Thursday, January 26, 2012

Maryland Stem Cell Companies Advance Therapies for Diabetes, Depression

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Two Maryland biotechs announced inroads in their efforts to develop stem cell treatments for depression and diabetes.

Neuralstem has gotten the regulatory go-ahead to advance to phase 1b in its ongoing clinical trial of its stem cell treatment for major depressive disorder.

NSI-189 stimulates new neuron growth in the brain’s hippocampus, which may be involved in depression and other conditions, including Alzheimer's disease and post-traumatic stress disorder, according to the Rockville biotech.

"Loss of hippocampal volume is a known characteristic in depressed patients,” Karl Johe, chairman and chief scientific officer, said in a statement. “NSI-189 stimulates neurogenesis and increases hippocampal volume in healthy adult mice, at the same time reversing behavioral symptoms in mouse depression models, so it could address depression at the source."

The next phase — expected to run six months — will test the safety of increasing doses in 28 daily administrations in 24 depressed patients.

Meanwhile, Osiris Therapeutics of Columbia reported that preliminary results from a phase 2 clinical trial of its stem cell therapy in patients with newly diagnosed type 1 diabetes showed the treatment to be well tolerated.

However, the treatment did not slow the progression of the disease, even though there was a trend toward fewer hypoglycemic events, compared with patients receiving a placebo, the company said in a statement.

The two-year trial, under way for a year, is being conducted with the Juvenile Diabetes Research Foundation. It involves infusions of the treatment, Prochymal, which is formulated from the adult mesenchymal stem cells of unrelated adults. A total of 63 adult and child patients are in the study.

“Importantly, no patients experienced a reaction to the infusions despite the cells being unrelated to the recipient, unmatched, and used without immunosuppression,” Osiris reported.

"The ability to safely use stem cells from unrelated donors is an important finding of this study and provides new possibilities for further development of stem cell therapies for type 1 diabetes," Jay S. Skyler, professor of medicine and deputy director of the Diabetes Research Institute at the University of Miami Miller School of Medicine, said in the statement.

Osiris is developing the treatment under a strategic alliance with Genzyme.

In other Maryland bioscience industry news:

Kenneth Carter, a veteran of Maryland’s bioscience industry who founded Noble Life Sciences more than a year ago, has transitioned into leading one of the startups that the Gaithersburg company was designed to help foster.

Carter is now president and CEO of NexImmune of Gaithersburg, which initially is focusing on developing a treatment for melanoma. The company also named Jonathan Schneck, a professor of pathology, medicine and oncology at Johns Hopkins School of Medicine, chairman of its clinical and scientific advisory board.

The startup launched in the fall, said Carter, who will remain chairman of Noble. He’s been succeeded as Noble’s president by Alain Cappeluti, its former CFO.

“It’s a close-knit environment,” Carter said.

Noble was established with two objectives: to operate as a contract research organization for the pharmaceutical and biotech industries and also to provide startup services and an incubator environment for startups with their own products.

“We’ve gotten down the path on that now,” Carter said.

He declined to disclose the startup’s backers, but said financing details will be announced later.

“There’s no big venture capital financing put together,” said Carter, who for a decade was CEO at Avalon Pharmaceuticals, before it was acquired by Clinical Data.

NexImmune also announced that it has licensed from Johns Hopkins the Artificial IMmune nanotechnology developed by Schneck and a colleague, Mathias Oelke.

"The AIM technology provides an exciting leap in immuno-therapy for a variety of cancer and other diseases whereby artificial cells direct specific immune responses against a specific disease,” Carter said in a statement. “I am very pleased to lead the team that will commercialize this exciting new technology.”

"It is terrific to work with Ken and his team on the commercial transformation of the AIM technology to a product development platform,” Schneck said in the statement. “If the products to be developed by NexImmune are successful in human clinical trials, I believe the AIM technology has the potential to provide a major step forward for immuno-therapy for a variety of diseases."

RegeneRx Biopharmaceuticals is exploring a handful of partnerships for its dry-eye drug candidate, RGN-259, after the Rockville biotech reported in November that the treatment failed to meet two primary outcome measures in a 72-patient, phase 2 clinical trial that ran 30 days.

Still, the data showed that the candidate “had statistically significant benefits over placebo in certain signs and symptoms of dry eye syndrome,” according to a RegeneRx statement, which also referred to the results as “very encouraging.”

“In this exploratory study with only 35 subjects per group, RGN-259 was shown to have clinically significant effects on dry eye, albeit not in the chosen pre-determined primary endpoints,” RegeneRx said in a later statement released Thursday. “The significant protective effect that RGN-259 was shown to have on ocular discomfort and corneal staining in response to adverse stimuli indicate that changes in response to the [controlled adverse environment] challenge are effective new endpoints for future clinical trials.”

“As we reported prior to the announcement of the dry eye data, we had signed a number of confidentiality agreements with ophthalmic companies prior to release of data from the trial,” CEO J.J. Finkelstein said in the earlier statement. “Since then, we have been engaged in discussions with two of these companies and we have been contacted by two additional companies that are evaluating our data and with whom we are also holding discussions. We have told all of them that our intended goal is to close a deal by Jan. 31.”

The company will “also be taking additional cost-cutting steps to preserve cash during this period, which we will announce in greater detail after Jan. 1,” Finkelstein said.

Citing information from Global Data, an industry market research firm, RegeneRx has said that the worldwide annual market for dry eye disorders was about $1.9 billion in 2010 and is estimated to reach $2.8 billion by 2017.

Twenty healthy adult volunteers have received Profectus BioSciences’ experimental HIV vaccine in a phase 1 study to test its safety and immunogenicity.

The first studies showing the potential of recombinant vesicular stomatitis virus as an HIV vaccine vector were performed at Yale University more than a decade ago, according to a statement from the Baltimore biotech. The recombinant version used in the new study can replicate in human cells, but has been attenuated so it won’t cause illness in animals or humans.

The trial is to involve 60 adults. The vaccine was found to be safe and immunogenic in non-human primates.

The vaccine program has been supported by a $22.5 million contract from the National Institutes of Health.

VirxSys of Gaithersburg reported raising $1 million of a new $30 million mixed offering from 10 investors.

The biotech is working on an HIV vaccine and genetic treatments for cardiovascular disease and hemophilia.

Novavax reported that CPL Biologics, its joint venture in India with Cadila Pharmaceuticals, is ready to begin clinical studies of flu and rabies vaccines this year and next.

Rabies is a “significant” public health hazard in India, where 36 percent of the world's rabies deaths occur, according to a statement from the Rockville biotech.

CPL Biologics also has made “rapid” progress with the validation of its vaccine manufacturing plant in Dholka, India, Novavax said. The facility uses the single-use vaccine bioprocessing system that Novavax uses at its pilot plant in Rockville.

Vanda Pharmaceuticals, which plans to move to Washington, D.C., by April, will recognize expenses totaling $1 million to $1.5 million by ending its current lease at 9605 Medical Center Drive, Rockville, by June 30, 2013, according to a regulatory filing.

The company also reported that the Internal Revenue Service has approved a change in its accounting method related to startup expenditures that will be reflected in a fourth-quarter tax benefit of about $0.3 million, an increase in its net operating loss carry forwards of about $35.7 million, and research and development credits of about $3.4 million.

Arcion Therapeutics of Baltimore reported that it received notice of allowance for a U.S. patent for its high-strength gel formulation of topical lidocaine, ARC-2022.

Arcion is developing the product for pain associated with post-herpetic neuralgia and shingles.

"With ARC-2022 our goal is to deliver lidocaine to the skin without the requirement of an occlusive patch,” James N. Campbell, president and CEO, said in a statement. “ With topical delivery, systemic toxicities and [central nervous system] side effects can be avoided and the abnormal pain signals are suppressed at the point of origination in the skin."

