To date, there are no current orally available compounds that promote bone formation for treating osteoporosis; most treatments act by inhibiting osteoclastic bone resorption, leading to increased bone mineral density and reduced hip fracture rate in a modest way. Analogues of parathyroid hormone (PTH) are the most frequently used bone anabolic agents, which even though effective, they require daily injections.

Invectys Inc. and CTMC, a joint venture between MD Anderson Cancer Center and National Resilience Inc., have announced FDA clearance of an IND application for a phase I/IIa study of IVS-3001, Invectys' lead engineered human leukocyte antigen A (HLA-G)-targeting chimeric antigen receptor (CAR) T-cell therapy for the treatment of solid tumors.

The histone-lysine N-methyltransferase 2A (KMT2A; also known as mixed-lineage leukemia 1 [MLL1])-fusion proteins require direct interaction with the nuclear scaffolding protein menin in order to form menin-KMT2A complex, which plays a key role in the transcription of multiple leukemogenic target genes. On the basis of this, it is hypothesized that blocking the menin-KMT2A interaction by small-molecule inhibitors could be a promising new strategy for the treatment of KMT2A-altered and NPM1-mutant acute myeloid leukemia (AML).

Type I JAK2 inhibitors improve symptoms and outcomes of patients with myeloproliferative neoplasms (MPNs), but mutant allele JAK2 VF remains unchanged with this therapy. Type II JAK2 inhibitors bind the inactive conformation of the kinase domain and reduce the fraction of JAK2 VF mutant allele in vivo, suggesting an improved approach for JAK2 inhibition. Ajax Pharmaceuticals Inc. presented preclinical data on the type II JAK2 inhibitor AJ1-10502 for the treatment of MPNs.

Chimeric antigen receptor (CAR) T cells are astounding. In B-cell cancers, they have been transformative. Yet engineering-wise, CAR T cells are in the equivalent of the Model T era. CAR T-cell engineering has already evolved, with the addition of costimulatory domains, which affect cell expansion and signaling. But once the cells are injected into a patient, there is really no way to affect their behavior.

Rearrangement of the KMT2A gene is a recurrent mutation in acute myeloid leukemia (AML) that leads to poor outcomes in patients due to increased rate of relapsed and refractory disease. The identification of novel targets and potential therapies is crucial for patients with KMT2A-rearranged leukemias.