Seeking a Cure for One of the Rarest Diseases: Progeria
More than 5,700 human conditions have now had their molecular causes defined—the majority of which are rare diseases that were molecularly characterized in the last 25 years.1 Yet, even today, approved therapies exist for only about 500 of these conditions.2 Clearly, an urgent need exists for therapeutic advances to help people suffering from rare diseases—a need fraught with many challenges.
The private sector's interest in developing molecularly-targeted therapies has been growing, but still remains quite limited for the rarest diseases that have very little market potential. Given the costs, it is difficult to embark upon such a therapeutic development effort from scratch; historically, the failure rate is very high and it has taken an estimated 14 years and several billion dollars to develop and gain approval for a therapy aimed at a molecular target.2 The National Institutes of Health is working to overcome these obstacles through a variety of innovative efforts at its National Center for Advancing Translational Sciences, with repurposing of compounds developed for other applications being one particularly attractive option.
- Received June 1, 2016.
- Accepted June 3, 2016.