Stem Cells are advancing everyday bringing new hope to so many...........MrCordBlood

Breakthrough In Alzheimer's Disease Research

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In vitro Alzheimer’s cells for research

Researchers are closer to understanding Alzheimer’s disease using a new stem celltechnique. They have successfully replicated Alzheimer’s disease neurons with stem cells for the first time.

Researchers out of UC San Diego School of Medicine created in vitro models of genetic and sporadic forms of Alzheimer’s disease, using induced pluripotent stem cells (iPSC) from patients who suffered from the neurodegenerative disorder. The neurons were purified, meaning they were separated from other types of cells, to reduce variability in the experiment. To create the neurons, the researchers extracted fibroblasts—cells from the skin—of two patients with familial Alzheimer’s, two patients with sporadic Alzheimer’s and two people with no known neurological problems. The researchers then reprogrammed the fibroblasts into stem cells, which then differentiated into working neurons.

“Creating highly purified and functional human Alzheimer's neurons in a dish – this has never been done before,” said senior study author Dr. Lawrence Goldstein, distinguished professor in the Department of Cellular and Molecular Medicine, Howard Hughes Medical Institute Investigator and director of the UC San Diego Stem Cell Program, in a press release.

The iPSC-derived neurons from Alzheimer’s patients exhibited normal cell activity, formed functional synaptic contacts and – most importantly – displayed indicators of Alzheimer’s disease, such as elevated production of beta-amyloid proteins and abnormal activation of the protein kinase GSK-3. Goldstein added the models aren’t “perfect” – they’re merely the first step. However, the research proves creating isolated Alzheimer’s neurons can be done and provides a blueprint for how to do so.

“Additional features of the model need to be developed,” Goldstein told “At this point, it’s purified neurons. So now, knowing what the purified neurons do, we want to add back defined qualities of other cells – like astrocytes. Astrocytes are normally a very important part of how neurons function, and they also play a role in resistance or susceptibility to disease.” “We don’t know what that role is yet, but we can start to piece that back together by mixing the astrocytes back in with the neurons,” he added.

The neurons may prove to be a crucial tool for studying the causes of Alzheimer’s, as well as developing and testing drugs to treat the disease. “We're dealing with thehuman brain. You can't just do a biopsy on living patients,” Goldstein explained. “Instead, researchers have had to work around, mimicking some aspects of the disease in non-neuronal human cells or using limited animal models. Neither approach is really satisfactory.” Now with this new technique with in vitro neurons, the researchers more deeply investigate the onset of Alzheimer’s disease and observe the initial processes that lead to the destruction of brain cells.

Currently, dementia research is mostly centered around studies of post-mortem tissues. “The way to think about it is, if you want to understand what goes wrong early – if you only get post-mortem tissue, a lot of the damage is already done,” Goldstein said. “Suppose you work for the NTSB and you have to study a plane crash,” he explained. “You can get a lot of information about the crash from the wreckage, but the black box tells you what went wrong early. That’s incredibly important information for preventing crashes. We’re looking for the black box of Alzheimer’s.”

He added that “we show that one of the early changes in Alzheimer's neurons thought to be an initiating event in the course of the disease turns out not to be that significant… What we observed is, it was not the beta-amyloid fragments causing biochemical abnormalities, but it was a pre-cursor to that, called beta-CTS.”

According to Goldstein, the next step for using this research would be to begin testing drugs and scaling up the technology to test more patients. “From the standpoint of drug development, here’s the core problem: we don’t have any drugs so we don’t exactly know what it’s going to take to develop them,” Goldstein said, “We think by having true human neurons to work with we can increase the speed and likelihood of finding effective drugs.”

Goldstein said continuing to research Alzheimer’s disease is critical in order to reduce the economical and emotional toll the disease takes on the nation. “People make this interesting mistake where they say it’s just a disease of the elderly – and who cares?” Goldstein said. “The truth is, a 70 year-old person who doesn’t have the disease can be very productive economically and socially, while those who have the disease can be a drain in terms of cost of care. Projections are that the cost of Alzheimer’s will go into the trillions. It’s a real substantial impact.”

