Summer 2017 Update to SMA Drug Pipeline is Released

We’ve recently released an update to the SMA drug pipeline. This latest version includes:

  • 19 active programs, including one approved therapy. 
  • 14 pharmaceutical partners. 
  • 5 programs in clinical trials. 
  • An ever-increasing breadth of potential treatment approaches to SMA.

Effective Treatments for All Ages, Stages and Types of SMA

Late last year, our community celebrated the approval of Spinraza, our first-ever FDA approval of a treatment for SMA. The updated version of this pipeline reflects our community’s progress in gaining access to Spinraza, as well as the progress of 18 additional programs currently in development.

Individuals with SMA don’t produce survival motor neuron (SMN) protein at high enough levels due to a mutation in the survival motor neuron 1 (SMN1) gene. Spinraza, and many of the other programs in the drug pipeline, work to address this loss of SMN protein by replacing SMN1 or by modulating SMN2, the low-functioning SMA “backup gene.” These are called “SMN-based” or “SMN-enhancing” approaches.

There are also a number of systems, pathways and processes that are affected in SMA, and there may be additional ways to treat SMA that work on these other areas—collectively referred to as “non-SMN” approaches. These non-SMN approaches include drugs that work on the muscles and nerves.

Research strongly suggests that a combination of SMN-enhancing and non-SMN approaches is the best route to treat all ages, stages, and types of SMA. This allows us to attack SMA from all sides and ensures that comprehensive, effective treatments are available. Because of this, Cure SMA’s strategic research model is focused on the complementary goals of increasing the total number of programs in the drug pipeline, and increasing the diversity of programs in the pipeline.

Advancing Our Research Goals

This latest drug pipeline update adds programs from both SMN-enhancing and non-SMN approaches. As the drug programs continue to move forward and as we continue to work through issues surrounding access to approved therapies, we also remain focused on continued funding for research to ensure continued growth of the pipeline.

Earlier this spring, we announced over $1 million in basic research grants, which also funded both SMN-enhancing and non-SMN approaches. Basic research investigates the causes and biology of SMA, often revealing more effective ways of making SMA drugs. These “seed ideas” can lead to drug development programs that are then added to the pipeline. Basic research is our investment in future drug development for SMA.

Thank You

The dedicated support of our community has made all of this possible. Whether families raising funds and awareness, researchers investigating new potential treatments, or pharmaceutical and regulatory partners helping us take the next steps—everyone has a role to play. Thank you to everyone for their hard work and dedication.

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