Day 1 began with two sets of First Look sessions—rapid-fire presentations showcasing the next wave of GCT breakthroughs from 16 investigators across the Mass General Brigham system.
Topics included host-microbiome interactions in health and disease; using reprogrammed stem cells to test strategies for the targeted degradation of tau as a treatment for Alzheimer’s disease; alternatives to immunosuppressive drugs to prevent rejection of transplanted organs and a 3D organoid platform to model the blood-brain barrier, among others.
A new feature for the WMIF in 2022, a series of lunchtime “The Doctor is In” sessions provided attendees with an opportunity to take deeper dives into the scientific challenges and opportunities in GCT.
Florian Eichler, MD, Director of the Center for Rare Neurological Diseases at Massachusetts General Hospital, discussed research and therapeutic efforts into single-gene correction in the brain.
In a question and answer session with Tazeen Ahmad, Managing Director, Global Research, BofA Securities, Eichler was asked what criteria should be used to determine the safety of treatments and the tolerability of side effects, particularly when it comes to diseases that are often fatal when untreated.
Lee Greenwood, whose child is being treated by Eichler for Canavan disease, expressed his heartfelt appreciation for Eichler’s efforts to identify and test new treatments and his openness about the risks involved.
“My family, we certainly know the pros and cons,” Greenwood said. “We’re going into this with our eyes wide open. But we know what the path is when there is no hope, there is no treatment, and that path is awful. To have something, anything, where we can give our child some hope—it was an easy choice, honestly.”
The afternoon session began with a conversation with Anne Klibanski, MD, President and CEO of Mass General Brigham, and Brian Moynihan, Chair and CEO of Bank of America that was moderated by CNBC reporter Bertha Coombs.
The discussion highlighted the need to improve data sharing within and across health care systems; the importance of preventive care and wellness initiatives to lower healthcare costs; and the need to work collectively to accelerate the development of new GCT treatments—and make them available to all patients.
Watch Opening Remarks
In a panel discussion of WMIF co-chairs, several themes emerged—the excitement surrounding new therapies for previously untreatable diseases, the importance of collaborating across disciplines to move new treatments forward in the most cost-effective ways, and the need to engage patients as partners in the research process.
“What’s so exciting about GCT is it is potentially curative,” said Marcela Maus, MD, PhD, Director of the Cellular Immunotherapy Program at the Mass General Cancer Center. “There are so many things in medicine where it is palliative, or where we treat symptomatically, or try to modify the outcome of disease without really getting at the fundamental mechanisms of the disease. You can get at that with gene and cell therapy.”
The sentiment was echoed by Ravi Thadhani, MD, Chief Academic Officer of Mass General Brigham. “That’s what exciting about these next few days,” Thadhani said. “The opportunity to make a difference in those diseases that otherwise have been neglected or we had no therapies to deliver.”
It was also clear from the discussion that more work needs to be done to accelerate the development process, identify new funding models and lower the cost of treatments.
“We can’t do it alone. We need partnership,” said Miceal Chamberlain, President of Massachusetts, Northeast Regional Executive, Bank of America. “Everyone has to be aligned in this ecosystem in order for this to work.”
Watch Co-Chair Kick Off