Leukemia

Treatments & research overview

What researchers are actively studying

Researchers are testing improved venetoclax-based combinations in AML. Multiple MyeloMATCH trials are comparing venetoclax with azacitidine versus cytarabine-based induction in younger intermediate-risk patients (NCT05554393) and in older FLT3-mutated AML patients with gilteritinib (NCT06317649). A Phase 3 trial (NCT06578247) is evaluating quizartinib, a FLT3 inhibitor, added to chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation → in FLT3-ITD-negative AML. A Phase 2 trial (NCT06497062) is testing sonrotoclax, a next-generation BCL-2 inhibitor, combined with chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation → in newly diagnosed AML. A Phase 3 trial of nilotinib combined with pegylated interferon alfa-2a in CML (PETALs) showed increased rates of deep molecular response with the combination, at the cost of additional toxicity. In CLL, a Phase 3 trial (NCT04608318) is directly comparing ibrutinib monotherapy, venetoclax plus obinutuzumab, and ibrutinib plus venetoclax as first-line options. A Phase 2 trial (NCT05536349) is evaluating the triplet combination of pirtobrutinib, venetoclax, and obinutuzumab in treatment-naive CLL. In ALL, a Phase 3 study (NCT02003222) is adding blinatumomab to standard chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation → for newly diagnosed BCR-ABL-negative B-cell ALL. Novel cellular therapies include CD70-targeted chimeric antigen receptorA type of immunotherapy that genetically engineers a patient's own T cells to recognise and destroy cancer cells.Click for full explanation → natural killer (CAR-NK) cell therapy for AML in Phase 1 (NCT06696846) and neoantigen-specific T cell receptor-transduced peripheral blood lymphocytes in hematologic malignancies (NCT06904066). A case report described zanubrutinib monotherapy achieving early responses in hairy cell leukemia variant, a rare B-cell malignancy with limited relapsed-setting options. Preclinical research identifies CD179a and CD81 as potential leukemia-specific therapeutic targets.

Where the evidence stands

Venetoclax-based regimens in AML and CLL have strong Phase 3 support and have shifted treatment practice, particularly for older patients. BTK inhibitors in CLL are backed by multiple Phase 3 trials. Targeted therapiesDrugs that block specific molecules that cancer cells need to grow.Click for full explanation → for CML have decades of evidence and long-term follow-up data showing durable molecular responses. CAR-T cell therapyA type of immunotherapy that reprograms a patient's own T cells to attack cancer.Click for full explanation → is well-established in relapsed or refractory B-cell ALL and is now being extended to AML and other leukemia subtypes, primarily at Phase 1 and Phase 2 stages. Next-generation inhibitors such as pirtobrutinib and sonrotoclax are at Phase 2 and Phase 3, with results still maturing. Neoantigen-targeted cellular therapies and CAR-NK approaches represent more experimental frontiers in early Phase 1 exploration.

What this means for people affected

Leukemia outcomes vary substantially by type, molecular subtype, and patient fitness. For CML, TKIs have made long-term disease control achievable for many people, with some reaching deep enough responses to attempt treatment discontinuation under careful monitoring. For CLL, newer targeted agents have largely replaced chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation → as the standard for many patients, offering more manageable long-term therapy. AML remains harder to treat, particularly in older patients, though venetoclax-based combinations have meaningfully expanded options for those unable to tolerate intensive chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation →. The active trial landscape suggests that combination strategies, next-generation targeted agents, and novel cell-based therapies will continue to advance outcomes, especially in high-risk and relapsed settings.

Last updated May 4, 2026

Recent research findings

Understanding evidence levels
PreclinicalLab or cell studies — no human data yet.
Animal StudyResults in animals only — may not apply to humans.
Phase 1 TrialFirst-in-human safety testing in small groups.
Phase 2 TrialEarly effectiveness testing in a larger group.
Phase 3 TrialLarge controlled trial — the strongest trial evidence.
Observational StudyPatterns observed in populations — not a controlled trial.
ReviewSummary analysis of multiple existing studies.
Meta-AnalysisStatistical pooling of results from multiple studies.
Phase 2 TrialTests early effectiveness in a larger group after phase 1 safety is established.Est. completion: October 20, 2029

Testing Nemtabrutinib and Lisocabtagene Maraleucel for Relapsed/Refractory CLL/SLL

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying the combination of nemtabrutinib and lisocabtagene maraleucel for people with chronic lymphocytic leukemia/small lymphocytic lymphoma that has come back or doesn't respond to treatment. The trial is currently recruiting participants. The results of this trial are not yet available.

