Speeding the Course of Clinical Trials

Speeding the course of clinical trials for Duchenne muscular dystrophy (DMD) and ALS (amyotrophic lateral sclerosis) is one of the main goals of MDA’s new clinical trial network – and the work already has begun.

In DMD ...

Investigators at five elite U.S. centers are working together to support trials and studies and collaborate on DMD-related projects. The centers are located at:

  • Washington University in St. Louis;
  • Boston Children's Hospital;
  • Nationwide Children's Hospital in Columbus, Ohio;
  • University of California Davis in Sacramento, Calif.; and
  • University of Minnesota in Minneapolis,

The combined efforts of network researchers should result in faster testing of new treatments and the development of a standardized set of measures that investigators will be use to assess how treatments work in trials.

(In addition to the five clinics focusing on DMD, five other clinics in the MDA Clinical Research Network are focused on ALS, also called amyotrophic lateral sclerosis or Lou Gehrig’s disease.)

New ways to measure DMD progress means more trial participants

US map
Clinics in the DMD clinical trial network are shown in yellow, and the ALS clinical network is in dark blue. Boston is home to two clinics participating in the network.

The DMD network research teams have identified several priority projects, one of which involves identifying measurements of disease progression in very young boys (ages 1 month through 5 years) and also in boys and young men who are unable to walk.

Pediatric neurologist Anne Connolly, principal investigator at the network center at Washington University, says having a standardized set of measurements for use with these groups will enable a much larger percentage of individuals to participate in research studies.

Most trials are geared toward individuals who can walk, and who can cooperate, but this often excludes many who are young, unable to walk, or who have cognitive impairment.

"Those who follow these men and children recognize that there are variables, other than the ability to walk, that can be tracked," Connolly explains. "The hope is that by developing these measurable outcomes, researchers may be able to demonstrate the effectiveness of treatments through, for example, measurements that show improved hand or arm function, even in trial participants who can't walk."

Another project identified by researchers involves finding the best methods to assess cardiac function in the DMD-affected heart, important because heart-muscle deterioration, or cardiomyopathy, is a leading cause of death in DMD.

In ALS ...

Investigators from the five centers that comprise the ALS-focused aspect of MDA's new Clinical Research Network also have begun selecting and prioritizing projects.

The MDA/ALS centers in the clinical trial network are:

  • California Pacific Medical Center, San Francisco;
  • Columbia University, New York;
  • Emory University, Atlanta;
  • Massachusetts General, Boston; and
  • Methodist Hospital, Houston.

Looking for connections

Among the identified projects is a study of hyperlipidemia (raised levels of fatty molecules, or "lipids," in the blood) as a predictor of disease progression.

Results from a French study, published in March 2008 in the journal Neurology, suggested that elevated levels of lipids in the blood, known to be risk factors for cardiovascular disease, actually may be a good thing in ALS.

Other ALS projects identified by the investigators as priorities include the development of meaningful measures for use in clinical trials, and development of a system to select, design and perform studies on treatments in phase 2 clinical trials.

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