Stuyvesant High School
New York, NY
Teacher: Maria Nedwidek-Moore
Driving Leukemia Away: The Future of CAR Therapy
Nearly 45 years ago, Theodore Friedmann and Richard Roblin of the Salk Institute proposed in Science magazine that “exogenous, ‘good’ DNA be used to replace defective DNA in those who suffer from genetic defects” such as phenylketonuria . They assumed that many genetic diseases were caused by “enzyme deficiencies” and could be cured simply by introducing correct genes into a patient’s afflicted cells…
…Chimeric antigen receptor T-cell (CAR-T) therapies are prime examples: they qualify as gene therapies (physicians introduce exogenous DNA into a patient’s cells with viral vectors) but, by transforming T-cells with fusion proteins ex vivo in order to treat leukemias, they represent the expansion of the field into new, promising territory.
…CAR-T therapies treat ALL indirectly by liberating the body’s own immune system to kill malignant cells. CAR-T is thus an immunotherapy: one of a new class of treatments that unleash the immune system on cancers.
The concept of CAR-T is straightforward — that a patient’s T cells may be extracted from the blood, genetically engineered ex vivo with a specially-designed fusion protein (a CAR), then infused back into the patient to mount a powerful, highly specific immune response . Transformed CAR-T cells will express the CAR on their cell membranes. A CAR is capable of streamlining and condensing an immune response that would normally occur in multiple cell types simultaneously into one signaling pathway within a single cell. This lets CAR-T cells efficiently target circulating cancer cells [12,13]…