A Stem Cell Treatment for Optic Neuritis
Some classes of first generation stem cell transplants are known to reduce inflammation, though the signaling mechanisms involved are still poorly understood. Nonetheless, this means that a range of conditions thought to have a strong inflammatory component to their pathology are potential targets for treatment. Here for example, a clinician has found that stem cell transplants produce benefits in some patients suffering from the blindness produced by optic neuritis, chronic inflammation of the optic nerve that can occur for reasons that are unclear in many cases:
Vanna Belton was in Washington in 2009 when, while stuck in traffic one day, she noticed the streetlights were blurry. Weeks later she had almost no vision and no explanation for why everything seemingly went dark. She was diagnosed with a sudden and perplexing case of optic neuritis, a general term meaning optic nerve inflammation. As Belton searched for alternatives, she found Dr. Jeffrey N. Weiss, who was enrolling blind patients in an unorthodox stem cell study. He wasn't affiliated with a university or government institute, but he was taking on all those who could afford the roughly $20,000 to pay for the study and injecting stems cells into their eyes in one of three ways -- around the retina, in the retina and directly into the optic nerve -- in hopes of restoring some people's sight. He made no promises.In early 2014, she had the surgery. During the four-hour procedure, Weiss and a medical team extracted bone marrow from Belton's hip, separated her stem cells in a machine and then injected the cells in and around her right eye's retina and directly into her left eye's optic nerve. Weiss is not following the usual steps of clinical studies. Among other things, he didn't test his treatment theories first on lab animals or using computer models, or randomize his trials by using either stem cells or placebos in study participants. He didn't test the procedure for safety on a small group before moving to a larger trial. Weiss, who is board-certified in ophthalmology, said he didn't have the patience for academic research, which is strictly governed by internal review boards and requires fundraising. Without a long history of stem cell research and a current academic appointment, he said, he sought legitimacy for his work by registering the trial with NIH, which scientific journals require to publish promising results.
The NIH also requires researchers to gain approval and oversight from an ethics review panel. Universities and government agencies have their own panels; Weiss tapped the International Cellular Medicine Society, an independent group that promotes stem cell therapies. Weiss said that 60 percent of his 278 patients with macular degeneration, glaucoma and other diseases have regained some sight. While he can't explain how it works, he believes that will become clear eventually. "We didn't know how penicillin worked for many years, but it saved many lives in the meantime. It is hubris to think that something can't work until you understand how it does. ... It is more important what the patient sees, not what I see." Sitting on the front steps of her home a year and a half ago, Vanna Belton was startled and thrilled when her eyes focused on a car's license plate. Essentially blind for more than five years, she could read the numbers and letters. No one disputes that Belton now sees well enough to, for example, read the menus in a restaurant. She can navigate the streets without the white cane she once used.
Link: http://www.baltimoresun.com/health/bs-hs-stem-cells-for-eyes-20160201-story.html
For the record, here's Tech Review's main article of the week:
"If all goes according to plan, sometime next month a surgeon in Texas will use a needle to inject viruses laden with DNA from light-sensitive algae into the eye of a legally blind person in a bet that it could let the patient see again, if only in blurry black-and-white.
The study, sponsored by a startup called RetroSense Therapeutics, of Ann Arbor, Michigan, is expected to be the first human test of optogenetics (...).
The trial, to be carried out by doctors at the Retina Foundation of the Southwest, will involve as many as 15 patients with retinitis pigmentosa, a degenerative disease in which the specialized light-sensitive photoreceptor cells in the eye die, slowly causing blindness."
Source: https://www.technologyreview.com/s/600696/in-first-human-test-of-optogenetics-doctors-aim-to-restore-sight-to-the-blind/#/set/id/600843/
The approach described in that article is quite different from the one you quote here, though, as RetroSense essentially bypasses the problematic elements (photoreceptors which have decayed) and aimes at repurposing another component of the eye (ganglion cells) to partially restore the patients' vision.
But this technique does make use of viral vectors ̶— like gene therapy ̶— and I find it smart; repurposing cells may not be an end in itself (we'd want to restore our degraded bodily function exactly as they were before) but sounds useful as an interim solution.