Acute lymphoblastic leukemia

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ORPHA:513OMIM:247640C91.0
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13FDA treatments175Active trials245Specialists8Treatment centers7Financial resources

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Acute lymphoblastic leukemia (ALL), also known as acute lymphocytic leukemia or acute lymphoid leukemia, is a cancer of the blood and bone marrow characterized by the uncontrolled proliferation of immature lymphoid cells (lymphoblasts). These abnormal cells accumulate in the bone marrow, crowding out normal blood cell production, and can spread to the blood, lymph nodes, spleen, liver, central nervous system, and other organs. ALL is the most common childhood cancer, though it also occurs in adults. The disease is classified into B-cell ALL (approximately 85% of pediatric cases) and T-cell ALL based on the lineage of the affected lymphoblasts. Key symptoms result from bone marrow failure and organ infiltration. Patients typically present with fatigue, pallor, and weakness due to anemia; recurrent or severe infections due to neutropenia; and easy bruising, petechiae, or bleeding due to thrombocytopenia. Other common features include bone and joint pain, fever, lymphadenopathy (swollen lymph nodes), hepatosplenomegaly (enlarged liver and spleen), and unexplained weight loss. Central nervous system involvement may cause headaches, vomiting, or cranial nerve palsies. The treatment landscape for ALL has evolved significantly, particularly in pediatric patients where cure rates now exceed 85-90%. Treatment typically involves multi-agent chemotherapy administered in phases: induction (to achieve remission), consolidation/intensification (to eliminate residual disease), and maintenance therapy, often spanning two to three years. CNS-directed therapy (intrathecal chemotherapy and/or cranial radiation) is a critical component. For relapsed or refractory disease, options include targeted therapies such as tyrosine kinase inhibitors for Philadelphia chromosome-positive ALL, bispecific T-cell engager antibodies (blinatumomab), antibody-drug conjugates (inotuzumab ozogamicin), and chimeric antigen receptor (CAR) T-cell therapy. Allogeneic hematopoietic stem cell transplantation remains an important option for high-risk or relapsed patients. Adult ALL carries a less favorable prognosis than pediatric ALL, though outcomes have improved with the adoption of pediatric-inspired treatment protocols.

Also known as:

Inheritance

Multifactorial

Caused by a mix of several genes and environmental factors

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Oct 2026HEM-iSMART E: Capivasertib + Venetoclax + Dexamethasone in Pediatric Patients With Relapsed or Refractory Hematological Malignancies

Princess Maxima Center for Pediatric Oncology — PHASE1, PHASE2

TrialNOT YET RECRUITING
Sep 2026A Study to Find the Highest Dose of SNDX-5613 (Revumenib) as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and Mixed Phenotype Acute Leukemia

Children's Oncology Group — PHASE1

TrialNOT YET RECRUITING
Sep 2026This Clinical Trial is a Single-arm, Non-randomized Pilot Trial to Determine the Safety of Administering Autologous Anti-C19 Cells (ARI-0001) and the Feasibility of Local CAR-T Cell Production in Patients Over 18 Years of Age With Relapsed/Refractory (R/R) CD19+ Hematologic Malignancies, Including R

GUSTAVO SALGUERO — NA

TrialNOT YET RECRUITING
Aug 2026Clinical Performance of Giomer-Based Composite Restorations in Class V Carious Lesions in Diabetic and Non-Diabetic Patients

Cairo University — NA

TrialNOT YET RECRUITING
Jun 2026Cannabidiol as an Adjunct Treatment for Alcohol Withdrawal and Craving

Johns Hopkins University — PHASE2, PHASE3

TrialNOT YET RECRUITING
Apr 2026Hospital Based Continuous Patient Monitoring System

University of Alabama at Birmingham — NA

TrialNOT YET RECRUITING
Apr 2026CD22 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

National Cancer Institute (NCI) — PHASE2

TrialNOT YET RECRUITING
Apr 2026Continuous Temperature Monitoring (CTM) for Cytokine Release Syndrome (CRS), an Immune-Related Adverse Event

National Cancer Institute (NCI) — NA

TrialNOT YET RECRUITING
Apr 2026Etoposide, Prednisone, Vincristine, Cyclophosphamide, and Doxorubicin (DA-EPOCH) With or Without Rituximab Plus Recombinant Erwinia Asparaginase (JZP458) for the Treatment of Newly Diagnosed Ph Negative B-Acute Lymphoblastic Leukemia or T Acute Lymphoblastic Leukemia

University of Washington — PHASE2

TrialRECRUITING
Mar 2026Study to Assess the Safety, Tolerability, and Treatment Response of GXV813 in Hospitalized Adults With Schizophrenia

