This invention is a novel strategy for differentiating stem cells into dopamine-producing neurons that can be efficiently engrafted in vivo. Such a method could be used both therapeutically, by implanting the cells directly into the brains of Parkinson’s patients who have lost the ability to produce dopamine on their own, or as a research tool by drug companies that want to test the effects of their Parkinson’s drugs in the laboratory.
The protocol results in the production of dopaminergic neurons in just three to four weeks. These functional neurons have a profile that mimics endogenous dopaminergic neurons. In vivo transplantation in mouse, rat, and primate models of Parkinson’s disease resulted in long-term, stably-engrafted functional neurons as well as improvements in forelimb use and voluntary movement. The investigators have an optimized system for manufacturing clinical grade neurons in a GMP-compliant manner. The technology is currently in trials in primates.
- This differentiation process is greatly accelerated, compared to prior methods, and easily scalable
- Both human embryonic stem cells and induced pluripotent stem cells can be used as starting material, increasing the amount of available source material
- Unlike other methods, this differentiation protocol does not result in formation of teratomas, or any other neural overgrowth
Parkinson’s disease is the second most common neurodegenerative disease, with an estimated four million patients worldwide ($2.7B market). There is currently no adequate treatment to restore proper dopaminergic neuron function.
Proof-of-concept in primate models
U.S. National Application 14/356,042 published; national applications pending in Europe, Canada, Australia, Japan, China, and elsewhere
Lorenz Studer, MD/PhD, Director, Center for Stem Cell Biology, Memorial Sloan Kettering Cancer Center
Imke Ehlers, PhD, CLP
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