Acute Lymphocytic Leukemia and CAR-T therapy
By: Rachel Daniel, Greenwood High International
Summary
This project explores explores the genetic basis and advanced treatments for Acute Lymphocytic Leukemia (ALL). ALL is an aggressive blood cancer primarily affecting children, characterized by the abnormal proliferation of immature lymphocytes. The ETV6-RUNX1 translocation, occurring in 25% of ALL cases, disrupts hematopoiesis and requires a "second hit" for leukemic transformation, highlighting its complex genetic origin. Symptoms of ALL range from fatigue and weight loss to severe immune dysfunction. Diagnosis involves blood tests, bone marrow analysis, and spinal fluid tests. Treatments include chemotherapy, targeted drug therapies, stem cell transplants, and advanced immunotherapies like CAR-T cell therapy. CAR-T therapy involves engineering a patient’s T-cells to target leukemia cells expressing CD19. Although highly effective, CAR-T therapy is expensive and primarily reserved for relapsed or refractory cases. The study emphasizes the need for more targeted therapies, including multi-protein targeting CAR-T therapies and exploring epigenetic therapies to prevent leukemic transformation. Further research into the genetic and epigenetic factors behind ETV6-RUNX1 could refine therapies and improve outcomes for pediatric ALL patients, making treatments safer, more specific, and widely accessible.
ETV6-RUNX1 translocation and its effects on the expression of genes (Figure representation created by the author: Rachel Daniel)
Video Presentation
Impact Statement
My name is Rachel Daniel and I am a student of Greenwood High International (India). My project delved into the topic of acute lymphocytic leukaemia and the genetic factors that control the onset of this disease. I also explored some of the most effective treatments for ALL, the main one being CAR-T cell therapy. The program itself specialized in understanding diseases from a broad overview (Epidemiology) to a molecular level (Genetics). I liked the progression of learning through the classes. We started by finding out the populations where the disease is prevalent and to the environment in that area that could effect the onset of the disease and finally the defect at the molecular level. This project helped me understand the research process and helped me gain insight in how to structure a paper. I loved the fact that our mentor was always available and answered my queries even at odd times of the day.
Report White Paper
_By: Rachel Daniel_
The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of Elio Academy.
