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Disease

Leukemia

Late-stage therapeutic developmentEmerging researchRising momentum
MeSHLeukemia
10
Publications
24
Clinical trials
3
Related proteins
2025
Latest publication
Current focus
Antibodies biologyTherapeutic developmentInflammation & immunity
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

Nanotechnology in leukemia therapy: revolutionizing targeted drug delivery and immune modulation.

Research2025-05-17Clinical and experimental medicine

Metastatic gastric cancer target lesion complete response with Claudin18.2-CAR T cells.

Research2024-02-05Journal for immunotherapy of cancer

Disruption of SUV39H1-Mediated H3K9 Methylation Sustains CAR T-cell Function.

Research2024-01-01Cancer discovery

Swarm Learning for decentralized and confidential clinical machine learning.

Research2021-05-26Nature

Monocyte-derived IL-1 and IL-6 are differentially required for cytokine-release syndrome and neurotoxicity due to CAR T cells.

Research2018-05-28Nature medicine

Adjuvant trastuzumab in HER2-positive breast cancer.

Research2011-10-01The New England journal of medicine

The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes.

Research2009-04-08Blood

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 5 clinical trials expected to report results, the earliest in Q3 2026.
Upcoming Milestones
Clinical Milestones10View all 10
Results posted 2
Results expected 5
Recently completed 1
Other updates 2
+2 more in the activity timeline below
Activity timeline10

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Zanubrutinibapproved

Approval — Brukinsa as monotherapy is indicated for the treatment of adult patients with Walden… (2021)

Melphalan hydrochlorideapproved

Approval — High-dose of Phelinun used alone or in combination with other cytotoxic medicinal product… (2020)

Posaconazoleapproved

Approval — Posaconazole Accord is indicated for use in the treatment of the following fungal infecti… (2019)

Clinical trials

14 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
24
All trials
6
Active
16
Late-stage
6
Completed
Late-stage studies

A Phase IIIb, Multi-center, Open-label, Randomized Study of Tolerability and Efficacy of Oral Asciminib Versus Nilotinib in Patients With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase.

Phase 3 · Active, not recruiting · Novartis Pharmaceuticals

A Phase 3, Randomized Study to Compare the Efficacy and Safety of Nemtabrutinib Versus Chemoimmunotherapy for Previously Untreated Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma Without TP53 Aberrations

Phase 3 · Active, not recruiting · Merck Sharp & Dohme LLC

A Phase 3, Double-Blind, Randomized, Placebo-Controlled Trial Of Quizartinib Administered in Combination With Induction and Consolidation Chemotherapy and Administered as Maintenance Therapy in Adult Patients With Newly Diagnosed FLT3-ITD Negative Acute Myeloid Leukemia

Phase 3 · Active, not recruiting · Daiichi Sankyo

Discontinuation of TyrosIne Kinase Inhibitors (ITK) in Chronic Myeloid Leukemia (LMC) and Impact on the Immune System: a Randomized Comparative Study of Two Therapeutic Strategies

Phase 3 · Active, not recruiting · Poitiers University Hospital

A Prospective, Multicenter Clinical Study on the Efficacy and Safety of PD-1 Inhibitors Combined With Venetoclax+CAG Regimens in the Treatment of Adult Refractory/Relapsed Acute T-lymphoblastic Leukemia (T-ALL)

Phase 3 · Active, not recruiting · Fujian Medical University Union Hospital

A Randomized Trial of Levocarnitine Prophylaxis to Prevent Asparaginase-Associated Hepatotoxicity in Adolescents and Young Adults Receiving Acute Lymphoblastic Leukemia Therapy

Phase 3 · Active, not recruiting · Children's Oncology Group
Recently completed

A Phase III Randomized Study of Farnesyl Transferase Inhibitor R115777 in Acute Myeloid Leukemia (AML) Patients in Second or Subsequent Remission or in Remission After Primary Induction Failure or Patients Over Age 60 in First Remission

Phase 3 · Completed · National Cancer Institute (NCI)

A Randomized, Placebo-Controlled, Double Blind, Trial of the Administration of the MDR Modulator, Zosuquidar Trihydrochloride (LY335979), During Conventional Induction and Post-Remission Therapy in Patients Greater Than 60 Years of Age With Newly Diagnosed Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts in Transformation or High-Risk Refractory Anemia With Excess Blasts

