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News, Research

Potential leukaemia treatment found in eye drops

08/01/2019By Callum Glennen
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Researchers have identified an active ingredient in an eye drop currently under development that has the potential to kill leukaemia cells without harming blood cells.

The discovery, made by researchers at the Wellcome Sanger Institute (WSI), University of Cambridge and University of Nottingham, followed a previous study that had identified more than 400 genes as possible therapeutic targets for different types of acute myeloid leukaemia (AML). One gene in particular, SRPK1, was found to be an essential element in the development of AML.

Fortunately, an inhibitor for SRPK1 was already in development at biopharmaceutical company Exonate, which is developing a variety of treatments for retinal vascular diseases.

“We have discovered that inhibiting a key gene with a compound being developed for an eye condition can stop the growth of an aggressive form of acute myeloid leukaemia without harming healthy cells,” Dr George Vassiliou, joint leader of the research from the WSI and the Wellcome-MRC Cambridge Stem Cell Institute, said.

“This shows promise as a potential approach for treating this aggressive leukaemia in humans.”

Following collaborations on a number of animal tests, the gene inhibitor was found to be effective at slowing the growth of cancer cells. The results of these tests were recently published in the journal Nature Communications.

“When Dr Vassiliou told me that SRPK1 was required for the survival of a form of AML, I immediately wanted to work with him to find out if our inhibitors could actually stop the leukaemia cells growing,” Exonate co-founder Professor David Bates said.

“The fact that the compound worked so effectively bodes well for its potential development as a new therapy for leukaemia. It will take some time, but there is real promise for a new treatment on the horizon for patients with this aggressive cancer.”

However, while results have been promising, the drug is yet to be tested on humans and will likely require much higher doses than what is currently being developed to combat retinal disease. No plans for human trials had been released at the time of publication.

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