StemCell Therapy

Wed May 9, 2012 3:35pm BST

(Reuters) - Pluristem Therapeutics Inc said a 7-year old girl suffering from a bone marrow disease experienced a reversal of her condition after receiving its experimental stem cell therapy, sending the Israeli company's shares up 32 percent.

The girl, suffering from aplastic bone marrow in which the patient has no blood-forming stem cells, had a significant rise in her red cells, white cells and platelets following an injection of Pluristem's therapy -- PLacental eXpanded cells.

"The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow," Reuven Or, the child's physician at Hadassah Medical Center, was quoted in a statement by Pluristem.

Last September, the company said animal studies showed that the therapy had the potential to treat blood tissue complications related with acute radiation syndrome, commonly called radiation sickness.

Last month, the U.S. health regulators gave a go ahead to the company to start a mid-stage trial of the therapy for treating Intermittent Claudication -- a subset of peripheral artery disease.

Pluristem shares, which have gained 5 percent since receiving the FDA nod for the mid-stage trial, were up 15 percent at $2.70 in morning trade on the Nasdaq. They touched a high of $3.10 earlier.

(Reporting by Esha Dey in Bangalore; Editing by Gopakumar Warrier)

Original post:
Pluristem stem cell therapy saves a patient, shares jump

ALBANY -- Almost halfway through a $600 million state program supporting stem cell research, eight medical schools around New York are reporting progress on projects such as replicating liver cells and eradicating leukemia cells.

A new report from Associated Medical Schools of New York updates work at the institutions where hundreds of researchers are starting to unravel causes and potential treatments for conditions ranging from autism to heart disease and cancer. Stem cells are self-renewing and have the ability to develop into other types of cells.

The Mount Sinai School of Medicine reported finding a method to transform human skin cells into stem cells and turned differentiated human stem cells into heart cells. Those findings are expected to result in better understanding of how heart disease develops and allow initial testing of new treatments on stem cells before they are used on human subjects.

The Empire State Stem Cell Program was intended to fund projects in early stages, including those that initially have been unable to get federal or private funding. Grants have also been used for capital projects like renovating labs and establishing new stem cell centers.

The Albert Einstein College of Medicine reported replicating liver cells that could help reduce the need for liver transplants using live donors and cadavers.

Dr. Allan Spiegel said 12 new researchers have been hired with state funding at the Bronx school, which also lists anemia, brain disorders, heart disease and obesity among its stem cell research subjects.

The 11-year program has awarded nearly $223 million in research grants since 2008, with medical schools awarded $137.5 million. This year's state budget includes $44.8 million, the same as last year and down from the $50 million originally planned.

According to the report, the funding has supported about 400 research and related positions from New York City to Buffalo and Rochester.

"This research has the potential to have significant impact on the treatment of patients with specific types of leukemia and will be useful in treating lymphoma and multiple myeloma," the report said.

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N.Y. medical schools chart progress with stem cells

Source: ISNA, Tehran

Iranian researcher and lecturer Radbod Darabi jointly with his collogues from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process which for the first time makes the production of human muscle cells from stem cells efficient and effective.

Radbod Darabi, MD, PhD with Rita Perlingeiro, PhD. (Credit: Image courtesy of University of Minnesota Academic Health Center)

The research outlines the strategy for the development of a rapidly dividing population of muscle-forming cells derived from induced pluripotent (iPS) cells.

IPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos.

This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy.

According to the researchers, there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human stem cells.

This lag has dramatically limited the development of cell therapies or clinical trials for human patients.

The latest research from the University of Minnesota provides the proof-of-principle for treating muscular dystrophy with human iPS cells, setting the stage for future human clinical trials.

As the researchers noted one of the biggest barriers to the development of cell-based therapies for neuromuscular disorders like muscular dystrophy has been obtaining sufficient muscle progenitor cells to produce a therapeutically effective response.

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Iranian researcher helps treating muscular dystrophy using stem cells

ALBANY Almost halfway through a $600 million state program supporting stem cell research, eight medical schools around New York are reporting progress on projects such as replicating liver cells and eradicating leukemia cells.

A new report from Associated Medical Schools of New York updates work at the institutions where hundreds of researchers are starting to unravel causes and potential treatments for conditions ranging from autism to heart disease and cancer. Stem cells are self-renewing and have the ability to develop into other types of cells.

