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

A healthcare professional holding a child's arm, preparing for a medical examination.
An elderly person seated with a medical profession who is placing a hand on their shoulder in a supportive gesture.

In adults, AML is the most common acute leukemia worldwide3

AML accounts for about 31% of the world’s leukemia cases per year3

There are about 144,000 new cases of AML per year globally4

In children and young adults under 20 years old, ALL makes up 76% of acute leukemia cases5

YOUR UNIQUE ACUTE LEUKEMIA6

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.

WHAT DRIVES YOUR AML?6-8

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 approved for patients with acute leukemia, while others are being studied in clinical trials. Two common genetic alterations are NPM1 mutations and KMT2A rearrangements.

A TAILORED TREATMENT PLAN9,10

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 THERAPY11

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

  • CHEMOTHERAPY11,12

    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.

  • BONE MARROW TRANSPLANT13

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

  • CLINICAL TRIALS13

    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

Overview

What is a clinical trial?

A clinical trial is a monitored study of volunteer participants that measures the efficacy and safety of an investigational medicine that a company or research organization is developing for the studied condition. Clinical trials may improve upon existing medicines or bring new treatment options to patients in a given disease, push the boundaries of science and potentially 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. For cancer trials, 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 an application for marketing 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, if approved.

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
  • Have a hand in supporting new scientific research that may benefit future patients

Talk to your doctor about whether participating in a clinical trial is right for you.

Common definitions to describe specific clinical trial settings and development approaches in AML

  • 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
STANDARD TREATMENT OF MANY LEUKEMIAS GENERALLY OCCURS IN PHASES14

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

PHASE OF THERAPY
OBJECTIVE/INTENT
TREATMENT OPTIONS

INDUCTION THERAPY1,13,15

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

CONSOLIDATION THERAPY1,13,15

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

  • Chemotherapy
  • Targeted therapy
  • Transplant*

MAINTENANCE THERAPY†,1,15,16

Prevent the leukemia from returning

  • Hypomethylating agents
  • Target therapy

THERAPY FOR RELAPSE2,15,17

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.

**Kura Oncology is providing this information to you for informational purposes only and does intend to provide medical advice. A patient should always consult with their treating doctor and care team to determine an appropriate treatment plan which may or may not include participation in a clinical trial.

† Not everyone will receive maintenance therapy. It may be recommended depending on your type of disease, consolidation, and risk of relapse.

ADVOCATING FOR YOUR CARE*

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 questions like the following and having someone with you.

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?

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.

* Kura Oncology cannot provide medical advice, treatment options, care, or make clinical recommendations. The information here is not intended as medical advice and you should always rely on your personal healthcare team for medical advice, diagnosis, and treatment plans.

PATIENT AND CAREGIVER RESOURCES

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.

AMERICAN CANCER SOCIETY

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

LEARN MORE

NCCN PATIENT GUIDELINES

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

LEARN MORE

LEUKEMIA & LYMPHOMA SOCIETY

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.

LEARN MORE

ACUTE LEUKEMIA ADVOCATES NETWORK (ALAN)

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

LEARN MORE

REFERENCES

  • 1. National Comprehensive Cancer Network. NCCN Guidelines for Patients: Acute Myeloid Leukemia. 2024. Accessed June 12, 2024. 2. Mayo Clinic. Acute lymphocytic leukemia. Accessed June 17, 2021. https://www.mayoclinic.org/diseases-conditions/acutelymphocytic-leukemia/symptoms-causes/syc-20369077 3. Yun Z, Zhang Y, Wang J, et al. Global, Regional and National Burden of Leukemia, 1990–2021: Analysis of Data from the Global Burden of Disease Study 2021. Blood. 2024;144(Suppl 1):7907-7908. Abstract 908 4. Zhou Y., Huang G., Cai X., Liu Y., Qian B., Li D. Global, regional, and national burden of acute myeloid leukemia, 1990–2021: A systematic analysis for the global burden of disease study 2021. Biomark. Res. 2024;12:101. doi: 10.1186/s40364-024-00649-y 5. Cancer.org. Cancer Facts & Figures 2024. Accessed June 12, 2024. https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2024/2024-cancer-facts-and-figures-acs.pdf 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.org. 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.org. 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:// www.cancer.org/treatment/treatments-and-side-effects/treatment-types/chemotherapy/how-chemotherapy-drugs-work.html 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. https://www.mayoclinic.org/diseases-conditions/acute-myelogenous-leukemia/diagnosis-treatment/drc-20369115 14. Harvard Health Publishing. Leukemia. Published December 12, 2014. Accessed June 17, 2021. https://www.health.harvard.edu/cancer/leukemia 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.