University of Minnesota tackles Friedreich’s ataxia, a progressive neurological condition

Wednesday, July 16, 2014

The University of Minnesota is pushing research efforts forward against the progressive neurological condition, Friedreich’s ataxia (FA) after receiving $150,000 in new funding from the Friedreich’s Ataxia Research Alliance (FARA), GoFAR, and Ataxia U.K.

These foundations launched an international call for projectsto develop new non-invasive approaches for evaluating how neurons in the brain and spinal cord are affected in people with FA, especially in the early stages of the disease. Another project was also selected and funded at the University of Newcastle, U.K. 

The University of Minnesota’s Center for Magnetic Resonance Research (CMRR) was selected because it is a world leader in magnetic resonance imaging (MRI) research in addition to its strong ataxia research program, and presence of the Ataxia Center and Bob Allison Ataxia Research Center (BAARC).

The CMRR research team is looking to identify biomarkers of disease progression in FA, and their goal is to develop new imaging tools to monitor changes in the brain and the spinal cord of patients over time.

Led by Christophe Lenglet, Ph.D., assistant professor of radiology, CMRR, and Pierre-Gilles Henry, Ph.D., assistant professor of radiology, CMRR, the team will use a novel approach to developing sensitive measures of brain and spinal cord degeneration. They plan to use two techniques: diffusion MRI and magnetic resonance spectroscopy (MRS), which provide complementary information on structural and neurochemical changes in the brain and spinal cord.

“Researchers now have a better understanding of the disease, and we will soon be ready to test potential therapeutic strategies in clinical trials. One significant hurdle is that such clinical trials would require large numbers of patients over many years,” said Henry. “We believe MRI and MRS could play an important role by detecting if a potential treatment is working more quickly and on smaller group of patients.”

The research team already has some data using these techniques from patients with FA and they now need additional information over a longer period of time to assess changes over a one year period. They also wish to extend this study by including people at an earlier stage to see if any changes measured using these techniques can be seen earlier in the course of the condition.

"We hope that these novel neuroimaging techniques will help improve our understanding of the disease, and eventually assess the efficacy of potential treatments in clinical trials,” said Lenglet.

Due to the relative rarity of FA (which affects 1-2 in 50,000 people), the study is enrolling patients from all over the U.S.

In a  joint statement Jen Farmer (FARA), Julie Greenfield (Ataxia U.K.) and Mina Ruggeri (GoFAR) said, “We are delighted to be working together in this important initiative that we hope will lead to new disease relevant ways of measuring the effect of treatments in Friedreich’s ataxia trials. Having consulted with researchers in academia, industry and regulators it became clear that there was a gap that needed some attention. We were pleased with the response the call generated and are grateful to the researchers’ constant efforts in developing new research tools for the Friedreich’s ataxia community, taking us one step closer to developing a much needed treatment for this currently incurable condition.”