The Cure SMA has organization awarded a research grant totaling $80,000 to support a project from Linda Lowes, PT, PhD, at Nationwide Children’s Hospital. The project is entitled “Development of An Innovative Outcome Measure to Define Disease Progression in SMA Type I for Use in the Home or Clinic” and aims to improve the progression of research on spinal muscular atrophy (SMA) into effective clinical trials by offering an accurate measuring method of the success of investigational treatments.
Lowes’ main purpose is to reduce the number of unsuccessful research projects by accurately measuring its probability in early stages of development. In order to do so, she is focused on developing a novel outcome measures tool that will be able to work with trial participants of all types, ages, and severity of SMA. With the new funding, Lowe is going to test the measuring tool in infants who suffer from SMA type 1.
“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,” explained Lowes in an interview on the Cure SMA website. “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.”
The project to be conducted in children is expected to help advance the measuring method, and it can be used by parents themselves in cases when children can not make regular visits to the study site. The movements of the participants will be recorded through the Microsoft Kinect program, and researchers will visit participants’ homes to instruct them on how to use the system once per month for a year.
Lowes has decided to implement the study on infants given the difficulties associated with clinical trials involving small children with SMA, as she believes that conducting the test at home can facilitate the process for both the participants and their families.
With a degree in physical therapy from Ohio State University and a PhD from Drexel University, Lowes is currently conducting her research at Nationwide Children’s Hospital in Columbus, Ohio with co-investigator Lindsay Alfano. “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,” she added.
The $80,000 grant awarded to Lowes is part of a basic research funding totaling $640,000 that Cure SMA is providing to researchers working on the biology and causes of the disease. Within the same program, the organization that supports research to find a treatment and an eventual cure for SMA has also awarded a $140,000 grant to the research project “mTOR and Protein Synthesis in SMA” currently being conducted by Mustafa Sahin, MD, PhD, at Boston Children’s Hospital.