Acute Leukemias

Strength in numbers

Acute myeloid leukemia (AML) is a type of blood cancer that starts in bone marrow, and affects the development of red blood cells, white blood cells, and platelets. There are many different subtypes of AML, which are categorized and treated based on genetic mutations and other features. Acute lymphoblastic leukemia (ALL) is another type of blood cancer that starts in bone marrow, primarily affecting white blood cells.1,2

In adults, AML is the most common acute leukemia worldwide3

AML accounts for about 23% of the world’s leukemia cases per year3

There are about 120,000 new cases of AML per year globally4


A number of genetic factors could be driving, or contributing to, your particular acute leukemia. Understanding which genes are involved in your cancer is important, because it can help your doctor decide on the treatment path that’s best for you.


Certain genetic alterations—including mutations and rearrangements—are known to “drive” acute leukemia, and are often used to predict outcomes and guide treatment selections. Some genetic alterations have targeted therapies indicated for patients with acute leukemia, while others are being studied in clinical trials. Two common genetic alterations are NPM1 mutations and KMT2A rearrangements.


According to NCCN—an alliance of cancer centers that are considered the leading authority in how cancer is managed—AML and ALL care can include any one or a combination of:


    Targeted therapies act on specific genetic mutations or molecules that may be helping cancer cells grow and survive.


    Chemotherapy is a drug treatment that uses chemicals to destroy fast-growing cells by disrupting the way they grow and divide.

    While this kind of treatment kills tumor cells, it can also destroy healthy cells in the process. Determining whether intensive chemotherapy or nonintensive chemotherapy is appropriate for a patient is often the first treatment decision point.


    A bone marrow transplant is a procedure that replaces damaged cells with healthy stem cells.


    For people who lack effective treatment options, clinical trials give early access to investigational therapies that may benefit them. Speak with your oncologist to see if you may be a candidate to participate in a clinical trial associated with acute leukemias, such as AML. Patient advocacy organizations can also be a helpful resource in navigating your treatment options. Additional resources to support you are included here.

Overview of Clinical Trials

What is a clinical trial?

A clinical trial is a monitored study of volunteer participants that measures if an investigational medicine that a company or research organization is developing is safe and effective for the studied condition. Clinical trials can improve upon existing medicines or bring new treatment options to patients in a given disease, push the boundaries of science and help current and future patients lead better, longer lives.

How does a clinical trial work?

Clinical trials are conducted based on a plan, called a protocol, that defines the clinical trial objectives and ensures that they are met. The doctors and professionals overseeing the trial follow strict guidelines to protect participant safety. Each trial site is supervised by a lead investigator, who is an oncologist, and may work with your cancer care team, as appropriate. The data reported from the trial may support approval of the investigational medicine by regulatory agencies, such as the U.S. Food and Drug Administration (FDA), allowing the medicine to be prescribed to future patients.

Why should I participate in a clinical trial?

All available medicines exist because of clinical trial participants. These are a few reasons to participate:

  • Obtain access to promising investigational treatments that may be more effective than those currently available
  • Have a hand in supporting new scientific research
  • Advance investigational medicines that may improve or save the lives of others

Common definitions to describe specific clinical trial settings and development approaches

  • AML that has returned after treatment or remission may be referred to as: relapsed
  • AML that has not responded or has become resistant to treatment may be referred to as: refractory
  • AML when first diagnosed by a licensed physician may be referred to as: newly diagnosed and treatment setting for this patient population may be referred to as frontline therapy
  • Some trials can be studied with one investigational medicine, commonly known as: monotherapy or single agent therapy
  • Other trials may combine more than one method of treatment, including targeted therapies, chemotherapy and/or other standards of care. This is commonly referred to as: combination therapy or multimodality therapy

Often the treatment of acute leukemia depends less on how far the disease has advanced than on the patient’s age and overall health.



Kill most of the leukemia cells in the blood and bone marrow and restore normal blood cell production; to go into remission

  • Chemotherapy
  • Targeted therapy


For people in remission following induction therapy to destroy any remaining leukemia in the body

  • Chemotherapy
  • Targeted therapy
  • Transplant*


Prevent the leukemia from returning

  • Hypomethylating agents
  • Target therapy


When leukemia returns after a period of remission (relapses), the goal of treatment is to achieve remission again

  • Targeted therapy
  • Aggressive chemotherapy followed by transplant*
  • Hypomethylating agents
  • Clinical trial
  • Best supportive care

*For people who meet certain criteria depending on their overall physical health.
†Not everyone will receive maintenance therapy. It may be recommended depending on your type of disease, consolidation, and risk of relapse.


Kura is currently conducting three studies of ziftomenib for patients with acute myeloid leukemias – those with NPM1 mutations and those with KMT2A-rearrangements.

