Last week, members of Cure SMA’s Scientific Advisory Board (SAB) gathered in Chicago, meeting to advance Cure SMA’s research goals.

Our SAB is made up of highly respected SMA researchers, including experts in child neuromuscular disorders, SMA biology, SMN splicing, motor neurons, mouse and other animal models, and more.

The SAB focuses on identifying the most promising projects for a Cure SMA basic research grant, in order to enhance our understanding into the causes and biology of SMA, and on setting the agenda for our yearly SMA Researcher Meeting, which is the largest SMA research meeting in the world.

The Importance of Basic Research Grants

The SAB will be awarding up to $815,000 in new basic research grants. Basic research is part of Cure SMA’s comprehensive research funding strategy. Basic research investigates the causes and biology of SMA.

Some basic research projects will identify seed ideas that can serve as the basis for the development of new SMA drugs. This allows the SMA drug pipeline to continue to grow and diversify, giving us both a breadth and a depth of options in our quest for an effective SMA therapy.

Some basic research projects will develop tools that facilitate SMA research, including new outcome measures for SMA clinical trials. Outcome measures help determine whether a treatment being tested is successful, and must be developed for individuals of all different types, ages, and severities of SMA.

By expanding the breadth of the SMA pipeline through different approaches—such as those targeting the muscles and nerves—and by developing the tools to conduct well-designed trials in all SMA populations, basic research is key to developing treatments for all ages, stages, and types of SMA.

How Basic Research Grants are Awarded

Earlier this year, we opened up a new basic research request for proposals (RFP). A basic research RFP is an invitation for scientists to submit their best ideas for projects that address the causes and biology of SMA. They explain what they want to study, how they plan to study it, and why they think it will help those affected by SMA.

During this meeting, the SAB evaluated all proposals to see which projects are the most intriguing, which have a well constructed study plan, and which match up with the most pressing unanswered questions about SMA. The best proposals will then receive a basic research grant from Cure SMA.

Planning for the 2016 SMA Researcher Meeting

The SMA Researcher Meeting is the largest SMA focused conference worldwide. The 2016 SMA Researcher Meeting set a record with over 300 attendees, and we anticipate another full slate for our 2017 meeting at Disney World in Orlando, FL. It is held in conjunction with the Annual SMA Conference, so that researchers can interact with families affected by SMA.

The SMA Researcher Meeting promotes sharing of unpublished scientific data to accelerate the pace of research. It helps build research collaborations, integrate new researchers and drug companies into the community, and educate future SMA researchers. The SMA Researcher Meeting also brings researchers in contact with the families in our community, through sharing research updates or fun activities like the researcher relay race.

Each year, sessions are planned around some of the most important questions in SMA research. During last week’s meeting, the SAB reviewed the current landscape of SMA research, as well as our priorities for future research, and used that to develop the agenda for the 2017 SMA Researcher Meeting.

Our thanks to the SAB for their time, dedication, and expertise.