FDA clears way for Phase 1/2a trial of stem cell therapy for Parkinson’s
ANPD001 to be tested at US sites in people with moderate to severe disease
The U.S. Food and Drug Administration (FDA) has given Aspen Neuroscience clearance to launch a clinical trial of ANPD001, an experimental stem cell therapy designed to replace the nerve cells that are lost in Parkinson’s disease.
As planned, the open-label Phase 1/2a trial will test the safety, tolerability, and early efficacy of ANPD001 at escalating doses in people with moderate to severe Parkinson’s.
FDA clearance “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,” Damien McDevitt, PhD, president and CEO of Aspen, said in a company 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,” McDevitt said.
Phase 1/2a trial in US will test stem cell therapy for Parkinson’s disease
Parkinson’s is caused by the dysfunction and death of dopaminergic neurons — nerve cells in the brain that are responsible for making dopamine, a chemical that nerves use to communicate with each other and the rest of the body. The loss of dopamine signaling ultimately leads to the development of most disease symptoms.
ANPD001 aims to replace the dopaminergic neurons that are lost in Parkinson’s. The stem cell therapy is made using a three-step process.
First, skin cells are collected from a patient. They then are brought to a laboratory, where they are engineered to create a type of human-made stem cell called induced pluripotent stem cells or iPSCs. Like the stem cells naturally found in the body, iPSCs are able to grow into other cell types with the right set of biochemical cues.
In the third and final step, the iPSCs are given cues so that they grow into dopamine neuronal precursor cells (DANPCs) — immature cells able to become mature dopaminergic neurons. After a series of quality control steps in the lab, the DANPCs are given as a transplant to the patient with the aim of growing to replace lost dopaminergic neurons.
The upcoming Phase 1/2a clinical trial is expected to take place at multiple centers in the U.S. Aspen launched a screening study last year to help identify patients for the trial.
With FDA clearance secured, the clinical trial will be the first in the U.S. to test a therapy that uses iPSCs derived from the person who is undergoing treatment, referred to as autologous iPSCs.
“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,” said 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.”