Clinical Trial
Diagnosis Population
How Ziftomenib is Administered

Currently Recruiting

For patients with relapsed or refractory AML that carries an NPM1 mutation (NPM1-mutant AML)

Phase 2

Ziftomenib given once daily to evaluate clinical activity, safety, and tolerability

Currently Recruiting

For patients with newly diagnosed AML or relapsed or refractory AML, with NPM1 mutations or KMT2A rearrangements (NPM1-mutant AML or KMT2A-rearranged AML)

Phase 1

Ziftomenib given in combination with venetoclax and azacitidine or chemotherapy to evaluate clinical activity, safety, and tolerability

Currently Recruiting

For patients with relapsed or refractory NPM1-mutant or KMT2A-rearranged AML

Phase 1

Ziftomenib given in combination with FLAG-IDA, LDAC, or gilteritinib to evaluate clinical activity, safety, and tolerability

Clinical trial participants may be compensated, including for travel, food and lodging costs. Share the clinical trial number with your oncologist if you’re interested in participating in a KOMET clinical trial:

We encourage you to contact patient advocacy organizations for further support.

Patient Resources

*Ziftomenib has not been approved by the FDA.


It’s important to advocate for your care and find the right tools and information for your unique needs. Many people feel empowered when they can play an active role in their treatment plan. To help ensure that you get care that’s suited to you, consider asking your doctor the following questions, and having someone with you to help listen and take notes:

Have I had any tests that show the genetic factors behind my cancer? If so, when and how often should testing occur?

What types of genetic tests are available for my type of cancer? Who administers these tests, and how are they performed?

Are there any targeted treatments available or in clinical trials that target these genetic factors?

Are there any clinical trials that I would be eligible for that we should consider?

View a variety of printable question guides from the Leukemia & Lymphoma Society (LLS).

These are just a few suggestions to get you started. Don’t be afraid to speak up, ask any questions you have, and discuss your care plan with your cancer care team.


During this time, arming yourself with information about your disease can be empowering. National cancer organizations and patient advocacy groups can provide a supportive community to help you get started. Their aim is to help people live longer, healthier lives by advancing research, raising awareness, and providing emotional and financial support to patients and their families.


Get in-depth information on AML and ALL, treatment costs and insurance coverage, finding support groups, and ways to help advance cancer research.


Consult comprehensive, up-to-date guidelines that detail the sequential management decisions and interventions that currently apply to AML and ALL.


Connect with the blood cancer community, along with other patients and caregivers through chats, support groups, and peer-to-peer programs, or speak with a specialist to get personalized disease and treatment information.


ALAN is a global network of patient alliances dedicated to fostering patient advocacy within acute leukemias.


  • 1. National Comprehensive Cancer Network. NCCN Guidelines for Patients: Acute Myeloid Leukemia. 2020. Accessed October 19, 2021. 2. Mayo Clinic. Acute lymphocytic leukemia. Accessed June 17, 2021. 3. Dong Y, Shi O, Zeng Q, et al. Leukemia incidence trends at the global, regional, and national level between 1990 and 2017. Exp Hematol Oncol. 2020;9:14. 4. Yi M, Li A, Zhou L, Chu Q, Song Y, Wu K. The global burden and attributable risk factor analysis of acute myeloid leukemia in 195 countries and territories from 1990 to 2017: estimates based on the global burden of disease study 2017. J Hematol Oncol. 2020;13(1):72. 6. DiNardo CD, Cortes JE. Mutations in AML: prognostic and therapeutic implications. Hematology. 2016(1):348-355. 7. Klossowski S, Miao H, Kempinska K, et al. Menin inhibitor MI-3454 induces remission in MLL1-rearranged and NPM1-mutated models of leukemia. J Clin Invest. 2020;130(2):981-997. 8. Papaemmanuil E, Gerstung M, Bullinger L, et al. Genomic classification and prognosis in acute myeloid leukemia. N Engl J Med. 2016;374(23):2209-2221. 9. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Myeloid Leukemia V.3.2021. © National Comprehensive Cancer Network, Inc. 2021. All rights reserved. Accessed October 19, 2021. To view the most recent and complete version of the guideline, go online to NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 10. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Lymphoblastic Leukemia V.2.2021. © National Comprehensive Cancer Network, Inc. 2021. All rights reserved. Accessed October 19, 2021. To view the most recent and complete version of the guideline, go online to NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 11. American Cancer Society. How chemotherapy drugs work. Revised November 22, 2019. Accessed June 17, 2021. https:// 12. Ferrara F, Barosi G, Venditti A, et al. Consensus-based definition of unfitness to intensive and nonintensive chemotherapy in acute myeloid leukemia: a project of SIE, SIES and GITMO group on a new tool for therapy decision making. Leukemia. 2013;27(5):997-999.13. Mayo Clinic. Acute myelogenous leukemia diagnosis and treatment. Accessed October 15, 2021. 14. Harvard Health Publishing. Leukemia. Published December 12, 2014. Accessed June 17, 2021. 15. National Comprehensive Cancer Network. NCCN Guidelines for Patients: Acute Lymphoblastic Leukemia. 2021. Accessed October 19, 2021. 16. Reville PK, Kadia TM. Maintenance Therapy in AML. Front Oncol. 2021;10:619085. 17. DeWolf S, Tallman MS. How I treat relapsed or refractory AML. Blood. 2020;136(9):1023-1032.