Ceftriaxone is best known as an antibiotic that treats Lyme disease but has also shown efficacy in models of ALS. Ceftriaxone- a semi-synthetic, third generation cephalosporin antibiotic- may block glutamate toxicity by increasing the levels of a protein that reduces excess glutamate. A small trial of Ceftriaxone was recently initiated to assess any side-effects that may appear with long term administration in people with ALS. The Center of Hope is participating in this trial to look at the effect of Ceftriaxone on survival in ALS. This study is coordinated by Massachusetts General Hospital and is funded by NIH.

Status: Currently enrolling.

The purpose of this research study is to test an experimental drug call dexpramipexole. While the precise mechanism of dexpramipexole is currently unknown, it appears to increase the level of functioning of mitochondrial cells, which in turn may protect the nerve cells. The results of the Phase 2 safety and tolerability study of this drug when it was under the name KNS-760704 by Knopp, showed it was safe and well tolerated for up to nine months. A large, long-term Phase 3 study is beginning in Spring 2011, under the new sponsor, Biogen Idec Inc., and the MDA/ALS Center of Hope is participating in this phase as well.

Xaliproden is a small molecule with neurotrophin-like effects. This study was a multi-centered Phase III trial sponsored by Sanofi.

Avanir Pharmaceuticals sponsored a 12 week study of an experimental drug, Zenvia, in order to assess its safety, tolerability and efficacy in the treatment of Pseudobulbar Affect (PBA) in patients with ALS. Zenvia is a novel combination product comprised of two approved drugs, dextromethorophan and quinidine. Dextromethorophan is an NMDA-selective receptor antagonist which will suppress the release of excitatory neurotransmitters. Quinidine blocks the metabolism of dextromethorophan in the body, thus maintaining sufficient dextromethorophan plasma levels.

Status: Received FDA approved October 29th, 2010.

This study is designed to study the safety, tolerability and feasibility of a high fat/high calorie diet versus high calorie diet versus normal diet.  Secondary outcome measures include lipid levels, weight, body mass, body composition and preliminary effects on disease progression. We will also test biomarkers of body composition, and lipid metabolism before and during diet intervention. 

Status: Open for enrollment.

This study examined whether the experimental drug called CK-2017357 can improve muscle strength and fatigue in ALS. CK-2017357 is an investigational drug being developed for the potential treatment of skeletal muscle function in conditions associated with muscular weakness. It improved muscle function in SOD1 (G93A) mice –the animal model of ALS. This research study compared two different single doses of CK-2017357 to placebo and demonstrated promising results.

Status: Actively enrolling Phase 2 Part B.