Novartis Pharmaceuticals — PHASE2

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

13 available

Xatmep

methotrexate oral solution· Silvergate Pharmaeuticals, Inc.■ Boxed Warning

Treatment of pediatric patients with acute lymphoblastic leukemia (ALL) as a component of a combination chemotherapy maintenance regimen

Leukine

Sargramostim· Immunex Corporation
For the acceleration of myeloid reconstitution following autologous peripheral blood progenitor cell (PBPC) or bone marrow transplantation in adult and pediatric patients 2 years of age and older with

For the acceleration of myeloid reconstitution following autologous peripheral blood progenitor cell (PBPC) or bone marrow transplantation in adult and pediatric patients 2 years of age and older with acute lymphoblastic leukemia (ALL)

Methotrexate

Methotrexate sodium· Lederle Laboratories■ Boxed Warning

treatment of adults and pediatric patients with acute lymphoblastic leukemia (ALL) as part of a combination chemotherapy maintenance regimen

Lipiodol

ethiodized oil injection· Guerbet LLC■ Boxed Warning

lymphography in adult and pediatric patients

Novantrone

Mitoxantrone· Serono, Inc.■ Boxed Warning

in combination with other approved drug(s) is indicated in the initial therapy of acute nonlymphocytic leukemia (ANLL) in adults

Aucatzyl

obecabtagene autoleucel· Autolus Incorporated, US subsidiary of Autolus Limited■ Boxed WarningOrphan Drug

treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)

Blincyto

blinatumomab· Amgen, Inc.■ Boxed WarningOrphan Drug
treatment of CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL) in the consolidation phase of multiphase chemotherapy in adult and pediatric patients on

treatment of CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL) in the consolidation phase of multiphase chemotherapy in adult and pediatric patients one month and older

Rylaze

asparaginase erwinia chrysanthemi (recombinant)-rywn· Jazz Pharmaceuticals Ireland LimitedOrphan Drug
As a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients 1 month or older who h

As a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients 1 month or older who have developed hypersensitivity to E. coli-derived asparaginase.

Marqibo

vinCRIStine sulfate LIPOSOME injection· Acrotech Biopharma LLCOrphan Drug
Treatment of adult patients with Philadelphia chromosome-negative (Ph-) acute lymphoblastic leukemia (ALL) in second or greater relapse or whose disease has progressed following two or more anti-leuke

Treatment of adult patients with Philadelphia chromosome-negative (Ph-) acute lymphoblastic leukemia (ALL) in second or greater relapse or whose disease has progressed following two or more anti-leukemia therapies.

Erwinase

Erwinia L-asparaginase· Jazz Pharmaceuticals, Inc.Orphan Drug

Treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity to E. coli-derived asparaginase.

Arranon

nelarabine· Novartis Pharmaceuticals Corp.■ Boxed WarningOrphan Drug
Treatment of patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma whose disease has not responded to or has relapsed following treatment with at least two chemotherapy r

Treatment of patients with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens

Clolar

clofarabine· Genzyme CorporationOrphan Drug

Treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens

Oncaspar; Asparlas

Pegaspargase· Servier Pharmaceuticals LLCOrphan Drug

Combination chemotherapy for the treatment of patients with acute lymphoblastic leukemia who are hypersensitive to native forms of L-asparaginase.

