HSCI, a network of the nation’s top stem cell and regenerative medicine researchers, gathers to exchange ideas and forge new collaborations to advance biomedical research.

Summary

• 2018 gathering of stem cell scientists provided rich ground for knowledge exchange.
• New projects, technologies, and ideas were presented and discussed by faculty and trainees.
• Trainees awarded prizes for best talks and posters, based on audience voting.

On May 16th 2018, around 350 members of the Harvard Stem Cell Institute (HSCI) gathered for the 13th Annual HSCI Malkin Retreat at Harvard University’s Northwest Building. The network’s biggest knowledge-exchange event was opened by Massachusetts Senator Elizabeth Warren, whose video address stressed the importance of pursuing and investing in fundamental science. The senator urged HSCI scientists to continue pushing the frontiers of stem cell biology, and searching for transformative therapies that will make a difference for patients, and benefit society. 

The keynote presentations of 2018 were delivered by Nick Leschly, CEO of leading gene-therapy company bluebird bio, and Sally Temple, Director of the Neural Stem Cell Institute. Each of them put patients and public trust first, and urged participants to keep reaching out to the many people looking for answers from stem cell science, including regulators.

Gene therapies: the timing is right

Leschly’s talk was highly personal, and indeed his company’s mantra is to make hope a reality, and always focus on the goal of benefiting patients. bluebird bio’s technology platform, based on gene editing of blood stem cells, is applicable to many diseases, most notably those of the brain and blood. 

Leschly said that while advancing gene therapy still requires investment in basic discovery, the timing for clinical and market success is right, because regulatory processes, approvals, pricing, reimbursement, and industry validation are all aligned. Accordingly, bluebird bio has four programs running in the clinic, will soon introduce two products to the market, and is bring two more closer to commercialization.

The keynote touched on the need to learn from clinical failures, to facilitate multi-disciplinary collaboration among scientists, clinicians, process engineers, and bioengineers, and maintaining the right corporate culture to ensure ethical and scientific decisions are intuitively aligned.

Promises, perils, and public trust

Temple’s keynote address spoke directly to the serious issue of predatory companies, both in the US and globally, that exploit desperate patients. She advocated ethical research and clinical care, and vigilance to ensure potentially harmful cell therapies are not foisted on the public. Temple exhorted her fellow scientists to get involved in educating regulatory authorities. Because stem cells are living products, many fundamental questions must be answered carefully for progress to be possible. For example, how can you characterize a biological product for the regulatory agencies when it is variable by its very nature?

Temple presented her work on creating a cell therapy for retinal repair for macular degeneration. The cell therapy she is developing brings together a range of solutions: the delivery of drugs to stimulate internal repair, and the delivery of cells (adult or pluripotent, in suspension or on a 3D matrix). Which type of therapy makes sense for which patients, and when, is an open question. Her talk inspired a lively discussion.

Updates from HSCI faculty

HSCI co-founder David Scadden was the first faculty member to present his lab’s current work. He focused on regenerating T-cell immunity: a central issue in both infectious disease and aging. For Scadden, unpacking what happens during aging led his group to identify the role of a specific cell population in the bone marrow that could be the target of therapeutic interventions. They also collaborated with scientists in the Harvard John A. Paulson School of Engineering and Applied Sciences (SEAS) to see how new biomaterials could improve T cell generation and function.

Paola Arlotta showed how the new technology of three-dimensional organoids is opening the door to understanding complex diseases such as autism spectrum disorder. The discovery of therapies for diseases like these has been held back by a lack of tools for studying them. Arlotta’s lab is creating ways to analyze the dynamics of these conditions—and potential ways to intervene—that are fit for purpose.

Biologist and bioengineer Jeff Karp shared his lab’s experience studing nature’s solutions to common problems, which sparked new ideas for clinical therapies. Snails, for example, produce a sticky substance that can be adapted into a tissue adhesive that works in a wet environment. One application is creating a tissue patch for healing cardiac septal wall defects. Karp’s group also identified a drug that could be delivered in the ear to regenerate the inner-ear hair cells and restore hearing.

Other talks demonstrated the breadth of expertise across the HSCI network. Several new faculty members presented their work on tendon repair, bone regeneration, blood stem cell growth, and Alzheimer’s disease modeling.

“HSCI helps my work because it creates an adventuresome and exciting atmosphere — one that encourages you to take chances," said Rich Lee, M.D., Head of the HSCI Cardiovascular Program. "The retreat was fantastic. It’s interesting that HSCI is going as strong as ever, in an age when people are trying to figure out how to make a viable therapy.”

Meet the faculty

The 13th Annual HSCI Malkin Retreat offered opportunities for delegates to meet with faculty—and guest speaker Sally Temple—at their lunch tables to discuss set topics. Choosing a scientific question, clinical applications of stem cells, and planning a career path were a few examples. These small gatherings made for conversation on a more intimate level, and allowed junior scientists to mingle with faculty they might not otherwise have a chance to meet.

Live voting

HSCI’s network fosters collaboration among exceptional scientists at many career stages. Several HSCI postdocs and graduate students presented their ideas on topics ranging from Zika to goosebumps, to the formation of blood. Retreat co-organizers Jonathan Hoggatt and Tracy Young-Pearse invited audience members to listen carefully, and cast their vote for the best talk at the end.

This set the scene for the poster session, where everyone was invited to interact with close to 50 presenters, learn about their work, and vote on the best poster. The session was full of energy, with members taking in as much of the work as possible.

Trainee prizes

After all the votes were counted and the presentations fully assessed by Hoggatt and Young-Pearse, it was time to present awards for four presentations and three posters. 

Meryem Gonzalez Celeiro took the Grand Prize for her presentation on how goosebumps form, which led to a new understanding of the interplay of nerve, muscle and hair stem cells in hair formation and regeneration.