Listen to Dr. Crystal Mackall on the Future of Medicine podcast — a fascinating conversation about the science, breakthroughs, and challenges shaping the next era of cancer treatment.

Dr. Crystal Mackall, a pioneer in cancer immunotherapy, traces her journey from a working-class Ohio upbringing to leading cutting-edge T-cell research at Stanford. Inspired by family members lost to cancer, she pursued medicine early, eventually training at the National Cancer Institute during the formative years of immunotherapy in the 1980s.

Her work centers on CAR T-cell therapy, particularly for pediatric cancers with no viable alternatives. A landmark collaboration with neurobiologist Michelle Monje led to a GD2-targeted CAR T-cell therapy for diffuse midline glioma — a devastating brainstem tumor affecting young children. With over 50 patients treated at Stanford, the results have been remarkable, with some wheelchair-bound children regaining the ability to walk.

Mackall speaks candidly about the barriers to access, noting that only 20% of eligible U.S. patients currently receive potentially curative CAR T-cell therapies due to cost and complexity. To address this, she co-founded the nonprofit Access for Kids and is exploring hybrid nonprofit/for-profit models to make cell therapies economically viable for rare pediatric cancers. She has also worked with the FDA — alongside partners including Friends of Cancer Research and the Parker Institute — to advocate for more flexible manufacturing requirements for small-batch cell therapies, recently resulting in new FDA guidance.

Looking ahead, Mackall believes the future of medicine lies in nucleic acid-based therapeutics delivered via lipid nanoparticles — essentially turning cell therapy into gene therapy.

https://medicine.stanford.edu/news/stories/episodes/crystal-mackall-cell-therapy.html