Acute myeloid leukemia: Clinical trials

During the latest Know AML Ambassador Meeting, Professor of Hemato-oncology and Know AML Ambassador, Charles Craddock, delivered an expert-led session providing an overview of clinical trials. 

Professor Craddock discusses the stages, purposes, and processes of clinical trials. Furthermore, he highlights the benefits of involvement, addresses patient concerns about being assigned to the control arm, and empowers patients to participate in clinical trials.

Expert-led session: Overview of clinical trials

During Blood Cancer Awareness Month, Know AML spoke to Gail Roboz, a world-leading doctor in acute myeloid leukemia who is based at Weill Cornell Medicine, New York, US. We asked, "Are there any clinical trials focusing on molecular relapses and treatment?" In this video, Gail describes what a molecular relapse is, and explains that although there are no specific drugs to target molecular relapse, there are ongoing trials into treating relapse in specific molecular diseases such as NMP1-mutated and core binding factor acute myeloid leukemia.

What clinical trials look at molecular relapse?

Treatment, side effects, and clinical trials

Know AML conducted a poll among patients with acute myeloid leukemia (AML), and their families and caregivers, revealing a concern regarding potential side effects that may be experienced during participation in a clinical trial. Know AML was pleased to speak with Gail Roboz, physician at Weill Cornell Medicine and Know AML ambassador, to address this concern. 

Roboz emphasizes that clinical trials can offer improved treatment options beyond the standard of care and reassures patients that they often do not involve completely novel drugs. She encourages patients to compare listed side effects in trial treatments to those of standard of care treatments, and to consider participation in clinical trials to potentially benefit from receiving the investigational drug and to contribute to advancing treatment options.

Addressing concerns around side effects in clinical trials

Know AML was pleased to speak with Harry Erba, Professor of Medicine and Director of the Leukemia Program at Duke University in Durham, North Carolina, US. We asked the following questions:

What sets the myeloMATCH trials apart from previous clinical trials in AML and MDS, and how could it help improve personalized treatment on a global scale?

Why is the speed of genetic testing so critical in the myeloMATCH trial, and how quickly can patients typically receive results?

Erba explains that the myeloMATCH program is a shared effort to develop new drug options for people with AML, by leukemia experts from hundreds of locations across the US and Canada. Since AML can be caused by a range of different genetic mutations which take place in the bone marrow, various drugs are being investigated that can target these specific mutations. The myeloMATCH program includes multiple clinical trials testing these drugs, to help develop the most appropriate treatments, personalized to each individual patient.

Erba also discusses the importance of rapidly identifying a patient’s specific genetic mutations, to help identify the optimal treatment for them as soon as possible after diagnosis. He highlights the value of the myeloMATCH program in helping to speed up this process – genetic testing results typically take up to 14 days to be processed, while patients in a myeloMATCH clinical trial may receive results within 72 hours.

This educational resource is independently supported by Thermo Fisher Scientific. All content was developed by SES in collaboration with an expert ambassador group. Funders were allowed no influence on the content of this resource.

The myeloMATCH clinical trials: Improving personalized treatment in AML

During Blood Cancer Awareness Month, Know AML spoke to Gail Roboz, a world-leading doctor in acute myeloid leukemia. We asked, "Have there been any clinical trials targeting the

gene specifically?" In this video, Gail describes the importance of 

 mutations and how this effects the type of treatment given. She mentions the role of menin inhibitors in early clinical trials for 

Are there clinical trials targeting the NPM1 gene specifically?

During Blood Cancer Awareness Month, Know AML spoke to Gail Roboz, a world-leading doctor in acute myeloid leukemia who is based at Weill Cornell Medicine, New York, US. We asked, "Have there been any ground-breaking discoveries recently with clinical trials in acute myeloid leukemia?" In this video, Gail explains that there have been several recent ground-breaking clinical trials, and focuses on those involving targeted agents to IDH1/2 mutations, FLT3 inhibitors, venetoclax, and the recent U.S. Food and Drug Administration (FDA) approval for CC-486 in acute myeloid leukemia maintenance.

