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- Gene Therapy for Inherited Retinal Dystrophy Gets FDA Clearance: The first gene therapy for a specific mutation leading to disease was approved by the FDA, the agency announced Tuesday. Spark Therapeutics' voretigene neparvovec-rzyl (Luxturna) is now the first gene therapy approved to treat those with confirmed biallelic RPE65 mutation-associated retinal dystrophy, a type of Leber congenital amaurosis. This is a rare disease affecting approximately 1,000-2,000 patients in the U.S. A loss of vision often begins during childhood, with certain patients turning completely blind over time. The therapy uses an engineered adeno-associated virus as vector to deliver a working copy of the RPE65 gene directly to retinal cells. It is administered via subretinal injection by a surgeon. Each eye must be treated at least 6 days apart. In November, results of a small phase III study were presented showing that 93% of patients receiving the treatment had improved vision to the point where they could navigate a maze in low-to-moderate light. 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, in a statement.
- Should you be smoking marijuana to treat your glaucoma? Although marijuana can lower eye pressure, recommending it does not make sense given its side effects, short duration of action, and lack of evidence that its use alters the course of glaucoma. Marijuana's effect on eye pressure only lasts 3-4 hours, meaning that to lower the eye pressure around the clock it would have to be smoked 6-8 times a day. In addition, marijuana's mood-altering effects prevent the patient who is using it from driving, operating heavy machinery, and functioning at maximum mental capacity. Marijuana cigarettes also contain hundreds of compounds that damage the lungs, and the chronic, frequent use of marijuana can damage the brain. Other means of administering the active ingredient of marijuana, tetrahydrocannabinol (THC), include by mouth and under the tongue. These methods avoid the harmful effect of marijuana smoke on the lungs, but are limited by the other systemic side effects, such as drowsiness and loss of judgment. The use of eye drops containing THC or related compounds has been investigated, but it has not yet been possible to formulate an eye drop that is able to introduce the drug into the eye in sufficient concentration to be effective. Although marijuana does lower the eye pressure, it also lowers blood pressure. Lower BP could result in reduced blood supply and possible harm to the optic nerve. Therefore, it is possible that even though marijuana does lower the eye pressure, its use could conceivably make the vision loss from glaucoma worse! For this reason, marijuana cannot be recommended without a long term clinical trial that evaluates the health of the optic nerve as well as the eye pressure.
- Managing Diabetes - New technologies can make It easier - excerpts. You might not notice all that your body does to keep you healthy until something goes wrong. Diabetes (DM) is a serious disease that happens when your body has trouble managing and using blood glucose (BG) a sugar that your body uses as fuel. When you have DM, you must actively take on this process yourself. A missed, or miscalculated, dose can lead to serious complications - DM increases your risk for blindness, heart disease, stroke, kidney failure, and amputation. Dr. GA-Rubin, NIH DM Tech Program says it is a significant burden to self-test sugar levels several times a day. and calculate the amount of insulin you need to inject. NIH funded-scientists are testing promising technologies to help people better manage DM. For example, artificial pancreas systems monitor BG levels and provide insulin automatically. The devices vary in how easy they are to set up and use. Dr. E Damiano, Boston University says their device only requires that you type in your body weight to get started. Other scientists are studying smart insulins which would become active only when needed. Researchers are also looking for ways to stop the body from attacking insulin producing cells. Others work on ways to regenerate or replace insulin-producing cells. While future tools may make it easier to manage your diabetes, glucose monitors, medications, and insulin pumps are all available now. Talk with your health care provider about your options.
Medpage Today 12/19/17
Henry D. Jampel, Wilmer Eye Institute Johns Hopkins. Last rev 10/29/17 Glaucoma Research Foundation
NIH News 12/2017