What is acute myeloid leukemia?

Acute myeloid leukemia (AML) is a rapidly evolving blood cancer that starts in the bone marrow. It is characterized by a swift buildup of immature white blood cells, called myeloblasts, which crowd out healthy blood-forming cells.

As leukemia cells build up in the bone marrow, patients can become increasingly vulnerable to serious infections, bleeding complications and other potentially life-threatening consequences, making prompt treatment critical.

Common symptoms may include ongoing fatigue, frequent infections, easy bruising or bleeding and shortness of breath.

Diagnosing AML

“Initially, the symptoms of AML can resemble everyday illnesses or the effects of aging, so getting a timely diagnosis is often challenging, but it’s also critical,” says Jessica Vermeulen, M.D., Ph.D., Vice President, Lymphoma & Leukemia Disease Area Stronghold Leader, Johnson & Johnson Innovative Medicine.

There is no routine screening test for AML, so diagnosis usually starts with a blood test to identify myeloblasts and a bone marrow biopsy. If AML is confirmed, doctors may also use biomarker testing to look for changes in genes or chromosomes.

“Clinicians often need to make immediate treatment decisions after diagnosis, which is why quickly understanding the biology of a patient’s leukemia is so important,” says Dr. Vermeulen. “We’ve also learned that AML isn’t a single disease, and what looks similar under a microscope can be driven by very different genetic changes. Those differences can influence prognosis, treatment decisions and research approaches.”

Biomarker testing plays a critical role because different genetic and molecular changes can affect how the disease behaves and responds to treatment. Some forms of AML are linked to specific genetic changes, including FLT3, IDH and NPM mutations and KMT2A rearrangements. Identifying these features can help inform prognosis and guide treatment planning.

Acute myeloid leukemia treatments

Once AML is diagnosed, immediate treatment is necessary to destroy the cancerous leukemia cells in the blood and bone marrow. Treatment depends on a person’s age, overall health and the genetic mutations driving their specific leukemia.

Intensive chemotherapy has traditionally been a cornerstone of AML treatment. However, older adults and patients with certain underlying health conditions may face greater risks from intensive treatment, and lower-intensity approaches may also be considered depending on the patient’s circumstances.

“Treating AML requires an individualized approach,” says Christina Loefgren, M.D., Ph.D., Senior Global Medical Affairs Director for Myeloid Diseases, Johnson & Johnson Innovative Medicine. “Clinicians must consider both the biology of the disease and the individual needs of the patient when determining the most appropriate treatment strategy.”

This first, rigorous course of chemo can help some patients reach initial remission, which means that their blood counts have recovered and the number of leukemia cells is greatly reduced. When this happens, high-risk AML patients can also consider a stem cell transplant to keep their AML at bay.

“AML treatment is often an ongoing process rather than a single decision,” says Dr. Loefgren. “Clinicians are continually evaluating how a patient is responding to therapy, their overall health and the risk of the disease returning to determine the best path forward.”

Unmet needs remain despite treatment advances

Even though AML is the most common type of acute leukemia in adults, it has the lowest survival rate. Many patients relapse after initial therapy. Treatment can also be physically demanding, especially for older adults and people with other health conditions.

“The goal isn’t simply to get patients into remission, it’s to help them stay there,” says Dr. Loefgren. “One of the biggest challenges in AML is that even after an initial response, the disease can return. That’s why, at Johnson & Johnson, we’re focused on finding new approaches to improve longer-term outcomes for patients.”

Specifically, more must be done to address the unmet needs in AML, including:

• Achieving more and longer-lasting remissions
• Strong treatment options if AML returns or doesn’t respond to initial treatment, when choices may be limited
• Tolerable approaches for patients who are older or medically fragile
• Treatments that address different AML subtypes, including forms of the disease that are harder to treat

How Johnson & Johnson is advancing acute myeloid leukemia research

Advances in the understanding of AML biology are creating new opportunities to develop more precise treatment approaches aimed at specific proteins or genetic changes that help drive the disease.

As a leader in the treatment of hematologic malignancies, Johnson & Johnson’s research in AML focuses on understanding the drivers of different subtypes, with the goal of developing more precise treatment strategies.

Across newly diagnosed and relapsed AML, the company is studying targeted and combination approaches aimed at improving outcomes for patients, including biologically defined subtypes such as KMT2A-rearranged and NPM1-mutated AML.

“Our goal is a future where an AML diagnosis comes with clearer options, more personalized treatment strategies and greater confidence in what comes next,” says Dr. Vermeulen. “That is why we continue to invest in research aimed at improving our understanding of AML biology and translating those insights into meaningful advances for patients.”