Related Experiment Video
Updated: Mar 12, 2026
Murine Model of Leukemia Relapse to Induction Chemotherapy for Acute Lymphoblastic Leukemia
Published on: October 17, 2025
Updates in the biology and therapy for infant acute lymphoblastic leukemia
1aDepartment of Pediatrics, Division of Hematology/Oncology/Bone Marrow Transplantation, Children's Mercy Hospital and Clinics, Kansas City, Missouri, USA bPrincess Máxima Center for Paediatric Oncology, Utrecht, The Netherlands.
Insights
Prognosis for infant acute lymphoblastic leukemia (ALL) remains poor. Advances in molecular profiling and targeted therapies offer new hope for improving survival rates in infant ALL patients.
Area of Science:
- Pediatric Oncology
- Hematology
- Molecular Biology
Background:
- Infant acute lymphoblastic leukemia (ALL) has a poor prognosis despite advances in childhood ALL treatment.
- Understanding the unique pathogenesis of infant ALL is crucial for improving outcomes.
Purpose of the Study:
- To review recent advances in understanding infant ALL pathogenesis.
- To discuss translating these findings into clinical trials for better treatment strategies.
Main Methods:
- Molecular profiling studies to characterize infant ALL subtypes.
- Analysis of KMT2A rearrangement (KMT2A-R) status.
- Review of current and emerging therapeutic strategies.
Main Results:
- Infant ALL is categorized into KMT2A-R and non-KMT2A-R types.
- KMT2A-R infant ALL is an epigenomic disease; intensified therapy has not improved survival.
- Non-KMT2A-R infant ALL shows modest survival improvement with chemotherapy intensification.
- Discovery of chemoresistance markers aids risk classification and treatment development.
Conclusions:
- Advances in molecular profiling and targeted therapies show potential for reducing toxicity and improving survival in infant ALL.
- Integrating molecularly targeted therapies into clinical trials is a key strategy for future treatment.
Purpose Of Review:
The prognosis for infants less than 12 months of age who are diagnosed with acute lymphoblastic leukemia (ALL) remains poor despite overall advances in the treatment of childhood ALL. In this review, we highlight the recent advances in the understanding of the pathogenesis of infant ALL and discuss opportunities for translating these findings into clinical trials.
Recent Findings:
Infant ALL can be divided into two major disease types, defined by the presence or absence of KMT2A (MLL) rearrangement (KMT2A-R). Recent molecular profiling studies have found that infant ALL with KMT2A-R is an epigenomic disease that lacks other somatic driver mutations. Strategies to intensify therapy have not improved survival for infants with KMT2A-R ALL. In contrast, infant ALL without KMT2A-R is more similar to ALL of older children and survival has improved modestly with intensification of chemotherapy. Discovery of clonal molecular markers that predict chemoresistance will allow further risk classification and development of novel treatment strategies. Modern clinical trials are integrating molecularly targeted therapies into the treatment of infant ALL.
Summary:
Advances in molecular profiling and integration of targeted therapy have the potential to reduce toxicity and improve survival for infants with ALL.
More Related Videos
10:49Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
06:08Assessment of Chimeric Antigen Receptor T Cell-Associated Toxicities Using an Acute Lymphoblastic Leukemia Patient-Derived Xenograft Mouse Model
Published on: February 10, 2023
Related Concept Videos
01:22Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
02:50Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
02:49Cancer Therapies
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...