The study was published Wednesday in the online version of the journal Nature.

New Hope being brought to the table for those with Alzheimer's courtesy of stem cells...........MrCordBlood

Tuesday, January 24, 2012

Breakthrough Therapy Using Cord blood Banking Driving Stem Cell Industry

Five Star Equities Provides Stock Research on Cord Blood America & Aastrom Biosciences

NEW YORK, NY, Jan 24, 2012 (MARKETWIRE via COMTEX) -- Given some positive trends, the stem cell market could be set to skyrocket this year. A recent report from Kalorama Information titled "Worldwide Markets for Transplantation, Cord Blood Banking and Drug Development," estimates that the market for stem cell technologies will rise to over $700 million this year and could reach over $1 billion. Five Star Equities examines the outlook for companies in the Biotechnology industry and provides equity research on Cord Blood America Inc. CBAI +30.82% and Aastrom Biosciences Inc. ASTM +1.02% . Access to the full company reports can be found at:

Kalorama finds that cell therapies are largely confined to a few conditions. Oncological conditions such as leukemia, multiple myeloma and non-Hodgkins lymphoma are some of the most popular areas of research, although certain non-cancerous diseases like aplastic anemia, immunodeficiency and lupus are also increasingly addressed with stem cells.

Kalorama finds that cord blood banking is a source of revenue growth in the stem cell market. U.S. researchers are currently undergoing a phase I safety study using a child's umbilical cord blood stem cells to try to restore hearing loss. "This study is exciting because it might offer a non-surgical option for some children with profound hearing loss," explains Dr. James Baumgartner, sponsor of the study and guest research collaborator at The University of Texas Health Science Center at Houston Medical School.

Five Star Equities releases regular market updates on the biotechnology industry so investors can stay ahead of the crowd and make the best investment decisions to maximize their returns. Take a few minutes to register with us free at and get exclusive access to our numerous stock reports and industry newsletters.

Stem cells from umbilical cords do not pose an ethical dilemma because the cells come from a source that would otherwise be discarded, Science Daily reports. Recent studies suggest that stem cells from umbilical cords have been converted into other types of cells, which may eventually lead to new treatment options for nervous system diseases. "This is the first time this has been done with non-embryonic stem cells," says James Hickman, a University of Central Florida bioengineer and leader of the research group.

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The adult stem cells industry is growing by leaps and bound year over year. Cord blood banking is becoming a more popular choice for parents and stem cells are being used for more and more medical treatments and therapies........MrCordBlood

Wednesday, January 18, 2012

Adult Stem Cell Research FAR AHEAD of Embryonic

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A few months ago, Dr. Thomas Einhorn was treating a patient with a broken ankle that wouldn't heal, even with multiple surgeries. So he sought help from the man's own body.

Einhorn drew bone marrow from the man's pelvic bone with a needle, condensed it to about four teaspoons of rich red liquid, and injected that into his ankle.

Four months later the ankle was healed. Einhorn, chairman of orthopedic surgery at Boston University Medical Center, credits "adult" stem cells in the marrow injection. He tried it because of published research from France.

Einhorn's experience isn't a rigorous study. But it's an example of many innovative therapies doctors are studying with adult stem cells. Those are stem cells typically taken from bone marrow and blood — not embryos.

For all the emotional debate that began about a decade ago on allowing the use of embryonic stem cells, it's adult stem cells that are in human testing today. An extensive review of stem cell projects and interviews with two dozen experts reveal a wide range of potential treatments.

Adult stem cells are being studied in people who suffer from multiple sclerosis, heart attacks and diabetes. Some early results suggest stem cells can help some patients avoid leg amputation. Recently, researchers reported that they restored vision to patients whose eyes were damaged by chemicals.

Apart from these efforts, transplants of adult stem cells have become a standard lifesaving therapy for perhaps hundreds of thousands of people with leukemia, lymphoma and other blood diseases.

"That's really one of the great success stories of stem cell biology that gives us all hope," says Dr. David Scadden of Harvard, who notes stem cells are also used to grow skin grafts.

