Modeling Research Lays the Foundation for Continued Advancements in Alagille Syndrome

Kameswaran Surendran, PhD, Associate Professor of Basic Biomedical Sciences at the University of South Dakota, discusses exciting endeavors in Alagille syndrome clinical trials.

Question:

Are there any clinical trials or studies in this area that you have your eye on and are interested in, related to Alagille syndrome?

Kameswaran Surendran, PhD:

The primary organ that Alagille syndrome affects is the liver, and there are quite a lot of good researchers who are studying the abnormalities in the liver, and they’ve created various model organisms that model the liver defects. Some of them have done screens and are actually testing ways of boosting the Notch signaling.

These are already basic studies that are ongoing, and some of them are quite promising that they might be able to slow down or actually even regenerate the liver. It’s unclear, at this point, whether those will actually translate or how well they will translate in humans, but those are very promising studies that I think we’re all excited about and sort of give a sense of hope for improving the lives of those with Alagille syndrome.

Rare Disease 360® is the Official Media Partner and Official Publication of The Alagille Syndrome Alliance (Alagille.org).