California Institute for Regenerative Medicine

14
May 2015
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Vision loss trial cleared in California

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Millions of people in North America live with varying degrees of irreversible vision loss. Some good news is that a novel stem cell therapy for retinitis pigmentosa (RP), an inherited condition that slowly damages the retina and can result in blindness, has been cleared by the U.S.…

Millions of people in North America live with varying degrees of irreversible vision loss. Some good news is that a novel stem cell therapy for retinitis pigmentosa (RP), an inherited condition that slowly damages the retina and can result in blindness, has been cleared by the U.S. Food and Drug Administration (FDA) to start a Phase I clinical trial.

The therapy for RP, which affects 1 in 3500 Canadians, was developed by Dr. Henry Klassen, Associate Professor at the Gavin Herbert Eye Institute, UC Irvine School of Medicine, and will consist of injecting patients with stem cells to help replace the cells destroyed by the disease and to save those cells that are not damaged yet.

The goal of the trial, which will involve up to 16 patients, is to evaluate the safety and identify side effects of the treatment. Although it is early to speak about a cure for RP, the researchers are hopeful.

“This milestone is a very important one for our project. It signals a turning point, marking the beginning of the clinical phase of development, and we are all very excited about this project.” said Dr. Klassen in the CIRM press release.

The trial has received almost $20 million in funds from the California Institute for Regenerative Medicine (CIRM).

“One of the goals of the agency is to provide the support that promising therapies need to progress and ultimately to get into clinical trials in patients. RP affects about 1.5 million people worldwide and is the leading cause of inherited blindness in the developed world. Having an effective treatment for it would transform people’s lives in extraordinary ways.” Jonathan Thomas, Ph.D., J.D., Chair of the Governing Board of the CIRM, said in the CIRM press release.

Click here to discover more about how stem cells are being used to understand and treat eye diseases on our Toward Treatments page.

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11
Apr 2014
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Stem cell research in California: ten years later

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Ten years ago, Californians voted in favor of Proposition 71 to support stem cell research, committing $3 billion to stem cell research and creating the California Institute for Regenerative Medicine (CIRM), the state agency that allocates the funds.…

Ten years ago, Californians voted in favor of Proposition 71 to support stem cell research, committing $3 billion to stem cell research and creating the California Institute for Regenerative Medicine (CIRM), the state agency that allocates the funds.

In 2011, CIRM announced it would fund its first human clinical trial using a stem-cell derived therapy, committing $25 million to a company called Geron to test the use of neural stem cells in patients in spinal cord injury. It was an inauspicious start: Geron shut down the trial after about six months and gave back the money, citing a desire to focus on experimental cancer therapies that were further along in the development pipeline.

However, CIRM has carried on. And according to the Sacramento Business Journal nine of the first 14 stem cell trials the agency has funded have either started enrolling patients or will begin doing so by the end of the year. Two trials are already underway: one testing therapies for HIV/AIDS, the other for congestive heart failure. Other trials set to begin include therapies for leukemia and solid tumor cancers, degenerative eye disease, Type I diabetes, sickle cell disease and a serious blood disorder.

“It’s the largest funding in the world for this particular science and it turned California into the epicenter for stem cell research,” said Dr. Jonathan Thomas, board chairman for the CIRM.

The process of transition from studies in mice to human trials is daunting and complex. (Read our recent blog entry on the state of stem cell trials about the state of stem cell clinical trials around the world.) But bringing more potential therapies to clinical trials is absolutely crucial.

“The world is full of cured mice,” says Dr. Jonathan Thomas. “You never know what you get in humans but this is how you find out. You have to get to clinical trials.”

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