Dr. Ethan Perlstein Discusses Drug Repurposing for Rare Diseases
“Drug repurposing for rare diseases requires building animal models to screen for activity. ”
One of our goals for Raising Rare is to introduce some of the scientists who are working on finding treatments for Raghav. Another goal is to educate our listeners about finding and developing treatments. In this episode Sanath and I spend some time with Dr. Ethan Perlstein talking about various animal models that can be used to screen for active drugs.
Dr. Perlstein is the CEO of Perlara, a Public Benefit Corporation that is dedicated to helping families seek treatments for rare genetic diseases. He is also one of the original members of the CureGPX4 Scientific Team.
One of the key strategies CureGPX4 is using is called drug repurposing. The animal models are one way to screen existing approved drugs to see if they may have a beneficial effect. However, it is not as easy as it sounds. Developing these animal models requires time and money. Selecting which model to invest limited funds in first is an extremely important decision. Dr. Perlstein shares his thoughts on how to approach it.
We hope you enjoy one of our more scientifically focused episodes.
NOTE: We are continuing our discussion with Ethan Perlstein on the Improbable Developments podcast. There we will talk about his career and what drew him towards his work with animal models.