Two weeks ago, the principals in a campaign known as End Blindness by 20/20 announced that they were putting up $2 million in gold for the individual, group, or institution that contributes most to eradicating blindness by the year 2020. Those principals are former Columbia University roommates Sanford Greenberg, Jerry Speyer and, yes, Art Garfunkel.

The reward, known as the Sanford and Susan Greenberg Prize, will be administered by FFB’s longtime partner the Wilmer Eye Institute at Johns Hopkins University, in particular a governing council which includes Dr. Morton Goldberg, a world-renowned retinal disease researcher who’s also chairman of the Foundation’s Clinical Research Institute and a member of its Scientific Advisory Board. So how, exactly, does Art Garfunkel fit in?

The singer explains his role in this video:

Long story short, Garfunkel befriended Greenberg when they were roommates at Columbia in the early 1960s. At age 19, Greenberg suddenly lost his vision to glaucoma and, despite being told he should go home to Buffalo and learn how to make screwdrivers, graduated Phi Beta Kappa. He then attained advanced degrees, worked for U.S. presidents and found success as a tech entrepreneur – all while he, Garfunkel and Speyer, a real estate mogul, remained close friends.

In 2005, during his commencement speech at Hopkins, the university’s then president, William Brody, told the story of how Garfunkel helped Greenberg in college by, among other things, reading his textbooks aloud to him. Years later, when a then-unknown act named Simon & Garfunkel needed money to record a demo of a song titled “The Sound of Silence,” Greenberg gave Garfunkel $500.

Greenberg, Garfunkel and Speyer came of age about the time President John Kennedy challenged the scientific community’s best minds to put a man on the moon within a decade. It took roughly 8 years, or 2,978 days. And, in that same spirit, Greenberg et al. announced their challenge on October 18 – 2,978 days from when the winner of the prize will be announced, in mid-December of 2020.

In another video on the site, Dr. Peter McDonnell, director of the Wilmer Eye Institute, explains that the $2 million prize is being offered to parties worldwide, so that not just any one agency or person, but an entire community of researchers, is working toward the same goal – “to find light for the millions who now awaken to darkness.”

While FFB is focused specifically on retinal diseases, which affect 10 million Americans, its approach runs parallel to that of the End Blindness by 20/20 campaign. FFB has cast an international net so as to find promising research opportunities and raise the funds needed to ferry potential treatments through clinical trials. Seeing as Messrs. Greenberg, Garfunkel and Speyer are doing the same, we applaud their efforts.

Art Garfunkel perhaps sums it up best in his video when he says that, although Greenberg has been able to achieve much in his life, he’s never given up on the vow, made at a young age, to end blindness for everyone after him. Then, with his arm around Greenberg, Garfunkel says that he once “tried to close my eyes for a day, in an effort to truly empathize with my dear friend here. I got up to an hour.”

To understand the implications of their research, imagine this: You’re losing your vision to a disease like retinitis pigmentosa (RP). You go into a clinic where doctors take a small sample of blood or skin. Back in their lab, they genetically reprogram that sample to become stem cells, which can then be developed into any type of cell or tissue in your body.

In your case, you need retinal cells to replace the ones you have lost to RP. So the doctors make new retina cells from the stem cells, which are then transplanted back into your eye to restore vision. And, by the way, they may have even corrected the genetic defect in those retinal cells that caused your RP in the first place.

Dr. Shinya Yamanaka received one of the 2012 Nobel Medicine prizes for figuring out how to induce mature cells from skin or blood to become stem cells for use in future treatments like the one I just described. He accomplished that feat just five short years ago.

Dr. John Gurdon, one of two recipients of this year’s Nobel Prize for Medicine, discusses his reaction
to winning the prize during a press conference.

What is amazing to me is that this potential treatment, known as induced pluripotent stem cells (iPSC), isn’t that far from the clinic, thanks to the efforts of researchers like Dr. David Gamm, of the University of Wisconsin-Madison, who is developing the technology in lab studies with the goal of launching a human study within a few years. The RIKEN Center, a Japanese institution, also plans to launch a clinical trial of iPSC for people with age-related macular degeneration.

The other Nobel Medicine recipient, Dr. John Gurdon, performed pioneering work in cloning and cell reprogramming back in the 1950s. In one series of experiments, he took the nucleus of a cell from a tadpole and transferred it into the egg cell of a frog to make a new tadpole identical to the one from which he pulled the nucleus.

This work greatly informed the work of Dr. Yamanaka, Dr. Gamm and other scientists involved in iPSC development and related research.

Dr. Gurdon discusses some of his work in the video I have included with this post, and also provides a compelling back story about his early science education. Let’s just say we are lucky he stuck to his guns in pursuing his passion.

Pictured, top of page: Dr. Shinya Yamanaka, co-winner of the 2012 Nobel Prize for Medicine.
Photo Copyright ©, Gladstone Institutes/Chris Goodfellow 

I’d be remiss if I didn’t mention that Michelle is under the care of Dr. Richard Weleber, the study’s principal investigator, an outstanding clinician-researcher and one of the most pleasant, unassuming people I know.

It will likely take several months — perhaps years — to know if the UshStat treatment is working. The treatment’s primary goal is to halt or slow the progress of vision loss, which can occur slowly in patients with Usher syndrome and related retinal conditions. So, we need to be patient awaiting results on efficacy. But I am optimistic about the treatment’s potential for saving vision.

In case you missed it, I also blogged recently about an emerging Usher 2A gene therapy, and there are two recent articles focused on Usher syndrome on the Foundation’s website. One’s about expansion of the UshStat trial in Paris, France; the other about the discovery of a new Usher gene.

He came up with the idea after a friend told him how dependent his visually impaired brother is on the family for help. Sharma then put together a team which, in six days, designed and built a prototype – just in time for the MIT Media Lab Design and Innovation Workshop in 2011.

Ducere is now testing the latest version of the shoe, with the intent of making it available later this year. Sharma provides updates on his own web site, and, according to The Economist, the company’s hoping to build in sensors that would also warn users of nearby obstacles – including, eventually, cars.

Such is the case with The Wall Street Journal, which, among other FFB associates, quotes our chief research officer, Dr. Steve Rose, in this article, focusing specifically on Stargardt disease. It’s accompanied by a blog post that gives a shout-out to Eye on the Cure while confirming the Foundation’s position as a leader in research-funding efforts.

It’s also worth noting that the WSJ article includes mention of a gene therapy developed, and being tested in a clinical trial, by the British company Oxford BioMedica. Known as StarGen, it’s one of a few of the company’s treatments-in-development that FFB has had a hand in funding. That includes a gene therapy for Usher syndrome which is undergoing a clinical trial in both the United States and France.

Thank you, Wall Street Journal, for getting the word out! Otherwise, visitors to Eye on the Cure can rely on our continual reports on research developments for various retinal diseases.