Cure SMA Announces New $140,000 Basic Research Grant to Chad Heatwole, MD, at the University of Rochester

By: PRLog
PRLog - March 25, 2015 - ELK GROVE VILLAGE, Ill. -- 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." This grant is supported by funding from The Spinal Muscular Atrophy Research Team (www.smarthope.com), Buffalo, NY.

As more spinal muscular atrophy (SMA) 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, Cure SMA's current round of funding includes a previously announced grant to Dr. Linda Lowes, who is studying outcome measures in infants with SMA type I.

The goal in supporting these different projects is to ensure that well-designed trials can be conducted in all SMA populations.

This grant to Dr. Heatwole is part of $640,000 in new basic research funding by Cure SMA. In addition to this grant and the $80,000 grant to Dr. Lowes, past grant announcements include $140,000 to Dr. Sara Custer at Indiana University, $140,000 to Dr. Francesco Lotti at Columbia University, and $140,000 to Dr. Mustafa Sahin at Boston Children’s Hospital.

Basic research is the first step in Cure SMA's comprehensive research model. Basic research investigates the biology and cause of SMA, in order to identify the most effective strategies for drug discovery and to develop tools that facilitate SMA research.

About 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:

1. Utilize a large cross-sectional study to identify those symptoms that are most important to SMA patients;
2. Develop and validate a reliable, responsive, and patient-meaningful patient reported outcome measure for SMA patients; and
3. 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.

###

About Cure SMA

Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)—a disease that takes away a person’s ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.

Since 1984, we’ve directed and invested in comprehensive research that has shaped the scientific community’s understanding of SMA. We are currently on the verge of breakthroughs in treatment that will strengthen our children’s bodies, extend life, and lead to a cure.

We have deep expertise in every aspect of SMA—from the day-to-day realities to the nuances of care options—and until we have a cure, we’ll do everything we can to support children and families affected by the disease.

Learn more about how you can help us reach a treatment and cure at www.cureSMA.org.

Read Full Story - Cure SMA Announces New $140,000 Basic Research Grant to Chad Heatwole, MD, at the University of Rochester | More news from this source

Press release distribution by PRLog

Data & News supplied by www.cloudquote.io
Stock quotes supplied by Barchart
Quotes delayed at least 20 minutes.
By accessing this page, you agree to the following
Privacy Policy and Terms and Conditions.