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Archive for April 15th, 2012|Daily archive page

MAKING SENSE OF THE FDA AND STEM CELLS – WSJ OPED

In STEM CELLS IN THE NEWS on April 15, 2012 at 11:06 pm


By ANDREW VON ESCHENBACH-WSJ 4/15/12-See
http://online.wsj.com/articleSB10001424052702303815404577331673917964962.html?mod=googlen
ews_wsj

When I was commissioner of the Food and Drug Administration (FDA) from 2005 to 2009, I saw firsthand how regenerative medicine offered a cure for kidney and heart failure and other chronic conditions like diabetes. Researchers used stem cells to grow cells and tissues to replace failing organs, eliminating the need for expensive supportive treatments like dialysis and organ transplants.

But the beneficiaries were laboratory animals. Breakthroughs for humans were and still are a long way off. They have been stalled by regulatory uncertainty, because the FDA doesn’t have the scientific tools and resources to review complex innovations more expeditiously and pioneer regulatory pathways for state-of-the-art therapies that defy current agency conventions. Fortunately, Congress may have an opportunity as soon as this week to begin changing that.

The FDA isn’t obstructing progress because its employees are mean-spirited or foolish. But for decades, Congress has starved the agency of critical funding, limiting its scientists’ ability to keep up with peers in private industry and academia. The result is an agency in which science-based regulation often lags far behind scientific discovery. This forces the FDA to slow the approval of new treatments—and at times
creates acrimonious litigation between the FDA and innovators, not to mention disillusionment among desperate patients.

For example, in August 2010, the FDA filed suit against a company called Regenerative Sciences. Three years earlier, the company had begun marketing a process it called Regenexx to repair damaged joints by injecting them with a patient’s own stem cells. The FDA alleged that the cells the firm used had been manipulated to the point that they should be regulated as drugs. A resulting court injunction halting use of the technique has cast a pall over the future of regenerative medicine.

From the agency’s perspective, it had only called a “time out” until it could apply its regulatory process designed to analyze the therapy’s effectiveness and potential risks. For the industry, however, government had intervened in a way that seemed to bar the established clinical practice of using an individual’s own cells to advance the healing process.

Lawyers—many lawyers—are now trying to resolve this dispute. But at a time when science and technology are creating marvelous medical breakthroughs, the FDA should be leading and guiding the development of state-of-the-art therapies like regenerative medicine. Instead, the agency’s process for regulating complex new technologies often starts too late, after companies and researchers have sunk millions of dollars and thousands of hours into painstaking research.

It makes far better sense for the FDA to work collaboratively with physicians, patients, companies and academic researchers to craft standards for evaluating new technologies while they are still being developed, not years later when a company makes a marketing application for a breakthrough product.

Until that time, FDA scientists typically have little contact with the scientists who know the most about these innovative technologies.

This is not because they don’t want to. But consumer groups distrustful of industry have led Congress to erect ever greater barriers between regulators and those they regulate. The FDA can convene advisory committees of outside experts, but these experts weigh in only at the end of the regulatory process.

Worse, congressionally mandated conflict-of-interest rules keep many of the most knowledgeable academic and industry scientists off advisory committees out of fear that industry ties might bias their judgment.

Meanwhile, budget constraints have eroded the agency’s scientific foundations. When I moved from director of the National Cancer Institute at the National Institutes of Health (NIH) to become FDA commissioner in 2005, I was surprised to learn there are no provisions for continuous education to acquire new skills in emerging fields such as stem-cell biology, nanotechnology or computational biology. Even sending agency staff to academic conferences provoked a congressional outcry over meeting and travel costs.

If we want the FDA to lead innovation, and not lag behind it, Congress must give the agency the resources to be the world’s foremost science-based regulatory agency. It should endorse formal career development programs and encourage more collaboration with scientists in academia, industry, NIH and other federal agencies.

Congress should also make conflict-of-interest restrictions more rational, to ensure that agency staff can get early access to external scientific expertise to evaluate emerging technologies. The FDA should be able to make greater use of scientific consultants as “Special Government Employees” to broaden the pool of qualified and approved advisers for the agency.