Phase 3 · Completed · Eastern Cooperative Oncology Group

ALINC #17 Treatment for Patients With Low Risk Acute Lymphoblastic Leukemia: A Pediatric Oncology Group Phase III Study

Phase 3 · Completed · Children's Oncology Group

A Phase III Study of Daunorubicin and Cytarabine +/- G3139 (Genasense, Oblimersen Sodium, NSC #683428, IND #58842), a BCL2 Antisense Oligodeoxynucleotide, in Previously Untreated Patients With Acute Myeloid Leukemia (AML) > / = 60 Years

Phase 3 · Completed · National Cancer Institute (NCI)

A PHASE III STUDY IN CHILDREN WITH UNTREATED ACUTE MYELOGENOUS LEUKEMIA (AML) OR MYELODYSPLASTIC SYNDROME (MDS)

Phase 3 · Completed · National Cancer Institute (NCI)

A Phase III Study Comparing Imatinib Standard Dose (400 mg/Day) Versus Imatinib High Dose (800 mg/Day) in the Treatment of Newly Diagnosed High Risk Chronic Myeloid Leukemia in Chronic Phase

Phase 3 · Completed · University of Bologna

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2021emaApproval — Zanubrutinib· Brukinsa as monotherapy is indicated for the treatment of adult patients with Waldenström’s macroglobulinaemia (WM) who have received at least one prior therapy, or in first line treatment for patients unsuitable for chemo-immunotherapy. Brukinsa as monotherapy is indicated for the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy. Brukinsa as monotherapy is indicated for the treatment of adult patients with chronic lymphocytic leukemia (CLL). Brukinsa in combination with obinutuzumab is indicated for the treatment of adult patients with refractory or relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies. source ↗
2020emaApproval — Melphalan hydrochloride· High-dose of Phelinun used alone or in combination with other cytotoxic medicinal products and/or total body irradiation is indicated in the treatment of: multiple myeloma, malignant lymphoma (Hodgkin, non-Hodgkin lymphoma), acute lymphoblastic and myeloblastic leukemia, childhood neuroblastoma, ovarian cancer, mammary adenocarcinoma. Phelinun in combination with other cytotoxic medicinal products is indicated as reduced intensity conditioning (RIC) treatment prior to allogeneic haematopoietic stem cell transplantation (allo-HSCT) in malignant haematological diseases in adults. Phelinun in combination with other cytotoxic medicinal products is indicated as conditioning regimen prior to allogeneic haematopoietic stem cell transplantation in haematological diseases in the paediatric population as: Myeloablative conditioning (MAC) treatment in case of malignant haematological diseases RIC treatment in case of non-malignant haematological diseases. source ↗
2019emaApproval — Posaconazole· Posaconazole Accord is indicated for use in the treatment of the following fungal infections in adults: Invasive aspergillosis; Fusariosis in patients with disease that is refractory to amphotericin B or in patients who are intolerant of amphotericin B; Chromoblastomycosis and mycetoma in patients with disease that is refractory to itraconazole or in patients who are intolerant of itraconazole; Coccidioidomycosis in patients with disease that is refractory to amphotericin B, itraconazole or fluconazole or in patients who are intolerant of these medicinal products. Refractoriness is defined as progression of infection or failure to improve after a minimum of 7 days of prior therapeutic doses of effective antifungal therapy. Posaconazole Accord is also indicated for prophylaxis of invasive fungal infections in the following patients:  Patients receiving remission-induction chemotherapy for acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) expected to result in prolonged neutropenia and who are at high risk of developing invasive fungal infections; Hematopoietic stem cell transplant (HSCT) recipients who are undergoing high-dose immunosuppressive therapy for graft versus host disease and who are at high risk of developing invasive fungal infections. source ↗
2019emaApproval — Posaconazole· Posaconazole AHCL oral suspension is indicated for use in the treatment of the following fungal infections in adults: Invasive aspergillosis in patients with disease that is refractory to amphotericin B or itraconazole or in patients who are intolerant of these medicinal products; Fusariosis in patients with disease that is refractory to amphotericin B or in patients who are intolerant of amphotericin B; Chromoblastomycosis and mycetoma in patients with disease that is refractory to itraconazole or in patients who are intolerant of itraconazole; Coccidioidomycosis in patients with disease that is refractory to amphotericin B, itraconazole or fluconazole or in patients who are intolerant of these medicinal products. Oropharyngeal candidiasis: as first-line therapy in patients who have severe disease or are immunocompromised, in whom response to topical therapy is expected to be poor. Refractoriness is defined as progression of infection or failure to improve after a minimum of 7 days of prior therapeutic doses of effective antifungal therapy. Posaconazole AHCL oral suspension is also indicated for prophylaxis of invasive fungal infections in the following patients: Patients receiving remission-induction chemotherapy for acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) expected to result in prolonged neutropenia and who are at high risk of developing invasive fungal infections; Hematopoietic stem cell transplant (HSCT) recipients who are undergoing high-dose immunosuppressive therapy for graft versus host disease and who are at high risk of developing invasive fungal infections. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

