Leukemia

Treatments & research overview

What researchers are actively studying

Research is active across all subtypes. In AML, trials include ziftomenib and bleximenib (menin inhibitors for KMT2A-rearranged and NPM1-mutated disease), revumenib in relapsed or refractory disease, mocravimod after allogeneic transplant, and navitoclax with venetoclax and decitabine. The national MYELOMATCH platform is assigning people to molecularly targeted studies. In CLL, pirtobrutinib is being compared with ibrutinib in a phase 3 trial and with idelalisib in another, and novel combinations of acalabrutinib with ceralasertib, zanubrutinib with sonrotoclax, and asciminib in the CML T315I-mutated setting are underway. Cellular therapies include CD7-directed allogeneic CAR-TA type of immunotherapy that reprograms a patient's own T cells to attack cancer.Click for full explanation → (WU-CART-007) for T-cell ALL, a CD19 CAR-TA type of immunotherapy that reprograms a patient's own T cells to attack cancer.Click for full explanation → in relapsed or refractory ALL, and allogeneic natural killer cell products such as SAR445419. Next-generation sequencing guided minimal residual disease monitoring, plasma-based DNA testing, and artificial intelligence assisted morphology are being developed to guide treatment intensity.

Where the evidence stands

Tyrosine kinase inhibitor therapy in CML and BTK inhibitors in CLL are supported by mature phase 3 trials and long-term follow-up. Venetoclax-based combinations in AML also have phase 3 evidence. Pirtobrutinib has phase 3 data in CLL, and multiple menin inhibitors are in late phase testing with early encouraging results in KMT2A-rearranged and NPM1-mutated AML. CAR-TA type of immunotherapy that reprograms a patient's own T cells to attack cancer.Click for full explanation → cell therapies in B-cell ALL have mature regulatory approval; CAR-TA type of immunotherapy that reprograms a patient's own T cells to attack cancer.Click for full explanation → and engineered natural killer cell products in other subtypes remain in phase 1. Minimal residual disease monitoring is clinically established in ALL and CML and is being adopted more routinely in AML.

What this means for people affected

The treatment picture varies enormously by subtype. CML is typically managed as a chronic condition with oral pills and a near-normal life expectancy. CLL has moved largely away from chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation → toward targeted oral drugs. AML and ALL remain more intensive, but mutationA change in DNA sequence that can drive cancer development.Click for full explanation → testing at diagnosis now directs which targeted drugs are added, and cellular therapies provide meaningful options in relapsed B-cell ALL and some lymphomas. Minimal residual disease monitoring is increasingly guiding how long to treat and when to intensify.

Last updated April 22, 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.
Observational StudyPatterns observed in populations over time, not a controlled experiment.Published: April 20, 2026

STIC Lesion Management in German-speaking Countries

Researchers surveyed doctors in Austria, Germany, and Switzerland about how they manage a rare type of cancer called serous tubal intraepithelial carcinoma (STIC). They found that doctors have different approaches to treating STIC, especially when it's found in women with or without a known genetic mutationA change in DNA sequence that can drive cancer development.Click for full explanation →. The study highlights the need for more consistent guidelines.

Why it matters: This study highlights discrepancies between guideline recommendations and real-world practices in managing STIC lesions.

Survey answered by 77 physicians in German-speaking gynecological centers.

PubMed
Observational StudyPatterns observed in populations over time, not a controlled experiment.Published: April 18, 2026

Diagnosing Cancer in Patients with Liver Disease

Researchers reported a case of a patient with both liver cirrhosis and chronic lymphocytic leukemia, a type of blood cancer. The patient's symptoms overlapped, making diagnosis and treatment challenging. The researchers highlighted the need for a multidisciplinary approach to manage such complex cases.

Why it matters: This case highlights the complexities of diagnosing and treating patients with concurrent liver disease and blood cancer, emphasizing the importance of individualized treatment.
PubMed
Phase 1 TrialFirst-in-human trial. Focuses on safety and dosing in small groups.Est. completion: June 24, 2026

Acalabrutinib Study for B-cell Malignancies in Chinese Adults

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying the safety and effectiveness of the medication acalabrutinib in Chinese adults with certain types of blood cancers, including mantle cell lymphoma and chronic lymphocytic leukemia. The trial is currently active but not recruiting participants. The trial aims to gather more information about the medication's effects in this population.

Why it matters: This trial may help doctors better understand how acalabrutinib works in Chinese adults with these types of blood cancers.

Active but not recruiting participants.

ClinicalTrials.gov
Phase 1 TrialFirst-in-human trial. Focuses on safety and dosing in small groups.Est. completion: August 26, 2026

Testing AZD6738 and Acalabrutinib for Relapsed CLL

This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying the combination of AZD6738 and Acalabrutinib in people with chronic lymphocytic leukemia (CLL) that has come back or doesn't respond to treatment. The trial is currently active but not recruiting participants. The goal is to understand the safety and effectiveness of this treatment combination.

Why it matters: This trial aims to provide more information about a potential new treatment option for people with relapsed or refractory CLL.

Active but not recruiting participants.

ClinicalTrials.gov
ReviewSummary and analysis of existing published studies.Published: April 17, 2026

Using AI to Improve Cancer Diagnosis with Microscope Images

Researchers looked at how artificial intelligence can help diagnose a type of blood cancer called acute leukemia. They found that AI can analyze microscope images more efficiently and accurately than human doctors, but there are still challenges to overcome. This is an early step in developing new diagnostic tools.

