Chasing a Cure for Macular Degeneration
When the SpaceX mission returns to earth in April, there will be 80 mice onboard who have spent a month living at the International Space Station. The rodents represent the first investigational ophthalmic therapy in space and the next step in Dr. Hema Ramkumar’s pursuit of a cure for age-related macular degeneration.
An ophthalmologist and retinal surgeon, Hema Ramkumar, MD, has developed a novel ocular regenerative gene therapy, designed to modify gene expression within the retina to stop macular degeneration’s progression toward blindness. Preclinical studies have been promising, with the experimental therapy making retina cells more resistant to oxidative stress and improving cell life span.
Spaceflight to microgravity offers a unique but valuable environment, as the oxidative stress and slow deterioration of the retina that would take months to create on earth can be created in a very short time in space. “There is no perfect model of the disease, but this takes us much closer,” explains Dr. Ramkumar, whose research is partially funded by the National Science Foundation and the International Space Station Laboratory. Once on earth, retinal scans of the 40 mice injected with the gene therapy will be compared against the control group.
While therapies have existed for decades to effectively seal the leaking blood vessels that characterize “wet” macular degeneration, an advanced version of the disease, there remains no cure and no effective early-stage interventions to prevent progression.
Dr. Ramkumar says it was her frustration with the lack of options she could offer patients with macular degeneration, the leading cause of irreversible blindness in those over 60, that started her down this path—and prompted her to spend 15 years pursuing research, including authoring more than 30 papers on retinal disease.
“Macular degeneration takes away so much,” she explains. “To have nothing to offer in the early stages of the disease or a cure is unacceptable.”
NEXT STEP? Applying to begin human clinical trials, which Dr. Ramkumar hopes to start later this year.
To make an appointment with Dr. Ramkumar, call 714-738-4620. To find a physician who is right for you, go to providence.org/findadoctor.