Learn about ALS
Research
A number of clinical trials are underway globally for ALS; a comprehensive listing
of trials in the US can be found at ClinicalTrials.gov.
KNS-760704 is under clinical investigation in ALS patients. It is hoped that the
drug will have a neuroprotective effect. It is one enantiomer of pramipexole, which
is approved for the treatment of Parkinson's disease and restless legs syndrome.
The single-enantiomer preparation is essentially inactive at dopamine receptors,
is not dose limited by the potent dopaminergic properties of pramipexole. Results
of a Phase II clinical trial conducted by Knopp Neurosciences and involving 102
patients were reported in 2010; the trial found a dose-dependent slowing in loss
of function.
Olesoxime (TRO19622) is being tested in a phase 3 clinical study, as part of the
MitoTarget Project. The molecule has a cholesterol-like structure and displays strong
neuroprotective properties, and it should be as effective as a cocktail of three
neurotrophic factors in keeping motor neurons alive in culture. The ongoing clinical
study aims to test efficacy, safety, tolerability and plasma levels in patients
with ALS, to see whether a single daily dose of two capsules – under certain circumstances
– can improve survival and symptoms of ALS patients. The trial started in May 2009,
all the patients are recruited and results are expected in the last quarter of 2011.
The study is taking place in France, Belgium, Germany, the UK and Spain.
The new discovery of RNAi has some promise in treating ALS. In recent studies, RNAi
has been used in lab rats to shut off specific genes that lead to ALS. Cytrx Corporation
has sponsored ALS research using RNAi gene silencing technology targeted at the
mutant SOD1 gene. Cytrx's orally-administered drug Arimoclomol is currently in clinical
evaluation as a therapeutic treatment for ALS.
Insulin-like growth factor 1 has also been studied as treatment for ALS. Cephalon
and Chiron conducted two pivotal clinical studies of IGF-1 for ALS, and although
one study demonstrated efficacy, the second was equivocal, and the product has never
been approved by the FDA. In January 2007, the Italian Ministry of Health has requested
INSMED corporation's drug, IPLEX, which is a recombinant IGF-1 with Binding Protein
3(IGF1BP3) to be used in a clinical trial for ALS patients in Italy.
Ultra-high-dose vitamin B12 analog Methylcobalamin by intravenous injection, which
has a protective effect on cultured cortical neurons against glutamate-induced cytotoxicity,
is being studied in Japan. Ultra-high-dose methylcobalamin slows down the progressive
reduction of the CMAP (compound muscle action potential) amplitudes in ALS.
