News Feature | May 29, 2014

UCL Researchers Eye Leukemia Drugs To Treat Vision Loss

By Marcus Johnson

UCL researchers have turned to leukemia drugs to find new ways to treat eye diseases. In a recent study, the researchers found that abnormal blood vessel growth in the eye, which can lead to blindness, can be treated with leukemia drugs. The researchers noted that leukemia drugs could also potentially be used to treat patients with a variety of eye diseases, including age-related macular degeneration, diabetic retinopathy, and other eye blood vessel diseases.

The UCL researchers study was published in the Journal of Experimental Medicine. Professor Christiana Ruhrberg, who works at the UCL Institute of Ophthalmology and led the study, commented on the research. “Blood vessels normally form in developing fetuses to deliver oxygen to growing organs, or in adults when organs are deprived of oxygen due to conditions such as diabetes, stroke, or heart attack,” she said. “Even though the growth of new blood vessels aims to restore organ health, blood vessels often grow abnormally in adults. For example, in diseases such as age-related-macular degeneration and diabetic retinopathy, abnormal eye vessels leak blood and fluids, causing vision loss.”

Current treatments for eye blood vessel growth problems consist of drugs that block the vascular endothelial growth factor signal, or VEGF. The VEGF drives the creation of new blood vessels in the eye. Drugs that block the VEGF are injected into the eye, and, for most patients, are effective. However, the effectiveness of the drugs can wane over time.

Ruhrberg’s research with leukemia drugs does not affect the VEGF. “We discovered an alternative mechanism that regulates blood vessel growth and does not depend on VEGF,” she said. “It involves the activation of a molecule called neuropilin 1 (NRP1) by connective tissue components in the eye. The NRP1 pathway then conveys signals inside the blood vessel cells via another molecule termed ABL1, and this process stimulates blood vessel growth.”

Because the leukemia drugs, which block the NRP1/ABL1 pathways and keep them from growing abnormally, are administered via tablet, patients could potentially avoid eye injections and could administer the drug from their own homes.