Clinical trial for AMN patients: test of an innovative drug replacing pioglitazone

Written on Friday 17 January 2014

Last june, we announced the launching of a clinical trial testing the effect of the pioglitazone for the treatment of adrenomyeloneuropathy (AMN). Since then, a new drug was identified by the scientific community and will be tested in France replacing pioglitazone.

Funded by ELA, the trial will be soon be available at the Bicetre and Pitie-Salpetriere hospitals in France, and possibly also in Germany and Spain. It will be coordinated by Pr. Patrick Aubourg, head of the reference center for Leukodystrophies at the Bicetre hospital.

Named “MD1003 in adrenomyeloneuropathy”, the trial paperwork will be shortly submitted to the French Medicine Agency (ANSM) and to the IRB. The ELA staff is actively involved in this study in order to get the legal authorizations as soon as possible. The trial should start during the first semester of 2014.



Adrenomyeloneuropathy is a rare genetic disease due to a defect in a gene called ABCD1. This gene is essential for the degradation of some fatty acids in the body. In AMN, the fatty acid accumulation in nerve cells induces harmful oxidative stress and impairments in the mitochondria, an important energy generator for cells. In the end, the nerve signal is altered and the patient progressively loses his balance and motor function.  No current therapy can halt the progress of adrenomyeloneuropathy.

The treatment

The MD1003 drug is developed by MedDay Pharmaceuticals founded by Drs. Frédéric Sedel and Guillaume Brion and located at the Brain and Spine Institute (ICM). MD1003 acts on the energetic metabolism of the brain. Contrary to pioglitazone, it bears no known risk for patients and shows very encouraging preliminary results on the motor function of patients dealing with a progressive form of multiple sclerosis. In the AMN trial,  a group of patients will be taking a placebo for a year. Concomitantly, the treated group of patients will be treated daily with the MD1003 drug. During the second year of the study, all the patients will be taking the drug. The conditions of the trial are being defined in order to satisfy the requirements for a market authorization and coverage by health insurances.

The trial

The clinical trial will evaluate the efficacy and tolerance of the MD1003 drug in AMN patients for 2 years. Efficacy means the possibility that this treatment slows or halts the motor deficits in the lower limbs that naturally occurs without treatment. It will be estimated by motor and biological tests as well as neuroimaging. Tolerance means the absence of adverse events during the treatment and will be assessed by clinical examinations (electrocardiogram) and biological tests (blood tests).

Once the legal authorizations are gathered, a full information about this new clinical trial will be published on our website.