"If we can recreate that success in other tissues, what can we possibly imagine for other people?"

That sort of promise has long been held out for embryonic stem cells, which were first isolated and grown in a lab dish in 1998. Controversy over their use surrounded the 2001 decision by former President George W. Bush to allow only restricted federal funding for studying them.

Proponents over the past decade have included former first lady Nancy Reagan and actors Michael J. Fox and the late Christopher Reeve. Opponents object that human embryos have to be destroyed to harvest the cells.

Embryonic cells may indeed be used someday to grow replacement tissue or therapeutic material for diseases like Parkinson's or diabetes. Just on Friday, a biotech company said it was going ahead with an initial safety study in spinal cord injury patients. Another is planning an initial study in eye disease patients later this year.

But in the near term, embryonic stem cells are more likely to pay off as lab tools, for learning about the roots of disease and screening potential drugs.

Observers say they're not surprised at the pace of progress.

As medical research goes, the roughly 10 years since the embryonic cells were discovered "is actually a very short amount of time," said Amy Rick, immediate past president of the Coalition for the Advancement of Medical Research. The group has pushed for embryonic stem cell research for about that long.

Hank Greely, a Stanford University law professor who works in bioethics and has followed stem cells since the 1990s, said: "Give it another five years and I'll be surprised if we don't have some substantial progress" beyond initial safety studies.

The Pro-Life Secretariat of the U.S. Conference of Catholic Bishops continues to oppose embryonic work. Deirdre McQuade, an official there, said that compared to adult stem cell research, work on embryonic cells is proving "fruitless."

Adult cells have been transplanted routinely for decades, first in bone marrow transplants and then in procedures that transfer just the cells. Doctors recover the cells from the marrow or bloodstream of a patient or a donor, and infuse them as part of the treatment for leukemia, lymphoma and other blood diseases. Tens of thousands of people are saved each year by such procedures, experts say.

But it is harnessing these cells for other diseases that has encouraged many scientists lately.

In June, for example, researchers reported they had restored vision to people whose eyes were damaged from caustic chemicals. Stem cells from each patient's healthy eye were grown and multiplied in the lab and transplanted into the damaged eye, where they grew into healthy corneal tissue.

A couple of months earlier, the Vatican announced it was funding adult stem cell research on the intestine at the University of Maryland. And on Friday, Italian doctors said they'd transplanted two windpipes injected with the recipients' own stem cells.

But these developments only hint at what's being explored in experiments across the United States.

Much of the work is early, and even as experts speak of its promise, they ask for patience and warn against clinics that aggressively market stem-cell cures without scientific backing.

Some of the new approaches, like the long-proven treatments, are based on the idea that stem cells can turn into other cells. Einhorn said the ankle-repair technique, for example, apparently works because of cells that turn into bone and blood vessels. But for other uses, scientists say they're harnessing the apparent abilities of adult stem cells to stimulate tissue repair, or to suppress the immune system.

"That gives adult stem cells really a very interesting and potent quality that embryonic stem cells don't have," says Rocky Tuan of the University of Pittsburgh.

One major focus of adult stem cell work for about a decade has been the ailing heart. While researchers remain committed, much of the early enthusiasm from patients, doctors and investors has slacked off because results so far haven't matched expectations, says Dr. Warren Sherman of Columbia University.

"Everyone, including myself, is impatient and would like to see positive results appear quickly," said Sherman, who hosts an annual international meeting of researchers. But he called for patience.

In treating heart attack, for example, studies show stem cell injections help the heart pump blood a bit better, Sherman said. But the research has not yet established whether injections cut the risk of death, more heart attacks or future hospitalizations, he said.

Sherman said he hopes a large study of those patient outcomes can be done in the next couple of years, and is "very optimistic that patients will benefit."

Similarly, in heart failure, research indicates stem cells can ease symptoms but larger studies are still needed to show how much good the treatments provide, he said. He noted that current studies are testing stem cells taken not only from bone marrow and leg muscle, but also from fat.

