details.

IBC Life Sciences' 2nd Annual Cell Therapy Bioprocessing Conference

Terrapinn 4th Annual Stem Cells USA and Regenerative Medicine Congress

About NeoStem, Inc.

NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a large role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT") with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cells product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a patent and patent pending (IP) portfolio, we believe we are well positioned to succeed.

For more information on NeoStem, please visit http://www.neostem.com. For more information on PCT, please visit http://www.pctcelltherapy.com.

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Progenitor Cell Therapy, a NeoStem Company, Invited to Present at Two Conferences in September

Spinal Stem Cell Injections Help Reverse Paralysis [Medicine]

A new medical intervention has allowed doctors to inject neural stem cells into the spines of paralyzed patients, successfully helping them recover feeling in previously unresponsive parts of their bodies.

New Scientist reports that a small trial involving three partially paralyzed patients saw injections of 20 million neural stem cells administered directly into their spinal cords. The stem cells were harvested from donated fetal brain tissue, and the patients received immunosuppressive drugs to minimize the risks of rejection.

Before treatment, all of the patients could feel nothing below their nipples. Six months on, two of them can now feel touch and heat as far down as their belly button. While that may not sound impressive, it's a massive leap forward in the treatment of paralysis. Stephen Huhn, one of the researchers, told New Scientist:

"The fact we've seen responses to light touch, heat and electrical impulses so far down in two of the patients is very unexpected. They're really close to normal in those areas now in their sensitivity."

These three patients are the first of 12 to undergo the therapy, and the positive results will see Huhn and his colleagues push on with the tests. The results were presented yesterday in London at the annual meeting of the International Spinal Cord Society.

While it's not clear exactly how the stem cells improve sensitivity, the researchers suggest that they may be helping restore myelin insulation of damaged nerves, or perhaps causing existing nerves to function better.

It's too early to tell for sure whether the treatment could represent a standalone treatment for paralysis. It does, however, offer hope that in the futureperhaps when combined with drugs and physical therapythe treatment could help make paralysis a temporary, rather than permanent, condition. [New Scientist]

Image by Sebastian Kaulitzki/Shutterstock

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Spinal Stem Cell Injections Help Reverse Paralysis [Medicine]

2 of 3 spinal injury patients see gains in stem cell trial

Interim results from three patients in an early-stage trial of StemCells Inc's experimental cell treatment for chronic spinal cord injury show that two of them experienced gains in "sensory function," the company said.

StemCells is using neural stem cells, technically adult stem cells, taken from the partly developed brains of fetuses and tested for qualities showing they are destined to form particular types of nerve cells.

The company said that six months after being infused with the cells all three patients have tolerated the transplantation well and there are no safety concerns.

"We clearly need to collect more data to establish efficacy, but we are encouraged," Stephen Huhn, vice president at StemCells, said in a statement. He also said the company is pushing ahead with plans to dose patients with incomplete spinal cord injuries.

The initial phase of the trial involved patients with complete injuries and no neurological function below the level of the spinal injury.

Changes in sensitivity to touch, heat and electrical stimuli were observed in areas below the level of injury in two of the patients, while no changes were seen in the third patient, the company said.

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2 of 3 spinal injury patients see gains in stem cell trial

NeoStem to Present at Three Investor Conferences in September

NEW YORK, Sept. 4, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NYSE MKT:NBS) ("NeoStem" or the "Company"), a rapidly emerging market leader in the fast growing cell therapy market, today announced that Company management will present at three investor conferences in September.

The 19th Annual Newsmakers in the Biotech Industry - BioCentury & Thomson Reuters

Rodman & Renshaw Annual Global Investment Conference

National Investment Banking Association Conference

About NeoStem, Inc.

NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a large role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT") with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cells product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a patent and patent pending (IP) portfolio, we believe we are well positioned to succeed.

Forward-Looking Statements for NeoStem, Inc.

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current expectations, as of the date of this press release, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company's business strategy, including with respect to the Company's or its partners' successful development of AMR-001 and other cell therapeutics, the size of the market for such products, its competitive position in such markets, the Company's ability to successfully penetrate such markets and the market for its CDMO business, and the efficacy of protection from its patent portfolio, as well as the future of the cell therapeutics industry in general, including the rate at which such industry may grow. Forward looking statements also include statements with respect to satisfying all conditions to closing the disposition of Erye, including receipt of all necessary regulatory approvals in the PRC. The Company's actual results could differ materially from those anticipated in these forward- looking statements as a result of various factors, including but not limited to (i) the Company's ability to manage its business despite operating losses and cash outflows, (ii) its ability to obtain sufficient capital or strategic business arrangement to fund its operations, including the clinical trials for AMR-001, (iii) successful results of the Company's clinical trials of AMR-001 and other cellular therapeutic products that may be pursued, (iv) demand for and market acceptance of AMR-001 or other cell therapies if clinical trials are successful and the Company is permitted to market such products, (v) establishment of a large global market for cellular-based products, (vi) the impact of competitive products and pricing, (vii) the impact of future scientific and medical developments, (viii) the Company's ability to obtain appropriate governmental licenses and approvals and, in general, future actions of regulatory bodies, including the FDA and foreign counterparts, (ix) reimbursement and rebate policies of government agencies and private payers, (x) the Company's ability to protect its intellectual property, (xi) the company's ability to successfully divest its interest in Erye, and (xii) matters described under the "Risk Factors" in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 20, 2012 and in the Company's other periodic filings with the Securities and Exchange Commission, all of which are available on its website. The Company does not undertake to update its forward-looking statements. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside its control.

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NeoStem to Present at Three Investor Conferences in September

PropThink: Interim Data In Spinal Cord Injury Driving STEM Higher; PSTI Reacting in Sympathy

This morning, StemCells, Inc. (STEM) released six-month interim results for the first group of patients in its Phase I/II trial, testing the company`s HuCNS-SC (human neural stem cells) treatment candidate in patients with spinal cord injury - previous coverage available here. Shares are trading up nearly 20% in pre-market, and are likely to maintain momentum on the positive news. The interim data showed that thus far, HuCNS-SC treatment led to considerable gains in sensory function in two of three patients vs. their baseline levels of sensation prior to cell transplantation. The company also noted that the treatment continues to exhibit a favorable safety profile. The trial represents the first time that neural stem cells have been transplanted as a potential therapeutic agent for spinal cord injury, and the sensory gains observed have developed in a progressive pattern below the level of injury. Impressively, gains in sensation are not generally expected in spinal cord injury patients, particularly given the severity of injury seen in patients admitted to the Phase I/II study. According to STEM, sensory function of all patients observed thus far was stable before cell transplantation, therefore, the reappearance of sensation is a very positive sign that the treatment is having a beneficial effect. While the third patient did not experience an increase in sensation, that patient`s level of sensation remains stable. PropThink notes that another small-cap stem cell company, PluriStem Therapeutics (PSTI) is also moving in pre-market trading, likely in sympathy with the STEM news.

To see this article at PropThink.com, click here.

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PropThink: Interim Data In Spinal Cord Injury Driving STEM Higher; PSTI Reacting in Sympathy