CIRM Board Approves Funding for Clinical Trial Targeting Painful Scarring of the Urethra

South San Francisco, CA - A urethral stricture is scarring of the tube that carries urine out of the body. If left untreated it can be intensely painful and lead to kidney stones and infections. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved investing more than $3.8 million in a Phase 1 clinical trial to create a stem cell-based therapy for the condition.

This is the 81^st clinical trial that CIRM has funded.

When a scar, or stricture, forms along the urethra it impedes the flow of urine and causes other complications. James Yoo, M.D., Ph.D., and his team at Wake Forest University Health Sciences will use epithelial and smooth muscle cells, taken from the patient's bladder, and layer them on to a synthetic tubular scaffold. The tube will then be surgically implanted inside the urethra.

The goal is for the progenitor cells to support self-renewal of the tissue and for the entire structure to become integrated into the surrounding tissue and become indistinguishable from it, restoring normal urinary function. Dr. Yoo and his team believe their approach has the potential to be effective for at least a decade.

"While not immediately life-threatening, urethral strictures lead to multiple health complications that impair quality of life and predispose to kidney dysfunction," says Dr. Maria T. Millan, President and CEO of CIRM. "Developing an effective and durable treatment would significantly impact lives and has the potential to decrease the cumulative healthcare costs of treating recurrent kidney stones, infections and downstream kidney complications, especially of long-segment urethral strictures."

The CIRM Board also approved almost $4 million for ImmunoVec's late-stage preclinical program developing a stem cell and gene-based therapy for X-linked Chronic Granulomatous Disease (XCGD). This is a rare genetic disorder that leaves people with a severely impaired immune system, one that is vulnerable to repeated bacterial and fungal infections. Over time those infections take a toll on the individual and the average life expectancy for someone with XCGD is between 30 and 40 years.

The ImmunoVec team is developing an approach that will remove some of the patient's own blood stem cells, genetically re-engineer them in the lab and then return those stem cells to the patient. The goal is for the new cells to lead to a sustained production of white blood cells that have the capacity to kill infectious microorganisms and eliminate or prevent severe recurrent infections

In this late-stage preclinical program the goal is to show they can manufacture the cells, do the toxicity testing to show they are safe, and to develop the other materials needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial.

About CIRM

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission.

To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies.

With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world's largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.

For more information go to www.cirm.ca.gov

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