Parkinson’s disease and glioblastoma are very different diseases of the brain but neither has very good treatment options and both clearly represent an unmet medical need. With that in mind, the governing Board of the California Institute for Regenerative Medicine (CIRM), the state’s stem cell agency, yesterday voted to invest almost $9.5 million in developing new approaches to both conditions.

John Zaia, M.D., at City of Hope, was awarded $3.68 million to do the late stage preclinical research needed to apply to the US Food and Drug Administration (FDA) to run a clinical trial targeting glioblastoma.

There is no cure for glioblastoma, the deadliest form of brain cancer. The standard treatment involves surgery, chemotherapy and radiation but even then, the median survival time is only around 15 months (meaning half the patients survive this long). Because chemotherapy can cause toxic side effects in the blood the strength of the dose that patients get is often limited. The City of Hope team plans to both genetically engineer the patient’s own blood stem cells to protect them from chemotherapy and sensitize the tumor cells to make them more vulnerable to the chemotherapy. This will hopefully enable patients to get a higher dose of chemotherapy and improve survival time and quality of life.

Dr. Maria Millan, CIRM’s President & CEO, says even people who have access to the best treatment rarely do well with this form of cancer:

“Glioblastoma is the most common, and the most aggressive, form of brain cancer that led to the death of U.S. Senator Ted Kennedy and former Vice President Joe Biden’s son Beau Biden. CIRM has supported a variety of stem cell-based approaches to target this devastating and currently untreatable condition.  The project approved by our Board today is unique in that it seeks to use gene modified stem cells to allow patients to tolerate the high doses of chemotherapy while also making these tumors more susceptible to the chemotherapy.”

The CIRM Board also awarded $5.8 million to Krystof Bankiewicz, M.D., Ph.D., at the University of California, San Francisco (UCSF).  In collaboration with Clive Svendsen, Ph.D. at Cedars-Sinai, this team is testing the potential for neural progenitor cells, engineered to express the growth factor GDNF, to impact Parkinson’s disease. With CIRM funding, the investigators will perform pre-clinical research that is aimed at enabling them to file an application with the FDA to test this approach in a clinical trial.

Dr. Millan noted that the cells used in this project are already being used in a CIRM-funded clinical trial:

“CIRM is currently funding a Phase 1 clinical trial at Cedars-Sinai with this neural progenitor cell product for the treatment of ALS, another devastating neurodegenerative disorder for which there is no cure.”

David Higgins, PhD, the CIRM Board Patient Advocate for Parkinson’s disease says there is a real need for something that can have a big impact on the disease:

“One of the big frustrations for people with Parkinson’s, and their families and loved ones, is that existing therapies only address the symptoms and do little to slow down or even reverse the progress of the disease. That’s why it’s important to support any project that has the potential to address Parkinson’s at a much deeper, longer-lasting level.”