Cure SMA Releases June 2016 Update to the SMA Drug Pipeline

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

  • 18 active programs.
  • 14 pharmaceutical partners.
  • 6 programs in clinical trials.
  • 28 programs in the cumulative pipeline total, including 10 failures to date.
  • An ever-increasing breadth of potential treatment approaches to SMA.

Effective Treatments for All Ages, Stages and Types of SMA

As the SMA drug pipeline has expanded and grown in diversity and complexity, the need for multiple therapeutic approaches and combination therapies has become increasingly apparent.

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. Much of the early research into SMA has focused on increasing SMN production, either by replacing SMN1 or by modulating SMN2, the low-functioning SMA “backup gene.”

Many of the programs in the pipeline rely on these SMN-based approaches, including gene therapy and antisense oligonucleotides. And as the pipeline shows, a number are already being tested in clinical trials. In future, these or other different approaches to increasing SMN levels could be used alone or together.

As research has progressed, it has also revealed that a number of systems, pathways and processes are affected in SMA, and there may be additional ways to treat SMA that work on these other areas. And perhaps most crucially, these other approaches could be used in combination with approaches that work on SMN levels, allowing us to attack SMA from all sides and giving us the best chance of a comprehensive, effective treatment. This is particularly important as we seek to develop treatments for all ages, stages and types of SMA.

In just the last six months since the last pipeline update in December, two new SMA programs have been added to the pipeline, both pursuing combination therapies. Both programs are funded by grants from Cure SMA.

In the next several weeks, we will also be announcing funding for existing programs on the SMA pipeline focused on improving the functioning of the SMN2 gene.

The results are impressive, but we still have more work to do. Only 10% of the drugs that make it to clinical trials will ultimately receive FDA approval, so we need to keep building and growing the pipeline. Beyond funding the pipeline, Cure SMA is committed to advocating for the patient voice in drug development to ensure regulators understand the SMA community and its needs.

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 toward approval—everyone has a role to play. Thank you to everyone for their hard work and dedication.

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