Breakthrough Discovery: Researchers Identify New Target for Leukemia Treatment

New Gene Target Identified for Leukemia Treatment

Overview of Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a severe cancer impacting blood cells, causing abnormal growths that disrupt normal blood function. Due to its aggressive nature, AML can lead to death within weeks or months if not treated. Current treatment options, including chemotherapy, radiation therapy, and occasionally stem cell therapy, only offer a cure for about one-third of diagnosed individuals. Consequently, the pursuit of novel treatment options for leukemia is crucial.

Research Findings on SRPK1 Gene

A recent study published in *Nature Communications* by Tzelepis and an international research team highlights the identification of a new gene, SRPK1, associated with acute myeloid leukemia. The researchers explored the gene’s influence on leukemia cells and assessed whether targeting a defective SRPK1 could hinder disease progression.

To investigate the role of the SRPK1 gene and its mutations, the team conducted experiments on isolated cancer cells and utilized mouse models.

Impact of SRPK1 Inhibition

The study revealed that reducing SRPK1 gene expression prompted significant changes in cell proliferation and apoptosis, leading to a decrease in the growth of both human and mouse leukemia cells. Additionally, this inhibition improved survival rates in mice diagnosed with leukemia. Notably, the reduction of SRPK1 expression did not interfere with the formation of normal blood cells over time or affect blood stem cells. Furthermore, the positive outcomes of inhibiting SRPK1 were evident regardless of whether the leukemia stemmed from genetic mutations in this gene.

Implications for Future Treatments

These findings suggest that SRPK1 represents a promising target for leukemia therapy. Developing methods to inhibit SRPK1 in humans, without adverse effects, could yield new, more effective treatment options for acute myeloid leukemia. Such targeted therapies may present fewer side effects compared to conventional chemotherapy and radiation treatments. Moreover, the implications of this research could extend beyond leukemia, potentially leading to advancements in treatments for neovascular eye diseases and other cancers where SRPK1 plays a significant role in gene regulation.

Written by C.I. Villamil
Reference: Tzelepis et al. 2018. SRPK1 maintains acute myeloid leukemia through effects on isoform usage of epigenetic regulators including BRD4. *Nature Communications* 9:5378.