The Mount Sinai School of Medicine reported finding a method to transform human skin cells into stem cells and turned differentiated human stem cells into heart cells. Those findings are expected to result in better understanding of how heart disease develops and allow initial testing of new treatments on stem cells before they are used on human subjects.

Dr. Ihor Lemischka, director of the Black Family Stem Cell Institute at Mount Sinai, said recreating heart cells in a dish from a patient with LEOPARD Syndrome, a disease caused by a genetic mutation, has opened ongoing avenues for researching the disease and screening potential drugs.

The shared research facility at Mount Sinai supports the work at 80 different labs, Lemischka said.

The Empire State Stem Cell Program was intended to fund projects in early stages, including those that initially have been unable to get federal or private funding. Grants have also been used for capital projects like renovating labs and establishing new stem cell centers.

The Albert Einstein College of Medicine reported replicating liver cells that could help reduce the need for liver transplants using live donors and cadavers.

Dr. Allan Spiegel said 12 new researchers have been hired with state funding at the Bronx school, which also lists anemia, brain disorders, heart disease and obesity among its stem cell research subjects.

It offers tremendous potential for understanding the causes of and developing better treatments for diseases like cancer, type 1 diabetes and Parkinsons, he said.

Einstein scientists also report advanced work on blood stem cell function. The school has opened a new $25 million research institute funded through private philanthropy. The report said state money has been used by the schools to leverage both federal and private grants.

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NY medical schools chart progress with stem cells

LEBANON Retired Dartmouth College professor Roger Smith said he had nothing to lose by joining a stem cell therapy clinical trial. In fact, if he didn't join, he did have something to lose possibly his leg.

In the end it appears the experiment saved his leg. And Smith, who'd already lost two toes to amputation, said he's proud to have played a part in a study that could dramatically improve the outcome for many other patients facing lower-limb amputations resulting from diabetes, high cholesterol, smoking, genetic predisposition and other causes.

The three-year study that ended last year was led by vascular surgeons at Dartmouth-Hitchcock Medical Center in Lebanon, who believe the treatment may offer new hope to sufferers of peripheral artery disease, a condition that causes nearly 60,000 lower-limb amputations every year.

Dr. Richard J. Powell, chief of vascular surgery at Dartmouth-Hitchcock, was the lead investigator of the second-phase national study, which included 72 patients from 20 different sites across the United States.

It's a winner, Powell said. For me, it was dramatic, because there has been nothing that has been shown to work. The results of the third-stage trial are to be presented to the Food and Drug Administration to be approved as a treatment for patients, Powell said.

Peripheral artery disease afflicts more than 9 million patients in the United States, according to Dartmouth-Hitchcock. The condition results from blockages in blood vessels caused by atherosclerosis hardening of the arteries. Options for these patients are limited to the insertion of stents or bypass surgery.

And for about 150,000 patients in the United States who have the most severe form of the disease, amputation is the only option. The results of this recent study suggest amputation could be prevented in the majority of these severe cases.

And if the third phase of the clinical trials confirms the earlier results, the lives of those patients with severe cases could be tremendously affected, Powell said.

This is the first potential treatment that is non-surgical for really severe cardiovascular disease in the legs, he said. Roger Smith's story

About six years ago, Smith, 79, said he was having pain in his leg and trouble sleeping. He was initially misdiagnosed as having the nerve-related condition referred to as spinal stenosis, more commonly known as sciatica. When his condition worsened, he was correctly diagnosed with advanced peripheral artery disease. He lost two of his toes to amputation and was in danger of losing his leg.

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Treatment spares Lebanon man from amputation

(SACRAMENTO, Calif.) -- UC Davis Health System researchers are a step closer to launching human clinical trials involving the use of an innovative stem cell therapy to fight the virus that causes AIDS.

In a paper published in the May issue of the Journal of Virology, the UC Davis HIV team demonstrated both the safety and efficacy of transplanting anti-HIV stem cells into mice that represent models of infected patients. The technique, which involves replacing the immune system with stem cells engineered with a triple combination of HIV-resistant genes, proved capable of replicating a normally functioning human immune system by protecting and expanding HIV-resistant immune cells. The cells thrived and self-renewed even when challenged with an HIV viral load.

