NANOSCALE COMBINATORIAL SYNTHESIS INC — Department of Health and Human Services SBIR Phase I: DESCRIPTION (provided by applicant): We will employ a novel approach to develop unique AL
NANOSCALE COMBINATORIAL SYNTHESIS INC — SBIR Phase I award from Department of Health and Human Services.
- Amount
- $295,299
- Agency
- Department of Health and Human Services · National Institutes of Health
- Program / Phase
- SBIR · Phase I
- Solicitation
- PA13-234
- NAICS
- —
- Place of performance
- CA
- Period
- —
Description
DESCRIPTION (provided by applicant): We will employ a novel approach to develop unique ALS therapeutics by focusing on drugs that activate a natural cellular protective mechanism, autophagy. Stimulating autophagy is a logical approach to treat the underlying neurodegeneration that causes ALS. It is the prime defense mechanism that neurons have for removing toxic misfolded aggregated proteins, and there is evidence that neurodegeneration of motor neurons (MNs) in ALS is caused by the buildup of toxic, misfolded, aggregated forms of SOD1, TDP43 and FUS. Mutations in these proteins cause familial ALS (fALS), and overexpression or abnormal post-translational modification of these proteins leads to MN degeneration in sporadic ALS (sALS). We will develop drugs that effectively stimulate autophagy in ALS MNs to remove the buildup of toxic proteins to block neurodegeneration and disease progression to treat sALS patients. Our collaborator, Dr. Finkbeiner, previously identified drugs that stimulate autophagy in