Retinitis pigmentosa (RP), is a slow, inexorable progression. You begin to stumble when entering dimly lit rooms. Driving at night becomes difficult, then impossible. Your peripheral vision narrows to the point where you feel like you’re looking through a pinhole. Bright lights become unbearable. Colors fade. Then, everything goes dark.
A Light in the Dark
RP is an inherited disease that destroys the photoreceptors in the back of the eyes. RP is so devastating, it’s considered “fatal” for vision. For 99% of those with RP, there’s no treatment. Nearly two million people suffer from the disease worldwide and it’s the leading cause of inherited blindness. But the groundbreaking research of UC Irvine professor of ophthalmology Henry Klassen, M.D., Ph.D. has given patients with RP hope.
Klassen and his team have successfully cultivated human retinal progenitor cells (hRPCs)—a type of stem cell of the eye. The cells are grown in the lab, stored in liquid nitrogen, and woken up when a patient is ready to receive them. In the comfort of an office, with nothing more than a topical anesthetic, an ophthalmologist injects the hRPCs into the eye. The hRPCs form a small cell cluster that secrete neurotrophic factors lacking due to the disease. By continuously secreting these factors, the cells restore photoreceptor activity and therefore visual function. Now, a patient’s photoreceptors that were once considered as good as dead, in a sense, come back to life.
From Possible to Practical
As an undergraduate at Berkeley, Klassen learned about Anders Björklund’s trailblazing work on transplanting embryonic brain tissue in rats and was excited by what this discovery could bring. What if it was possible to restore parts of the central nervous system in a human? What if we could regenerate parts of our body similar to how a lizard can grow back the end of their tail?
Klassen’s fascination in regenerative medicine persisted and he pursued a dual M.D./Ph.D. at the University of Pittsburgh. There, in Ray Lund’s lab, Klassen worked on rats in which embryonic retinas were transplanted into their brains. While Klassen proved he could create a new functional pathway in a rat brain sufficient to make its pupils constrict, he couldn’t imagine anyone lining up for this particular procedure.
After graduate school, Klassen looked for more practical applications for his research, specifically about how to approach different forms of neural degeneration. Interestingly, the retina is part of the central nervous system, yet it is much easier to access than the brain or spinal cord. When Klassen learned about RP he made the connection and found his life’s work.
Today, Klassen is a prominent figure in the field of regenerative medicine and stem cell therapies. The intervening years in his lab were exhilarating, but also anxiety provoking. Klassen and his team spent countless hours propagating, testing, harvesting and transplanting stem cells.
A startup’s survival depends on the merits of the science behind it, the expertise of business and industry partners, and the unwavering commitment of academic researchers to make an impact on society. Klassen’s advice to his fellow researchers is to see their idea through.
“I was getting nervous for a while because having nothing to offer to RP patients is a disheartening experience,” says Klassen.
Fortunately, Klassen’s anxiety didn’t have to linger for too long. Not only did his revolutionary research hold up, but he received hefty financial support. In 2005, the California Institute of Regenerative Medicine (CIRM) was established, and Klassen eventually received $14.9 million in funding from CIRM to fuel his research. In addition, UCI secured enough funding to construct the Sue and Bill Gross Stem Cell Research Center on campus. With financial backing and a new cutting-edge facility, Klassen and his team were able to accelerate their groundbreaking work and usher in a new generation of regenerative medicine.
Collaboratively Building Momentum
Seeing the impact his therapy could make in restoring vision convinced Klassen to expand his research beyond academia. In 2012, he launched jCyte, a start-up based on his research. Headquartered in Newport Beach, jCyte currently has 12 full-time employees.
JCyte’s relationship with UCI was critical to its success. The Research Translation Group has helped navigate the protection of intellectual property behind jCyte, and UCI was used for housing jCyte’s clinical trials.
A startup’s survival depends on the merits of the science behind it, the expertise of business and industry partners, and the unwavering commitment of academic researchers to make an impact on society. Klassen’s advice to his fellow researchers is to see their idea through.
Another UCI collaborator partnering with jCyte was The UCI Center for Statistical Consulting, which provided brainpower.
“The UCI Center for Statistical Consulting provided much more than just help calculating results. In addition to their expertise, they provided us with sophisticated analytical packages and Jump software. I would say statistics is the backbone of not only basic science but clinical science as well,” Klassen says.
The benefits of being a part of the UCI community extend beyond the walls of academia. The jCyte team has also taken advantage of the access to leaders in the local industry. One such leader is Gavin Herbert, the visionary co-founder of Allergan (the pharmaceutical company headquartered in Irvine) and the benefactor of the UCI eye center that bears his name. (The Gavin Herbert Eye Institute serves as jCyte’s central clinical facility where they conduct their clinical trials.) Herbert’s decision to move his company to Irvine in 1989 helped plant a flag for ophthalmology in Orange County. Since then, the area has grown into a mecca for ophthalmic companies.
The Promise of Regeneration
The jCyte team is currently preparing for Phase III clinical trials for the RP indication after which they will undergo FDA review. What is especially exciting about Klassen’s work is that its impact could reach far beyond the treatment of RP, and jCyte is developing treatments for other degenerative retinal disorders and optic nerve conditions. The potential to apply this technology to treat degeneration in other parts of the central nervous system could truly be revolutionary. Klassen’s data will likely serve as a baseline trove of information for other researchers.
The future for people with RP and other retinal disorders has never been brighter as jCyte works to bring Klassen’s groundbreaking therapy to all who need it. What’s taking place on UCI’s campus is forever changing the landscape of ophthalmic care and regenerative medicine.
Discover more at: http://jcyte.com