Close Success Stories Treatment for X-linked adrenoleukodystrophy (X-ALD) www.idibell.cat

Project leader

Aurora Pujol

Start year

2017

Participating organizations

IDIBELL, SOM Biotech

Project aims

Providing a treatment for the hereditary neurometabolic disease X-linked adrenoleukodystrophy (X-ALD) based on repositioning commercial drugs indicated for other diseases. X-ALD is a rare disease that currently has no treatment, with an incidence rate of 1:17,000 in newborns, although poor diagnosis means the real number may actually be higher. Patients with X-ALD, which is more common in men than women, undergo progressive destruction of myelin, the substance that insulates nerves in the brain and bone marrow.

Collaboration framework

Dr. Pujol's research group (IDIBELL) has contributed satisfactory results from experiments treating X-ALD in animal models. These have been the basis for 4 patent applications. On the other hand, Catalan company SOM Biotech is collaboration to co-develop some of these drugs to take them to market as a therapy.

Most significant challenges

One of the most important challenges this project has had to face is the pharmaceutical industry's lack of interest in this type of diseases. The fact that it is a rare disease means it takes a lot of investment in R&D to market a treatment, while the return on that investment is quite low.

Results

IDIBELL has licensed three of the four patents on repositioned drugs to treat X-ALD to SOM Biotech. They have signed a collaboration agreement to fund the project and a first-view contract, through which SOM Biotech will have first priority in seeing any new technology developed by Dr. Pujol's research group for this disease. Two of the three drugs are now about to move into phase II/III clinical trials and, if completed successfully, may hit the market in 2 to 3 years.