Clinical Trials

20 recruitingView all trials with filters →
Phase 31 trial
Interfant-21 Treatment Protocol for Infants Under 1 Year With KMT2A-rearranged ALL or Mixed Phenotype Acute Leukemia
Phase 3
Actively Recruiting
PI: Janine Stutterheim, Dr (Princess Maxima Center for Pediatric Oncology in T) · Sites: Buenos Aires; Clayton, Victoria +110 more · Age: 01 yrs
Phase 44 trials
Ciprofloxacin vs Ceftazidime for Empirical Treatment of High-Risk Neutropenic Fever in Children With Hematologic Malignancies
Phase 4
Actively Recruiting
· Sites: Sleman, DI Yogyakarta · Age: 018 yrs
Identification of Necessary Information for Treatment Induction in Newly Diagnosed Acute Lymphoblastic Leukemia/Lymphoma
Phase 4
Actively Recruiting
PI: Seth E. Karol, MD (St. Jude Children's Research Hospital) · Sites: San Diego, California; Tulsa, Oklahoma +1 more · Age: 118 yrs
Hepatoprotective Measures for Children at High Risk of NAFLD
Phase 4
Active
PI: Manal H El-Sayed, Professor of pediatrics (Ain Shams University-Faculty of Medicine-Pediatric) · Sites: Cairo; Cairo · Age: 518 yrs
ALL SCTped FORUM - Pharmacogenomic Study (add-on Study)
Phase 4
Actively Recruiting
PI: Christina Peters, Prof. MD PhD (St. Anna Kinderspital, Vienna, Austria) · Sites: Geneva, Cansearch Laboratory; Basel +2 more · Age: 018 yrs
Other15 trials
InterNatIonal CHildhood Leukemia Microbiome/MEtabolome Cohort
Actively Recruiting
· Sites: New York, New York; Barretos +6 more · Age: 318 yrs
Study Protocol: Study on Incidence and Risk Factors of Mold Infections in Children During Leukemia Treatment
Active
PI: Rasmus Møller Duus, MD (University of Southern Denmark) · Sites: Odense · Age: 017 yrs
Pharmacogenomics of Asparaginase Induced Hepatotoxicity
Actively Recruiting
PI: Houda Alachkar, Ph.D. (University of Southern California) · Sites: Los Angeles, California · Age: 1899 yrs
Backtracking Leukemia-Typical Somatic Mutations in Cord Blood
Actively Recruiting
PI: Adam de Smith, PhD (University of Southern California Keck School of M) · Sites: Minneapolis, Minnesota · Age: 025 yrs
Collection of Biological Samples From Patients Treated With CAR-T Cells for Hematological Malignancies
Actively Recruiting
· Sites: Montpellier · Age: 1899 yrs
Cardiovascular Reserve Evaluation in Survivors of Transplant, CREST Study
Active
PI: Saro H Armenian (City of Hope Medical Center) · Sites: Duarte, California · Age: 1899 yrs
Retrospective Observational Study on Infective Complications and Outcome of Patients With ALL Treated With INO
Actively Recruiting
PI: Livio Pagano (Policlinico Universitario A. Gemelli, IRCCS-Univer) · Sites: Roma · Age: 1899 yrs
A Multi-site Study to Evaluate the Persistence of Protective Immunity to Routine Childhood Vaccinations in Participants With B-ALL/Ly Who Have Received Blinatumomab
Actively Recruiting
PI: Lauren Appell, MD (Arkansas Children's Research Institute) · Sites: Little Rock, Arkansas; Springdale, Arkansas · Age: 123 yrs
Determining the Mechanisms of Loss of CAR T Cell Persistence
Actively Recruiting
PI: Persis Amrolia, BSc,MBBS,PhD (UCL Institute of Child Health) · Sites: London; London +1 more · Age: 025 yrs
A Long-Term Safety Follow-Up Study for Patients Treat With WU-CART-007
Enrolling by Invitation
PI: Cherry Thomas, MD (Wugen, Inc.) · Sites: Duarte, California; Los Angeles, California +3 more · Age: 1299 yrs
Investigating Cognitive Impairment in Young Patients With Cancer Prospectively
Actively Recruiting
· Sites: Copenhagen · Age: 729 yrs
Collecting Blood Samples From Patients With and Without Cancer to Evaluate Tests for Early Cancer Detection
Actively Recruiting
PI: Marie Wood, MD (University of Colorado, Denver) · Sites: Anchorage, Alaska; Anchorage, Alaska +738 more · Age: 4075 yrs
Long Non-coding RNAs and Their Role on Epigenome as Diagnostic Markers in Childhood Acute Lymphoblastic Leukemia of T Cells.
Actively Recruiting
PI: Giovanni Smaldone, PhD (IRCCS SYNLAB SDN) · Sites: Naples · Age: 118 yrs
Tracjectories and Predictors of Chemotherapy Induced Peripheral Neuropathy in Children With Acute Lymphoblastic Leukemia
Actively Recruiting
PI: Ka Yan Ho (The Hong Kong Polytechnic University) · Sites: Zhengzhou, Henan; Shanghai +1 more · Age: 817 yrs
Incidence of Invasive Fungal Infections Among Patients With Acute Lymphoblastic Leukemia Ph-negative
Actively Recruiting
PI: Marianna Criscuolo (Fondazione Policlinico Universitario A. Gemelli, I) · Sites: Rome · Age: 1899 yrs

Specialists

Showing 25 of 245View all specialists →
LS
Lewis B Silverman
Birmingham, Alabama
Specialist

Rare Disease Specialist

10 Acute lymphoblastic leukemia publications
SM
Srdan Verstovsek, MD
HOUSTON, TX
Specialist
PI on 9 active trials
RC
Richard E Champlin
Houston, Texas
Specialist

Rare Disease Specialist

PI on 2 active trials5 Acute lymphoblastic leukemia publications
SM
Sarah Wall, MD
Columbus, Ohio
Specialist