What are the recent discoveries in clinical trials for acute myeloid leukemia?

During the 63rd ASH Annual Meeting and Exposition, Know AML spoke with Mycal Casey, Augusta University, Augusta, US. We asked, Do patients enrolled in clinical trials really represent the acute myeloid leukemia population? 

Casey begins by discussing underrepresentation of Hispanic and African American populations as well as women in clinical trials. He goes on to outline the impact of mortality on clinical trials that lead to drug approval and global disparities in trial populations. Finally, Casey makes 3 suggestions that could improve the applicability of data and representation in clinical trials.

Do patients enrolled in clinical trials really represent the acute myeloid leukemia population?

During the 4th National Cancer Research Institute (NCRI) AML Academy Meeting, Know AML was pleased to speak to Ian Thomas, Cardiff University, Cardiff, UK. We asked, How are acute myeloid leukemia (AML) clinical trials designed to maximize inclusivity and provide data about a wide variety of patients? 

Thomas discusses strategies used to formulate clinical trials to allow for accurately reliable data to be gathered. Thomas goes on to explain how using varying genders, age groups, and trial locations are necessary factors in ensuring the data gathered provides an accurate representation of the general population.

How are clinical trials designed to maximize inclusivity & provide data about a variety of patients?

For AML World Awareness Day 2026, Know AML was pleased to speak with Know AML Ambassadors Erik Aerts, a specialist hematology nurse based in Switzerland, and Charles Craddock, an acute myeloid leukemia (AML) physician based in the United Kingdom. We asked, When might a clinical trial be right for patients, and how can they get involved?

Erik explains that clinical trials can be an option at any stage of the AML journey, including at diagnosis, during remission (when signs of leukemia are no longer detectable), or if the disease comes back (relapses). He encourages patients to ask their doctor whether a clinical trial might be suitable for them. He highlights that clinical trials are closely monitored for safety, completely voluntary, and can give patients access to new treatments which may not be available otherwise.

Charles agrees that clinical trials are essential for improving how AML is treated. Patients who take part in clinical trials often have better results than expected, whether they receive the standard treatment (control drug) or the new treatment being studied. Charles explains that patients can contribute to AML research in other ways, such as by joining patient registries. These registries collect information from patients outside of clinical trials to help doctors to understand more about AML and improve care for future patients.

Thank you to our official sponsors, Thermo Fisher Scientific, Kura Oncology, and Sumitomo Pharma. All content was developed by SES in collaboration with an expert ambassador group. Sponsors were allowed no influence.

This video is for informational or educational purposes only, and does not substitute professional medical advice or consultations with healthcare professionals.

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When might a clinical trial be right for patients, and how can they get involved? 

For AML World Awareness Day 2026, Know AML was pleased to speak with Know AML ambassadors Charles Craddock, an acute myeloid leukemia (AML) physician based in the United Kingdom, and Erik Aerts, a specialist hematology nurse based in Switzerland. We asked, How likely is relapse, and what treatment options are there?

Charles discusses treatments that aim to lower the risk of a person’s AML coming back (relapsing) once they have reached remission (i.e. when signs of leukemia are no longer detectable). For patients under 75 years old who are able to tolerate strong chemotherapy, this treatment – sometimes followed by a stem cell transplant – can lead to long-lasting remission. However, some patients with AML have certain changes in their genes (sections of DNA with instructions for cells on how to work properly) that can make chemotherapy less effective and increase the risk of AML coming back. In these cases, a stem cell transplant is often recommended to help lower this risk.

Charles explains that for patients older than 75 years who are unable to tolerate strong chemotherapy, gentler treatments are often used, such as venetoclax combined with azacitidine. Many patients are able to reach remission with these treatments, but the risk of AML coming back months or years later is high. He emphasizes that preventing relapse is one of the main challenges in AML and highlights the importance of clinical trials that are testing new treatment options. He encourages patients to discuss with their doctor whether a clinical trial might be an option for them.