10 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20082025
Major themes8
  • Leukemia3
  • Receptors, Chimeric Antigen2
  • Antineoplastic Agents1
  • Blockchain1
  • Confidentiality1
  • Drug Delivery Systems1
  • Erb-b2 Receptor Tyrosine Kinases1
  • Machine Learning1
Leading journals6
  • Blood2
  • Journal for immunotherapy of cancer2
  • British journal of cancer1
  • Cancer discovery1
  • Clinical and experimental medicine1
  • Nature1
Leading researchers8
  • Altmüller J1
  • Antelope C1
  • Arber DA1
  • Aschenbrenner AC1
  • Augustin M1
  • Aziz NA1
  • Backes M1
  • Bals R1
Affiliations (unnormalised)6
  • Center for Cell Engineering and Immunology Program1
  • Center for Epigenetics Research1
  • Center for Molecular Medicine Cologne (CMMC)1
  • Center of Integrated Protein Science Munich (CIPS-M) and Division of Clinical Pharmacology1
  • Children's Hospital1
  • Christian-Albrechts-University and University Hospital Schleswig-Holstein1

Disease biology

3 matches

Key proteins & gene products studied in this disease. Number shows shared papers.

Receptors, Chimeric Antigen4Antibodies, Monoclonal, Humanized2Receptors, Antigen, T-Cell2

Disease profile

A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.

Overview

Leukemia is a progressive malignant disease of the blood-forming organs in which leukocytes and their precursors proliferate abnormally in the blood and bone marrow. It is classified by the maturity of the malignant cells, with acute leukemias dominated by immature cells and chronic leukemias composed of more mature cells.

Causes

The supplied grounding does not identify a specific cause or aetiology for leukemia. It notes that modern classification includes entities defined principally by genetic features, but does not specify causal mutations or exposures.

Pathophysiology

Leukemia involves distorted proliferation and development of leukocytes and their precursors in the blood and bone marrow. The literature grounding also points to the tumor microenvironment and to immunologic mechanisms, including immune recognition and targeting of malignant cells, as relevant to the disease biology.

Risk factors

The supplied grounding does not support specific risk factors for leukemia. It only indicates that some leukemia entities are defined by genetic features, without linking these to risk.

Current standard of care

The grounding supports drug therapy and immunology as major treatment areas for leukemia. It specifically mentions chimeric antigen receptor T-cell therapy for CD19-positive leukemias, as well as monoclonal antibodies and targeted drug delivery approaches aimed at improving treatment and immune modulation. The review material also frames current management within evolving WHO classification and broader conventional therapies such as chemotherapy and immunotherapy, but does not provide regimen-level details.

AI-generated summary grounded in MeSH and 4 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A progressive, malignant disease of the blood-forming organs, characterized by distorted proliferation and development of leukocytes and their precursors in the blood and bone marrow. Leukemias were originally termed acute or chronic based on life expectancy but now are classified according to cellular maturity. Acute leukemias consist of predominately immature cells; chronic leukemias are composed of more mature cells. (From The Merck Manual, 2006)

Identifiers
MeSHLeukemia
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.