Three announcements in a single week from separate research teams report progress against motor neuron disease (MND). These take us closer to being able to understand, detect, and treat this awful condition. Those ice buckets may have really made a difference.
For people with MND (also known as amyotrophic lateral sclerosis), the week's most exciting news is probably publication in the British Journal of Pharmacology that the anti-inflammatory drug PMX205 slows the progress of the inherited form of the disease in rats and mice. “In animal models, PMX205 made a visible difference to tremors, muscle strength and mobility, and if this is reflected in people, it could make a real difference to patients.” Dr John Lee of the University of Queensland said in a statement.
Since around 90 percent of people with MND have what is known as the “sporadic” version, success against inherited MND is not such big news, but Lee and his colleagues think the chances are high that the majority of MND patients will also benefit. “At the moment, the only drug available for patients prolongs survival by two to three months at most,” Lee said. He hopes PMX205 can do better.
It takes a long time for drugs to get from the laboratory to the marketplace, but PMX205 has the advantage of already being used for other anti-inflammatory purposes, so some of the safety testing is already done. Lee's team are collaborating with a private company to undertake pre-clinical safety trials, which will run in parallel with efforts to confirm their suspicion of its relevance for sporadic MND. If both go to plan, clinical trials will start in 2019.
PMX205 is unlikely to prove a magic bullet, however, which makes the discovery of a genetic marker for the most common form of MND important. In Science Translational Medicine, the Mayo Clinic's Dr Tania Gendron has announced that people with the mutations most commonly associated with MND accumulate a protein known as polyGP in their cerebrospinal fluid and blood cells.