Why it matters: This trial may help researchers understand if the combination of nemtabrutinib and lisocabtagene maraleucel is effective for treating relapsed or refractory CLL/SLL.

Recruiting participants

ClinicalTrials.gov
Phase 1 TrialFirst-in-human trial. Focuses on safety and dosing in small groups.Est. completion: May 5, 2040

Testing a New Treatment for Relapsed B-Cell Malignancies

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying a new treatment called CD19.CAR Allogeneic NKT cells for patients with certain types of B-cell cancers that have come back or don't respond to other treatments. The trial is currently active but not recruiting new participants. The goal is to see how safe and effective this treatment is.

Why it matters: This trial may help us better understand how to treat patients with relapsed B-cell malignancies.

Active but not recruiting new participants.

ClinicalTrials.gov
Phase 2 TrialTests early effectiveness in a larger group after phase 1 safety is established.Est. completion: August 10, 2031

Testing Acalabrutinib and Venetoclax for Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying the combination of two medications, acalabrutinib and venetoclax, to treat people with chronic lymphocytic leukemia or small lymphocytic lymphoma who have not responded well to previous treatments. The trial is currently recruiting participants. The results of this trial are not yet available.

Why it matters: This trial aims to find a more effective treatment option for people with refractory or recurrent chronic lymphocytic leukemia or small lymphocytic lymphoma.

Recruiting participants

ClinicalTrials.gov
Phase 1 TrialFirst-in-human trial. Focuses on safety and dosing in small groups.Est. completion: May 19, 2028

Testing New Treatment for High-Risk Blood Cancers

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying a new treatment for patients with high-risk acute myeloid leukemia, acute lymphoblastic leukemia, and myelodysplastic syndrome. The treatment involves a monoclonal antibody and chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation →, followed by a stem cell transplant. The trial is currently recruiting participants.

Why it matters: This trial may help doctors develop a more effective treatment for patients with these blood cancers.

Recruiting participants, early-stage trial with limited data available.

ClinicalTrials.gov
Phase 1 TrialFirst-in-human trial. Focuses on safety and dosing in small groups.Est. completion: December 1, 2043

Testing a New Treatment for High-Risk T-Cell Cancers

Researchers are studying a new treatment called CD7-Specific Car T Cells for people with high-risk T-cell malignancies. This treatment uses a patient's own immune cells to fight cancer. The trial is currently recruiting participants.

Why it matters: This trial may help find a more effective treatment for people with high-risk T-cell cancers.

Recruiting participants

ClinicalTrials.gov
Phase 1/2 TrialCombined safety and early effectiveness trial in humans.Est. completion: December 31, 2026

Study on Heart Health After Cancer Treatment

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is investigating how a medication called Dexrazoxane Hydrochloride affects heart health in people who have had cancer treatment. The study is currently recruiting participants. Researchers will be monitoring various biomarkersA measurable biological signal used to detect disease or predict treatment response.Click for full explanation → associated with cardiomyopathy and heart failure.

Why it matters: This trial aims to improve our understanding of heart health after cancer treatment, which is crucial for patients who have undergone chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation → or other cancer therapies.

Recruiting participants

ClinicalTrials.gov
Phase 1 TrialFirst-in-human trial. Focuses on safety and dosing in small groups.Est. completion: January 1, 2027

Testing a New Treatment Combination for Higher-Risk Myelodysplastic Syndromes

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying a new combination of medications, venetoclax and azacitidine, in people with higher-risk myelodysplastic syndromes who have not received treatment before. The trial is currently active but not recruiting participants. The goal is to learn more about the safety and effectiveness of this treatment combination.

Why it matters: This trial may help researchers understand if the combination of venetoclax and azacitidine is a safe and effective treatment option for people with higher-risk myelodysplastic syndromes.

Active but not recruiting participants.

