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CEFTRIAXONE TRIAL TO BEGIN ENROLLING ALS PATIENTS Ceftriaxone is an intravenously administered antibiotic approved for meningitis and certain other infections, but it has additional properties that give it promise for ALS. The trials are the fruits of a drug screening program that began back in 2002, run by the National Institute of Neurological Disorders and Stroke (NINDS) in collaboration with the Packard Center, ALSA, and several other research organizations. Packard researchers did their share of screening the 1,040 drugs and substances already approved for a variety of other medical uses. Based on research by Packard scientists in this country and investigators in Europe who showed that high levels of the neurotransmitter glutamate and a parallel loss of glutamate transporter molecules are common to ALS patients, the scientists looked for drugs that could amend those conditions. Specifically, they developed a way to spot anything that would stimulate or increase the body’s natural transporter proteins that pull glutamate away from nerve cell synapses, where it accumulates. Using their assay, which tested the agents on both cell and spinal cord cultures, the Packard scientists came up with a short list of drugs. Highest on the list were ceftriaxone and structurally similar antibiotics, at dosages considered safe in their original uses. The Food and Drug Administration required additional testing to show that ceftriaxone would be safe when used for extended periods, as might be necessary in ALS treatment. After the success in lab screening, the drug then showed promise in a mouse model of ALS in experiments co-funded by the Packard Center, ALSA, NINDS and Project A.L.S. Animal testing was performed by the firm, PsychoGenics. The Northeast ALS Consortium, an organization supported, in part, by the Packard Center and the one most used collaboratively for its clinical studies, will conduct the multi-center trials. The initial drug screening effort that discovered possible benefits of ceftriaxone in ALS was funded jointly by ALSA, NINDS, the Hereditary Disease Foundation (HDF), and the Huntington's Disease Society of America (HDSA). Investigators from 26 laboratories took part in the initial 6-month, $1.3 million project, which tested 1,040 compounds using 29 different assays or tests. Approximately eight of the assays were related to ALS disease pathways. Ceftriaxone and related beta lactam antibiotics were the most active drug class in over half of the ALS assays. The principal investigator of the clinical trial, nationwide, is Merit Cudkowicz, M.D., from the Massachusetts General Hospital. Co-Principal investigators are Dr. Jeremy Shefner from SUNY at Upstate and Dr. Allitia Dibernardo at Massachusetts General Hospital. *This article is a modified version of one appearing on ALSA’s website.
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