CurePSP has awarded four grants totaling $320,000 to research potential therapies for prime-of-life neurodegenerative diseases caused by the accumu
CurePSP has awarded four grants totaling $320,000 to research potential therapies for prime-of-life neurodegenerative diseases caused by the accumulation of tau in the brain.
The grants support studies that will investigate gene therapy, neuroprotective enzymes, activation of autophagy, and identification of microglia associated with toxic protein accumulation to treat PSP, CBD, FTD, and related diseases.
The awards go to:
Dr. Rachel Bailey, Center for Alzheimer’s and Neurodegenerative Diseases, UT Southwestern Medical Center, Dallas, Texas
Dr. Daniel H. Geschwind, Department of Neurology, University of California, Los Angeles
Dr. Maria Catarina Lima da Silva, Department of Neurology of Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts
Dr. Marta Olah, Department of Neurology, Columbia University, New York City
Dr. Bailey will study gene replacement therapy to combat toxic accumulation of the tau protein. She will test a way to use a virus to deliver two types of RNA: one to prevent the manufacture of abnormal tau and the other to encode an aggregation-resistant form of tau.
Dr. Geschwind and his team will test four new, orally available drugs in mice genetically engineered to produce abnormal tau protein. The drugs enhance the activity of an enzyme called puromycin-sensitive aminopeptidase. This enzyme cuts up tau protein and enhances the brain cells’ autophagy system, which disposes of some types of abnormal proteins, including tau.
Dr. Lima da Silva will also investigate small-molecule activators of autophagy to clear toxic protein accumulation in the brain. Rather than mice, Dr. Lima da Silva’s approach will use neurons grown from stem cells derived from skin biopsies of human patients. She will study orally available compounds that activate an enzyme called ULK1, an autophagy enhancer.
Dr. Olah will study microglia, the resident immune cells of the brain, which recently have shown to be a major participant in the neurodegenerative process. Dr. Olah will use a new method to sequence the RNA in individual microglial cells, creating a map of which cells are encoding which proteins in proximity to degenerating neurons. This study could generate new insights into the disease process and new targets for drugs.
CurePSP awards Venture Grants twice a year. Applications are reviewed and recommended to the nonprofit's board of directors by an independent scientific advisory board. The next application deadline is December 17, 2021.