Dosing Starts in Canada in Trial of DA01 for Advanced Parkinson’s
The therapy consists of surgically injecting neurons (nerve cells) that make dopamine — and which are damaged and die with Parkinson’s — into the putamen, an area of the brain that controls movement. The cells are derived from human embryonic pluripotent stem cells — ‘primitive’ cells from which other types of cells can form — and converted in the lab into the dopamine-making neurons.
This first surgery, performed at University Health Network in Toronto, marks the closure of a first enrolled patient group in this Phase 1 trial (NCT04802733). Enrollment is still open for a second and final high-dose group in the U.S. and Canada. Information on contacts and locations can be found here.
“With this surgery, our objective is to use authentic cells, to have them integrate into the brain and restore lost physiologic function. If successful, this treatment could have implications for the Parkinson’s community both in Canada and beyond,” Andres Lozano, MD, PhD, principal investigator at the Krembil Brain Institute at University Health Network, said in a press release.
The trial’s goal is to test whether DA01 is safe, and to monitor for potential side effects.
Safety will be determined through the number of serious side effects reported during the year following the surgery or through evidence of abnormal tissue overgrowth related to the presence of the injected cells.
Secondary goals (endpoints) are evidence that cells can survive after being injected into the brain, changes in motor function and the number of waking hours in ‘off’ states (when levodopa is no longer working well), and safety up to two years following the surgery.
To be eligible to enter the open-label study, patients must between ages 60 and 76 in the U.S. and ages 50 to 76 in Canada, have been diagnosed with Parkinson’s for 5–15 years, and be on levodopa but not fully respond to the medication, with complications like off states and/or dyskinesia (involuntary jerky movements).
A device that is used to inject fluids into the brain will be used to deliver DA01. During the surgery, the patients will be under general anesthesia. Post-surgery, they need to take immunosuppressants for one year to prevent their body from rejecting the cells.
Open-label trials have no placebo group; all enrolled are given the experimental treatment.
“It is incredibly exciting to be a part of delivering this first-of-its-kind authentic cellular medicine to patients in Canada, with the hope of regenerating lost function and possibly improving and reversing Parkinson’s disease,” said Joachim Fruebis, PhD, chief development officer of BlueRock, a Bayer subsidiary.
The U.S. Food and Drug Administration granted fast track status to DA01 as a potential treatment of advanced Parkinson’s in July 2021.
In the U.S., the first patients were enrolled at Weill Cornell Medicine in New York City and the first surgeries performed at the Memorial Sloan Kettering Cancer Center. An additional site was added at the University of California in Irvine.