13 December 2023

The prospects for acute myeloid leukaemia (AML) patients are changing for the better as new drugs and targeted therapies are introduced, a haematologist has said.

Dr Justin Watts, of Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine, USA, said there has been more progress in the past 10 years than the previous four decades combined – especially for older AML patients.

Leading an educational session at ASH 2023, the annual meeting of the American Society of Hematology, in San Diego, Dr Watts highlighted new drugs, such as venetoclax plus azacitidine, and new targeted therapies resulting from research advances.

AML patients have been treated with intensive chemotherapy and a bone-marrow or stem-cell transplant, which tend to be more effective in people under age 60.

“We can cure about 60% of younger patients now, which is significantly better than just two decades ago,” said Dr Watts. “But older patients, depending on their fitness level, don’t usually tolerate these treatments and historically less than 10% were cured, but this is now pushing 30% with the advent of venetoclax plus azacitidine and targeted inhibitors.”

Until recently, next steps for these patients were limited to supportive care and blood transfusions.  However, the outlook has improved, especially for older patients, with the emergence of new drugs and targeted inhibitors for the mutations driving AML.

“AML is almost always driven by mutations acquired over time,” said Dr Watts. “That’s why the risk of AML increases as we age.”

Although there are hundreds of mutations that can cause AML, there are five more common ones that are targetable: IDH1, IDH2, FLT3, NPM1 and MLL, all of which have approved therapies – or have ones in development.

“We’re seeing very promising results in our studies and trials, combining venetoclax, azacitidine and targeted therapies, often as frontline therapy for AML,” said Dr Watts.

Combining venetoclax and azacitidine is producing good outcomes in about 52% of older patients, and the median survival is more than two years in these patients, with some patients living much longer.

Targeted therapies are also proving effective, even in relapsed patients, and these are better tolerated than chemotherapy.

Dr Watts told the conference the future direction for AML treatment will involve targeted therapy combined with the “best backbone we have, possibly chemotherapy for younger patients or the drugs like venetoclax and azacitidine generating good results for older adults”.

He also said there could be potential in immunotherapy. “I can see us getting the immune system more involved in treating these blood cancers, as it has done with solid tumours and lymphoma,” he added.

Source: ASH December 2023

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