Also known as amyotrophic lateral sclerosis (ALS), MND is a fatal, rapidly progressive disease that affects the brain and spinal cord. It attacks the nerves that control movement; people can still think and feel, but their muscles refuse to work. It can leave people locked in a failing body, unable to move, talk, and eventually, breathe. Every year in Europe some 13,000 people die with MND, 30% of these within a year, and more than 50% within 2 years of diagnosis. ALS has a dramatic impact upon individuals and families and care is largely palliative.
Despite progress in understanding ALS pathogenesis, promises of new therapies remain unfulfilled.
Our pioneering work on riluzole showed that it is possible to modify ALS progression but all subsequent trials of potential treatments aimed at slowing down the disease have failed. Thus, drug development in ALS, including trial design, patient selection, and outcome measures need to be re-thought if we are to break the current impasse. MIROCALS will overcome this therapeutic impasse by validating a new phase II trial design testing the efficacy and safety of low-dose IL-2 in ALS patients.