On June 5, the United States Food and Drug Administration (FDA) held a cell and gene therapy roundtable discussing rare disease and cancer therapies and the opportunity to consider product development from the patient and family point of view. Leading experts, including ACGT Scientific Advisory Council members and Research Fellows Crystal Mackall, MD (Stanford University) and Carl June, MD (University of Pennsylvania), were invited to share with the FDA the progress and challenges and, most importantly, the incredible potential in curing diseases through cell and gene therapies.
Dr. Mackall made a compelling case for building new therapeutic development and financial models to allow innovative cell and gene therapies for pediatric cancers to succeed, while Dr. June made a powerful call to action to FDA to move fast to modernize and adapt the regulatory process to secure US leadership in the field.
ACGT continues to fund the research that is driving the cancer cell and gene therapy pipeline and we are committed to continuing to bring curative new options to the patients who need them the most.
“A regulatory environment that nurtures innovation and gives investors confidence is critical to keep industry and academia working together on US soil,” Dr. June said. “Chinese momentum should be a wake-up call. In 2024, China surpassed the US in new drug clinical trials with 7,100 in China versus 6,000 in the US, reflecting their streamlined regulatory environment. The US still leads in the basic biomedical science, but we must not become complacent. We must get a more enabling regulatory climate. I urge the FDA to implement regulatory reforms that will maintain US leadership in this field.
Embracing a dual tier approach for early-stage trials like in China will allow FDA and IRB approved pilot trials to proceed and involving only the FDA later in larger regulatory trials would maintain safety through local oversight while accelerating innovation. This approach would not lower standards but would right-size them. So, I believe this adaptive framework is essential for the US to preserve leadership in cell and gene therapy. Autologous T- cell trials should not require FDA oversight in initial exploratory trials. The future of medicine with cell and gene therapy is at stake. We must act now to modernize our regulatory pathways.”
“The scientific advances that we have witnessed are just nothing short of spectacular,” Dr. Mackall said. “It’s not hyperbole. It’s not overstated. We could have never imagined at the time the power and precision that we can now endow into human immune cells. Even human immune cells taken from patients with endstage heavily pre-treated cancers. One dose of autologous gene modified cells can cure about half of large B-cell lymphoma patients. About a third of all patients with B-ALL and it looks like a third of now multiple myeloma patients whose diseases in all those cases were resistant to all other therapeutic classes. I mean, these are truly miraculous responses.
The glass is half full. But despite this unconditional scientific success, the field is really struggling to deliver these therapies to all patients who can benefit. I mean, even in the adult space, we only have market penetration at about 20%. But in pediatrics, we have essentially a complete market failure. Not only have we not seen any other approvals come down the pike, what we know is in the developmental pipeline of Biopharma today; there aren’t pediatric indications there. It’s just empty. Clinical trials, however, have demonstrated impressive results in pediatric brain tumors, solid cancers, neuroblastoma, T- cell leukemia, many of the biggest killers of children with cancer. But they’re not moving beyond research studies. And why? It’s mainly due to the high costs of development and the regulatory risk.”
