Leukemia drops
A cure for an aggressive type of cancer has been found in eye drops
Yulia Vorobyova, Vesti
The active component of eye drops created for the treatment of retinal diseases will help to overcome one of the most aggressive types of cancer. This discovery was made by researchers from several universities and research centers in the UK.
Experts report that the eye drops in question are not yet available on the consumer market, since they have not yet passed the stage of clinical trials. However, scientists were interested in one compound in the composition of the drug. It targets a gene capable of suppressing the progression of a disease called acute myeloid leukemia (AML).
This is one of the most dangerous types of leukemia, a malignant blood disease in which altered white blood cells multiply rapidly. They accumulate in the bone marrow, gradually displacing healthy red blood cells, platelets and leukocytes. This, in turn, leads to concomitant complications: it becomes increasingly difficult for the body to resist infections.
The disease affects people of all ages, but is more common in adults. Treatment requires hospitalization and prolonged chemotherapy, but unfortunately, it is not yet possible to completely cure the disease.
At the same time, experts note that the subtype of acute myeloid leukemia, caused by rearrangements in the MLL gene, has a particularly poor prognosis.
Earlier, researchers from the Sanger Institute, who participated in the new work, using CRISPR gene editing technology, identified more than 400 genes – potential therapeutic targets in the fight against AML.
Scientists have found that one of them – the SRPK1 gene – is associated with the progression of the same subtype of leukemia associated with rearrangements in the MLL gene. When the SRPK1 gene is active, malignant cells multiply. If you suppress its activity, respectively, the expansion of cancer will slow down.
When the researchers found out that the biotech company Exonate was already creating an SRPK1 inhibitor, they were interested in this development.
The compound in question is called SPHINX31. It helps in the treatment of neovascular diseases of the retina, in which new blood vessels grow on its surface. Such a "breakdown" causes spontaneous bleeding and is fraught with loss of vision for patients.
Representatives of Exonate say that when researchers contacted them and informed them about the possible potential of the compound, they decided to help colleagues and shared their developments.
As a result, it turned out that SPHINX31 can slow down the growth of several lines of MLL-rearranged malignant cells. And, importantly, the compound does not affect the reproduction of healthy hematopoietic stem cells in any way.
In the course of testing the "abilities" of SPHINX31, specialists transplanted cells obtained from AML patients to mice whose immunity was weakened. Then they were processed by SPHINX31.
As a result, the development of leukemia really slowed down a lot, and no side effects were observed in rodents.
According to scientists, this approach opens up huge prospects for the development of a new method of treating AML in humans.
Experts also explain what exactly caused such an "amazing", as they put it, effect. The fact is that the SRPK1 gene controls RNA splicing – this is the stage of preparing RNA for the synthesis of proteins, which are associated, among other things, with the processes of cell growth and reproduction.
When the SRPK1 gene is blocked, another gene, called BRD4, is also affected. He is notorious as an "accomplice" of acute myeloid leukemia.
Meanwhile, inhibition of the SRPK1 gene using the SPHINX31 compound changes the BRD4 gene, and as a result, the latter no longer promotes the growth of malignant cells, but on the contrary, begins to damage them.
"Our study describes a new mechanism necessary for the survival of leukemic cells and highlights the therapeutic potential of SRPK1 inhibition in aggressive type of AML. Targeting this mechanism can also be effective in other cancers where the BRD4 and SRPK1 genes play a role, such as metastatic breast cancer," adds one of the lead authors of the work, Konstantinos Tzelepis from the Sanger Institute.
His team notes: it will certainly take some time to create new treatments for AML, as well as to explore the potential of a new compound in the treatment of other cancers. However, this discovery gives hope that in the future the diagnosis of acute myeloid leukemia will cease to be a verdict for patients.
A scientific article based on the results of this work was published in the journal Nature Communications.
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