FDA scientists I have encountered do care deeply about patients and want to say “yes” to safe and effective new therapies. Regulatory approval is the only bridge between miracles in the laboratory and lifesaving treatments. Yet until FDA reviewers can be scientifically confident of the benefits and risks of a new technology, their duty is to stop it—and stop it they will.

Congress has an opportunity to improve all this as it considers renewing FDA user-fee legislation this year. The temptation will be to reauthorize the fees as quickly as possible and move on as usual for another five years. This would be a grave mistake. Reforms that allow the FDA to say “yes” to innovative therapies serve all the agency’s stakeholders, including current and future patients.

Otherwise we had better get used to the agency saying no by calling “time out” or, worse, “game over” for American companies developing new, vital technologies like regenerative medicine.

Dr. von Eschenbach, a former director of the National Cancer Institute and commissioner of the Food and Drug Administration from 2006 to 2009, is chairman of the Manhattan Institute’s Project FDA.

Engineered stem cells seek out and kill HIV infection in living organisms

In VICTORIES & SUCCESS STORIES on April 15, 2012 at 1:52 pm

“…stem cells can be genetically engineered into HIV-fighting cells…”

is there anything they can’t do???

http://cbssanfran.files.wordpress.com/2010/10/hiv_aids_generic.jpg?w=300

Engineered stem cells seek out and kill HIV infection in living organisms

Washington, Sun, 15 Apr 2012 ANI

Washington, Apr 15 (ANI): In a new study, scientists have expanded on previous research that had provided proof-of-principal that human stem cells can be genetically engineered into HIV-fighting cells.

This time, they have demonstrated that these cells can actually attack HIV-infected cells in a living organism.

The study by UCLA researchers demonstrate for the first time that engineering stem cells to form immune cells that target HIV is effective in suppressing the virus in living tissues in an animal model.

“We believe that this study lays the groundwork for the potential use of this type of an approach in combating HIV infection in infected individuals, in hopes of eradicating the virus from the body,” Scott G. Kitchen, lead investigator of the study, said.

In the previous research, the scientists took CD8 cytotoxic T lymphocytes – the “killer” T cells that help fight infection – from an HIV-infected individual and identified the molecule known as the T cell receptor, which guides the T cell in recognizing and killing HIV-infected cells.

However, these T cells, while able to destroy HIV-infected cells, do not exist in great enough quantities to clear the virus from the body. So the researchers cloned the receptor and used this to genetically engineer human blood stem cells.

http://www.differencebetween.co.in/wp-content/uploads/2011/05/Mouse.jpg

They then placed the engineered stem cells into human thymus tissue that had been implanted in mice, allowing them to study the reaction in a living organism.

The engineered stem cells developed into a large population of mature, multi-functional HIV-specific CD8 cells that could specifically target cells containing HIV proteins.

The researchers also discovered that HIV-specific T cell receptors have to be matched to an individual in much the same way an organ is matched to a transplant patient.

In this current study, the researchers similarly engineered human blood stem cells and found that they can form mature T cells that can attack HIV in tissues where the virus resides and replicates. They did so by using a surrogate model, the humanized mouse, in which HIV infection closely resembles the disease and its progression in humans.

In a series of tests on the mice’s peripheral blood, plasma and organs conducted two weeks and six weeks after introducing the engineered cells, the researchers found that the number of CD4 “helper” T cells – which become depleted as a result of HIV infection – increased, while levels of HIV in the blood decreased.

CD4 cells are white blood cells that are an important component of the immune system, helping to fight off infections. These results indicated that the engineered cells were capable of developing and migrating to the organs to fight infection there.

The researchers did note a potential weakness with the study: Human immune cells reconstituted at a lower level in the humanized mice than they would in humans, and as a result, the mice’s immune systems were mostly, though not completely, reconstructed.

Due to this, HIV may be slower to mutate in the mice than in human hosts. So the use of multiple, engineered T cell receptors may be one way to adjust for the higher potential for HIV mutation in humans.

“We believe that this is the first step in developing a more aggressive approach in correcting the defects in the human T cell responses that allow HIV to persist in infected people,” Kitchen added.

The study has been published in the journal PLoS Pathogens. (ANI)

Engineered stem cells seek out and kill HIV infection in living organisms.

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