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Archive - Dec 31, 2017

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FDA Approves Gene Therapy for Genetic Disease Causing Vision Loss & Often Leading to Blindness; Luxturna™ Therapy, Developed by Spark Therapeutics, Is Designed to Treat Biallelic RPE65 Mutation-Associated Retinal Dystrophy

On December 19, 2017, the U.S. Food and Drug Administration approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene. “Today’s approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb, MD. “We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing the right policy framework to capitalize on this scientific opening.

Bacterial Fats May Contribute to Heart Disease

Heart disease and fatty clogs in the arteries go hand in hand. But new evidence suggests the fatty molecules might come not only from what you eat, but from the bacteria in your mouth, report University of Connecticut (UConn) scientists in the August 2017 issue of the Journal of Lipid Research. The research may explain why gum disease is associated with heart trouble. The article is titled “Deposition and Hydrolysis of Serine Dipeptide Lipids of Bacteroidetes Bacteria in Human Arteries: Relationship to Atherosclerosis.” Heart attacks and strokes are the crises we notice, but they result from a slow process of atherosclerosis, the hardening and clogging of the arteries with fatty substances called lipids. Immune cells stick to the walls of blood vessels, scavenge lipids, and multiply. The blood vessel walls inflame and thicken as the smooth muscle cells lining them change, swelling and dividing to create plaques, clogs, and warty growths called atheromas. For a very long time, doctors and researchers assumed that the lipids came from eating fatty, cholesterol-rich food. But the research hasn’t borne this out; some people who eat large amounts of the foods we thought were the sources of the fat, such as eggs, butter, fatty fish, and meat, don’t necessarily develop heart disease. UConn researchers believe they may have solved part of the puzzle. Using careful chemical analysis of atheromas collected from patients by a colleague at Hartford Hospital, they found lipids with a chemical signature unlike those from animals at all. Instead, these strange lipids come from a specific family of bacteria. “I always call them greasy bugs because they make so much lipid. They are constantly shedding tiny blebs of lipids. Looks like bunches of grapes,” on a bacterial scale, says Dr.