Cure SMA Grants More Than $2 Million in New Research Funding

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by Charles Moore |

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CureSMAlogoCure SMA granted just over $2 million in new research funding in 2015-16. The non-profit organization dedicated to advocacy and treatment research funding for spinal muscular atrophy (SMA), reports that the SMA drug development spectrum has expanded dramatically over the past decade, with six of the 18 programs currently in the pipeline in clinical trials, several at Phase 2 and Phase 3 trials stages.

Cure SMA’s research funding strategy comes from its strong connections to patients, SMA families, and researchers. It enables support for a comprehensive research program that attacks SMA from all sides.

Designed to fund a wide range of projects in the drug pipeline, the program focuses on four general aspects of the drug development process:

1) Basic research into the bio-chemical dysfunctions that cause SMA is fundamental to developing effective treatments and tools to help facilitate SMA research.

2) Drug discovery converts what is learned about the causes and biology of SMA into a basic research phase about drug candidates.

3) Clinical and regulatory research facilitates and guides the conduct of clinical trials that pertain to all types and stages of SMA; leads to testing drug candidates for safety and effectiveness that could lead to regulatory approval.

This can be a long and difficult process, fraught with frequent failure due to the difficulty of predicting which drugs will successfully combine efficacy and safety. An estimated 80% to 90% of experimental drugs that reach the human clinical trials do not make it to U.S. Food and Drug Administration (FDA) approval.

4) The fourth leg of Cure SMA’s research strategy is clinical care research. Funding helps increase understanding of issues like breathing and nutrition that impact quality of life for people with SMA.

SMAmeetingCure SMA funding also supported the recent 20th Annual SMA Researcher Meeting and 2016 Annual SMA Conference. Both were held concurrently at The Disneyland Hotel in Anaheim, California. The meetings brought together leading SMA scientists, clinicians, and drug developers from around the world to discuss Cure SMA research strategies.

Click on the links below for details about recent Cure SMA grant fund recipients.

Basic Research

$140,000 Grant to Christine Beattie, PhD, Ohio State University

$140,000 Grant to Arthur Burghes, PhD, The Ohio State University

$90,000 Grant to Antoine Clry, PhD, ETH Zurich

$30,000 Grant to Jocelyn Cote, PhD, University of Ottawa

$70,000 Grant to Christine DiDonato, PhD, Lurie Children’s Hospital of Chicago b

$140,000 Grant to Megerditch Kiledjian, PhD, Rutgers

$140,000 Grant to Rashmi Kothary, MD, Ottawa Hospital Research Institute

$140,000 Grant to Charlotte Sumner, MD, Johns Hopkins University

Drug Discovery

$304,000 Grant to Livio Pellizzoni, PhD, at Columbia University

$300,000 Grant to Imago Pharmaceuticals/Charlotte Sumner, MD
*Two additional drug discovery grants totaling $100,000 will be announced later this month.

Clinical and Regulatory Research

$100,000 to help fund the launch of a collaborative industry consortium working on clinical trials, regulatory issues, and education

Clinical Care

$50,000 Grant to Tariq Rahman, PhD, Alfred I. DuPont Hospital for Children

SMA Researcher Meeting

$250,000 to help bring leading SMA researchers together from around the world


Cure SMA
2016 Annual SMA Conference