Rare Disease Specialist

CC
Cheng Cheng
Specialist
3 Acute lymphoblastic leukemia publications
SJ
Sima Jeha
MEMPHIS, TN
Specialist
3 Acute lymphoblastic leukemia publications
MB
Michael J Borowitz
BALTIMORE, MD
Specialist
3 Acute lymphoblastic leukemia publications
KR
Karen R Rabin
KENSINGTON, MD
Specialist
3 Acute lymphoblastic leukemia publications
MD
Meenakshi Devidas
Specialist
3 Acute lymphoblastic leukemia publications
SH
Stephen P Hunger
PHILA, PA
Specialist
3 Acute lymphoblastic leukemia publications
TC
Ti-Cheng Chang
Specialist
4 Acute lymphoblastic leukemia publications
BW
Brent L Wood
Specialist
3 Acute lymphoblastic leukemia publications
HK
Hagop Kantarjian
HOUSTON, TX
Specialist
4 Acute lymphoblastic leukemia publications
CM
Charles G Mullighan
Specialist
6 Acute lymphoblastic leukemia publications
CP
Ching-Hon Pui
MEMPHIS, TN
Specialist
4 Acute lymphoblastic leukemia publications
ER
Elizabeth A Raetz
NEW YORK, NY
Specialist
3 Acute lymphoblastic leukemia publications
GW
Gang Wu
Specialist
5 Acute lymphoblastic leukemia publications
ML
Mignon L Loh
SAN FRANCISCO, CA
Specialist
4 Acute lymphoblastic leukemia publications
JY
Jun J Yang
Specialist
5 Acute lymphoblastic leukemia publications
FR
Farhad Ravandi-Kashani
HOUSTON, TX
Specialist
PI on 7 active trials2 Acute lymphoblastic leukemia publications
CM
Corey Cutler, MD, MPH
PARK CITY, UT
Specialist
PI on 3 active trials

Treatment Centers

8 centers
⚗️ Trial Site

Arkansas Children's Hospital

📍 Little Rock, Arkansas

⚗️ Trial Site

Children's Hospital of Orange County

📍 Orange, California

👤 Richard Neibeger, MD

⚗️ Trial Site

Banner University Medical Center - Tucson

📍 Tucson, Arizona

⚗️ Trial Site

Cedars Sinai Medical Center

📍 Los Angeles, California

👤 Janssen Research & Development, LLC Clinical Trial

⚗️ Trial Site

UCSF Benioff Children's Hospital Oakland

📍 Oakland, California

👤 Neeta Thakur, MD, MPH

⚗️ Trial Site

University of California Davis Comprehensive Cancer Center

📍 Sacramento, California

👤 Ann (Annie) W Silk

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

Financial Resources

7 resources
Xatmep(methotrexate oral solution)Silvergate Pharmaeuticals, Inc.
Leukine(Sargramostim)Immunex Corporation
Blincyto(blinatumomab)Amgen, Inc.
Rylaze(asparaginase erwinia chrysanthemi (recombinant)-rywn)Jazz Pharmaceuticals Ireland Limited
Erwinase(Erwinia L-asparaginase)Jazz Pharmaceuticals, Inc.
Arranon(nelarabine)Novartis Pharmaceuticals Corp.

Besponsa

Pfizer

Acute Lymphoblastic Leukemia

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Latest news about Acute lymphoblastic leukemia

1 articles
Clinical trialUNITERAREApr 5, 2026
New Recruiting Trial: Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies
Researchers are testing a new cancer treatment that takes a patient's own immune cells, modifies them in a lab to recognize and attack their specific cancer, an
See all news about Acute lymphoblastic leukemia

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Acute lymphoblastic leukemia

What is Acute lymphoblastic leukemia?

Acute lymphoblastic leukemia (ALL), also known as acute lymphocytic leukemia or acute lymphoid leukemia, is a cancer of the blood and bone marrow characterized by the uncontrolled proliferation of immature lymphoid cells (lymphoblasts). These abnormal cells accumulate in the bone marrow, crowding out normal blood cell production, and can spread to the blood, lymph nodes, spleen, liver, central nervous system, and other organs. ALL is the most common childhood cancer, though it also occurs in adults. The disease is classified into B-cell ALL (approximately 85% of pediatric cases) and T-cell ALL

How is Acute lymphoblastic leukemia inherited?

Acute lymphoblastic leukemia follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Are there clinical trials for Acute lymphoblastic leukemia?

Yes — 20 recruiting clinical trials are currently listed for Acute lymphoblastic leukemia on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Acute lymphoblastic leukemia?

25 specialists and care centers treating Acute lymphoblastic leukemia are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Acute lymphoblastic leukemia?

13 patient support programs are currently tracked on UniteRare for Acute lymphoblastic leukemia. See the treatments and support programs sections for copay assistance, eligibility, and contact details.