Erik adds that hearing AML has returned after reaching remission can be emotionally devastating. Although many patients often feel responsible for their relapse, he emphasizes that when AML returns, it is because of the nature of the disease and is not caused by patients’ actions.

How likely is relapse, and what treatment options are there? 

Know AML was pleased to speak with Lizzie Dean, a patient with acute myeloid leukemia (AML) and patient ambassador for Know AML, and Hayley Ellis, senior hematology research nurse, Queen Elizabeth Hospital, Birmingham, UK. They discussed: “What can patients expect when joining a clinical trial?”

Lizzie shares her experiences of participating in two clinical trials: when she was newly diagnosed with acute leukemia (in her case AML) and then again following two relapses. She discusses how she joined the trials, her experiences of attending hospital appointments and treatment side effects, and what her key concerns and questions were through the process.

Hayley provides information on where patients can find information about participating in clinical trials if they are interested in taking part; for example, by discussing with a healthcare professional, contacting cancer charities, or registering with organizations online. She also explains how research nurses and other healthcare professionals can support patients through their treatment and clinical trial journey following diagnosis and gives an overview of how clinical trials might be conducted. For example, the HOVON Foundation has established a network of clinical trials and treatment centers, to advance research in the field of hematologic disease and to expand access to clinical trials for patients across Europe and the UK.

Hayley and Lizzie also provide their key advice for patients considering enrolling in a clinical trial, encouraging patients to fully familiarize themselves with any patient information materials provided by healthcare professionals and undertake their own further research online. They highlight the importance of asking their team of healthcare professionals any questions they might have, so that they can make an informed choice about enrolling in a trial and be fully supported throughout the process.

This educational resource is independently supported by Syndax. All content was developed by SES in collaboration with an expert steering committee. Funders were allowed no influence on the content of this resource.

Hovon. About Hovon. https://hovon.nl/en/about-hovon (https://hovon.nl/en/about-hovon). Accessed September 29, 2025.

What can patients expect when joining a clinical trial? 

Know AML was pleased to speak with Harry Erba, Professor of Medicine and Director of the Leukemia Program at Duke University in Durham, North Carolina. We asked, What are 

mutations, and how do they influence the treatment of acute myeloid leukemia (AML)? Dr Erba explains that 

 mutations are a specific genetic change in the 

gene, which causes over-production of stem cells in the bone marrow and blocks the normal process of stem cells developing into blood cells. This causes a specific subtype of AML, called 

Dr Erba discusses a new group of drugs called menin inhibitors, which block this abnormal process from taking place. He talks about the progress that has been made in clinical trials researching these drugs and explains that one of these menin inhibitors, ziftomenib, has been shown to work well and leads to high rates of remission when given to patients alongside chemotherapy. Dr Erba highlights that research is ongoing but that various drugs are being tested, which will hopefully have positive results for the treatment of people with 

This educational resource is independently supported by Kura Oncology. All content was developed by SES in collaboration with an expert ambassador group. Funders were allowed no influence on the content of this resource.

NPM1 mutations in AML: What are they, and how do they influence the treatment of AML? 

Know AML hosted a webinar for patients and healthcare professionals (HCPs) on April 23, 2025, titled ‘Mutation testing in AML: What you need to know’. Here, we share a discussion between Gail J. Roboz, physician at Weill Cornell Medicine and Know AML ambassador, and Ralph Hills, Know AML chair, where they consider how physicians and patients can communicate more clearly about mutation testing in AML.

Professor Roboz emphasized the importance of providing patients with clear, evidence-based information in a supportive manner, avoiding overwhelming technical language unless requested (

). She highlighted the need for open communication, encouraging questions, and creating a space where patients feel comfortable and empowered to make decisions. Hills enquired about the application of artificial intelligence (AI) in guiding treatment. Professor Roboz explained that though online tools such as Google and AI may seem helpful, they can often mislead, and put forward that, regardless of how much information patients have, patients want to feel genuinely cared for and to know someone is looking out for them, while Professor Craddock noted that AI could be useful in streamlining clinical trials and improving their accessibility for patients. Professor Roboz identified the value of staying informed about the latest treatments, especially for serious conditions such as AML, and encouraged patients to ask critical questions about their disease, diagnosis, and treatment plan (

). Professor Craddock added that collaboration among physicians is also crucial to ensure patients receive the best possible care.

