ACGT Scientific Advisory Council member Carl June, MD, (University of Pennsylvania) and ACGT Board of Directors member Marc Engelsgjerd, MD (Royalty Pharma) speak to assembled experts and guests at ACGT’s Lunch Symposium at SITC 2025.
Alliance for Cancer Gene Therapy (ACGT) had a strong presence at this year’s Society for Immunotherapy of Cancer (SITC) 2025 Annual Meeting in National Harbor, Maryland, hosting a Lunch Symposium that brought together leading experts in cancer cell and gene therapy to connect and share knowledge and insights.
The ACGT Lunch Symposium included a fireside chat with cell and gene therapy trailblazer and ACGT Scientific Advisory Council (SAC) member Carl H. June, MD (University of Pennsylvania), moderated by Marc Engelsgjerd, MD (Royalty Pharma), a member of ACGT’s Board of Directors.
Dr. June reflected on a career that has left a singular imprint on cancer cell and gene therapy, sharing his perspective on the current state of the field and offering insights into potential future directions for cancer immunotherapy.
Dr. June focused on the development of in vivo CAR T approaches, in which genetic payloads are delivered directly to T cells within the patient. Dr. June drew on his experience with small patient cohorts in prior cell and gene therapy trials, including his 2010 trial in refractory leukemia, which enrolled only three patients.
This is particularly relevant today, as a recent German study involving only 15 patients with refractory autoimmune disease achieved a 100 percent response rate, potentially opening the way for similar studies in in vivo CAR T therapy.
“You can have paradigm shifts with very few patients in cell and gene therapy,” Dr. June said. “In the space of just a year, because of what happened in Germany, the regulatory authorities are allowing us to inject integrated viruses into humans. I never thought that would happen in my lifetime. Those few patients that showed the possibility of response have completely changed that equation on risk balance.”
Dr. June also shared his take on the current US regulatory and funding environment for medical research, how it has changed significantly in recent years, and what researchers may expect in the future.
“If we don’t have a stable and nimble regulatory environment, investments are not going to happen in US-based pharma and biotech unless investors and companies have confidence that they can get their products to market,” Dr. June said. “So, we have a huge issue here and pressure to move investments and clinical trials offshore. There is also a long-term international issue regarding our competitiveness in the US pharmaceutical industry relative to China. So, we need to fix that.”
Dr. June observed that advances in China’s regulatory system have allowed the country to become a hotbed for innovative medical research.
“China has a two-tier clinical trial system where at a local level they can get approval with one ethical review board and not have to go to the equivalent of China’s FDA,” Dr. June said. “That has made it so that trials can start rapidly. It’s much cheaper and faster to run trials in China. I think the real threat is that US biotech is now doing their new first-in-human trials in China or Australia, where costs are lower and early-stage trials are more efficient.”
Lastly, Dr. June addressed access to CAR T therapies and how to make cell and gene therapies more affordable and accessible for all patients who qualify.
“We have a healthcare system that hasn’t figured out how to deal with the high costs of cell and gene therapies,” Dr. June said. “We need to have a way to have second-party and third-party insurance incentivized to manage curative therapies. With scale, the in vivo delivery of cell therapies will be much cheaper. Ex vivomanufacturing can be made more efficient through robotics; a lot of that technology is replacing expensive human labor. So, the cost is going to come down with scale, but we’re still in the very early days of that.”
The ACGT Lunch Symposium also featured a scientific presentation by Stephen Gottschalk, MD, and Giedre Krensciute, PhD (St. Jude Children’s Research Hospital), both members of the ACGT-funded STAR (Synthetic T-cell therapy against recurrent pediatric brain tumors) collaboration, which was honored at the SITC meeting with the SITC 2025 Collaboration Award.
The STAR collaboration aims to develop new T cell-based immunotherapies for pediatric brain cancers and was funded by the ACGT-Barbara Netter Fund Collaboration Challenge Award. This collaboration focuses on the pediatric brain tumors medulloblastoma and ependymoma, which combined are diagnosed in approximately 700 children in the United States each year.
The research project aims to target these cancers in children who have relapsed or not responded well to chemotherapy by genetically engineering synthetic T cells that achieve higher T-cell persistence against immunosuppressive brain tumors with lower toxicity.
At the SITC annual meeting, several ACGT SAC members and Research Fellows received honors and awards. Leading the way was Ira Mellman, PhD (Parker Institute for Cancer Immunotherapy and Medici Therapeutics), who received the prestigious Richard V. Smalley, MD Memorial Award and Lectureship, the highest award at SITC, recognizing his pioneering work in cancer immunology.
Dr. Mellman earned the honor for his long career as a leader in cancer immunology research. Dr. Mellman previously led Genentech’s early cancer vaccine programs against targeting patient-specific mutant neo-antigens using mRNA and DNA, and has since made contributions such as elucidating how dendritic cells initiate immunity or maintain immune tolerance, and how T cell signaling is regulated by immune checkpoints, among other achievements.
Dr. Mellman’s subsequent talk, entitled “The Coming Renaissance of Cancer Immunotherapy,” reviewed his career at the forefront of immuno-oncology research while offering insights into forthcoming breakthroughs, with particular focus on TILs (tumor-infiltrating lymphocytes) and cancer vaccines.
“The biggest win will be to figure out how to adapt forms of cell therapy to treat solid tumors,” Dr. Mellman said. “We have the opportunity to build on the modalities that today have significant clinical proof of concept, but so far, limited research and development activity. To me, this is a gift. In the case of TILs and therapeutic cancer vaccines, we know these things work. We know they can elicit clinical responses in patients. We need to be able to reproduce these responses and make these therapies cost-effective.”
Dr. Mellman presented data on the efficacy and, in some cases, curative effects of TIL therapy across multiple tumor types and discussed the potential to combine TILs with cancer vaccines.
In closing, Dr. Mellman highlighted the remarkable breakthroughs that are on the horizon using TCR-based and in vivo cell therapies that will allow companies to scale and provide these products to patients around the world at an incredibly reduced cost, leaving the SITC audience with an inspiring and motivating message of continued successes in cell therapies in the near future.
During the Awards Ceremony following Dr. Mellman’s lecture, ACGT SAC Chair Michael Lotze, PhD (University of Pittsburgh), and ACGT Research Fellow George Coukos, MD, PhD (University of Lausanne), were both recognized as 2025 Fellows of the Academy of Immuno-Oncology, which honors individuals who have significantly advanced the field of cancer immunotherapy.
