Buck Institute Awarded $6 Million to Lead Effort to Get Stem Cell Treatment for Parkinson’s Disease Ready for Clinical Trials

The promise of stem cell therapies to treat disease will remain just a promise unless methods are developed to get discoveries from the lab bench into clinical trials. The Buck Institute for Age Research has been awarded $6 million from the California Institute for Regenerative Medicine (CIRM) to fund a three-year project aimed at getting a stem cell derived treatment for Parkinson's disease (PD) ready for testing in humans.

The effort, led by Buck Institute faculty Xianmin Zeng, PhD, addresses the critical need for a robust system to efficiently produce the type of neurons needed to treat patients – under processes that meet standards for good manufacturing practice.  City of Hope, a leading biotech research and treatment center located in Duarte, CA is a partner on the award.

“It’s one thing to grow neurons from human embryonic stem cells in a petri dish,” said Zeng.  “It’s another matter entirely to scale up the production of neurons needed to treat patients in a manner that can be tested and validated throughout each stage of the manufacturing  process.”  The purpose of the “CIRM Early Translational II Research Award” is to get potential treatments to the point where they can be approved by the Federal Drug Administration as Investigational New Drugs (IND) – a step required prior to the beginning of clinical trials.

CIRM’s review of the grant application said Zeng’s proposal “addresses a significant, unmet medical need and, if successful, could have a profound impact.”  Zeng’s lab is focused on coaxing human pluripotent stem cells into dopamine-producing neurons. Dopamine is a neurotransmitter produced in the mid-brain which facilitates many critical functions, including motor skills. Patients with PD lack sufficient dopamine; the disease is a progressive, incurable neurodegenerative disorder that affects 1.5 million Americans and results in tremor, slowness of movement and rigidity.

A team lead by Larry A. Couture, Ph.D., Senior Vice President of City of Hope’s Center for Applied Technology Development (CATD), will produce the dopaminergic neurons from a clinically compliant embryonic stem cell line  using protocols developed  by the Buck Institute. Scientists in the Zeng lab will test the function of the neurons produced under GMP-compatible processes at City of Hope to determine if they are functionally equivalent to neurons generated from a lab bench, which were demonstrated to survive and correct behavioral deficits  in PD in the past by Dr. Zeng’s team.

“We are very excited about the opportunity to combine our expertise in translational science and manufacturing with Dr. Zeng’s leadership and state-of-the art stem cell research facilities at the Buck institute,” said Dr. Couture. “This project has a high probability of success and its potential to result in a treatment for Parkinson’s disease make it particularly important.”  Dr. Couture has over 20 years of experience in developing biologic therapies and currently oversees more then 30 active Investigational New Drugs (IND).

“It is essential that these neurons have the exact same characteristics and that they can be tracked back to their origin,” said Zeng.  “I am confident that our teams will be able to accomplish this essential step for future IND-enabling studies prior to FDA approval. I am grateful for CIRM’s approval of this project.”

The California Institute for Regenerative Medicine, the state stem cell agency created by proposition 71, approved a total 19 awards worth $67 million in its second round of awards designed to move good ideas out of the lab and into the clinic. “This second round of Early Translational Awards will strengthen CIRM’s portfolio of future therapies,” said Alan Trounson, CIRM President. “We are looking for ways to complement our leading edge of stem-cell based treatments for patients and these projects will load our frontline portfolio with promising studies on autism, muscular dystrophy, Canavan disease and liver disease.  These projects will enhance the potential medical options available for patients and hopefully in the near future produce cures for such debilitating handicaps and diseases.”

“We consider CIRM a major partner in our efforts to develop treatments aimed at alleviating age-associated diseases,” said Brian K. Kennedy, PhD, Chief Executive Officer of the Buck Institute. “Translating basic research into actual therapies is our ultimate goal; I congratulate Dr. Zeng on her award and thank CIRM for their support of this important work.”