Gene Therapy for Lipoprotein Lipase Deficiency Effective and Safe

The first patient who received the gene therapy that was recently approved in Europe to treat lipoprotein lipase deficiency (LPLD) had no abdominal pain or episodes of pancreatitis, following a history of 37 pancreatitis attacks. The gene therapy called alipogene tiparvovec allowed the patient to discontinue plasmaphere and described improved quality of life. The report is published in Human Gene Therapy. The article "Gene Therapy in Lipoprotein Lipase Deficiency (LPLD): Case Report on the First Patient Treated with Alipogene Tiparvovec under Daily Practice Conditions" was coauthored by Ursula Kassner, Tim Hollstein, Thomas Grenkowitz, Marion Wühle-Demuth, Bastian Salewsky, Ilja Demuth, Elisabeth Steinhagen-Thiessen, from Chari-té - Universitätsmedizin Berlin, and Michaela Dippel, MD Medscript, Bad Dürheim, Germany.

LPLD is a rare inherited disorder associated with an increased incidence of inflammation of the pancreas due to elevated triglyceride levels. Gene therapy with alipogene tiparvovec offers a potential cure for LPLD, which is currently treated with plasmapheresis when maintenance of an ultra-low fat diet, use of fibrates, and other triglyceride-lowering therapies prove inadequate. Alipogene tiparvovec is an adeno-associated virus 1 (AAV1) gene therapy administered via intramuscular injections together with immunosuppression.

Uniqure, the manufacturer of alipogene tiparvovec (marketed as Glybera), has ceased post-marketing studies and withdrawn the product from the market in the European Un-ion due to very limited demand for its use. The withdrawal of Glybera was not related to any risk-benefit concerns.

"While Glybera has not continued in commercial sales, it is very gratifying to gene therapy translational researchers to see a case report of a safe and effective gene therapy product in clinical use," 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.



Source-Eurekalert