Amyotrophic Lateral Sclerosis (ALS)

UPDATE: FDA Approves Testing Multiple Sclerosis Drug in ALS

The U.S. Food and Drug Administration (FDA) has given the go-ahead to the nonprofit biotech ALS Therapy Development Institute (ALS TDI) to conduct a clinical trial of TDI132  — also known as fingolimod, or the brand name Gilenya— in people withALS (amyotrophic lateral sclerosis).

National ALS Biorepository Pilot Study Launched

ALS Briefs: Stem Cells, Risk Factors, Emotion Processing Abnormalities

This roundup of recent amyotrophic lateral sclerosis (ALS) research and clinical trials news includes:

MDA Study Reveals ‘Cost of Illness’ for ALS, DMD, MMD

"We now have numbers to point to," said Annie Kennedy, MDA's senior vice president of advocacy. "It's not just a back-of-the-envelope calculation anymore. It's real data."

Trial to Evaluate the Hormone Drug Acthar in ALS

A 40-person phase 2 clinical trial to test the safety and tolerability of Questcor Pharmaceuticals’ H.P. Acthar Gel (Acthar) in people with amyotrophic lateral sclerosis (ALS) has opened at a trial site in Phoenix, Ariz.

ALS — James Shorter, Ph.D.

James Shorter, associate professor of biochemistry and biophysics at the University of Pennsylvania in Philadelphia, was awarded an MDA research grant totaling $300,000 over a period of three years to develop an experimental approach targeted at protein aggregates in amyotrophic lateral sclerosis (ALS).

SBMA/ALS — Constanza Cortes, Ph.D.

Constanza Cortes, a postdoctoral researcher at the University of California, San Diego, was awarded an MDA development grant totaling $177,410 over a period of three years to study the role of a cell recycling system in spinal-bulbar muscular atrophy (SBMA or Kennedy disease) and amyotrophic lateral sclerosis (ALS).

'Sugar' Molecule Delays Disease Progression in ALS Mice

Treatment with a small molecule called trehalose was associated with slower disease progression and longer life span in mice with a disease mimicking amyotrophic lateral sclerosis (ALS), an MDA-supported research team has reported.

Trehalose is a compound composed of two glucose (sugar) molecules.

ALS — NeuRx Diaphragm Pacing System (DPS)

The Muscular Dystrophy Association has committed $750,000 to help support a phase 2 clinical trial assessing the ability of the NeuRx Diaphragm Pacing System (DPS) to improve respiratory function and quality of life in people with amyotrophic lateral sclerosis (ALS).

Multicenter Trial to Test Safety of Mexiletine in Sporadic ALS

The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking participants for a phase 2 clinical trial that will evaluate the safety and tolerability of mexiletine in people with the sporadic form of amyotrophic lateral sclerosis (ALS).

Approximately 90 to 95 percent of ALS is sporadic, in which there is no known family history of the disease.