Another heart-related condition under study is critical limb ischemia, where blood flow to the leg is so restricted by artery blockage it causes pain and may require amputation. The goal here is to encourage growth of new blood vessels by injecting stem cells into the leg.

Sherman said limb ischemia research is moving fast and the results "are very, very encouraging."

The injected cells may serve as building blocks while also stimulating local tissue to grow the vessels, said Dr. Douglas Losordo of Northwestern University. His own preliminary work suggests such a treatment can reduce amputation rates.

Dr. Gabriel Lasala of TCA Cellular Therapy also has reported positive preliminary results. One success is Rodney Schoenhardt of Metairie, La.

Schoenhardt had already had surgery on both legs for the disease, and his surgeon was talking about amputating his left leg. Schoenhardt suffered so much pain in his left leg while standing that he used a wheelchair instead.

For Lasala's research, Schoenhardt got 40 shots in each leg about 18 months ago, with stem cells going into his left calf and a placebo dose into the other. Soon, he said, the pain in his left leg was gone.

Schoenhardt, 58, now mows his lawn, and he remodeled his living room to fix damage from Hurricane Katrina. "My wheelchair is in my garage, collecting dust," he said.

"I'm even thinking about taking up a little tennis again."

With all the heart-related stem cell studies, the former president of American Heart Association says, "We should be enthusiastic, but cautiously so." Beyond the promising indications of early studies, researchers need definitive evidence that the treatments not only make patients better but also don't cause unintended harm, says Dr. Clyde Yancy.

Among the other diseases being studied for stem cell treatments:

Multiple Sclerosis

In MS, the body's immune system repeatedly assaults brain and spinal cord tissues, which can cause numbness in the limbs, paralysis or vision loss.

Last year, Dr. Richard Burt of Northwestern reported a small trial in patients with early MS that was aimed at rebooting the immune system to stop the attacks. He removed stem cells from the patient's blood, destroyed their immune systems, and then re-injected them with their own cells to build a new immune system.

To his surprise, most patients actually improved. He's now conducting another trial to provide firmer evidence of improvement.

Dr. Jeffrey Cohen of the Cleveland Clinic is trying a different and less-researched approach. In a preliminary trial he is just starting, he'll use a different kind of stem cell from patients' marrow that he hopes can slow nervous system damage but also promote repair.

Lessons learned from this approach might eventually reveal some clues for treating other conditions like Parkinson's or spinal cord injury, he said.

Type 1 Diabetes

It's also caused by a misguided attack by the immune system, this time on insulin-producing cells. Burt and colleagues reported last year that the "rebooting" strategy allowed some patients to go without insulin for four years. However, some experts call his approach too risky for that disease. Burt is now doing another study in newly diagnosed adults.

Another study, at about a dozen medical centers around the country, is testing whether an off-the-shelf preparation of marrow stem cells can calm the immune system of diabetics. It's still early work, says C. Randal Mills, chief executive officer of Osiris Therapeutics.

Cancers such as melanoma and kidney cancer

The idea is to transplant cells to produce a new immune system that will attack the diseases. Earlier work around a decade ago failed to give lasting benefit, but new approaches aim for better results, said Dr. Michael Bishop of the National Cancer Institute.

Even as scientists hope adult stem cells will produce new treatments, they are concerned about clinics that make claims about unproven stem cell therapy.

"Clinics have sprung up all over the world ... that are essentially selling snake oil, that are preying on the hopes of desperate patients," said Sean Morrison, a stem cell expert at the University of Michigan.

Morrison suggests patients consult their own doctors about going to a clinic.

I feel that scientists are realizing that they can harness the amazing powers stem cells possess from adult stem cells with more success than embryonic stem cells. Adult stem cells have no ethical, moral, or religious debate. Adult stem cells found in in for example umbilical cord blood, placental stem cells, adipose tissues are even supported by countries around the globe and even "The Pope." It is very exciting times for the research and medical community right now and what the next 5-10 years will bring could be nothing short of spectacular.................MrCordBlood