|IN BRIEF |
• The National Multiple Sclerosis Society presented its 2015 Barancik Prize for Innovation in MS to a trio of research ophthalmologists from New York University Langone Medical Center, Johns Hopkins School of Medicine and University of Texas Southwestern Medical Center. The organization honored Laura Balcer, MD, MSCE, of NYU, Peter Calabresi, MD, of Johns Hopkins and Elliott Frohman, MD, PHD, of UT Southwestern for “novel, groundbreaking and impactful research” of the retina and other ocular structures in people with multiple sclerosis (MS). “Thanks in large part to this team’s efforts, [optical coherence tomography] has transitioned from a tool for ophthalmologists who treat glaucoma patients to a mainstream tool used to study disease mechanisms underlying MS in particular, but new evolving research suggests that such observations may apply to neurodegenerative disorders in general,” the society said in announcing the prize.
• Patients with diabetic macular edema who had one to three years of monthly ranibizumab (Lucentis, Genentech) therapy maintained their vision gains with a notable reduction in treatment frequency, according to an open-label extension phase of the Phase III RIDE and RISE trials. In results published online in Ophthalmology, subjects had an average of 4.5 injections over a follow-up of 14.1 months. Approximately 25 percent of subjects did not require further treatment, and mean best-corrected visual acuity was sustained or improved through the end of follow-up.
“Ophthalmology is a promising specialty for implementing precision medicine because of the eye’s amenability to intervention and the significant human and economic burdens it incurs,” says Stephen H. Tsang, MD, PhD, an associate professor in ophthalmology, pathology and cell biology at Columbia University in New York. Dr. Tsang’s clinic has genotyped more than 800 patients with retinitis pigmentosa and juvenile macular degeneration.
The National Institutes of Health (NIH) advisory committee in September released a framework for building a cohort of 1 million or more Americans that researchers can draw on for data and specimens. The Precision Medicine Initiative stands to receive $215 million in government funding fiscal year 2016. NIH will lead efforts in cancer genomics, as well as the development of the participant cohort.
The Precision Medicine Working Group developed the framework to form and manage the large research cohort. Among the goals of the framework are to help investigators discover biological markers that signal disease risk and create platforms for trials.
“Ophthalmic precision medicine is facilitated by the eye’s relative immune privilege and accessibility, and the effects of treatment can be precisely monitored non-invasively at the resolution of a single cell with adaptive optics imaging,” Dr. Tsang said. Because the eye is a “pair organ,” it provides ideal treatment-control conditions and carries a low risk of rejection of gene and stem cell therapies.
“In fact, therapies involving embryonic stem cell transplants for macular degenerations are the only FDA-approved regenerative medicine trials currently,” Dr. Tsang said. Ocata Therapeutics is conducting the Phase II clinical trial of its proprietary RPE cells in patients with dry age-related macular degeneration.
Dr. Tsang also noted that Spark Therapeutics, a late-stage gene therapy company, received both breakthrough therapy and orphan product designation from the U.S. Food and Drug Administration last year for its lead product candidate, SPK-RPE65, for the treatment of genetic retinal disorders. SPK-RPE65 is now in a fully enrolled, pivotal Phase III clinical trial, and Spark has said it plans to file for FDA biologics license application in 2016. (More on page 38.)
“Supporting vision research will be essential to making therapies based on precision medicine at Columbia a reality,” Dr. Tsang said. “An office to liaise with the FDA on approving gene therapy and autologous stem cell transplantations provides just one example of the additional resources that will be needed.”
He is hopeful that the NIH framework for precision medicine is a step in that direction.
With Synergetics Acquisition, Valeant to Integrate Two Vitrectomy Platforms
Valeant completed the Synergetics acquisition in October. The acquisition brings two vitrectomy platforms, B+L’s Stellaris and Synergetics VersaVIT, under one corporate structure. Synergetics also has other products for ophthalmology and neurosurgery.
Mr. Chang hinted that Stellaris and VersaVIT would be integrated into a single product.
“We are particularly excited to pair the extensive breadth of accessories featured in the Synergetics platform with our Stellaris PC microsurgical system to increase the procedural opportunities for our customers,” he said. “We also look forward to integrating the VersaVit and Stellaris PC portfolios to provide one unified product offering very soon.”
Synergetics has been “fully integrated” with B+L, Mr. Chang said. The combination more than doubles B+L’s vitreoretinal sales force.
“As a result, we’ve combined the Synergetics and Bausch + Lomb research and development teams to leverage their efforts in identifying and developing new retina-focused advancements and improvements to help address the ever evolving needs of the eye-care community,” he said.
Synergetics, based in the St. Louis suburb of O’Fallon, Mo., expects its operations to remain there, David M. Hable, Synergetics president and CEO, previously told the St. Louis Post-Dispatch. RS