At the Harvard Stem Cell Institute, time and timing are a key aspect of both our science and our approach for bringing discoveries to patients. Stem cells are crucial to the early stages of the body’s developmental processes, with the potential to become many different types of cells. As a result, they can teach us how to turn back the clock and regenerate tissues that are damaged by disease. But these passages through time are just the beginning: transforming the promise of stem cells into cures for patients requires bold, innovative thinking, so we support our researchers’ promising ideas at the earliest stages when they would not otherwise be funded by more traditional sources.
In 2020, the power of our approach became clear when the novel coronavirus pandemic demanded urgent solutions. Over a decade ago, HSCI funded an early project investigating how to deliver mRNA into cells, with the goal of reprogramming them into
an earlier stem cell state. That discovery ultimately led to the creation of the biotechnology company Moderna, which adapted the approach for therapeutics and was able to develop a coronavirus vaccine in record time. With the support of our donors, HSCI will continue to make such early investments to enable the next generation of transformative therapies.
We are proud that during the pandemic, HSCI scientists quickly adapted their research and applied their expertise to tackle the coronavirus. Our researchers originally developed lung organoids — miniature 3D cell cultures — to study diseases such as cancer, but are now using them to model coronavirus infection and rapidly screen potential drugs. HSCI researchers have also been part of an effort to catalog every cell type in the lung, and were able to mine the preexisting data to find exactly which cells are targeted by the virus.
Beyond these important contributions, HSCI researchers continued the momentum we have built over our 16 years and made advances in many areas. For instance, we now have better tools than ever before to study how the body develops. When HSCI was first founded, one of our earliest projects involved reprogramming a patient’s skin cells to an earlier developmental state, then converting those stem cells into the type of neuron affected by amyotrophic lateral sclerosis (ALS). That reprogramming technology has now come of age, and is used in different cell therapies in the clinic and as the basis for drug discovery.
But even as we refine these therapies, HSCI researchers are studying development in ever more detail to fuel the next wave of medicines. For example, recent advances in DNA sequencing technology enable us to track how the physical structure of each chromosome in an individual cell changes over time.
Over the years, HSCI scientists have also changed the way we think about disease, by discovering more connections between diseases that were previously considered separate. As the world population ages, we are seeing higher rates of diseases such as cancer and neurodegenerative conditions. HSCI researchers are identifying common links that underlie these diseases of aging, such as a weaker immune system that causes more inflammation, in order to better treat them.
Along with the research showcased in this report, these are just a few highlights of what HSCI accomplished in 2020 and where our passages will take us in the years to come. Our work would not be possible without the support of our donors, and we hope you will join us as we continue to harness the power of stem cells to improve patients’ lives.
Douglas Melton, Ph.D.
David Scadden, M.D.
Brock Reeve, M.Phil., M.B.A.