This independent educational activity is supported by Thermo Fisher Scientific.

All content is developed independently by the faculty. The funder is allowed no influence on the content.

Know AML webinar | How can physicians and patients communicate more clearly about mutation testing in AML going forward?

Know AML hosted a webinar for patients and healthcare professionals (HCPs) on April 23, 2025, titled ‘Mutation testing in AML: What you need to know’. Here, we share a discussion between Gail J. Roboz, physician at Weill Cornell Medicine and Know AML Ambassador, and Ralph Hills, Know AML Chair, where they considered the range of genetic mutations in AML and what they mean for treatments, and also how mutation testing methods used in the diagnosis of AML have changed in over the years.

They went on to debate challenges faced by patients during an AML diagnosis, including access to mutation testing and discussions between physicians and patients. They concluded by describing the importance of shared decision-making, and the role of physicians and patients in enabling clearer conversations about mutation testing in AML going forward.

The estimated number of new cases of AML in the US in 2024 was 20,800 – 1% of all new cancer cases in the US. Survival rates have improved steadily over the past 50 years.

Since 2017, many drugs designed to target specific mutations have been approved by the U.S. Food and Drug Administration (FDA) and the European Union (EU).

Patients face several challenges during the process of being diagnosed with AML, including lack of awareness of testing methods, the wait time for test results, and accessing appropriate testing methods; therefore, clear communication between physicians and patients is essential. 

Several mutation testing methods are available, including conventional cytogenetics (karyotyping), fluorescence 

 hybridization (FISH), polymerase chain reaction (PCR), and next-generation sequencing (NGS), and these techniques continue to evolve (

Physicians play a vital role in guiding patients towards appropriate diagnostic tests needed to inform treatment decisions. Providing access to testing methods, whether as part of diagnosis and monitoring in clinics or through clinical trials, is important. For example, the US myeloMATCH trial uses NGS mutation testing to assign patients to treatments that target their specific mutations and then tests how well and safely these treatments work.

Shared decision-making between physicians and patients is crucial to set realistic expectations for treatment and recovery, improve results following treatments, and help patients to feel more confident, independent, and in control of their health (

All content was developed independently by the faculty. The funder was allowed no influence on the content.

Know AML webinar | Empowering patients to ask their physician the right questions during their AML journey

During the latest Know AML community meeting, Professor of Haemato-oncology and Know AML ambassador, Charles Craddock, and fellow Know AML ambassadors discuss important topics from the long term success of stem cell transplants, new research opportunities surrounding the gut microbiome and the impact and importance of psychological monitoring and intervention throughout a patient's journey with AML and during clinical trials.

Community meeting Q&A

In this video, Ralph Hills, a Know AML ambassador who was diagnosed with AML 9 years ago, provides his insights on the quality of life of caregivers, family, and friends of those with AML. 

Ralph leads the discussion with other Know AML ambassadors on the importance of patients having access to specific information that will help them through their AML journey, the time required for patients and caregivers to understand diagnosis, clinical trials, and finances after an AML diagnosis, and the emotional burden on the caregiver journey and effects on caregiver quality of life.

Quality of life of caregivers, family, and friends (including patients' self-perceived burden)

Know AML is happy to share a presentation from Ralph Hills, a Know AML ambassador who was diagnosed with acute myeloid leukemia in 2014. 

In this video, Ralph provides us with a patient perspective of clinical trials by discussing the personal experience of participating in one; from the initial decision to enter a clinical trial, to the impact the trial had on life afterwards.

Experience of participating in a clinical trial

In this video, Dr Karen-Sue Carlson, Medical College of Wisconsin, Wisconsin, US, discusses the purpose of clinical trials and what to expect when participating in one. 

