Dexpramipexole shows efficacy in ALS

Newly published data confirm that in a two-part phase 2 clinical trial, the experimental therapy dexpramipexole showed dose-related slowing of symptom progression and increased survival time in people with ALS.

Fight smart, not just hard. That’s the motto of Richard Bedlack, a world-renowned ALS research warrior who directs the ALS Clinic at Duke University, Durham, N.C. “Everyone I’ve ever met who is affected by ALS wants to roll up their sleeves and do something about it,” he says.

One of the first things that comes with a diagnosis of ALS is the determination of which “form” a person has: familial or sporadic.

Sometimes it appears obvious. If there’s a known history of the disease “running in the family,” then the person is said to have familial, or inherited, ALS. If the disease seems to have “come out of nowhere,” then it’s sporadic, or noninherited.

But in reality it’s far more complicated than that.

The Muscular Dystrophy Association has awarded a $278,850 grant to the ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass., to support testing of a mouse version of a compound called CTLA4-FC in the SOD1 research mouse model of amyotrophic lateral sclerosis (ALS).

The ALS Therapy Development Institute (ALS TDI), an MDA-supported, nonprofit biotechnology organization dedicated to developing effective treatments for amyotrophic lateral sclerosis (ALS), will collaborate with two major biotechnology companies to investigate a potential treatment that modulates the immune system.

In the first stage of an ongoing phase 2 clinical trial to test the experimental therapy CK-2017357 in amyotrophic lateral sclerosis (ALS), the drug was found to be safe and well-tolerated.

Participants who received the highest dose showed improved scores on tests that measure motor and breathing function, muscle strength and fatigue.

The Merfeld Family Foundation Scholarship Fund — a national scholarship to help children of parents with amyotrophic lateral sclerosis (ALS) pay for their college education — is now accepting applications for the fall 2012 term.

The deadline to apply for one of four $2,500 scholarships is Jan. 6.

The scholarship fund was established by Greg Merfeld, who received an ALS diagnosis in 2010 at age 49. Merfeld has lost more than 20 relatives to the disease, which runs in his family.

Newly published data confirm that in a two-part phase 2 clinical trial, the experimental therapy dexpramipexole showed dose-related slowing of symptom progression and increased survival time in people with amyotrophic lateral sclerosis (ALS).

The findings were published online Nov. 20, 2011, in Nature Medicine by principal investigator Merit Cudkowicz (director of the MDA/ALS Center at Massachusetts General Hospital in Boston and a member of MDA’s Medical Advisory Committee) and colleagues.

ALS experts talked about the state of research, and industry leaders discussed ways to improve the pace and efficiency of ALS drug development at the 2011 ALS TDI Leadership Summit, Nov. 4, 2011. The Summit, an annual conference hosted by the MDA-supported ALS Therapy Development Institute (ALS TDI), took place in Cambridge, Mass., and was broadcast live over the Internet.

Editor's note 3/19/12: This story has been updated to reflect that Neuralstem has begun testing its stem cells in the cervical (neck) region of the spinal cord.

Two human clinical trials to test therapies in amyotrophic lateral sclerosis (ALS) are moving forward.