January 6, 2022: “BlueRock Therapeutics, a clinical stage biopharmaceutical company and wholly-owned subsidiary of Bayer AG, announced the closeout of the first of two cohorts in its ongoing Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with advanced Parkinson’s disease.
The purpose of the Ph1 clinical trial is to evaluate the safety, tolerability and preliminary efficacy of DA01 in patients with Parkinson’s disease.
Enrollment of the first cohort of patients was completed at three active sites in the United States and Canada: Weill Cornell Medicine, University of California, Irvine and University Health Network.
Patients in the first cohort received a bilateral surgery to transplant dopaminergic neurons in the midbrain region.
No severe adverse events were observed in any participating patients.
“By replacing lost neurons in the midbrain region of patients with Parkinson’s disease, we have the potential to regenerate lost function and possibly improve and reverse the disease,” states Joachim Fruebis, Ph.D., Chief Development Officer of BlueRock Therapeutics.
“This is an incredible step forward as we execute on our goal of bringing authentic cellular medicines to patients in desperate need of treatment.”
About the DA01 Phase 1 Trial
Enrollment is currently open for the second and final high-dose cohort in both the United States and Canada.
The primary objective of the Ph1 trial is to assess the safety and tolerability of DA01 cell transplantation at one-year post-transplant.
The secondary objectives of the trial are to assess the evidence of transplanted cell survival and motor effects at one- and two-years post-transplant, to evaluate continued safety and tolerability at two years, and to assess feasibility of transplantation.
The trial is being conducted at Weill Cornell Medicine, under the guidance of Dr. Sarva, M.D., Principal Investigator (PI), University of California, Irvine, under the guidance of Dr. Henchcliffe, M.D., D.Phil., F.A.A.N., F.A.N.A. (PI) and at the University Health Network, under the guidance of Dr. Lozano, O.C., M.D., Ph.D., F.R.C.S.C., F.R.S.C., F.C.A.H.S. (PI).
More information about this trial is available at clinicaltrials.gov (NCT#04802733).
About Parkinson’s Disease
Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels.
The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons.
At diagnosis, it is estimated that patients have already lost 60-80% of their dopaminergic neurons.Parkinson’s disease often starts with a tremor in one hand.
Other symptoms are rigidity, cramping and slowness of movement (bradykinesia). According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease, with one million living in the United States.
DA01 is being clinically evaluated in a subset of patients with advanced Parkinson’s disease, where regular medications are no longer fully effective.”