Dr Carlson begins by discussing the different types of clinical trials, how they are regulated, and the ethical principles that drive clinical trial development. Dr Carlson also outlines the process of enrolling in a clinical trial, what it is like to participate in a clinical trial from the patient’s perspective, and the factors that should be considered when deciding to participate.

What is it like to participate in a clinical trial?

During the EHA 2022 Congress, Know AML spoke with Jordi Esteve, Hospital Clínic de Barcelona, Barcelona, ES. We asked, What are targeted therapies and when can they be used in the treatment of acute myeloid leukemia? 

Esteve begins by explaining what targeted molecules are, and what these agents can achieve, including their clinical benefits compared to other therapies. Esteve discusses venetoclax, new therapeutic combinations, and molecular targets being explored in clinical trials to address unmet needs in the acute myeloid leukemia field.

What are targeted therapies and when can they be used in the treatment of acute myeloid leukemia?

During the 61st American Society of Hematology (ASH) meeting in Orlando, US, Know AML were pleased to speak to Charles Craddock, Queen Elizabeth Hospital, Birmingham, UK. We asked Charles Craddock: How can we work to accelerate clinical trials so that more patients can access novel agents sooner? Charles Craddock discusses scientific advances such as genomic stratification to guide treatment pathways and the emergence of novel agents in the field of acute myeloid leukemia. He talks about enhancing and reforming clinical trials so that more patients have access to drugs as soon as possible.

How can more patients access novel agents sooner?

During Blood Cancer Awareness Month, Know AML spoke to Gail Roboz, a world-leading doctor in acute myeloid leukemia. We asked, "Is there any prospect of CAR T-cell immunotherapy treatments becoming available for acute myeloid leukemia?" In this video, Gail describes how CAR T-cells have proved to be a promising modality for other hematological malignancies, before describing their use in clinical trials in acute myeloid leukemia and the results so far.

Are CAR T-cell treatments likely to become available for acute myeloid leukemia?

During Blood Cancer Awareness Month, Know AML spoke to Gail Roboz, a world-leading doctor in acute myeloid leukemia who is based at Weill Cornell Medicine, New York, US. We asked, "What research or clinical trials are ongoing in patients with currently ‘incurable’ acute myeloid leukemia?". In this video, Gail explains what is meant by 'incurable' acute myeloid leukemia, and how these patients might be ideal candidates for clinical trials if they are physically well enough.

You can find additional patient journey resources via our 

What research is ongoing in patients with ‘incurable’ acute myeloid leukemia?

Know AML is delighted to share a series of videos telling the story of Ralph Hills and Dr Gail Roboz. Ralph was diagnosed with acute myeloid leukemia in December 2014 and shortly after he met Dr Gail Roboz, a Professor of Medicine at Weill Cornell Medicine, New York, US.

Diagnosis, signs and symptoms, and initial steps

In this first video, they talk about Ralph's initial diagnosis, signs and symptoms of acute myeloid leukemia, and initial steps for patients who are embarking on their acute myeloid leukemia journey.

In this video, Ralph and Dr Roboz talk about the different treatment options for acute myeloid leukemia, including those that Ralph received, as well as any side effects he experienced along the way. Recent advances in the diagnosis and treatment of acute myeloid leukemia now play an important role in treatment decisions, and Ralph made the choice to take part in a clinical trial (a medical study involving people with the aim to optimize treatments for different diseases).

In this video, Ralph talks about his support network and how a number of factors have helped him through his acute myeloid leukemia journey. Dr Roboz outlines the need for routine follow-up of patients with acute myeloid leukemia in order to achieve the best possible long-term outcomes.

In this video, they talk about what it means to be in 'remission' and what can happen if patients with acute myeloid leukemia 'relapse'. Dr Roboz concludes by highlighting the impact of the COVID-19 pandemic on the availability of treatment and care for patients with acute myeloid leukemia.

