Cure SMA has awarded a $140,000 research grant to Chad Heatwole, MD, at the University of Rochester for his project, “Development of a Clinically Relevant Outcome Measure for SMA Therapeutic Trials.”
As more spinal muscular atrophy drug programs progress to clinical trials, it becomes critically important to develop ways of accurately measuring whether or not the treatment is successful. These “outcome measures” must be developed for individuals of all different types, ages, and severity of SMA.
Dr. Heatwole’s project will focus on developing a reliable method for adults with SMA to report on clinical trial outcomes that are important to them. Along with this grant to Dr. Heatwole, our current round of funding includes a previously announced grant to Dr. Linda Lowes, who is studying outcome measures in infants with SMA type I.
Our goal in supporting these different projects is to ensure that well-designed trials can be conducted in all SMA populations.
This project is supported by funding from The Spinal Muscular Atrophy Research Team (SMART), Buffalo, NY.
Meet Chad Heatwole
Who are you?
I am a member of the neuromuscular faculty at the University of Rochester Medical Center with 14 years of clinical experience treating and managing patients with neuromuscular disease. I currently lead an international network for the design and creation of disease-specific outcome measures and am the CEO and founder of The Neuromuscular Institute of Quality-of-Life Studies and Outcome Measure Development. My work has led to the development of multiple patient relevant outcome measures for use in drug labeling trials.
How did you first become involved with SMA research?
As a neurologist with subspecialty training in neuromuscular medicine I care for a variety of patient’s with neuromuscular disease; some have spinal muscular atrophy. There is a need for therapeutic advances in SMA. The development of the infrastructure to better study and evaluate SMA promising therapeutics is essential in preparation for successful clinical trials in SMA. There is a clear need to develop quality outcome measures that are reliable, valid, relevant, responsive to clinical change, and capable of capturing a patient’s insight on their health status. This need, and more importantly the desire to better provide for my SMA patients, has led me to become involved in SMA research.
What is your current role in SMA research?
While my prior research has extensively focused on other neuromuscular disorders, this will be our first research initiative specifically focused on SMA. I look forward to becoming a part of the SMA research community and utilizing the experience of our research team to develop useful disease-specific instruments for the benefit of SMA patients.
What do you hope to learn from this research project?
The objective of this project is to develop, validate, and utilize a reliable, responsive, and patient-meaningful disease-specific patient reported outcome measure for SMA clinical trials. Patient-reported outcomes are typically required by the FDA in pivotal drug trials.
How will this project work?
This research will:
- Utilize a large cross-sectional study to identify those symptoms that are most important to SMA patients;
- Develop and validate a reliable, responsive, and patient-meaningful patient reported outcome measure for SMA patients; and
- Implement this instrument in SMA clinical trials and in clinic settings as a means to track patient-meaningful responses to treatment.
What is the significance of your study?
At the completion of our work the SMA research community will have a valid outcome measure to aid in therapeutic assessment that will encourage therapeutic development for adult SMA patients.
Basic Research Funding
This grant to Dr. Heatwole is part of $640,000 in new basic research funding that we’ve recently announced. Please see below for links to other recent announcements.
Basic research is the first step in our comprehensive research model. We fund basic research to investigate the biology and cause of SMA, in order to identify the most effective strategies for drug discovery. We also use this funding to develop tools that facilitate SMA research.
Dr. Sara Custer
Dr. Francesco Lotti
Dr. Mustafa Sahin
Dr. Linda Lowes