ALS - Eric Huang, M.D., Ph.D.

MDA awarded a grant totaling $412,500 over a period of three years to Eric Huang, professor of neuropathology at the University of California in San Francisco. The funds will help support Huang’s work to create cellular and mouse models of amyotrophic lateral sclerosis (ALS) that is caused by mutations in the fused in sarcoma (FUS) gene.

Genetic data have shown that mutations in the FUS gene can be identified in more than 5 percent of people with familial (inherited) ALS.

ALS - Kenneth Hensley, Ph.D.

MDA awarded a research grant totaling $328,153 over a period of three years to Kenneth Hensley, associate professor in the departments of pathology and neuroscience, and research director in the department of pathology at the University of Toledo Medical Center in Ohio. The funds will help support Hensley’s study of a potential new target and treatment strategy for amyotrophic lateral sclerosis (ALS).

ALS - Brian Kraemer, Ph.D.

MDA has awarded a research grant totaling $316,557 over a period of three years to Brian Kraemer, a research biologist in the Geriatrics Research Education and Clinical Center at the Veterans Affairs Puget Sound Health Care System in Seattle.

The funds will help support Kraemer's study of the connection between mutated TDP43 protein and motor neuron (nerve cell) degeneration in amyotrophic lateral sclerosis (ALS).

Indications of Safety, Efficacy in BrainStorm Stem Cell Trial

Preliminary data reported Jan. 17, 2012, by BrainStorm Cell Therapeutics demonstrate that the biotechnology company's experimental NurOwn stem cell technology has not caused any significant adverse side effects in a phase 1-2 clinical trial in amyotrophic lateral sclerosis (ALS). In addition, clinical follow-up of trial participants indicates that the treatment appears to have improved breathing, swallowing and muscle strength.

ALS Biomarkers Study Seeks 250 Participants

The Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) is actively seeking participants for a study designed to identify and validate biomarkers in amyotrophic lateral sclerosis (ALS).

MDA Funds Development of Utrophin 'Magnet' for DMD/BMD

 

Physicians Push for Proof of Diaphragm Pacer Benefit

The U.S. Food and Drug Administration approved the NeuRx Diaphragm Pacing System as a humanitarian use device (HUD), a designation given to medical devices intended for use in rare diseases. For HUD approval, the FDA requires sufficient evidence the device does not pose “an unreasonable or significant risk of illness or injury, and that the probable benefit to health outweighs the risk of injury or illness from its use.”

A Closer Look: Diaphragm Pacing System

Pat Dwyer of Kenmore, Wash., uses three different devices to make sure he’s getting enough air. A “sip-and-puff” ventilator supplements his breathing efforts during the day; bilevel positive airway pressure (BiPAP) helps him breathe at night; and the NeuRx Diaphragm Pacing System, or DPS, stimulates his diaphragm (the primary breathing muscle) 23 out of every 24 hours.

Why Does It Take So Long To Go from Mouse to Man?

John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported, where a treatment appears to cure one neuromuscular disease or another in a mouse — yet there are few treatments available today for people with any of these diseases, and only a few treatments in human clinical trials. Why does it take so long?

Induced Stem Cells Require Caution

Stem cells have been much in the news lately, including for neuromuscular diseases.

One extremely promising approach has been to create stem cells from the adult (differentiated) cells of humans or animals, and then reprogram them back to a stemlike state, after which they can be redifferentiated into a desired cell type, such as muscle or nerve cells. Stem cells made from differentiated cells are called induced pluripotent stem cells, or iPSCs.

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