What did you think of this patient story? Let us know at 

ses_knowaml@scientificeducationsupport.com

Ralph and Gail’s story

Acute myeloid leukemia causes changes to blood cell counts. The aim of any treatment for acute myeloid leukemia is to help control the abnormal leukemia cells and restore a balance of healthy blood cells.

Acute myeloid leukemia cannot be defined as a single disease, your 

 will vary depending on the type of acute myeloid leukemia you have. Therefore, a variety of therapies are used for different 

 of acute myeloid leukemia and result in different outcomes.

Know AML has put together the animation below summarizing the treatment options available to people with acute myeloid leukemia.

The standard treatment for most people with acute myeloid leukemia is chemotherapy. Chemotherapy involves using drugs to destroy abnormal leukemia cells, hopefully leading to 

. Chemotherapy is usually given in two main phases: induction and consolidation, which are explained in more detail below.

Induction chemotherapy is used in the first phase of treatment, it aims to kill abnormal leukemia cells and stop them from multiplying in the blood and bone marrow. The most common induction chemotherapies include cytarabine and an anthracycline drug (daunorubicin, idarubicin, or mitoxantrone); these are mostly offered to people under the age of 60 years, or people aged over 60 years who are considered ‘fit’. Another treatment used for induction chemotherapy is the combination of daunorubicin and cytarabine (CPX-351), which is approved in the United States and Europe to treat people with specific types of acute myeloid leukemia, including therapy-related acute myeloid leukemia and acute myeloid leukemia with myelodysplasia-related changes. Induction chemotherapy can sometimes be administered with other drugs that target specific changes on or in leukemia cells, known as targeted therapies. 

Consolidation chemotherapy aims to kill and remove abnormal leukemia cells that may remain after induction chemotherapy, but that cannot be detected. This reduces the risk of the leukemia cells multiplying and the subsequent return of acute myeloid leukemia. The consolidation phase involves further treatment with chemotherapy, often with the same drugs that were used in the induction phase.

Allogeneic stem cell transplantation, using a matched sibling or unrelated donor, is increasingly offered to patients with acute myeloid leukemia, with disease likely to relapse if they receive only intensive chemotherapy. In patients with no suitable matched sibling, or when an unrelated donor cannot be found, transplantation using stem cells from either a family member who is not a complete match, or from umbilical cord blood, may be considered. While transplantation reduces the chances of relapse, it can cause several side effects, such as infections, graft-versus-host disease, and other medical issues that can occur months or years after the transplant (“late effects”). However, recent advances in transplant technology, such as improvements in the prevention of GvHD prophylaxis and the use of reduced-intensity conditioning regimens, are resulting in fewer side effects.

What treatments are available for people who are ineligible for intensive chemotherapy?

Intensive chemotherapy is often not well tolerated by patients over the age of 75 years, or patients with other medical issues (comorbidities), which represents the majority of patients diagnosed with acute myeloid leukemia. The most important treatment advance has been the introduction of venetoclax, a BCL-2 inhibitor, which alongside either azacitidine or low-dose cytosine arabinoside, has led to more patients achieving remission and prolonged survival. 

relapsed or refractory acute myeloid leukemia

Currently, treatment approaches for relapsed or refractory acute myeloid leukemia depend on a risk–benefit assessment, which analyzes the likely benefit of further therapy as well as the potential risks associated with it. The treatment decision is based on a number of factors, including age, length of remission, genetics, any previous stem cell transplant, general health status, and the patient’s wishes.

If considered eligible for intensive chemotherapy, treatment options include the following:

IDAC (intermediate-dose cytarabine) with G-CSF (granulocyte colony-stimulating factor), and cladribine, with or without mitoxantrone or idarubicin.

FLAG (fludarabine, cytarabine, and G-CSF), with or without idarubicin and/or venetoclax.

IDAC with etoposide, with or without mitoxantrone.

Clofarabine, with or without cytarabine and/or idarubicin.

CLIA (cladribine, idarubicin, and cytarabine) with venetoclax.

HDAC (high-dose cytarabine) with or without idarubicin, daunorubicin, or mitoxantrone (for people who have not previously received this combination).