"We envision this as a potential functional cure for patients infected with HIV, giving them the ability to maintain a normal immune system through genetic resistance," said lead author Joseph Anderson, an assistant adjunct professor of internal medicine and a stem cell researcher at the UC Davis Institute for Regenerative Cures. "Ideally, it would be a one-time treatment through which stem cells express HIV-resistant genes, which in turn generate an entire HIV-resistant immune system."

To establish immunity in mice whose immune systems paralleled those of patients with HIV, Anderson and his team genetically modified human blood stem cells, which are responsible for producing the various types of immune cells in the body.

Building on work that members of the team have pursued over the last decade, they developed several anti-HIV genes that were inserted into blood stem cells using standard gene-therapy techniques and viral vectors (viruses that efficiently insert the genes they carry into host cells). The resulting combination vector contained:

These engineered blood stem cells, which could be differentiated into normal and functional human immune cells, were introduced into the mice. The goal was to validate whether this experimental treatment would result in an immune system that remained functional, even in the face of an HIV infection, and would halt or slow the progression toward AIDS.

The results were successful on all counts.

"After we challenged transplanted mice with live HIV, we demonstrated that the cells with HIV-resistant genes were protected from infection and survived in the face of a viral challenge, maintaining normal human CD4 levels," said Anderson. CD4+ T-cells are a type of specialized immune cell that HIV attacks and uses to make more copies of HIV.

"We actually saw an expansion of resistant cells after the viral challenge, because other cells which were not resistant were being killed off, and only the resistant cells remained, which took over the immune system and maintained normal CD4 levels," added Anderson.

The data provided from the study confirm the safety and efficacy of this combination anti-HIV lentiviral vector in a hematopoietic stem cell gene therapy setting for HIV and validated its potential application in future human clinical trials. The team has submitted a grant application for human clinical trials and is currently seeking regulatory approval, which is necessary to move on to clinical trials.

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Stem cell therapy to battle HIV?

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The Advances news section in April's issue of Scientific American included stories on digital textbooks, the promise of using gene therapy to fight blindness and how fragile orchids survive. To learn more about any of the stories, follow these links.

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Free text in electronic health records, with the help of natural language processing (NLP) technology, can be used to create accurate clinical decision support (CDS) tools, according to a study published this week in the Journal of the American Medical Informatics Association

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The California
stem cell agency today unveiled initial details of how it plans to
run its $30 million bank of reprogrammed adult stem cells.

"This permits
CIRM to have complete control of this valuable resource and is
consistent with the practice of NIH’s Center for Regenerative
Medicine which is also creating a repository for iPSC lines and
derived materials."

"The (current) IP
regulations were drafted to address conventional drug discovery
activities and did not contemplate creation of a comprehensive
repository of cell lines intended for broad distribution. As a
result, the IP regulations contain a number of provisions which are
either not applicable or worse could impede the success of the hiPSC
bank. For instance, IP regulations permit the exclusive licensing of
CIRM funded inventions and technology. This would be
counterproductive to the goals of the hiPSC repository which are
predicated on wide spread access."

"These lines
will serve as valuable tools in drug discovery and will be available
to researchers worldwide. The Tissue Collection RFA No. 12-02 will
fund clinicians and other scientists to identify, recruit and consent
sufficient numbers of affected individuals within a disease
population so as to effectively represent the disease’s
manifestations. Tissues will be collected and appropriate clinical,
medical or diagnostic information, will be obtained to enable
informed discovery of disease-related phenotypes and drug development
activities using hiPSC-based models. These tissue samples will be
provided (without charge) to the recipient of the CIRM hiPSC
Derivation Award (RFA No. 12-03) for the production of the hiPSC
lines. Once derived, characterized and released, the lines will be
deposited in the CIRM hiPSC bank funded under RFA No. 12-04."

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Gary Rabin, CEO of ACT

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Free text in electronic health records, with the help of natural language processing (NLP) technology, can be used to create accurate clinical decision support (CDS) tools, according to a study published this week in the Journal of the American Medical Informatics Association

See on jamia.bmj.com

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Read about why a Qualcomm Life executive says mobile health doesn’t yet have an Instagram, and why it eventually will.

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