ClinicalTrials.gov
PreclinicalLab or cell studies. No human data yet.Est. completion: July 1, 2027

Evaluating Quality of Life in Older Adults with Acute Myeloid Leukemia

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying the quality of life in older adults with acute myeloid leukemia. The trial is currently active but not recruiting participants. The results of this study have not been published yet.

Why it matters: This trial aims to improve our understanding of the quality of life in older adults with acute myeloid leukemia.

The trial is currently active but not recruiting participants.

ClinicalTrials.gov
Observational StudyPatterns observed in populations over time, not a controlled experiment.Published: May 13, 2026

New Imaging Test Improves Breast Cancer Diagnosis

Researchers studied a new imaging test called positron emission mammography (PEM) to see if it helps diagnose breast cancer more accurately. They compared PEM to two other imaging tests, MRI and CESM, and found that PEM improved diagnosis in some cases. However, the study had limitations and more research is needed.

Why it matters: This finding may help doctors make more accurate diagnoses and choose the best treatment options for patients with breast cancer.
Efficacy

PEM demonstrated 100% sensitivity and higher specificity compared to MRI and CESM.

This was a retrospective study with a small sample size of 108 women.

PubMed
Phase 2 TrialTests early effectiveness in a larger group after phase 1 safety is established.Est. completion: July 1, 2035

Evaluating Asciminib for Newly Diagnosed Chronic Myeloid Leukemia

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying the effectiveness and safety of a new treatment called asciminib for patients with newly diagnosed chronic myeloid leukemia in chronic phase. The trial is currently recruiting participants. The results of this study will help determine if asciminib is a viable treatment option.

Why it matters: This trial aims to provide more information about the potential benefits and risks of asciminib for patients with chronic myeloid leukemia.

Recruiting participants

ClinicalTrials.gov
PreclinicalLab or cell studies. No human data yet.Est. completion: September 30, 2030

Evaluating Asciminib for Chronic Myeloid Leukemia in Germany

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying the safety, effectiveness, and tolerability of asciminib in patients with a specific type of leukemia in Germany. The trial is currently recruiting participants. The results of this study have not been determined.

Why it matters: This trial may provide valuable information about the potential benefits and risks of asciminib for patients with chronic myeloid leukemia.

Recruiting status

ClinicalTrials.gov
Phase 1 TrialFirst-in-human trial. Focuses on safety and dosing in small groups.Est. completion: May 1, 2029

Testing Blinatumomab and Asciminib for Acute Lymphoblastic Leukemia

Researchers are studying the combination of Blinatumomab and Asciminib in patients with a specific type of leukemia. This trial is currently recruiting participants. The goal is to understand the safety and effectiveness of this treatment combination.

Why it matters: This trial may provide new insights into treating Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia.

Recruiting participants

ClinicalTrials.gov

Related clinical trials

PHASE2RECRUITING

Acalabrutinib in Combination With Venetoclax for the Treatment of Refractory or Recurrent Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma, The AVENUE-2 Trial

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PHASE1ACTIVE_NOT_RECRUITING

CD19.CAR Allogeneic NKT for Patients With Relapsed or Refractory B-Cell Malignancies (ANCHOR)

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Nemtabrutinib and Lisocabtagene Maraleucel for the Treatment of Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

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CD22 CAR T-cells to Extend Remission Following Commercial CD19 CAR T-cells in Children, Adolescents, and Adults With Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia

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Acalabrutinib Monotherapy vs Investigator's Choice of Treatment in Patients With CL Leukaemia and Heart Failure

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A Study of Obecabtagene Autoleucel in People With B-cell Acute Lymphoblastic Leukemia

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A Study to Evaluate Next-Generation Sequencing (NGS) Testing and Monitoring of B-cell Recovery to Guide Management Following Chimeric Antigen ReceptorA type of immunotherapy that genetically engineers a patient's own T cells to recognise and destroy cancer cells.Click for full explanation → T-cell (CART) Induced Remission in Children and Young Adults With B Lineage Acute Lymphoblastic Leu...

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Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies

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Inotuzumab Ozogamicin and Blinatumomab With or Without Ponatinib in Treating Patients With Newly Diagnosed, Recurrent, or Refractory CD22-Positive B-Lineage Acute Lymphoblastic Leukemia

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Novel Unedited Allo Cell Therapy For High Risk T-Cell Malignancies Using CD7-Specific Car T Cells

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