The 2019 HSCI retreat, held at Harvard Medical School’s Joseph B. Martin Conference Center, welcomed 300 scientists. The event was opened by Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, who talked about the agency’s efforts to harmonize gene and cell therapy regulation internationally, reflected on the rapid acceleration of the field, and addressed the serious issues caused by the spread of misinformation.
HSCI scientists Kevin Eggan, Brian Wainger, and Kasper Roet shared a story that has been over 10 years in the making. Together, they developed human stem cell models of ALS in the lab, used them to identify a biological mechanism of ALS and a potential drug to treat it, and brought the drug to a successful clinical trial in patients.
Following a day packed with scientific presentations, panel discussions, and poster browsing, retreat co-
organizers Jonathan Hoggatt of Massachusetts General Hospital and Vikram Khurana of Brigham and Women’s Hospital presented awards for the best oral presentation to Alicia McConnell, best poster presentation to Sekyu Choi and Nick van Gastel, and best video presentation to Yulia Shwartz.
HSCI hosted the 12th annual Business of Regenerative Medicine conference in 2019, exploring how to define and create “value” in a field that is set to transform human health. Over three days, 150 scientists, CEOs, biotech pioneers, venture capitalists, and patient advocates gathered to share their perspectives on social, economic, and operational challenges in this emerging field.
Panelists at BRM 2019 discussed the issue that while scientific progress is rapid, health care markets remain unprepared to manage the one-time cost of cures. Featured speakers fired the imagination with presentations about the potential to print cells ‘at the bedside’ using 3D bioprinting that combines existing technologies; accelerating drug discovery with an ‘intestine on a chip’ that exposes human gut cells to complex biological forces; and using multi-layered biomaterials to deliver antibiotics, nucleic acids, and drugs within the same complex.
HSCI research featured prominently, including work from the David Scadden lab that is being taken forward by Magenta Therapeutics. Their revolutionary approach to bone marrow transplants would remove stem cells from a patient in a targeted manner with a single dose, with no side effects. If successful, the clinical trial slated for 2020 will be a major step towards making stem cell transplants an outpatient procedure.
The conference was opened by George Q. Daley, dean of Harvard Medical School and HSCI Principal Faculty member, who said: “We are at an inflection point in regenerative medicine, when CAR-T cells, dopaminergic neurons for Parkinson’s disease, beta cells for type 1 diabetes, and treatments for the retinal epithelium are making history. But transformative therapies typically take between 30 to 40 years to mature, and we are one decade into our investments in regenerative medicine products.”