New Hope for Treating Rare Genetic Disease

A new public-private partnership may reportedly provide a mechanism for developing drugs to treat rare genetic diseases.

Such drugs are relatively rare because of their low return on investment. But the European Rare Diseases Therapeutic Initiative might remedy the situation.

In an article in the open access international medical journal PLoS Medicine, Alain Fischer, chief of the department of pediatric immunology at the Necker University Hospital in Paris and colleagues describe how ERDITI brings drug companies and academic researchers together to find new treatments for rare diseases.

The thousands of compounds that have been developed by pharmaceutical companies for more common diseases, but that were abandoned or failed to achieve registration for several reasons — such as biopharmaceutical properties, toxicity, lack of efficacy, or strategic reasons — represent a treasure worth exploiting, said the authors.

The idea behind ERDITI is that drug companies give the abandoned compounds to academic researchers who then investigate whether the compounds might be effective for treating rare diseases.

Companies benefit from the relationship with academia in many ways, including the fact the research might lead to the discovery of drugs for the treatment of more common diseases, which could be highly profitable to industry.