Pelizeus-Merzbacher’s disease (PMD) is an inherited disease affecting the central nervous system characterized by physical and mental impairment. In the majority of cases, PMD is due to an increase in the expression of the PLP1 gene (gene encoding Protein Proteolipid 1 or lipophilin, the primary constituent of myelin). Currently, there is no cure for patients with PMD.

As part of an experimental therapeutic approach, we were able to reduce the gene expression of PLP1. We have been able to improve the course of the disease by administering an experimental progesterone receptor antagonist in mice mimicking the most common form of PMD.

In order to facilitate the application of our results to patients with PMD, we will attempt to answer the following questions in PMD mice:

Do progesterone antagonists currently authorised in other indications reduce PLP1 gene expression?

How can we optimise the treatment to obtain the best possible therapeutic effects?

What is the long-term impact of treatment acting on the progesterone receptor?

Can we identify other potential classes of drugs that reduce the expression ofPLP1?