New Vulnerability in Blood Cancer Cells Offers Hope for Targeted Therapies

Researchers at Karolinska Institutet have uncovered a critical weakness in specific blood cancer cells that could lead to more precise treatment options. By identifying a genetic mutation in the SF3B1 gene, scientists discovered that these cancer cells improperly process their genetic material, resulting in the defective production of an essential protein called UBA1. This deficiency impairs the cells' ability to manage other proteins, rendering them more susceptible to targeted drugs.

In their study, the team used a drug called TAK-243, which inhibits UBA1. They observed that while the drug effectively eliminated the mutated cancer cells, healthy cells with normal UBA1 levels remained unaffected. These findings were validated across multiple experimental models, including cells derived from patients with myelodysplastic syndrome (MDS), a challenging blood cancer primarily affecting older adults.

MDS is difficult to treat, with current options mainly aimed at symptom relief, such as managing anemia. Stem cell transplants can be curative but involve significant risks and are often not suitable for many patients. The discovery of this vulnerability opens the possibility for developing targeted therapies that specifically attack cancer cells carrying the SF3B1 mutation.

The research utilized advanced laboratory models, transforming induced pluripotent stem cells from MDS patients into blood cells, enabling detailed study without dependence on scarce patient material. Moving forward, the team plans to explore combination therapies to enhance effectiveness and hopes to translate these findings into clinical treatments for MDS.

This breakthrough offers hope for more precise and less invasive treatments for blood cancer patients, addressing an urgent need for targeted options beyond current practices.

Source: https://medicalxpress.com/news/2025-09-weakness-blood-cancer-cells-pave.html