Why it matters: This research could lead to more accurate and efficient diagnosis of acute leukemia, which is a complex and diverse group of cancers.
PubMed
Lab StudyLaboratory experiments on cells or tissue. No human or animal data.Published: April 17, 2026

Researchers Identify a Potential Protector Against ChemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation →-Induced Leukemia

Scientists discovered a peptide that may help prevent a type of leukemia caused by chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation →. They found that this peptide, called Ku3, can reduce the risk of chromosomal rearrangements in lab experiments. However, more research is needed to confirm these findings.

Why it matters: This discovery could lead to new treatments that reduce the risk of secondary leukemia, a severe side effect of chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation →.

These findings are based on lab experiments and have not been tested in humans.

PubMed
Phase 3 TrialLarge controlled trial comparing treatments. The strongest level of trial evidence.Est. completion: March 31, 2027

Study of Tisagenlecleucel in Patients with Leukemia or Lymphoma

Researchers are conducting a phase 3 clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → to study the effectiveness and safety of Tisagenlecleucel in patients with B-cell Acute Lymphoblastic Leukemia or Diffuse Large B-cell Lymphoma. The trial is currently recruiting participants. The results of this study are not yet known.

Why it matters: This trial aims to gather more information about the potential benefits and risks of Tisagenlecleucel for patients with these conditions.

Recruiting participants

ClinicalTrials.gov
Lab StudyLaboratory experiments on cells or tissue. No human or animal data.Published: April 17, 2026

Researchers Identify Metabolic Differences in Leukemia Patients

Researchers studied the metabolic profiles of 49 leukemia patients to see if they could identify differences in how their cells responded to a new type of treatment. They found that some patients had different metabolic profiles than others, which might help doctors predict how well they'll respond to the treatment. However, more research is needed to confirm these findings.

Why it matters: This study could help doctors develop more personalized treatment plans for leukemia patients.
PubMed
Observational StudyPatterns observed in populations over time, not a controlled experiment.Published: April 17, 2026

EMR vs Paper Records in Leukemia Care

Researchers compared patient outcomes in two Mexican hospitals using different record-keeping methods. They found that patients in the hospital with electronic medical records (EMR) had lower 60-day mortality rates and faster treatment times. However, the study had limitations and more research is needed.

Why it matters: This study highlights the potential benefits of digital record-keeping in cancer care, but more research is needed to confirm these findings.
Efficacy

Sixty-day mortality was lower in the EMR hospital (5.8%) than in the TPR hospital (35.9%; P<.001).

Retrospective natural experiment with a 2-center design and potential for residual confounding.

PubMed
Lab StudyLaboratory experiments on cells or tissue. No human or animal data.Published: April 17, 2026

New Marker for Predicting Leukemia Treatment Response in Children

Researchers studied whether a protein called Semaphorin 4D can help predict how well children with a type of leukemia called B-ALL will respond to initial treatment. They found that higher levels of this protein were associated with a poorer response to treatment. However, more research is needed to confirm these findings.

Why it matters: This finding could help doctors make more informed decisions about treatment for children with B-ALL.
Efficacy

Sema4D expression > 18% significantly identifies patients at risk for induction failure.

PubMed
PreclinicalLab or cell studies. No human data yet.Est. completion: June 7, 2030

Asciminib RMP Study for 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 Asciminib in patients with Chronic Myeloid Leukemia. The trial is currently recruiting participants. The results of this study are not yet available.

Why it matters: This trial may provide new insights into the treatment of Chronic Myeloid Leukemia.

Recruiting participants, status not specified

ClinicalTrials.gov
Phase 2 TrialTests early effectiveness in a larger group after phase 1 safety is established.Est. completion: December 30, 2028

Testing a New CAR-T Cell TherapyA type of immunotherapy that reprograms a patient's own T cells to attack cancer.Click for full explanation → for T-Cell Leukemia and Lymphoma

Researchers are studying a new treatment called WU-CART-007 for people with T-cell Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma. This treatment uses a type of immune cell called a CAR-TA type of immunotherapy that reprograms a patient's own T cells to attack cancer.Click for full explanation → cell. The trial is currently recruiting participants.

Why it matters: This trial may help determine if WU-CART-007 is a safe and effective treatment option for people with these types of cancer.

Recruiting participants

ClinicalTrials.gov

Related clinical trials

NARECRUITING

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...

NCT05621291
PHASE1RECRUITING

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

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PHASE1RECRUITING

First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia

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PHASE2RECRUITING

Open-label Study of Asciminib for CML-CP or CML-AP Patients With T315I MutationA change in DNA sequence that can drive cancer development.Click for full explanation → Who Are Resistant, Intolerant or Ineligible to Ponatinib.

NCT06514534
PHASE2RECRUITING

A Phase 2 Study of WU-CART-007, an Anti-CD7 Allogeneic CAR-T Cell TherapyA type of immunotherapy that reprograms a patient's own T cells to attack cancer.Click for full explanation → in T-Cell Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma (T-RRex)

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PHASE1ACTIVE_NOT_RECRUITING

A Study of AZD6738 and Acalabrutinib in Subjects With Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL)

NCT03328273
RECRUITING

Asciminib RMP Study

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PHASE3RECRUITING

Study of Out of Specification for Tisagenlecleucel

NCT04094311
PHASE1ACTIVE_NOT_RECRUITING

Study of Acalabrutinib in Chinese Adult Subjects With Relapsed or Refractory Mantle Cell Lymphoma, Chronic Lymphocytic Leukemia or Other B-cell Malignancies

NCT03932331
RECRUITING

Tissue Collection for Studies of Lymph Cancer

NCT01676805