Drug ‘cures’ 33% of patients with aggressive leukaemia in early trial

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An experimental pill for leukaemia has shown promise in early clinical trials — sending a third of patients with aggressive disease into complete remission. The drug — revumenib — was also credited with putting half of patients with acute myeloid leukaemia (AML) into partial remission during phase one clinical trials.

An experimental pill for leukaemia has shown promise in early clinical trials — sending a third of patients with aggressive disease into complete remission.

He said if more rigorous trials are successful, his team could apply for Food and Drug Administration approval by the end of this year. The initial trial did not include a control group, meaning the drug was not tested against current treatments. They also divide quickly and spread to different areas of the body, which leaves doctors struggling to eliminate them without harming healthy tissue.

In leukaemia with these mutations, a protein called menin binds to genes that trigger cancerous cells to keep growing and dividing. Another 32 patients were also shown to have partial remission, or a decrease in the size of tumors or the extent of cancer in the body.Overall, patients survived for about seven months after the start of the study.

Doctors say that those who have NPM1 leukaemia tend to have better responses to chemotherapy treatments. For all types combined, 9,900 people in the UK were diagnosed with leukaemia in 2015, Cancer Research UK statistics reveal. Symptoms are generally vague and get worse over time. These can include tiredness, frequent infections, sweats, bruising, heavy periods, nose bleeds or bleeding gums, palpitations, and shortness of breath.

Pluvicto, used to treat advanced prostate cancer, and injectable methotrexate, cisplatin, and fluorouracil — chemotherapy drugs that target many different cancers — are all becoming scarce, according to the United States Food and Drug Administration . A fifth drug, BCG, used to treat bladder cancer, is also dwindling, according to the University of Minnesota.

“Current drug shortages also are impacting ever more important medications, not just older generics that are no longer first-line therapies. For 2023, the number of drug shortages is forecast to reach a record number,” says Pilz. Pluvicto is currently made in just a single factory in Italy, notes Pilz. “Because it is a radiopharmaceutical , it can only be produced in small batches within a few days of when it needs to be delivered to patients. Any interruption in production or delivery may delay doses,” he says.

 

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