While the FDA-approved drug omaveloxolone (Skyclarys) improves neurological symptoms in FA, its effect on heart function remains unclear. Dr. Sharma proposes to test a novel stem cell-based therapy STM-01 and its derivatives in a translational, inducible FRDAkd mouse model, that captures both the cardiac and neurological features of Friedreich’s ataxia (FA). STM-01 is comprised of cardiac progenitor cells from human neonatal heart tissue, which releases molecules that reduce inflammation, tissue scarring and oxidative stress.
In collaboration with Dr. Elena Dedkova, this group will use a doxycycline-inducible FA mouse model to suppress frataxin and then treat mice with STM-01 or a placebo. Heart function will be assessed by echocardiography and electrical recordings, while neurological effects will be measured through balance, coordination, and sensory tests. Tissue analysis will examine nerve preservation and inflammation reduction.
The goal is to determine whether this therapy can:
1. Improve heart function and structure
2. Reduce scarring and inflammation
3. Enhance exercise capacity and motor coordination
4. Protect sensory function
Positive results would provide strong evidence for advancing this treatment toward clinical trials, addressing both the cardiac and neurological challenges of FA.