December 19, 2018 - 17:15 AMT
PanARMENIAN.Net - Scientists in the UK have found a potential new treatment for aggressive types of leukemia in a rather unexpected place, eye drops, Forbes says.
Although some types of leukemia have a high survival rate, such as childhood acute lymphoblastic leukemia, acute myeloid leukemia (AML) is still very hard to successfully treat, particularly if the leukemia is caused by certain genetic alterations. One of these groups of genetic alterations are called MLL-rearrangements where the MLL gene gets wrongly fused to a variety of other genes during cell replication, leading to leukemia. These MLL-rearranged AMLs are amongst the most difficult to treat and have a very poor prognosis.
The new study, published on Wednesday, December 19 in Nature Communications shows how a drug targeted at a protein called SPRK1 has successfully treated human leukemias in mice.
Previous work by the scientists using a well-known technique called a CRISPR screen, which in this case used CRISPR-Cas9 gene editing technology to disrupt thousands of genes in cancer cells to see which ones are vital for cell growth, had identified 400 genes which were potential drug targets in different types of AML.
SRPK1 was found to be particularly important in MLL-rearranged AML and is important in a process called angiogenesis, which is the production of new blood vessels. Targeting SRPK1 has previously been touted as a potential treatment for solid tumors including prostate cancer where the idea is to limit the blood supply to the tumor, inhibiting its growth. FDA-approved Avastin is already approved for inhibiting angiogenesis in a number of solid tumors, but targets a different gene.
The cancer research scientists found out that SRPK1 was also involved in some retinal diseases and that a company called Exonate was developing a drug called SPHINX31 to target it for a disease where the blood vessels in the eye bleed spontaneously, causing vision loss.
Importantly, the drug stopped the growth of several MLL-rearranged AML cell lines in lab experiments, but had no effect on normal blood cells. The scientists next transplanted samples from AML patients into mice with compromised immune systems, meaning the leukemia cells start to replicate inside the bone marrow of the mice, giving them leukemia. When the mice were given the new, experimental drug which targets SRPK1, it strongly inhibited the growth of the human AML without any noticeable side-effects on the mice.