Highlights from the 2019 HSCI Annual Retreat

May 30, 2019

Boston-area stem cell scientists convene to discuss regulation, future research directions, career paths

The Harvard Stem Cell Institute (HSCI) convened for its 14th annual retreat on May 21st at Harvard Medical School in the Joseph B. Martin Conference Center. The event was a mix of presentations and panels, with poster sessions and topic tables in the New Research Building’s sunny atrium and courtyard.

Program Highlights

Around 300 HSCI scientists welcomed keynote speaker Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. Marks talked about the agency’s drive for the international harmonization of regulation for advanced therapy medicinal products, including gene and cell therapies. He urged researchers to reach out to the agency before heading down the regulatory path, promoting the agency’s INTERACT program.

“We want to control manufacturing processes but we don’t want to be excessive. By having a dialogue with us very early on, we can make sure that the regulation makes sense,” said Marks. “If you are a PI and you have a concept, you can come in to talk about preclinical development and getting through to clinical trial. The FDA is committed to advancing the development of cell-based therapies. We have enough experience now that we can help streamline things and help people overcome limitations in manufacturing.”

Peter Marks, FDA, speaking at a podium at HSCI — Peter Marks, M.D., Ph.D., Director of the FDA’s Center for Biologics Evaluation and Research, speaking about regulation of stem cell products and the need for regulators and researchers to work together more closely. Harvard Stem Cell Institute annual retreat 2019

Regarding concerns about consumers who fall prey to misinformation, Marks touched on increased support for the regulation of stem cell products.

“We are going to make it much easier for people to ask whether their product is ‘minimally manipulated and homologously used.’ If people are legitimately just outside the line, they generally want to do the right thing. But the reality is there are people using the term ‘stem cells’ to sell products that often don’t even contain cells. People are paying out of pocket for stem cell therapies with no cells.”

Scientific presentations

Kevin Eggan, Brian Wainger, and Kasper Roet presented an HSCI story that has been over 10 years in the making: developing stem cell models of ALS in the lab, using the models to identify a biological mechanism of ALS and a potential drug to treat it, and bringing the drug to a successful clinical trial in patients. The presentation highlighted the large number of collaborators involved in the process, as well as HSCI’s dedication to shepherding the treatment through the translational journey from lab bench to clinic.

Three HSCI scientists smiling at the camera — Brian Wainger, Kasper Roet, and Kevin Eggan talked about their collaboration, which resulted in a successful clinical trial of an ALS drug in patients.

The closing keynote was presented by Beth Stevens, associate professor of neurology at Harvard Medical School and Boston Children’s Hospital. Her talk focused on microglia — the immune cells of the brain — and how their interactions with other neurons could play a role in neurodegenerative disease. Her presentation sparked a lively discussion with HSCI scientists, with multiple researchers proposing to exchange information about new techniques and data sets.

Beth Stevens speaking at an HSCI podium — Keynote speaker Beth Stevens, Ph.D., Associate Professor, Harvard Medical School, FM Kirby Neurobiology Center at Boston Children's Hospital, presenting to HSCI scientists on "Unraveling Microglia Function and Dysfunction in Neurodegenerative Disease."

The importance of risk

Scientists who previously trained in HSCI labs talked about their career choices and the value of taking risks. Felicia Pagliuca, Ph.D., scientific co-founder and vice president of cell biology research and development at Semma Therapeutics, said that her time with HSCI had been “life changing.”

“One thing I’ve taken to heart from Doug [Melton’s] lab is the value of taking on big questions and pursuing them fearlessly. Things that most people say will never work, that will take 10 years and hold your career back, that are too difficult. Maybe they’re right 99% of the time, but that is not what moves the field forward and it’s not what is going to help patients.

"We have the luxury as part of the HSCI community to take on those big questions and tackle them. What we were able to do by turning stem cells into insulin-producing cells would not have been possible without bringing together so many people – postdocs, graduate students, undergraduates – to achieve something difficult together. One thing that changed the course of my career as part of HSCI was seeing that incredible science didn’t just live in the lab, it lived in biotech, pharma, venture. All these players were part of the puzzle of how you get from a discovery to something that has a real impact on patients… We are grateful to the companies that funded us so that we are moving into our first clinical trial for cell therapy.”

Four panelists in an auditorium — HSCI summer internship program alumni, speaking about taking risks and the importance of considering different career paths. From left to right: Steven Curtis, Joel Schneider, Felicia Pagliuca, and Jennifer MacDonald.

Awards

Retreat co-organizers Jon Hoggatt of Massachusetts General Hospital and Vikram Khurana of Brigham and Women's Hospital presented awards for the best oral presentation, poster presentation, and video pitch. In the run-up to the conference, early-career scientists were invited to create short ‘elevator pitch’ videos, explaining their research idea. The HSCI Executive Committee presented their top choices, and the audience was invited to vote for their favorite.

Yulia Shwartz from the Hsu Lab won the prize for Best Video Presentation.

Alicia McConnell of the Zon Lab took the top prize for her oral presentation. Runners up were Christine Lian (Lian Lab), Karin Gustaffson (Scadden Lab), and Nayara Leite (Melton Lab).

Co-recipients for the General Poster Session prize were Sekyu Choi (Hsu Lab) and Nick van Gastel (Scadden Lab).

Four scientists show their awards — Sekyu Choi, Ya-Chieh Hsu, Yulia Shwartz, and Meryem Gonzalez Celeiro at the 14th Annual HSCI Retreat in 2019. Sekyu and Yulia received prizes for their presentations. Meryem took the Grand Prize for her presentation in 2018.

14th annual HSCI Retreat co-organizers Vikram Khurana of Brigham and Women's Hospital (left) and Jon Hoggatt of Massachusetts General Hospital (right) at the poster session.

Two challenges to the HSCI scientific community

Doug Melton, who founded HSCI with David Scadden, put forward a bold proposal: to join forces and tackle the challenge of making unlimited amounts of healthy hematopoietic cells.

“There is no cell type with broader application,” he said. “No one person or lab could solve this problem, which demands everything from chemical screening to better lineage analysis.”

Doug Melton standing at an HSCI podium — Doug Melton addresses the 14th annual HSCI retreat, asking delegates to consider joining forces to focus on two major goals.

Melton also challenged HSCI scientists to work toward a universal donor stem cell.

“Let’s imagine we could make a universal donor stem cell – a pluripotent stem cell that can be differentiated into any cell and would be immune protected. Like a pill, it could go into any person. If we want to achieve this, we could engage more with the reproductive immunology and cancer immunology communities. Cancer cells have figured out how to do what we want to do with our stem cells. A tumor cell that can hide from the immune system – that could represent anything we want to transplant. We could learn as a community from people who study reproductive immunology, how the mother’s body knows the baby won’t harm it. This can tell us a lot about how to modify cells to protect them from rejection.

"The history of science is replete with problems like this. It’s much more fun to take on a hard problem and solve it than to do something you already know. And this is a powerhouse of energy for people who want to do things together. Much of what we heard about this morning was about collaboration. The strength of the pack is in the wolf, and the strength of the wolf in in the pack. We should do this together.”