FDA Clears IND for New Parkinson’s Disease Treatment Using IPSCs

Today, Aspen Neuroscience announced that the United States Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for a new Parkinson’s Disease gene therapy. With this clearance secured, the company has the green light to begin clinical trials.

"The IND clearance of ANPD001 sets in motion a path toward a new treatment for the more than one million Americans and 10 million people worldwide with Parkinson's disease," said Damien McDevitt, PhD, Aspen president, and CEO, in the press release. "Our visionary team is working to make personalized regenerative medicine a reality, and we look forward to advancing this cell therapy for patients who are waiting."

ANPD001 is an autologous gene therapy that was developed by Aspen’s co-founders, Jeanne Loring, PhD, Professor Emeritus and Director of The Center for Regenerative Medicine, Department of Molecular Medicine at the Scripps Research Institute, and Andres Bratt-Leal, PhD, Aspen senior vice president, research and development.

The drug attempts to replace damaged neuronal cells in patients with Parkinson’s disease. According to the press release, the treatment process has three components. First, clinicians take a small sample of the patient’s stem cells.

The company will develop induced pluripotent stem cells (iPSCs) that will differentiate into dopamine neuronal precursor cells using these samples. Once these cells have been developed for each patient, the researchers can surgically replace lost or damaged neurons with these reprogrammed cells.

"This is a major milestone in Aspen's mission to develop and deliver personalized iPSC-derived cell replacement therapies for people with unmet medical needs, starting with Parkinson's disease," added Faheem Hasnain, chairman of the Aspen board of directors. "This is an exciting time for the Aspen team and the patients who have been so instrumental in enabling the company's development."

The company will start its phase 1/2a clinical trial in the coming years. The study will be a single-arm, open-label, dose-escalation clinical trial. Researchers have already screened patients to enroll in the clinical trial, which will test the treatment’s safety, tolerability, and efficacy.