Cure SMA Awards $80,000 Grant to Linda Lowes, PT, PhD, of Nationwide Children’s Hospital

ELK GROVE VILLAGE, Ill. - Feb. 18, 2015 - PRLog -- Cure SMA has awarded an $80,000 research grant to Linda Lowes, PT, PhD, at Nationwide Children’s Hospital for her project, “Development of An Innovative Outcome Measure to Define Disease Progression in SMA Type I for Use in the Home or Clinic.”

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. Otherwise, well-designed trials become difficult to conduct in all SMA populations.

Dr. Lowes’ project will test a new way of measuring results in infants with SMA type I. This measurement could be used in the home by parents, if the child isn’t able to make regular visits to a clinic.

This grant to Dr. Lowes is part of $640,000 in new basic research funding by Cure SMA. 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. This funding is also used to develop tools that facilitate SMA research.

About Linda Lowes

Who are you?

I received my physical therapy degree from The Ohio State University and my PhD from Drexel University. My co-Investigator, Lindsay Alfano and I are both currently researchers at Nationwide Children’s Hospital in Columbus Ohio.

How did you first become involved with SMA research?

I work with an amazing group of physicians and researchers in the Center for Gene Therapy at Nationwide Children’s Hospital. I provide the outcome measures for the Centers current gene therapy study in SMA type I.

What is your current role in SMA research?

Matching the correct outcome measure to the changes you expect to see during a clinical trial is crucial or you may reject a potential treatment because you weren’t quantifying the correct changes. SMA is such a unique disease that we wanted to develop an assessment tool specifically for measuring abilities in infants, children and adults with spinal muscular atrophy. We are working on a software suite that measures movement abilities in infants, people who can stand and those who are in a wheelchair.

What do you hope to learn from this research project?

This project will develop an outcome measure to help advance clinical trials in infants with SMA type I that can be used in the clinic or the patient’s home.

How will this project work?

The project team will utilize Microsoft Kinect to record the infant’s movement. Investigators will visit the homes to instruct families on how to use our system, teaching them to record the baby’s movement, once a month for 12 months.

What is the significance of your study?

It is difficult for fragile infants with SMA to participate in clinical trials. At home testing would ease the burden on the infant and family.

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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.