Single gene therapy for lipoprotein lipase deficiency reduces severity and frequency of pancreatitis

Over a 6-year period, patients with the genetic disease lipoprotein lipase deficiency (LPLD) who received a single gene therapy treatment of alipogene tiparvovec had a marked reduction in the severity and frequency of pancreatitis. No cases of severe pancreatitis and only one admission to the intensive care unit for an LPLD-related abdominal event were reported in the study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com/).

Daniel Gaudet and coauthors from Université de Montréal, Canada, Academic Medical Center and uniQure BV (Amsterdam, the Netherlands), Chiesi Farmaceutici (Parma, Italy), Beaujon Hospital, Denis Diderot University (Paris, France), University Hospital of North Midlands (U.K.), and Erasmus Medical Centre (Rotterdam, the Netherlands) present their findings in the article entitled "Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis (http://online.liebertpub.com/doi/full/10.1089/hum.2015.158)."

The researchers assessed LPLD-related acute abdominal events that required hospital care in a small group of patients treated with a single dose of the gene therapy product Glybera®. The results support an association between gene therapy for this rare genetic disease and overall reductions in healthcare costs and resource utilization.

"As gene therapy moves forward into the mainstream of medicine, it will be critical to define the evidence of its benefit to patients with specific diseases," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. "This work with alipogene tiparvovec provides an evidence-based guideline for its use in patients with LPLD going forward."