A recent approval from the U.S. Food and Drug Administration will allow MSK researchers and their collaborators to open the first clinical trial testing an investigational stem cell therapy aimed at restoring lost brain cells called neurons in people with advanced Parkinson’s disease (PD).
“This is a big step for the stem cell field – to finally test a truly “off-the-shelf” dopamine neuron product in patients with PD,” said Lorenz Studer, MD, Director of MSK’s Center for Stem Cell Biology and a co-inventor of the new therapy.
Parkinson’s disease occurs when neurons making dopamine, a chemical that coordinates movement, stop working or die, causing tremor, slowness, stiffness, and walking and balance problems, among other symptoms. PD is a lifelong and progressive disease, with symptoms slowly worsening over time, and it affects nearly 1 million people in the United States and more than 6 million people worldwide.
In the last 50 years, very little progress has been made in PD. The main treatment is still a drug called L-DOPA, which was first used in the 1960s and was revolutionary at the time. But it is not a cure, and eventually it stops working.
“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, Chair of MSK’s Department of Neurosurgery and an investigator for the upcoming trial. “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.”
Dr. Studer added, “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.”
The phase 1 clinical trial, with support from BlueRock Therapeutics, will assess the safety, tolerability, and preliminary efficacy of the new therapy in 10 patients. On February 4, 2021, the research that led to the approval of the IND and upcoming human trial was described by Drs. Studer and Tabar in two papers they had published in the journal Cell Stem Cell.
Researchers at Memorial Sloan Kettering Cancer Center, including Dr. Studer, developed stem cell-derived dopaminergic neurons for the treatment of neurodegenerative diseases, and MSK licensed this intellectual property to BlueRock Therapeutics. MSK has institutional financial interests related to this intellectual property and BlueRock. Dr. Studer has intellectual property rights and interests and financial interests related to BlueRock. Dr. Tabar has financial interests related to BlueRock.