Cure SMA awarded a $50,000 drug discovery grant to researcher Barrington Burnett of Uniformed Services University of the Health Sciences for the project “Slowing SMN degradation to treat SMA,” which aims to characterize and validate a new survival motor neuron (SMN) protein modulator for the treatment of spinal muscular atrophy (SMA).
Drug discovery is a research area via the Cure SMA organization that takes scientific discoveries concerning the causes or biology of SMA, and converts the knowledge into new drug candidates that can then be followed for assessment in clinical trials. Burnett’s grant is part of Cure SMA’s overall $704,000 discovery grant program this year.
Because people with SMA don’t produce enough SMN protein due to a mutation in the survival motor neuron 1 (SMN1) gene, most early research focused on increasing SMN protein levels by targeting the underlying genetics of the disease. The goal is to boost SMN protein production by replacing or correcting SMN1 or by modulating SMN2, SMA’s low-functioning back-up gene.
Burnett and colleagues from the Bethesda, Maryland federally operated university, are looking into ways to slow the degradation of the SMN protein so that it stays around for longer periods of time. The researchers expect to eventually increase the overall levels of SMN protein in cells.
The team will use cell-based assays and animal models to assess the safety, efficacy and selectivity of a newly identified compound, using a high throughput screen that modulates SMN protein degradation. The project aims to develop molecules with an innovative action that could treat the disease.
According to a press release, Burnett and the team believe the approach could be used in combination with other therapies that increase SMN levels, like SMN2 splicing modulators, that will allow the patients to not only produce more protein, but also make the protein last longer.