There are now two treatments approved by the U.S. Food and Drug Administration (FDA) that target the underlying genetics of spinal muscular atrophy (SMA) and several others in clinical trials. The advent of these new treatments for SMA has led to community discussion around the possibility of combining therapies in the hopes of better outcomes. This discussion is especially relevant because of the different ways single therapies may impact individual patients as a result of their age, age at the start of treatment, disease type, stage of disease, and severity of symptoms. While there’s great promise in the research landscape, there’s also a pressing need for continued and growing investment. In the end, it will likely take a combination of therapies to achieve the greatest possible impact for people with all stages and types of SMA.

To better understand the complex topic of combining treatments, Cure SMA developed a new resource booklet for families and caregivers, “Scientific Considerations for Drug Combinations.” In this, we define the term “combination therapy” in the context of SMA, discuss SMN-independent and SMN-dependent therapeutic options, and outline potential risks and benefits to consider when thinking about combination therapies. Many researchers believe that it will take a combination of SMN-based and non-SMN treatments to provide the most benefit for those affected by SMA. This could mean that affected individuals will take two drugs together simultaneously.

Cure SMA has a strategic goal of finding novel targets beyond SMN to develop therapies that will provide benefit for symptomatic patients, either in combination or alone. As we pursue this goal, we will consider all the elements discussed in this booklet. By outlining these considerations, we aim to help patients, families, and caregivers better understand potential options. As always, Cure SMA recommends patients consult with their healthcare providers regarding any treatment considerations or decisions.

This booklet was produced in partnership with the Cure SMA Industry Collaboration. The members of the Cure SMA Collaboration are Avexis Inc, a Novartis company, Astellas Pharma Inc, Biogen, Cytokinetics Inc, Genentech/Roche Pharmaceuticals, and Scholar Rock.