Researchers at the University of Cambridge in the United Kingdom have discovered an active ingredient from a remedy for eye diseases that can be used to treat malignant blood cancer without side effects. This achievement was published in the Nature Newsletter published on the 19th.
Acute myeloid leukemia (AML) is a very malignant blood cancer. For more than 30 years, the main treatment method is chemotherapy, but the effect is poor, and most patients are difficult to cure. In a previous study, researchers at the University of Cambridge developed a new approach based on CRISPR gene editing technology. Using this approach, they identified more than 400 genes that are therapeutic targets for different subclasses of AML, and found that one of the genes, SRPK1, is critical for AML growth in MLL gene rearrangements. SRPK1 is involved in a process called RNA splicing, which prepares RNA for translation into proteins, while proteins are molecules that direct most normal cell growth and growth processes.
In the new study, they found that genetic disruption of SRPK1 prevented the growth of MLL-rearranged AML cells; whereas using SRPK1 inhibitor SPHINX31, a compound extracted from eye drops for the treatment of retinal neovascular disease, The growth of several MLL rearranged AML cell lines was strongly inhibited, but it did not inhibit the growth of normal blood stem cells.
The researchers transplanted patient AML cells into immunocompromised mice and treated them with this compound. It is striking that the growth of experimental AML cells was strongly inhibited and did not show any significant side effects.
Project leader Dr. George Vasilyu believes that this compound extracted from eye drops can inhibit a key gene, thereby preventing the growth of key genes that cause malignant blood cancer, without harming healthy cells, and is expected to become a treatment for human malignant leukemia. Potential method.
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