ALS Research Roundup March-April 2010

by ALSN Staff on Mon, 2010-03-01 13:45
Article Highlights:

Updates on new and ongoing ALS research as of February 2010

This article reports news about clinical trials in ALS as of March 1, 2010, including: high-calorie diets, SOD1 blocker, altered communication, ceftraxione


Value of high-fat, high-calorie diets to be tested in ALS

An MDA-supported study comparing three tube-feeding formulas — one high-calorie, normal-fat; one high-calorie, high-fat; and one average-calorie, average-fat — in people with ALS is now being conducted at the five centers that make up the MDA/ALS Clinical Research Network.

The trial will test the hypothesis, suggested by observational studies and studies in mice, that a high-fat and/or high-calorie diet can increase survival in ALS. Participants must be tolerating tube feedings and must meet other study criteria.

The centers that make up the MDA/ALS Clinical Research Network are California Pacific Medical Center, San Francisco; Columbia University, New York; Emory University, Atlanta; Massachusetts General, Boston; and Methodist Hospital, Houston.

For more information, contact Katy Shaver at Massachusetts General Hospital at (617) 643-7434 or; or see the Clinical Trials section of the MDA Web site.

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Trial of SOD1 blocker now open

An MDA-supported, phase 1 clinical trial of the experimental drug ISIS-SOD1-Rx in people with the SOD1 familial (inherited) form of ALS opened in February 2010.

This study is being performed at Massachusetts General Hospital in Boston and five other U.S. sites. Timothy Miller at Washington University has received MDA support to work with Isis Pharmaceuticals of Carlsbad, Calif., to develop ISIS-SOD1-Rx.

The trial will test the safety and tolerability of this “antisense” compound, which is designed to block production of the SOD1 protein in people who have developed ALS because of inherited mutations in the SOD1 gene. Such mutations result in ALS in approximately 1 percent to 3 percent of cases.

The investigators will infuse ISIS-SOD1-Rx into the fluid that surrounds the brain and spinal cord, a delivery method that targets the cells that produce the toxic SOD1 protein.

Details of genetic testing and other study requirements will be discussed with potential study participants. Contact Pat Andres at (617) 724-8995 or (877) 458-0631 (toll-free) or

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Researcher studying altered communication in ALS, other diseases

Speech-language pathologist Carolyn Baylor at the University of Washington in Seattle is seeking help from people with ALS and other diseases that can affect speech and communication in developing a questionnaire for health care providers to use. Participants must use speech as their primary communication method, although they may use writing or augmentative devices at times.

“Our goal is that this questionnaire will be one of several tools that clinicians can use to help people with communication disorders improve their participation in activities they want to do,” Baylor explains on her Web site.

Glutamate is a necessary carrier of signals between nerve cells. However, it has to be cleared away from cells regularly to avoid toxic buildup. Ceftriaxone may aid in that process.

Participation involves about an hour of filling out questionnaires at home, online or on paper. Contact Carolyn Baylor at (206) 221-3563 or

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Ceftriaxone study remains open

A 540-person trial of intravenous ceftriaxone in people with ALS remains open at locations throughout the United States and Canada.

Ceftriaxone is an antibiotic that may increase the levels of a protein that helps clear potentially toxic glutamate from the areas surrounding nerve cells. Laboratory studies suggest the drug may help protect motor neurons, the main cells damaged in ALS. In this study, ceftriaxone is being administered intravenously.

Contact Sarah Titus at (617) 726-1398 or

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ALSN Staff
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