If intensive chemotherapy is not considered appropriate, the following treatment options are recommended:

Venetoclax in combination with an HMA (hypomethylating agent) or LDAC (low-dose cytarabine).

Enrollment into a clinical trial may also be an option for people with relapsed or refractory acute myeloid leukemia.

Maintenance therapy refers to lower intensity treatments that may be offered to you following an intensive treatment course or if you are unable to complete intensive treatment. The aim of maintenance therapy is to lower the risk of relapse and prolong survival, while minimizing side effects.

What are the advances in formulations of chemotherapy?

When talking about medicines, a formulation refers to how the different components are combined to make a product that can be given easily. Advances in chemotherapy for acute myeloid leukemia include attempts to make the treatment safer, more effective, or more convenient. With this in mind, improvements to traditional chemotherapy have been developed, such as:

: An oral formulation of azacitidine that was the first approved oral maintenance treatment for acute myeloid leukemia. 

: A formulation of combined daunorubicin and cytarabine that is more easily absorbed by the body.

 A formulation of combined oral decitabine and cedazuridine that is equivalent to intravenous decitabine.

In recent years, a number of new drugs have been developed to target specific markers found in or on leukemia cells. These advances have resulted in targeted drugs, which work differently to standard chemotherapy and can be useful if particular abnormalities have been identified. Generally, targeted therapies have higher specificity and aim to minimize side effects. Some approved targeted treatments for different types of acute myeloid leukemia are shown below.

Approximately 30% of people with acute myeloid leukemia have abnormal changes in a gene called 

. These genetic changes give rise to different forms of the FLT3 protein, which can lead to the growth of leukemia cells.

 A drug that blocks the function of the FLT3 protein. Quizartinib can be used in combination with standard induction and consolidation, and alone as a maintenance therapy following consolidation therapy in patients with a specific type of alteration in the 

gene referred to as internal tandem duplication.

: A drug that targets the abnormal FLT3 protein and blocks its function. Midostaurin can be used with standard chemotherapy to treat people who have abnormal changes in the 

: A drug that binds to the FLT3 protein and blocks its function. Gilteritinib can be used to treat people with relapsed or refractory acute myeloid leukemia who have abnormal changes in the 

In some people with acute myeloid leukemia, abnormal changes are detected in the 

 genes, which interfere with normal cell processes.

: A drug that blocks the function of the IDH1 protein in leukemia cells. Ivosidenib can be used to treat people who have abnormal changes in the 

Another drug that blocks the function of the IDH1 protein in leukemia cells, which can be used to treat people who have relapsed or refractory acute myeloid leukemia with abnormal changes in the 

: A drug that blocks the function of the IDH2 protein in leukemia cells. Enasidenib can be used to treat people who have relapsed or refractory acute myeloid leukemia with abnormal changes in the 

BCL2 is a protein found inside cells that helps leukemia cells to survive.

: A drug that blocks the BCL2 protein, resulting in increased leukemia cell death. Venetoclax can be used to treat people who are aged 75 years and older or those who have other medical conditions that prevent the use of standard chemotherapy.

 genes, a protein called menin interacts with the KMT2A protein to encourage leukemia cells to grow. Mutations in the 

 gene are present in nearly 1 in 3 patients with acute myeloid leukemia, while alterations in the 

 gene occur in 1 in 10 to 1 in 20 patients. Drugs called menin inhibitors prevent menin from interacting with the KMT2A protein and limit the growth of leukemia cells.

A drug that blocks the interaction between the menin and KMT2A proteins, preventing the growth of leukemia cells. Revumenib can be used to treat people who have relapsed or refractory acute myeloid leukemia with abnormal changes in the 

Another drug that blocks the interaction between the menin and KMT2A proteins, which can be used to treat people with relapsed or refractory acute myeloid leukemia who have abnormal changes in the 

In a cell, the ‘hedgehog pathway’ is a finely tuned network of signaling molecules that are important in early-life development. In acute myeloid leukemia, disturbances in this pathway can give rise to cells that are resistant to certain treatments, helping leukemia cells survive.

