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.
Observational StudyPatterns observed in populations over time, not a controlled experiment.Published: August 15, 2026
NPM1 MutationsA change in DNA sequence that can drive cancer development.Click for full explanation → in Leukemia: New Insights into Treatment Outcomes
Researchers studied 396 patients with a specific type of leukemia called NPM1-mutated AML. They looked at how different treatments affected their survival rates. The study found that some treatments worked better than others, but more research is needed to understand why.
Why it matters: This study provides new information that could help doctors make better treatment decisions for patients with NPM1-mutated AML.
Efficacy
Those treated with a combination of cladribine, low-dose cytarabine, and venetoclax alternating with azacitidine and venetoclax yielded a 5-year OS rate of 74%.
✦ Significant findingA meaningful signal from later-stage research, or a strong phase 2 result that stands out from routine findings.Phase 3 TrialLarge controlled trial comparing treatments. The strongest level of trial evidence.Published: August 14, 2026
Oral Cryotherapy May Reduce Oral Mucositis in Children with Leukemia
Researchers tested whether rinsing with ice-cold saline could prevent mouth sores in children with leukemia who received a high-dose chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation → drug. They found that this treatment may reduce the incidence and severity of mouth sores in these patients. However, more research is needed to confirm these findings.
Why it matters: This finding could improve the treatment experience for children with leukemia undergoing chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation →.
Efficacy
The incidence of any-grade oral mucositis was significantly lower in the oral cryotherapy group (22.0% vs. 44.1%).
Observational StudyPatterns observed in populations over time, not a controlled experiment.Published: August 14, 2026
Leukemia Mortality Trends in Asia from 1992 to 2021
Researchers analyzed leukemia deaths in 34 Asian countries from 1992 to 2021. They found that while the age-standardized mortality rate decreased, the absolute number of deaths increased due to population growth and aging. Acute myeloid leukemia led deaths in most regions, except East Asia where acute lymphoblastic leukemia was more common.
Why it matters: This study highlights the need for targeted leukemia control strategies in Asia, particularly in Southeast Asia and East Asia.
Lab StudyLaboratory experiments on cells or tissue. No human or animal data.Published: August 14, 2026
New Insights into Acute Myeloid Leukemia: OIP5-AS1's Role in Diagnosis and Prognosis
Researchers studied a type of RNA called OIP5-AS1 in patients with acute myeloid leukemia (AML). They found that OIP5-AS1 levels were higher in AML patients and were associated with lower survival rates. In lab experiments, knocking down OIP5-AS1 led to increased levels of a microRNA called miR-15a-5p, which in turn affected cell growth and death.
Why it matters: This finding may provide a new potential target for improving the prognosis of AML patients.
Observational StudyPatterns observed in populations over time, not a controlled experiment.Published: August 14, 2026
Elevated DNA Damage in Children with Leukemia
Researchers studied 32 children with leukemia and 30 healthy children to see if DNA damage was higher in those with leukemia. They found that children with leukemia had higher levels of DNA damage before treatment, but these levels decreased after chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation →. However, the levels were still higher than in healthy children after treatment.
Why it matters: This finding could help researchers understand how leukemia develops and how chemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation → works, which could lead to better treatments in the future.
Efficacy
Serum 8-OHdG levels significantly decreased after chemotherapy in children with ALL (P < .001).
This study was small and only looked at children with leukemia, so it's not clear if these findings apply to adults or other types of cancer.
Lab StudyLaboratory experiments on cells or tissue. No human or animal data.Published: August 14, 2026
Combining Herbs and Medicine May Help Fight Leukemia
Researchers tested a combination of a common leukemia medicine and an herbal extract in lab experiments. They found that the combination was more effective at killing leukemia cells than either treatment alone. However, more research is needed to confirm these findings.
Why it matters: This study suggests that combining herbal extracts with traditional medicines may be a useful approach for treating leukemia.
Efficacy
The combination therapy induced 47.54% apoptosis.
This study was conducted in a lab using a specific type of leukemia cell line, and the results may not apply to all patients with leukemia.
Lab StudyLaboratory experiments on cells or tissue. No human or animal data.Published: August 14, 2026
New Linkers May Improve Cancer Imaging
Researchers designed new molecules to help create more accurate cancer images. They tested these molecules in lab experiments and found some worked better than others. More work is needed to improve their effectiveness.
Why it matters: This finding could lead to better cancer imaging techniques, which are crucial for diagnosis and treatment planning.
