Rare Disease Report

Emerging FCS Treatments and Research

MAY 04, 2018
Alan S. Brown
Alan S. Brown, MD, FNLA, FACC, FAHA, is a Cardiologist at Advocate Lutheran General Hospital and the President-Elect of the National Lipid Association (NLA). While at the NLA’s 2018 Scientific Sessions in Las Vegas, Nevada, he reviewed the emerging treatments and research for familial chylomicronemia syndrome (FCS).




Brown: Good news is, amazingly, there is a medication and research that looks like it may work for these patients. What’s amazing about it is one wouldn’t have guessed this medication would work, but it actually blocks the production of a compound, a protein, that inhibits lipoprotein lipase, and as I mentioned, lipoprotein lipase usually doesn’t work in these patients. You wouldn’t think that removing an inhibiter for an enzyme that doesn’t work would suddenly make the patient better, but it does.

It turns out that this protein, APOC3, probably does a lot of things, not just inhibit life approaching lipase. For whatever reason, when you remove it, the patients with FCS (familial chylomicronemia syndrome) have a significant improvement in their triglycerides. That treatment is in clinical trial, and the hope is these patients, who have suffered for a long time, will have an opportunity for treatment.   

For more, follow Rare Disease Report on Facebook and Twitter.
 

Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.