The first patients at the University of Pennsylvania to receive an experimental CAR T cell therapy for leukemia achieved results that surpassed all expectations, ultimately paving the way for the world's first approved CAR T cell therapy, Kymriah. The remarkable outcomes from these early trials demonstrated the transformative potential of engineered immune cell treatments for cancer patients.
Unprecedented Clinical Responses
The initial results from the University of Pennsylvania trials were nothing short of extraordinary. "The first patient's biopsy came back with no evidence of leukemia," according to accounts from the research team. When Carl June received the email from David Porter about these results, his reaction captured the disbelief shared by the scientific community: "I don't believe it – it's too good to be true."
The skepticism was understandable given the novelty of the approach, but a repeat biopsy confirmed the initial findings. The success continued with subsequent patients, as the same remarkable outcomes were observed in the second and third patients treated. Among the first three patients, the trial achieved two complete responses and one partial response – results that would reshape the landscape of cancer immunotherapy.
Key Scientists Behind the Breakthrough
Bruce Levine, now the Barbara and Edward Netter professor in cancer gene therapy at the University of Pennsylvania, was among the key scientists involved in these pioneering studies alongside Carl June and others. Levine's continued involvement in the field extends beyond his research role, as he serves as a former president of the International Society for Cell and Gene Therapy (ISCT) and currently chairs ISCT's committee on ethics in cell and gene therapy.
Reflecting on his ongoing commitment to the field, Levine notes: "Once you're with ISCT, it's kind of an addiction to stay with them." This dedication to advancing cell and gene therapy reflects the passion that drove the early CAR T cell research at the University of Pennsylvania.
Path to the First Approved CAR T Therapy
The exceptional results from these early University of Pennsylvania trials provided the foundation for what would become Kymriah, the world's first CAR T cell therapy to receive regulatory approval. The journey from experimental treatment to approved therapy represents a milestone in cancer treatment, offering new hope for patients with previously limited therapeutic options.
The development of Kymriah marked a pivotal moment in oncology, demonstrating that genetically modified immune cells could achieve sustained remissions in patients with treatment-resistant leukemia. The success of these initial trials validated the CAR T cell approach and opened the door for additional CAR T therapies that would follow.
