CAR-T for Acute Lymphocytic Leukemia
Current treatments using CAR T-cells for leukemia primarily focus on Acute Lymphocytic Leukemia (ALL), a type of cancer affecting the blood and bone marrow, specifically targeting lymphocytes (a type of white blood cell). ALL is the most common form of cancer in children, though it also occurs in adults. In children, treatment often leads to a high chance of cure. However, when ALL affects adults, the likelihood of a cure is significantly lower.
ALL develops in the bone marrow, where blood cells are produced, and is characterized by the uncontrolled growth of unhealthy lymphocytes. These immature cells, known as lymphoblasts, accumulate in the blood and bone marrow, disrupting normal bone marrow function. As a result, they impair the production of healthy blood cells and fail to perform their intended immune functions.
ALL progresses rapidly, producing immature blood cells instead of mature, functional ones. The term “lymphocytic” in Acute Lymphocytic Leukemia refers to the affected white blood cells called lymphocytes. This condition is also known as acute lymphoblastic leukemia.
Before the advent of CAR T-cell therapy, the prognosis for patients with relapsed or refractory (R/R) ALL was poor, with a 5-year survival rate of only 21% in children (1) and 10% in adults (2). However, CAR T-cell therapy has dramatically improved outcomes, achieving high complete remission (CR) rates ranging from 57% to 93%.
CAR T-Cells for B-Cell Acute Lymphoblastic Leukemia
- 42 leukemia patients have been treated with CAR T-cell therapy.
- The 3-month best overall response rate is 85.7% (36 out of 42 patients).
- The first patient treated with this therapy:
- Achieved complete remission within the first two months.
- Has remained relapse-free for 42 months to date.
The side effects of CAR T-cell therapy vary depending on the specific treatment used. Several large companies and a few smaller ones have developed technologies with differing approaches. Newer generations of CAR T-cell therapies tend to have fewer side effects compared to the first-generation treatments.
The most notable side effect of CAR T-cell therapy is Cytokine Release Syndrome (CRS), a potentially serious condition frequently associated with this treatment. CRS occurs when cytokines—chemical messengers that help T cells perform their functions—are released in large quantities as CAR T-cells multiply in the body and attack cancer cells.
CRS symptoms can range from mild, flu-like effects to more severe reactions. Common mild symptoms include:
- Nausea
- Fatigue
- Headache
- Chills
- Fever