Doctors for the first time edit genome in a living human patient

(c) AP Photo/Eric Risberg

For the first time ever, doctors have edited the genome of a living human patient. A 44-year-old man received the treatment for Hunter's syndrome at a hospital in Oakland, California on November 13.

FDA approves the first digital pill that tracks compliance

Source: Proteus Digital Health

The Food and Drug Administration has approved the first drug in the U.S. with a digital ingestion tracking system. Abilify MyCite (aripiprazole tablets with sensor) has an ingestible sensor embedded in the pill that records that the medication was taken. The product is approved for the treatment of schizophrenia, acute treatment of manic and mixed episodes associated with bipolar I disorder and for use as an add-on treatment for depression in adults.
 

Studies show stem cell-based treatment safe in macular degeneration

Results from two early clinical trials show that it may be possible to use human embryonic stem cells as treatment for the dry form of macular degeneration, according to presentations at the American Academy of Ophthalmology Annual Meeting 2017 in New Orleans, LA. Stem cells injected into the eye appear to have replaced the missing cells damaged by the disease, with no serious side effects.

Does performing/undergoing surgery in the afternoon reduce the risks?

(c) Genetic Literacy Project

A new study suggests patients undergoing open heart surgery in the afternoon have a lower risk of potentially fatal complications than those undergoing operations in the morning. The study found that events including heart attacks and heart failure were less common among those who had undergone a valve replacement operation in the afternoon. The finding appears to be linked to the ability of the heart tissue to recover after being starved of blood supply during surgery – an effect the researchers say is influenced by the cells’ biological or “circadian” clock.

The first dual-action Glaucoma drug, Vyzulta, approved by FDA

(c) AAO

Food and Drug Administration (FDA) has approved Vyzulta™ (latanoprostene bunod ophthalmic solution, 0.024%), which is the first prostaglandin analog with one of its metabolites being nitric oxide (NO), indicated for the reduction of intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension. Following topical administration, Vyzulta, a once daily monotherapy with a dual mechanism of action, works by metabolizing into two moieties, latanoprost acid, which primarily works within the uveoscleral pathway to increase aqueous humor outflow, and butanediol mononitrate, which releases NO to increase outflow through the trabecular meshwork and Schlemm's canal.

Orion, the Cortical Visual Prosthesis System, gets full FDA approval

(c) medgadget.com

The FDA has granted full approval to Second Sight Medical Products (SSMP) to begin the feasibility study for the Orion-I^TM Cortical Visual Prosthesis System. The approval allows two US sites, the University of California at Los Angeles (UCLA) and Baylor College of Medicine in Houston, to enroll up to five total patients. The company’s new Orion I Visual Cortical Prosthesis goes even further by bypassing the optic nerve, along with the entire visual system, and stimulates the brain’s visual cortex directly. This would allow even people who lack any functionality within their eyes to be able to see.

Converting to Eylea from Avastin may benefit Patients with Diabetic Macular Edema

(c) British Journal of Ophthalmology

A study from Portugal, published in Graefe's Archive for Clinical and Experimental Ophthalmology, finds changing from bevacizumab (avastin) to aflibercept (eylea) for persistent diabetic macular edema resulted in functional and anatomical improvements. Central macular thickness previous to starting aflibercept was found to be a predictor of anatomical changes after aflibercept.

Beware: Using colored contact lenses on Halloween may lead to blindness!

Whether you’re a ghost or zombie, vampire or witch, poor costume choices—including decorative (colored) contact lenses and flammable costumes—and face paint allergies can cause injuries that haunt you long after Halloween. Also remember what eating lots of sweet candy can do to your or your children's body.

It always pays to be aware and careful. Happy Halloween!

DNA & RNA editing - the future of disease management

Scientists at Harvard University and the Broad Institute of MIT and Harvard have developed a new class of DNA base editor that can alter genomic structure to help repair the type of mutations that account for half of human disease-associated point mutations. These mutations are associated with disorders ranging from genetic blindness to sickle-cell anemia to metabolic disorders to cystic fibrosis.

Suprachoroidal injection - Clearside's approach to treating Diabetic Macular Edema

(c) NEI

Clearside Biomedical, Inc. has completed patient enrollment in the Phase 2 clinical trial (referred to as "TYBEE") of CLS-TA, the company's proprietary suspension formulation of the corticosteroid triamcinolone acetonide (TA) for suprachoroidal administration, used in combination with intravitreally administered Eylea^TM (aflibercept) for treatment of diabetic macular edema (DME).  Patient follow-up in the TYBEE trial is 6 months.

Leber Hereditary Optic Neuropathy - The Gene Therapy race to treatment

GenSight Biologics, which is in advanced stages of clinical development of gene therapy for Leber Hereditary Optic Neuropathy, has a new Chief Medical Officer. Barrett Katz, M.D., M.B.A., who until recently was the Professor of Ophthalmology, Neurology and Neurosurgery at the Montefiore Medical Center and the Albert Einstein College of Medicine in New York. Dr. Katz has worked in both academic medicine and the industry as a neuro-ophthalmologist, working as CEO of ophthalmic biotech Danube Pharmaceuticals, as well as CMO of Fovea Pharmaceuticals and VP of medical affairs and strategy at Eyetech.

Biosimilars and clinical care - information for healthcare providers - Part I

A significant number of people, both in clinics and in pharmaceutical industry, mention the fact that they keep hearing of the word 'biosimilars' in academic meetings and in discussions, but they have no idea what they are, and what they do. Considering that biosimilars are likely to affect the way we practice medicine in the days ahead, it is important that we are aware of what biosimilars mean, and what they do. Also, it is important from the pharmaceutical industry perspective that everyone is knowledgeable of the process for an approval. Lastly, there is a minuscule, but very relevant question, about legal implications, which may or may not break your/your company's back in getting the drug to the market! The last two topics will be covered in subsequent posts.