A patient being treated with the experimental blood cancer treatment tosedostat along with other drugs has died of myocarditis, an inflammation of the heart usually caused by infection. As a result, the Food and Drug Administration (FDA) has placed a partial hold on research of the drug.
Tosedostat was in mid-stage clinical testing when the death occurred. The FDA has asked for more information on patients being treated with the drug, including more details on the deceased patient, and any cardiac-related effects on patients given the drug.
The clinical trial was being conducted by an investigator-sponsor and not by Cell Therapeutics. The hold federal regulators placed on the drug involves only part of the clinical work. The company says it has started to comply with the FDA’s request.
Tosedostat is a novel, orally administered aminopeptidase inhibitor that deprives tumor cells of the amino acid building blocks they need to make proteins necessary for tumor cell survival. According to Cell Therapeutics, the drug has demonstrated significant anti-tumor responses in blood-related cancers and solid tumors in Phase 1-2 clinical trials.
There are currently two ongoing Phase 2 investigator-sponsored trials examining the clinical activity of tosedostat in combination with other drugs on patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome.