Targeted Therapy in Acute Lymphoblastic Leukaemia

Area of Science:

• Hematology
• Oncology
• Genetics

Background:

• Significant advancements in understanding genetic lesions in acute lymphoblastic leukaemia (ALL) have occurred.
• Next-generation sequencing has identified driver mutations impacting prognosis and defining subtypes like BCR-ABL-like ALL.

Purpose of the Study:

• To review available and emerging targeted therapeutics for ALL.
• To explore the mechanisms of action for these targeted therapies.
• To discuss the current evidence supporting their clinical use.

Main Methods:

• Literature review of recent studies on targeted therapies in ALL.
• Analysis of genetic mutations and their therapeutic implications.
• Evaluation of clinical trial data for novel agents.

Main Results:

• Tyrosine kinase inhibitors (TKIs) and JAK inhibitors show promise for BCR-ABL-like ALL.
• Targeted agents like monoclonal antibodies and CAR T cells are active in B-ALL, with specific toxicities.
• Advances in T-ALL treatment include nelarabine, bortezomib, and CAR T cell therapy.

Conclusions:

• Targeted therapies are revolutionizing ALL treatment, with significant progress in B-ALL.
• Further research is needed to optimize T-ALL therapies and manage treatment-related toxicities.
• Understanding ALL biology enables targeting of pathways like apoptosis and epigenetic regulation.