Parkinson’s disease (PD) is the second most common neurodegenerative disorder and is estimated to affect more than four million patients worldwide — a number predicted to more than double by 2030. A fundamental characteristic of PD is progressive, severe, and irreversible loss of specific dopamine-producing neurons (DA neurons) in the midbrain that ultimately may result in disabling motor dysfunction. Multiple therapies have been developed for PD, but none can replace the lost cells. Cell transplantation has been considered a promising therapy, but in spite of extensive efforts to develop it in laboratories across the world, this approach has faced multiple challenges, including the absence of an appropriate cell source that can match the lost cells in function and safety.
In 2011, our team made a major discovery that enables the derivation of nearly unlimited numbers of authentic, engraftable midbrain DA neurons from human embryonic stem cells (hESCs). In recent publications, we have demonstrated that these cells can survive in three independent PD models and can reverse motor deficits of the disease.
In addition, the cells have an excellent safety profile with no evidence of tumor or excessive growth in any of the animals tested.
The investigators anticipate that by the end of the project period in 2017, our team will be ready to submit an Investigational New Drug (IND) application to the US Food and Drug Administration for a clinical trial in Parkinson’s patients. The team consists of scientists, neurologists, surgeons, industry leaders, ethicists, trial experts, and patient advocates who are dedicated to the achievement of this goal. The project further harnesses the expertise and strength present within Memorial Sloan Kettering at the Center for Cell Engineering (CCE) and the Center for Stem Cell Biology (CSCB) to deliver a first-in-man embryonic stem cell therapy for PD.
Investigators from Memorial Sloan Kettering, along with colleagues from Weill Cornell Medical College, Northwestern University, and Rush University Medical Center, have received a contract from New York State Stem Cell Science (NYSTEM) for almost $15 million over four years to develop a stem-cell-based therapy for Parkinson’s disease. NYSTEM works to further the agenda of the Empire State Stem Cell Board, whose mission is to foster a strong stem cell research community in New York State.
NYSTEM aims to accelerate the growth of scientific knowledge about stem cell biology and promote the development of therapies and diagnostic methods to alleviate disease and improve human health. The NYSTEM contract has enabled the creation of a multidisciplinary consortium with the overarching goal of developing an optimized, clinical-grade source of human DA neurons for cell therapy in PD by 2017.