BlueRock Therapeutics, a wholly-owned subsidiary of Bayer AG, in collaboration with Memorial Sloan Kettering Cancer Center (MSK), announced the U.S. Food and Drug Administration (FDA) has cleared their Investigational New Drug (IND) application to proceed with a Phase 1 (Ph1) study in patients with advanced Parkinson’s disease (PD). This is the first trial in the United States to study pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease. Under the IND, BlueRock and MSK will execute a Ph1 clinical trial to evaluate the safety, tolerability and preliminary efficacy of DA01 in patients with PD.

“This is a big step for the stem cell field – to finally test a truly “off-the-shelf” dopamine neuron product in human PD patients,” said Lorenz Studer, MD, scientific co-founder of BlueRock and Director, Center for Stem Cell Biology at MSK. “We are also grateful for the visionary support by NYSTEM, the NY state-sponsored stem cell program that supported the earlier stages of this project.”

“This trial is the culmination of a decade of arduous collaborative work that is based on very rigorous science. It is an important milestone on the road towards regenerative brain repair,” said Viviane Tabar, MD, founding investigator of BlueRock and Chair of MSK’s Department of Neurosurgery. “It is a real privilege and very exciting to be able to participate in both the bench science and the actual surgical intervention, here at MSK. Our collaborators at Weill Cornell Neurology will also be an integral part of the trial.”

“Today, there is no disease-modifying treatment for Parkinson’s. Through this trial and those to follow, we hope to change that,” stated Emile Nuwaysir, Ph.D., President and Chief Executive Officer of BlueRock. “Our therapy is intended to replace the midbrain dopaminergic neurons lost in the degenerative condition to rebuild the neural circuit, and thereby restore motor control to Parkinson’s patients. This could shift the treatment paradigm for millions of PD patients, as well as demonstrate for the first time that degenerative disease is, in principle, reversible. We believe this would represent an enormous step for the PD community worldwide, and for medicine.”

The trial plans to enroll ten patients starting with a first clinical site at Weill Cornell Medicine in the initial open-label study. The primary objective of the Ph1 study is to assess the safety and tolerability of DA01 cell transplantation at one-year post-transplant. The secondary objectives of the study 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.

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 dyskinesias. Parkinson’s disease is the second most common neurodegenerative disorder, impacting more than 7.5 million people, including 1.3 million people in North America.