Latest advancements in treatment for acute myeloid leukemia

By Nicole Ferrara • March 2, 2023

Estimated reading time: 4 minutes

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By Jessica Saenz

Acute myeloid leukemia, also called acute myelogenous leukemia (AML), is the most common type of acute leukemia in adults, and it can progress quickly if not treated. Chemotherapy can sometimes successfully treat AML, but new treatments and tailored chemotherapy approaches are helping even more people achieve remission.

James Foran, M.D., a Mayo Clinic oncologist, explains these treatments and who can benefit from them.

Understanding acute myeloid leukemia

Acute myeloid leukemia is a blood and bone marrow cancer that most often affects myeloid cells. Healthy myeloid cells mature to become red or white blood cells or platelets. But when DNA mutations develop in these cells, they can sometimes grow and divide when they shouldn't, which results in crowding of healthy cells.

Unlike chronic myeloid leukemia, which develops more slowly, AML progresses rapidly. "Acute leukemias come on more quickly," says Dr. Foran. "People come in sick, worn down, and their blood counts are very off. They often have risks of infection or bleeding because of how the leukemia grows. So, it's the acuity of the situation that lends itself to the name 'acute leukemia.'"

Tailoring chemotherapy for acute myeloid leukemia to each patient

Chemotherapy is an effective treatment for many types of cancer, including AML. "We still use chemotherapy-based treatments because they work. We have a history, dating back almost four decades, showing that you can get people with AML into remission," says Dr. Foran.

But chemotherapy can be hard on the body, and how it is delivered can greatly improve how well people tolerate it. "Younger adults traditionally benefit from chemotherapy and tolerate treatment very well. In older adults, we've learned that some new lower-dose strategies can be almost as effective in achieving remission as intensive chemotherapy. And they're much more tolerable for someone 60 or 70 years old — especially if they have comorbid diseases like heart disease," he says.

He adds that because chemotherapy is so effective in treating AML, it's almost always worth trying. This new approach to chemotherapy delivery can often reduce fatigue, damage to the immune system and other side effects.

Targeting treatment by acute myeloid leukemia subtype

Like other types of cancer, AML can behave differently according to its genetic characteristics and subtype. But through genetic testing of leukemia cells, cancer experts are learning how to target them better. "There have been massive advances in the last five or seven years. We now understand that almost everybody with acute myeloid leukemia will have some genetic abnormality in the cells," says Dr. Foran. "In some patients, we can target a particular mutation with available new drugs."

Dr. Foran says he and his team routinely test leukemia cells for genetic mutations and other unique characteristics. "At the time of diagnosis, we do a bone marrow biopsy to see what chromosomal abnormalities or DNA mutations are in the leukemia cells. That helps us determine if the cells will be sensitive to chemotherapy or resistant to it. And there are some mutations we can target with specific drugs — either on their own or in addition to chemotherapy."

When specific mutations are present, Dr. Foran says combining targeted drugs with chemotherapy can make treatment more effective and improve long-term survival. If a mutation indicates that standard treatment might be less successful or that the subtype of AML has a higher likelihood of recurrence, mutations can guide a more effective treatment strategy using other types of chemotherapy or immunotherapy.

The future of acute myeloid leukemia treatment

Dr. Foran anticipates that targeted therapy will continue to improve AML treatment. "As we learn more about the mutations that contribute to AML, we're studying treatments that target cells with those mutations," he says.

In addition to targeted drugs, Dr. Foran says therapies that harness the power of the immune system to attack cancer cells will also play a more significant role. One treatment that has proven results is a bone marrow transplant. "For many patients who go into remission with AML, a bone marrow transplant from a donor is the best way to prevent it from returning. Bone marrow transplant is a form of immunotherapy; it provides new bone marrow and a new immune system," he says.

Dr. Foran says cancer researchers at Mayo Clinic are also exploring how CAR-T cell therapy, which can be used to treat other types of leukemia and lymphoma, could help patients with AML. "I think there's going to be an increasing role in leveraging someone's immune system to help it recognize the cells and treat them," he says. "I think that will be the real focus in the next five to 10 years."

Watch Dr. Foran discuss the latest treatment for acute myeloid leukemia on this "Mayo Clinic Q&A" podcast video: