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Immunotherapy for Leukemia

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  • Leukemia
  • Treatment Options
  • CRI's Impact
  • Clinical Trials

What Makes Immunotherapy a Promising Treatment for Leukemia?

Reviewed By: John C. Byrd, M.D.
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D. Warren Brown Chair of Leukemia Research and Professor, The Ohio State University
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Leukemia is cancer of the bone marrow and lymphatic system, and affects both children and adults.  In leukemia, abnormal cells, produced by the bone marrow, begin to overtake and replace the normal blood and marrow cells.

There are four main types of leukemia, each named for the rate at which the disease develops and worsens as well as the affected blood cell type.

  • acute myeloid leukemia (AML): fast-growing cancer that starts in myeloid cells—the precursor to red blood cells, platelets (cells that clot the blood), or white blood cells known as granulocytes; also known as acute myelogenous or myeloblastic leukemia
  • chronic myeloid leukemia (CML): slower-growing cancer that starts in myeloid cells—the precursor to red blood cells, platelets (cells that clot the blood), or white blood cells known as granulocytes; also known as chronic myelogenous or myeloblastic leukemia
  • acute lymphocytic leukemia (ALL): fast-growing cancer that starts in lymphoid cells, which make different types of white blood cells; also known as acute lymphoblastic leukemia
  • chronic lymphocytic leukemia (CLL): slower-growing cancer that starts in lymphoid cells, which make different types of white blood cells; also known as chronic lymphoblastic leukemia

Leukemia occurs most often in adults older than 55 years, but it is the most common cancer in children younger than 15 years. Among adults, the most common types are CLL (35%) and AML (32%). Among children and teens, ALL is the most common, accounting for 75% of pediatric leukemia cases.

In the United States, over 60,300 new cases of leukemia are expected in 2018, and more than 24,400 deaths will occur. Survival rates vary substantially by leukemia subtype, ranging from a current 5-year relative survival rate of 27% for adults diagnosed with AML to 86% for those with CLL, and 66% for children, adolescents, and young adults diagnosed with AML to 92% for those with ALL. In 2018, there are an estimated 381,774 people living with or in remission from leukemia in the United States.

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Leukemia Treatment Options

Treatment for leukemia depends on an individual’s age, cancer type, and the severity of the disease. In most cases, chemotherapy is the first line of treatment for this disease, though some patients require a stem cell transplant to eliminate the leukemia entirely.

In addition to stem cell transplants, there are currently ten approved immunotherapy options for leukemia.

Targeted Antibodies

  • Alemtuzumab (Campath®): a monoclonal antibody that targets the CD52 pathway; approved for subsets of patients with chronic lymphocytic leukemia (CLL)
  • Blinatumomab (Blincyto®): a bispecific antibody that targets CD19 on tumor cells as well as CD3 on T cells; approved for subsets of patients with acute lymphoblastic leukemia (ALL)
  • Gemtuzumab ozogamicin (MyloTarg®): an antibody-drug conjugate that targets the CD33 pathway and delivers toxic drugs to cancer cells; approved for subsets of adult and pediatric patients with CD33-positive acute lymphoblastic leukemia (ALL)
  • Inotuzumab ozogamicin (Besponsa®): an antibody-drug conjugate that targets the CD22 pathway and delivers toxic drugs to cancer cells; approved for subsets of patients with advanced acute lymphoblastic leukemia (ALL)
  • Obinutuzumab (Gazyva®): a monoclonal antibody that targets the CD20 pathway; approved for subsets of patients with CD20-positive chronic lymphocytic leukemia (CLL), including as a first-line therapy
  • Ofatumumab (Arzerra®): a monoclonal antibody that targets the CD20 pathway; approved for subsets of patients with CD20-positive chronic lymphocytic leukemia (CLL), including as a first-line therapy
  • Rituximab (Rituxan®): a monoclonal antibody that targets the CD20 pathway; approved for subsets of patients with chronic lymphocytic leukemia (CLL), including as a first-line therapy

Adoptive Cell Therapy

  • Tisagenlecleucel (Kymriah®): a CD19-targeting CAR T cell immunotherapy; approved for subsets of children and young adult patients with acute lymphoblastic leukemia (ALL)

Immunomodulators

  • Interferon alfa-2a (Roferin®-A): a cytokine that targets the IFNAR1/2 pathway; approved for subsets of patients with hairy cell leukemia and Philadelphia chromosome positive chronic myeloid leukemia (CML)
  • Interferon alfa-2b (Intron A®): a cytokine that targets the IFNAR1/2 pathway; approved for subsets of patients with hairy cell leukemia and aggressive follicular non-Hodgkin lymphoma

New immunotherapies are currently being tested in leukemia clinical trials.

Find a leukemia clinical trial

CRI's Impact in Leukemia

Throughout our history, the Cancer Research Institute has funded numerous studies and clinical trials involving the treatment of leukemia with immunotherapy. Some of these studies have led to discoveries that have the potential to cure several subtypes of leukemia.

Current and past CRI-funded studies on immunotherapy for leukemia include:

  • The first toxin-linked monoclonal antibody targeted toward CD33 on leukemic blasts for acute myeloid leukemia (gemtuzumab ozogamicin, or Mylotarg), was developed by Irving Bernstein, M.D., a CRI postdoctoral fellow from 1972-1974, and was approved by the FDA in 2000.
  • Paola Betancur, Ph.D., a CRI postdoctoral fellow from 2014-2016 in the laboratory of Irving L. Weissman, M.D., at Stanford University School of Medicine, is working to validate and test therapeutic strategies targeting the CD47 protein a “don’t eat me” signal that stops macrophages, a type of immune cell that engulfs and destroys its targets) to treat cancer.
  • Kristen E. Pauken, Ph.D., a CRI postdoctoral fellow at the University of Pennsylvania from 2013-2016, along with Michael A. Farrar, Ph.D., a former CRI postdoctoral fellow and investigator award recipient at the University of Minnesota, found BCR-ABL leukemia actively suppresses immune responses by converting leukemia-specific T cells into inhibitory, regulatory T cells, aiding leukemia’s progression.
  • CRI investigator Hiroyoshi Nishikawa, M.D., Ph.D., at Osaka University found that several cancer-testis antigens, including NY-ESO-1, were highly expressed in adult T cell leukemia/lymphoma (ATLL) and that they could be recognized by killer T cells, providing proof-of-principle that cancer-testis antigens are a promising target for ATLL immunotherapy.
  • CRI investigator Ryan Teague, Ph.D., at Saint Louis University School of Medicine has shown that blockade of CTLA-4, PD-1, and LAG-3 could restore anti-tumor activity in adoptively transferred T cells and result in durable and more effective anti-tumor immunity in advanced leukemia.

Explore CRI’s current funding for leukemia research in our funding directory.

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Featured Patient

Once you choose hope, anything is possible.

Kristin Kleinhofer
Leukemia  |  Diagnosed 2010
Read My Story
Featured Scientist
Zhaoqing Ba, Ph.D.
Boston Children’s Hospital
Postdoctoral Fellow  |  2016
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Leukemia Statistics

4 Main types of leukemia
>55 Years Age most commonly affected by leukemia
#1 Most common cancer in children <15 years old

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Leukemia Clinical Trial Targets

Discover the different proteins, pathways, and platforms that scientists and physicians are pursuing to develop new cancer treatments. Use this information to consider your clinical trial options.

Therapies
  • Targeted Antibodies
  • Cancer Vaccines
  • Adoptive Cell Therapy
  • Immunomodulators
  • Oncolytic Virus Therapy

Targeted antibodies are proteins produced by the immune system that can be customized to target specific markers on cancer cells in order to disrupt cancerous activity, especially unrestrained growth. Antibody-drug conjugates (ADCs) are equipped with anti-cancer drugs that they can deliver to tumors. Bispecific T cell-engaging antibodies (BiTEs) bind both cancer cells and T cells in order to help the immune system respond more quickly and effectively. Antibody targets under evaluation in leukemia clinical trials include:

  • CD19: a receptor found on the surface of almost all B cells that influences their growth, development, and activity; often expressed by leukemia, lymphoma, and myeloma cells
  • CD20: a receptor found on the surface of B cells during their development; often expressed by leukemia, lymphoma, and myeloma cells
  • CD22: a receptor found primarily on the surface of mature B cells; often expressed by leukemia and lymphoma cells
  • CD25 (also known as IL2-R):a cytokine receptor involved in the growth and expansion of immune cells; often expressed by leukemia and lymphoma cells
  • CD30: a receptor that is expressed on certain types of activated immune cells; often expressed by leukemia and lymphoma cells
  • CD33: a surface receptor found on several types of immune cells; often expressed by leukemia cells
  • CD37: a protein found on many types of immune cells; often expressed by leukemia and lymphoma cells
  • CD38: an immune cell surface protein that plays roles in cell adhesion and signaling; often expressed by leukemia and myeloma cells
  • CD52: a protein found on the surface of mature immune cells as well as other cell types
  • CD123 (also known as IL-3R): a receptor found on immune cells that is involved in cellular proliferation and differentiation and is often expressed by leukemia and lymphoma cells

Cancer vaccines are designed to elicit an immune response against tumor-specific or tumor-associated antigens, encouraging the immune system to attack cancer cells bearing these antigens. Cancer vaccines can be made from a variety of components, including cells, proteins, DNA, viruses, bacteria, and small molecules. Cancer vaccine targets under evaluation in leukemia clinical trials include:

  • NY-ESO-1: a protein that is normally produced only before birth, but is often abnormally expressed in cancer
  • Personalized neoantigens: these abnormal proteins arise from mutations and are expressed exclusively by tumor cells; they are unique to each patient
  • Toll-like receptors (TLRs): activation of these innate immune receptors can help stimulate vaccine-like responses against tumors
  • Tumor-associated antigens (TAAs): proteins often expressed at abnormally high levels on tumor cells that can be used to target them; also found on normal cells at lower levels
  • WT1: a protein that is often mutated and abnormally expressed in patients with cancer, especially Wilms’ tumor (WT)

Adoptive cell therapy takes a patient’s own immune cells, expands or otherwise modifies them, and then reintroduces them to the patient, where they can seek out and eliminate cancer cells. In CAR T cell therapy, T cells are modified and equipped with chimeric antigen receptors (CARs) that enable superior anti-cancer activity. Natural killer cells (NKs) and tumor infiltrating lymphocytes (TILs) can also be enhanced and reinfused in patients. Cell-based immunotherapy targets under evaluation in leukemia clinical trials include:

  • CD19: a receptor found on the surface of almost all B cells that influences their growth, development, and activity; often expressed by leukemia, lymphoma, and myeloma cells
  • CD20: a receptor found on the surface of B cells during their development; often expressed by leukemia, lymphoma, and myeloma cells
  • CD22: a receptor found primarily on the surface of mature B cells; often expressed by leukemia and lymphoma cells
  • CD33: a surface receptor found on several types of immune cells; often expressed by leukemia cells
  • CD123 (also known as IL-3R): a receptor found on immune cells that is involved in cellular proliferation and differentiation, and often expressed by leukemia and lymphoma cells
  • Epstein-Barr Virus (EBV)-related antigens: foreign viral proteins expressed by EBV-infected cancer cells
  • ROR1: an enzyme that is normally produced only before birth, but is often abnormally expressed in cancer and may promote cancer cell migration as well as prevent cancer cell death
  • WT1: a protein that is often mutated and abnormally expressed in patients with cancer, especially Wilms’ tumor (WT)

Immunomodulators manipulate the “brakes” and “gas pedals” of the immune system. Checkpoint inhibitors target molecules on immune cells to unleash new or enhance existing immune responses against cancer. Cytokines regulate immune cell maturation, growth, and responsiveness. Adjuvants can stimulate pathways to provide longer protection or produce more antibodies. Immunomodulator targets under evaluation in leukemia clinical trials include:

  • CD47: this surface protein acts as a “don’t eat me!” signal that protects cancer from being consumed by immune cells called macrophages; blocking CD47 can improve the cancer-eating activity of macrophages
  • CD137 (also known as 4-1BB): activating this co-stimulatory pathway can help promote the growth, survival, and activity of cancer-fighting T cells
  • CSF1/CSF1R: blocking this pathway can help reprogram cancer-supporting macrophages
  • CTLA-4: blocking this pathway can help promote expansion and diversification of cancer-fighting T cells
  • CXCR4: blocking this pathway can promote the migration and recruitment of immune cells
  • IDO: blocking this enzyme’s activity may help prevent cancer-fighting T cells from being suppressed
  • IL-2/IL-2R: activating this cytokine pathway can help promote the growth and expansion of cancer-fighting T cells
  • OX40: activating this co-stimulatory pathway can help promote T cell survival after activation
  • PD-1 / PD-L1: blocking this pathway can help prevent cancer-fighting T cells from becoming “exhausted,” and can restore the activity of already-exhausted T cells
  • STAT3: activating this intracellular signaling protein can help stimulate adaptive immune responses

Oncolytic virus therapy uses modified viruses that can infect tumor cells and cause them to self-destruct. This can attract the attention of immune cells to eliminate the main tumor and potentially other tumors throughout the body. Viral platforms under evaluation in leukemia clinical trials include:

  • Adenovirus: a family of common viruses that can cause a wide range of typically mild effects including sore throat, fatigue, and cold-like symptoms
  • Vesicular stomatitis virus: a virus that belongs to the same family as the rabies virus; can cause flu-like symptoms in humans
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Sources: American Cancer Society Cancer Facts & Figures 2018; GLOBOCAN 2012; Leukemia and Lymphoma Society; SEER Cancer Statistics Factsheets: Leukemia (National Cancer Institute); What You Need To Know About™ Leukemia (National Cancer Institute); ClinicalTrials.gov; CRI grantee progress reports and other CRI grantee documents

Updated August 2018

*Immunotherapy results may vary from patient to patient. Consult a healthcare professional about your treatment options.

*Immunotherapy results may vary from patient to patient.

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