Drug Discovery

Drug discovery converts what we have learned about the causes and biology of SMA through basic research into new drug candidates that can be tested in clinical trials.

It can be a long and difficult process. Failures happen often, so it’s difficult to predict which drugs will be successful. An estimated 80% to 90% of experimental drugs starting human clinical trials never gain FDA approval. 

Our Approach to Drug Discovery

Our unique approach minimizes the most common challenges of drug discovery by building a diverse “pipeline” of drug candidates.

  • We invest in a number of projects at a time, which means that if a drug candidate fails, several others can take its place.
  • We fund projects that represent a variety of therapeutic approaches, attacking SMA from all sides. 
  • We provide seed funding for new projects. Traditionally, it has been difficult to get pharmaceutical companies to invest in research for rare diseases like SMA. By providing early-stage funding, we lower the risk and attract larger investments from industry and government as drug candidates move through the process.
  • We are unbiased as we evaluate all possible treatment opportunities, and we prioritize, select, and manage our drug discovery projects through a Translational Advisory Committee, comprised of industry experts.

Our Achievements in Drug Discovery

Our diverse and broad-based approach has led to important breakthroughs:

  • We are getting closer to an FDA-approved therapy for people with SMA. Fifteen years ago, we had just two potential drugs in the beginning stages of preclinical discovery. Today we have 18, including six now in clinical trials. One of those six, nusinersen, is now being submitted for FDA approval, meaning there could be a decision on the first-ever FDA-approved treatment for SMA in 2017.
  • We have identified four different therapeutic approaches that show promise in treating SMA: replacement or correction of the mutated gene in SMA called SMN1, modulation of backup gene in SMA called SMN2, neuroprotection, and muscle protection. 
  • Important biotech and pharmaceutical partners are committed to SMA research. Today, 14 companies are engaged in SMA research, including some of the biggest pharmaceutical companies in the world.

While there is real promise in the research landscape right now, which gives us great reason for hope, we know that much work remains to be done. We’re optimistic about the possibilities, but we’re also committed to helping families interpret all of the information and understand what they can realistically expect for their children.

Our Drug Discovery Funding

Cure SMA has funded over $20 million in drug discovery grants, converting basic research seed ideas into candidates that can be tested in the next stage of research: clinical trials.

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