Targeting rearranged during transfection (RET) proto-oncogene may be an effective therapeutic strategy for acute myeloid leukemia (AML) patients with mutations in FLT3 and activated RET. However, few RET-targeting agents have been approved for clinical use, and no FLT3/RET dual-targeting drugs have been identified.

Hoth Therapeutics Inc. has reported promising results from sponsored preclinical research conducted at NC State University (NCSU) with HT-KIT, the company’s new molecular entity for the treatment of advance systemic mastocytosis. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene c-Kit by inducing mRNA frame shifting.

Researchers from the University of Oxford and Oxstem Ltd. have discovered small-molecule candidates with the ability to induce differentiation of acute myeloid leukemia (AML) cells.

Xnk Therapeutics AB has entered into a research collaboration with the Karolinska University Hospital to evaluate the suitability of the company's preclinical autologous natural killer (NK) cell therapy candidate XNK-02 as a novel therapy for treatment of acute myeloid leukemia (AML).

Leukemic cells rely on excessive mitochondrial respiratory and energy metabolism. Therefore, targeting mitochondrial proteases has been proposed as a potential approach to improve therapeutic regimens for acute myeloid leukemia (AML). The mitochondrial caseinolytic protease P (ClpP), located in the mitochondrial matrix, maintains protein quality by mediating the proteolytic hydrolysis of damaged proteins. The chaperone ClpX regulates this hydrolysis and is overexpressed in AML, thus providing a rationale for using ClpP agonists to disrupt AML proliferation.

Researchers at City of Hope were awarded $32.3 million from the California Institute for Regenerative Medicine (CIRM) to support three novel phase I clinical trials evaluating innovative cell and gene therapy treatments for patients with HIV, acute myeloid leukemia (AML) and severe aplastic anemia.