A particular form of leukaemia, acute myeloid leukaemia, or AML, is hard to treat at the best of times – but some patients have a certain genetic mutation that makes their AML more likely to recur and harder to treat.
But since November, a new drug has been available.
Gilteritinib safely treats AML relapses and improves survival rates by more than a third, a study by the University of Pennsylvania found.
Around 6,100 Britons are diagnosed with AML each year, and about half will relapse after a course of treatment.
Their best chance of a cure is a bone marrow transplant, but it only helps between a quarter to a half of patients – if they even survive long enough to receive the live-saving stem cells.
“We’re excited about the survivals we’ve seen,” said lead study author Professor Alexander Perl, oncologist at the University of Pennsylvania.
The three-year survival rate for people who have this kind of leukaemia is less than half. It presents doctors with a problem as it’s extremely hard to treat.
Some treatments only help to keep the cancer in check, but not cure it.
Plus, current drugs have side effects such as nausea and extreme tiredness.
Added to which they sometimes don’t work for very long and the cancer can then develop new mutations and become resistant to treatment.
In short, drugs for this form of AML either work well, but have terrible side effects, don’t work for very long, or don’t target mutations well enough.
“We wanted a drug that checked all the boxes,” said Dr Perl. “Gilteritinib basically does all those things.
“It’s very potent and it’s an oral drug, so you can take it once a day by mouth at anywhere from 10 to 300mg doses.”
And many patients don’t have to take it at that highest dosage for gilteritinib to work, making it truly personalised medicine, Dr Perl says.
Compared to patients on the standard chemotherapy regime, those who took gilteritinib survived two-thirds longer.
But just as importantly, the majority of patients in the gilteritinib trial no longer needed blood transfusions, and could continue taking their cancer drug at home, instead of getting their chemotherapy as an outpatient at a hospital.
All of this suggests that the drug should be “the new standard of care for this population”, said Dr Perl.
“We’ve moved this subgroup from inpatient to outpatient care, and the toxicity is very compatible with outpatient management,” he added.
This is quite a substantial step forward for patients, by any criteria.
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