The results of a Phase 1 clinical trial of the experimental drug ivosidenib, led by researchers at The University of Texas MD Anderson Cancer Center, have been published in the New England Journal of Medicine. Ivosidenib is being developed as a cancer treatment, and the study looked at its effect on acute myeloid leukaemia. The full article can be found here, at the MD Anderson Cancer Center website.

Acute myeloid leukaemia (AML) is an aggressive form of cancer that affects the myeloid cells. Myeloid cells are a type of white blood cell that carries out multiple functions, including fighting infections and preventing tissue damage. AML develops when stem cells found in the bone marrow produce too many blast cells (immature white blood cells), causing a decrease in other important cells such as oxygen-carrying red blood cells and platelets that help blood to clot. Approximately 2,600 people in the UK are diagnosed with AML each year, with the most commonly affected age group being those over 65.

The experimental drug ivosidenib has been found in a Phase 1 study to lead to durable remissions in some AML patients. The drug functions by inhibiting an enzyme protein called isocitrate dehydrogenase 1 (IDH1). In 6 to 10% of AML patients, the IDH1 protein is altered. It is thought that using drugs to inhibit the mutated protein may be an effective treatment for this sub-group of AML patients.

The Phase 1 trial was carried out at several centres on patients who had relapsed or refractory AML and the IDH1 protein alteration. A total of 258 patients were enrolled between 2014 and 2017. They each received a daily dose of 500mg of the IDH1 inhibitor drug. The study examined the safety and efficacy of ivosidenib.

The results showed that, in the primary analysis cohort of 125 patients, 41.6% had an overall response (tumour reduction) and 21.6% showed complete remission. 30.4% of this group were in complete remission, although their blood counts had not been fully restored. The study also found that patients had an increased survival rate. The overall survival rate was half of the patients at 18 months, whereas the historical overall survival is under 5 months for people with relapsed AML and two previous therapies.

The researchers wrote, “In the current study, ivosidenib resulted in encouraging rates and durations of complete remission.”

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