Dr Jill Jarecki Discusses the Current Pipeline for SMA Therapies

Combination treatments may be the future for treating patients with spinal muscular atrophy (SMA) at all stages and ages, explained Jill Jarecki, PhD, chief scientific officer at Cure SMA and research director of TREAT-NMD Neuromuscular Network.

Transcript

What else is in the pipeline for SMA and how do those therapies differ from what is already approved?

So, at CureSMA, we're working to fund a number of drugs in the SMA drug pipeline and the reason for that is that there has been dramatic changes with the FDA approved drugs in our presymptomatic in our younger patient population. And we also get clinically meaningful, but more modest gains, in our older patients who are chronically living with SMA.

So, we believe that we need a cocktail approach to achieve maximally effective benefit in all stages and ages of SMA. And this likely means combination therapies, and combination therapies that target either muscle function or motor neuron function. Muscle drugs are the furthest along with this sort of combination, or cocktail, approach. And we have a drug in clinical trials with Scholar Rock that helps with the muscle side of things.

And in our pipeline, we have many early stage drugs that focus on more diverse approaches, and that's where CureSMA is currently directing our funding, to help identify novel drug approaches for SMA that can be layered on with our approved SMN [survival of the motor neuron] upregulating drugs.