Moving the Needle on Appendiceal Cancer

Watch the workshop sessions, including a fireside chat featuring Greg Simon and Christopher Austin and a keynote presentation by David Weinstock.

Cancer of the appendix is a rare disease, several types of which have a poor prognosis. Depending on the type of appendiceal cancer, the 5-year survival rate may be as low as 27%. As for many rare diseases, there are few effective treatments, with little in the drug development pipeline. Currently, there are only about 10 clinical trials for appendiceal cancer therapies.

Last spring, however, stakeholders across academia, industry, patient advocacy groups, and regulatory spaces gathered at MIT for “New Approaches to Accelerating Biomedical Innovation: Case Study on Appendiceal Cancer,” a workshop exploring new avenues for developing novel therapies and moving them through clinical trials and FDA approval to benefit patients.

Co-hosted by MIT’s Laboratory for Financial Engineering and Koch Institute for Integrative Cancer Research, the workshop also featured a keynote presentation by David Weinstock, Vice President of Discovery Oncology at Merck Research Laboratories, and a fireside chat with Greg Simon, who co-founded Faster Cures and served as executive director of the initial Cancer Moonshot and later as president of the Biden Cancer Initiative, and Christopher Austin, CEO of Vesalius Therapeutics and CEO-partner at Flagship Pioneering.

The goal of the workshop was to develop actionable plans to accelerate the development of new treatments, increase funding by reducing the financial risk and uncertainty of transformative research, and to provide a template for addressing these same needs in other rare diseases and in more prevalent cancers.

While the consensus among presenters and attendees was that the road to new treatments for appendiceal cancer would be long and difficult, there was hope for success and a sense of resolve for making an impact on patient survival. When discussing his work on the Cancer Moonshot and Biden Cancer Initiative, Simon recalled that he had to frequently answer the question of “What can you really get done in just a few months?” with “not much.”

He offered a different question for framing intractable problems in treating disease: “What can be started right now that will make an enormous difference in the life expectancy of the people with this disease?” Then he continued, “Let this gathering be a call to action to collaborate and start activities that will make a profound difference.”

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