This study was conducted in lab experiments and has not been tested in humans.
PreclinicalLab or cell studies. No human data yet.Est. completion: November 1, 2027
New Trial for Acute Myeloid Leukemia Treatment in India
This clinical trialA research study that tests a medical intervention in human volunteers.Click for full explanation → is studying the combination of oral Venetoclax and subcutaneous or intravenous Azacitidine in newly diagnosed adult participants with Acute Myeloid Leukemia who cannot receive standard treatment. The trial aims to assess the safety and effectiveness of this treatment combination. The trial is currently recruiting participants.
Why it matters: This trial may provide new insights into the treatment of Acute Myeloid Leukemia in patients who are ineligible for standard therapy.
PreclinicalLab or cell studies. No human data yet.Est. completion: May 25, 2027
Evaluating Heart Health in Cancer Survivors
The CREST Study is investigating how cancer survivors' hearts adapt to physical activity. Researchers are using various tests to assess cardiovascular health in participants who have undergone cancer treatment. The study is currently active but not recruiting participants.
Why it matters: This trial aims to improve our understanding of cardiovascular health in cancer survivors, which could inform future treatments and care.
ACTIVE_NOT_RECRUITING, conditions include Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and others
Observational StudyPatterns observed in populations over time, not a controlled experiment.Published: August 13, 2026
Study Helps Plan Better Use of Cancer Prevention Program in Clinics
Researchers studied how to make a successful cancer prevention program work in clinics. They talked to clinic leaders and workers to find out what helps and what gets in the way. They found that support from clinic staff and existing partnerships are key to success.
Why it matters: This study's findings can help improve the implementation of cancer prevention programs in clinics, which is important for reaching underserved communities.
Lab StudyLaboratory experiments on cells or tissue. No human or animal data.Published: August 13, 2026
Eucalyptus Extract May Help Treat Leukemia
Researchers studied how Eucalyptus camaldulensis leaf extract affects leukemia cells in a lab. They found that the extract can reduce the viability of these cells, especially when combined with the medication thalidomide. However, more research is needed to confirm these findings.
Why it matters: This study may lead to new ways to treat acute myeloid leukemia, a type of blood cancer.
Efficacy
The combination of thalidomide and E. camaldulensis extract demonstrated an additive cytotoxic effect (CI = 1) and the IC₅₀ values were 197.80 µg/mL for thalidomide, 30.26 µg/mL for the extract, and 28.26 µg/mL for the combination.
This study was conducted in a lab using a single cell line and more research is needed to confirm these findings.
Animal StudyResults in animals only. May or may not apply to humans.Published: August 13, 2026
New Cancer Treatment Target Identified from Marine Compound
Researchers discovered a new potential treatment for acute myeloid leukemia (AML) by modifying a compound found in the ocean. This compound, called ap-a48, targets a protein called HSP90 that helps cancer cells grow. In lab experiments, ap-a48 showed promise in treating AML without causing severe side effects.
Why it matters: This finding could lead to the development of new treatments for AML, a type of blood cancer.
Efficacy
In HL-60 xenograft mouse models, ap-a48 suppressed tumor growth by 71.2% at intraperitoneal 40 mg/kg and 67.3% at oral 60 mg/kg.
This study was conducted in rats and mouse models, and more research is needed to confirm its effectiveness in humans.
Testing the Addition of Venetoclax or Gemtuzumab Ozogamicin (GO) to Usual Treatment Regimen (Cytarabine and Daunorubicin, "7+3") for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) to Improve Response (A MYELOMATCH Treatment Trial)
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
Testing the Addition of the Anti-cancer Drug Venetoclax and/or the Anti-cancer ImmunotherapyTreatments that use the body's immune system to fight cancer.Click for full explanation → Blinatumomab to the Usual ChemotherapyDrugs that kill rapidly dividing cells, including cancer cells.Click for full explanation → Treatment for Infants With Newly Diagnosed KMT2A-rearranged or KMT2A-non-rearranged Leukemia
A Study to Assess Adverse Events and Change in Disease Activity When Intravenous (IV) Pivekimab Sunirine is Given in Combination With Oral Venetoclax and IV or Subcutaneous Azacitidine in Adult Participants With Acute Myeloid Leukemia (AML)
A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia
A Clinical TrialA research study that tests a medical intervention in human volunteers.Click for full explanation → of MK-1045 in People With B-cell Acute Lymphoblastic Leukemia (MK-1045-005)
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