: A drug that blocks the hedgehog pathway, which can be used to treat people aged 75 years and older or with medical conditions that prevent the use of standard chemotherapy.

Antibody–drug conjugates contain an antibody (a synthetic immune protein that recognizes a specific target on leukemia cells) and an anticancer drug. The antibody is used to guide the drug to the leukemia cells, where it can destroy them.

: A drug made up of an antibody that specifically recognizes CD33 (a protein found on leukemia cells) and a chemotherapy drug.

 are medical studies involving people, they are a key step towards optimizing treatments for different diseases. A clinical trial may be designed with the aim of improving current therapies or to investigate how safe and effective a new treatment is. There are various ongoing clinical trials investigating treatment options for acute myeloid leukemia.

Researchers are also studying whether combining treatments can help even more. In some clinical trials, three medicines are used together (sometimes called “triplet therapy”). These combinations aim to attack leukemia cells in different ways at the same time. For example, a chemotherapy drug might be combined with a targeted therapy and another drug that helps leukemia cells die.

A patient with acute myeloid leukemia may not respond to the therapies that are currently approved, and for certain types of acute myeloid leukemia, there are very few treatment options available. Therefore, taking part in a clinical trial may be the best option for some people and could provide the opportunity for effective disease treatment/management.

What treatment side effects may be expected?

Treatments for any medical condition can cause side effects. The side effects you may experience can vary depending on the treatment (i.e., chemotherapy/targeted therapy) and your overall health. For most people, side effects are temporary and will go away after treatment has finished or following changes to the treatment regimen, such as altered dosing.

Common side effects of treatment for acute myeloid leukemia include tiredness, increased risk of infection (impaired immune system), nausea, vomiting, hair loss, rashes, and low levels of red blood cells (anemia). Your leukemia specialist should advise you of the common side effects associated with any treatment you may receive.

If you have any questions or concerns regarding treatment options, clinical trials, or side effects, reach out to your leukemia specialist.

American Cancer Society. Typical Treatment of Acute Myeloid Leukemia (Except APL). 

https://www.cancer.org/cancer/types/acute-myeloid-leukemia/treating/typical-treatment-of-aml.html

. Updated May 4, 2025. Accessed Feb 10, 2026.

American Cancer Society. Chemotherapy for Acute Myeloid Leukemia (AML). 

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Blood Cancer United. Acute Myeloid Leukemia in Adults: In Detail. 

https://llsorg.widen.net/view/pdf/1s3flqe0dl/en-booklet-adult-aml-detail-ps32.pdf?t.download=true

American Cancer Society. Stem Cell Transplant for Acute Myeloid Leukemia (AML). 

https://www.cancer.org/cancer/types/acute-myeloid-leukemia/treating/bone-marrow-stem-cell-transplant.html

Leukaemia Care. Relapsed or refractory acute myeloid leukaemia (AML). 

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. Updated Mar 31, 2025. Accessed Feb 10, 2026.

National Comprehensive Cancer Network® (NCCN®). Acute Myeloid Leukemia (Version 2.2026). 

https://www.nccn.org/guidelines/guidelines-detail?category=1&id=1411

. Published Nov 24, 2025. Accessed Feb10, 2025. 

American Cancer Society. Targeted Therapy Drugs for Acute Myeloid Leukemia (AML). 

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. Updated Oct 28, 2025. Accessed Feb 10, 2026.

https://www.cancer.org/cancer/types/acute-myeloid-leukemia/treating/targeted-therapy.html#

Leukaemia Care. Relapsed and refractory acute myeloid leukaemia (AML). 

https://media.leukaemiacare.org.uk/wp-content/uploads/Relapsed-and-refractory-AML-Leaflet.pdf

American Cancer Society. Clinical Trials for Cancer Treatment. 

https://www.cancer.org/content/dam/cancer-org/cancer-control/en/booklets-flyers/clinical-trials.pdf

. Updated Jun 3, 2025. Accessed Feb 10, 2026.

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