Overview of Lower-Risk Myelodysplastic Syndromes (LR-MDS)

In general, mutations can occur in several genes in cytologically normal AML cells, including NPM1, FLT3, CEBPA, MLL, NRAS.

Secondary malignancies following CAR T-cell therapy have been a known risk, but an FDA investigation and call for labeling change have renewed interest.

The FDA granted a rare pediatric disease designation to a promising new drug, SLS009, for the treatment of pediatric leukemia.

Patients with chronic myeloid leukemia can have a near-normal life expectation when treated with BCR::ABL1 tyrosine kinase inhibitors.

SL-172154 is being studied in a phase 1a/1b study in acute myeloid leukemia and higher-risk myelodysplastic syndrome, as well as other phase 1 studies in platinum-resistant ovarian cancer.

An investigational new drug application for lomonitinib has been cleared by the FDA for FLT3-mutated relapsed/refractory AML treatment, and a phase 1 trial evaluating the agent will begin in the US.