CAR T-Cells: The Future of Personalized Medicine?
Written by Sophia Gerrans
Edited by Isabel Louie
In 2012, Tom and Kari Whitehead were looking for a miracle-- their 6-year-old daughter, Emily, had once again relapsed with acute lymphoblastic leukemia. By this point, the disease had spread rapidly, chemotherapy had failed and she was no longer eligible for bone marrow transplants. Tom and Kari were told by doctors the only thing left to do was to take her home, and let the family say goodbye.Kari and Tom refused, instead taking Emily to the Children’s Hospital of Philadelphia, where Dr. Stephan Grupp was starting phase 1 trials for CAR T-cell therapy in pediatric patients. Emily became the very first patient to enroll. In 2022, Emily and her family celebrated the 10-year anniversary of her recovery.
Photos by Julia Leavitt (eks92@cornell.edu)
Emily’s seemingly miraculous treatment started as just a simple question. In 1992, scientists began asking: What if we could train a patient’s T-cells to become specialized cancer-fighting immune cells? They investigated the possibility of extracting a patient’s T-cells, modifying them, and then returning the now super-charged immune cells.
A year later, scientists developed just the weapon needed: chimeric antigen receptors, or CARs. Regular T-cells detect threats using antigen receptors. When these detectors identify a threat, the T-cell kicks into gear, multiplying and recruiting other immune cells. Scientists realized they could create their own CARs to recognize specific cancer markers, like CD19 for leukemia, sharpening the immune system’s recognition of a patient’s unique cancer cells.
In 2010, after almost two decades of development, CAR T-cells were tested in the first human patient. Following a successful trial in adults, two years later Emily became the first pediatric patient.
Shortly after, both Science magazine and the Food and Drug Administration (FDA) officially recognized CAR T-cells as a medical breakthrough, which were awarded their first FDA approval for Acute Lymphocytic Leukemia. In 2024, over 10,000 patients received this life-saving treatment. Today, there are six FDA-approved CAR T-cell therapies.
Photo from iStock. Curated by Kayla Vance (kmv53@cornell.edu)
However, experts report that for a single patient the entire treatment can cost between $500,000 to $1,000,000. As Dr. Micheal Sadelain, Director of the Columbia Initiative in Cell Engineering and Therapy, puts it “if [CAR-T cell therapy] remains expensive, we won’t be able to give CAR T-cells to everyone who needs it.” CAR T-cells are a groundbreaking innovation, but the reality is that the costs are staggering. This estimate is only for the procedure, and doesn’t account for the cost of travel and accommodations to access the few CAR T-cell facilities in the world.
This reveals the unfortunately contradictory nature of personalized medicine within the American healthcare system. Despite being a paradigm that values an individual, empathetic approach to health, it struggles to champion it for everyone. The costs, not only on the individual, but also on the institution, already make these therapies a risky research gamble, and once they enter the pharmaceutical industry they will become primarily dictated by marketplace laws.
Recognizing this, some researchers are developing an alternative: “universal” CAR T-cells. Donor-derived CAR T-cells could be designed with more generic antigen receptors. These could then be administered to any cancer patient. The hope is that this off-the-shelf option could expand availability and drop costs.
CAR T-cell therapy is a testament to scientific ingenuity, but is also a warning. For Emily, CAR T-cells undoubtedly saved her life, but thousands of others still suffer without even a hope of accessing this treatment. As the medical field upholds personalized medicine as the next frontier, it leaves us to wonder: who will be left behind?
Sophia Gerrans ’27 is in the College of Agriculture and Life Sciences. She can be reached at sg2388@cornell.edu.
