Myeloproliferative neoplasms (MPNs) can only be cured, to date, using allogeneic stem cell transplantation which, in turn, only works for up to 20% of patients. As calreticulin (CALR) frameshift mutations are the second most common cause of MPNs, targeting this endoplasmic reticulum resident protein is one of the strategies emerging at the forefront of hematological malignancies research.

Cholangiocarcinoma accounts for about 3% of all gastric tumors. Mutant KRAS is the most prevalent oncogene in cholangiocarcinoma, suggesting a potential role for KRAS inhibitors as a therapeutic approach.

Researchers from Taipei Medical University have presented data from a study that assessed the role of KH-type splicing regulatory protein (KHSRP, also called KSRP) in clear cell renal cell carcinoma (ccRCC).

Even though the treatment options that exist for acute myeloid leukemia (AML) are growing, the clinical outcome of patients is still unfavorable. In AML dysregulation of the tyrosine kinase receptors, including RAS, RAF, MEK and ERK, and of the Aurora kinase family (AURK) exists, are both tied to AML development and progression.

Variants in several subunits of the Mediator protein complex are responsible for MEDopathies, which present variable clinical manifestations and modes of inheritance. Researchers from Université de Paris Cité have investigated the role of MED16, a subunit of the evolutionary-conserved Mediator complex, in MEDopathies.

Atrial septal defect (ASD) is a congenital heart condition where the formation of the connection between the atrial chambers is defective, thus allowing left-to-right shunting and the consequent risk of atrial fibrillation, stroke and heart failure, among others.

Septerna Inc. scientists have reported proof-of-concept data on a novel oral small-molecule thyroid-stimulating hormone receptor (TSHR) antagonist, SP-1351, with potential for the treatment of Graves’ disease (GD), including ophthalmic manifestations.

The therapeutic response of approved KRAS inhibitors is not always durable and patients develop resistance frequently. To enhance this durability, the search for KRAS inhibitors in combination with drugs that target downstream or upstream components of RAS is crucial.