Eye Drops For Leukemia Treatment


Researchers at Wellcome Sanger Institute at the University of Cambridge, University of Nottingham and their collaborators have discovered that an active ingredient in eye drops developed for the treatment of retinal neovascular disease has shown promise for treating Leukemia. Not only that, the results so far show that this compound can kill leukemia blood cells without harming non-leukemic blood cells.

The results, published recently in Nature Communications reveal a potential new treatment approach for an aggressive blood cancer with a poor prognosis. Mainstream AML treatments have remained unchanged for over 30 years, with the current treatment being chemotherapy, and the majority of people’s cancer cannot be cured. A subtype of AML, driven by rearrangements in the MLL gene has a particularly bad prognosis.

In a new study, Sanger Institute researchers and their collaborators set out to work out how inhibition of SRPK1 can kill AML cells and whether it has therapeutic potential in this disease. They first showed that genetic disruption of SRPK1 stopped the growth of MLL-rearranged AML cells and then went on to study the compound SPHINX31, an inhibitor of SRPK1, which was being used to develop an eye drop treatment for retinal neovascular disease – the growth of new blood vessels on the retinal surface that bleeds spontaneously and causes vision loss.

The team found that the compound strongly inhibited the growth of several MLL-rearranged AML cell lines, but did not inhibit the growth of normal blood stem cells. They then transplanted patient-derived human AML cells into immunocompromised mice and treated them with the compound. Strikingly, the growth of AML cells was strongly inhibited and the mice did not show any noticeable side effects.

“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 leukemia without harming healthy cells. This shows promise as a potential approach for treating this aggressive leukemia in humans”, according to Dr. George Vassillou, joint leader of the research from the Wellcome Sanger Institute and the Wellcome-MRC Cambridge Stem Cell Institute.

The new eye drops were still at a pre-clinical stage of development when the additional uses were discovered, so no human trials have been conducted as of yet. The eye treatment is expected to move into human clinical trials around 2020, but more work is still needed to explore dosage and delivery in terms of cancer treatment.


The Wellcome Sanger Institute is one of the world’s leading genome centres. Through its ability to conduct research at scale, it is able to engage in bold and long-term exploratory projects that are designed to influence and empower medical science globally. Institute research findings, generated through its own research programmes and through its leading role in international consortia, are being used to develop new diagnostics and treatments for human disease. To celebrate its 25th year in 2018, the Institute is sequencing 25 new genomes of species in the UK. Find out more at www.sanger.ac.uk or follow @sangerinstitute on TwitterFacebookLinkedIn and our blog


Source: Wellcome Sanger Institute




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