The NIH Parkinson's Disease Repurposing Program is a coordinated federal initiative to identify existing drugs that can be repurposed for Parkinson's disease therapy development. Led by the National Institute of Neurological Disorders and Stroke (NINDS) in collaboration with the National Institute on Aging (NIA), this program leverages the NIH's unique position to facilitate rapid clinical testing of approved compounds for new indications.
The program emerged from the recognition that the drug development pipeline for Parkinson's disease lacks sufficient disease-modifying therapies, and that drug repurposing offers a faster path to clinical translation given known safety profiles of existing drugs.
The program screens existing compounds based on:
| Criterion | Description |
|---|---|
| Mechanistic Rationale | Drugs targeting PD-relevant pathways |
| Blood-Brain Barrier Penetration | CNS penetration for neuronal targets |
| Known Safety Profile | Established human safety data |
| Preclinical Evidence | Animal model support for neuroprotection |
Promising compounds undergo:
Clinical trial phases include:
The NIH has supported multiple clinical trials of GLP-1 receptor agonists for Parkinson's disease:
| Drug | Trial Phase | Status | Mechanism |
|---|---|---|---|
| Exenatide | Phase III | Completed | GLP-1R activation, neuroprotection |
| Liraglutide | Phase II | Completed | GLP-1R activation, anti-inflammatory |
| Semaglutide | Phase II/III | Ongoing | GLP-1R activation, mitochondrial function |
Clinical Evidence:
| Drug | Target | PD Rationale |
|---|---|---|
| Nilotinib | BCR-ABL, c-KIT | Autophagy induction, alpha-synuclein clearance |
| Dasatinib | BCR-ABL | Senolytic effects, anti-inflammatory |
NIH-Supported Trials:
| Drug | Mechanism | Trial Status |
|---|---|---|
| Minocycline | Microglial inhibition | Phase II completed |
| Ibuprofen | COX inhibition | Observational studies |
The program works with leading research institutions:
| Trial Name | Drug | Phase | N | Primary Outcome |
|---|---|---|---|---|
| EXENATIDE-PD | Exenatide | Phase II | 60 | Motor scores |
| NILO-PD | Nilotinib | Phase II | 75 | Motor scores |
| AMBRA-PD | Ambroxol | Phase II | 120 | GBA activity |
The program is expanding to include: