Precision Medicine and the Power of Stem Cells: Highlights of the 2019 NYSCF Conference

In 2006, Shinya Yamanaka, MD, PhD, made a discovery that revolutionized biomedical research and earned him a Nobel Prize. His lab found that stem cells, the body’s ‘building blocks,’ can be made from just a small sample of skin. These cells, called induced pluripotent stem cells (iPSCs), have changed the way we research disease, bringing us closer than ever to cures — Dr. Yamanaka’s (and NYSCF’s) ultimate goal.

“I am doing whatever it takes to bring this technology to patients,” remarked Dr. Yamanaka, in his keynote address at the 2019 NYSCF Conference. “When I was training as a young surgeon, my father passed away. I couldn’t do anything to help him, and that had a huge impact on me. I truly believe that it is science that will help patients suffering from intractable diseases.”

Dr. Yamanaka, Director of the Center for iPS Cell Research and Application (CiRA), Kyoto University, and a Senior Investigator at the Gladstone Institutes, delivers his keynote presentation

During the 2019 NYSCF Conference held at The Rockefeller University on October 22–23, over 500 researchers, students, and thought leaders shared their latest findings in translational stem cell research, highlighting how stem cells are being used to illuminate disease mechanisms, discover new therapies, and advance precision medicine.

A Vision for Precision Medicine

In his plenary lecture, biotech pioneer Lee Hood, MD, PhD, described his approach to modernizing medicine. Instead of only intervening once a disease has begun to manifest — which he calls ‘20th century medicine’ — Dr. Hood urged a more proactive and preventive approach.

Dr. Lee Hood (SVP and Chief Science Officer, Providence St. Joseph Health; Chief Strategy Officer, Co-founder and Professor, Institute for Systems Biology), Dr. Antony Rosen (Vice Dean for Research, Professor of Medicine, Johns Hopkins University), Susan Solomon (NYSCF CEO), Dr. Craig Thompson (President, Memorial Sloan Kettering Cancer Center), Dr. Feng Zhang (Core Member of the Broad Institute of Harvard and MIT and NYSCF – Robertson Investigator), Dr. Raeka Aiyar (NYSCF Senior Director of Scientific Outreach)

“21st century medicine is really about studying wellness and the dynamics of disease, but most importantly, it’s about identifying the transitions from wellness to disease and reversing them at the earliest point,” explained Dr. Hood.

His team uses ‘deep phenotyping’ — that is, integrating vast amounts of clinical and molecular data on individuals — to identify these points of transition. This approach enables earlier diagnosis, identifies how diseases manifest differently across patients, and defines subgroups that would be more responsive to certain drugs.

A panel discussion on precision medicine moderated by Raeka Aiyar, PhD, and featuring Dr. HoodSusan Solomon, JDCraig Thompson, MDAntony Rosen, MBChB, MS, and Feng Zhang, PhD, further explored this topic and announced a new partnership between NYSCF, Johns Hopkins, and Bloomberg Philanthropies.

“Diseases are heterogenous, but within that heterogeneity are homogenous subgroups. Finding these homogenous subgroups at scale is a major opportunity,” explained Dr. Rosen. “With NYSCF, we are creating stem cells from these subgroups and making them available to investigators around the world to see if we can understand the driving mechanisms behind disease.”

“We are eager to move into 21st century medicine,” added Ms. Solomon. “This collaboration not only allows us to bring large-scale automation and AI to create precise biological models, but it allows us the information we need to discover what causes people to become unwell.”

Bringing Stem Cell Technology to Women’s Reproductive Cancers

Women’s reproductive cancers are a group of diseases that could greatly benefit from precision medicine. Most patients receive the same therapies, and the standard of care has not changed much in decades.

A panel discussion examined these issues and highlighted how they are being addressed by NYSCF’s Women’s Reproductive Cancers Initiative. The panel was chaired by Siddhartha Mukherjee, MD, DPhil, and featured Hans Clevers, MD, PhD, Laura Andres-Martin, PhD, Michael Birrer, MD, PhD, and Ursula Matulonis, MD. Dr. Andres-Martin leads the Initiative, and Drs. Mukherjee, Matulonis, and Birrer serve on the Initiative’s Scientific Advisory Board.

Dr. Hans Clevers (group leader at the Hubrecht Institute for Developmental Biology and Stem Cell Research and Professor of Molecular Genetics at the University Medical Center Utrecht and Utrecht University), Dr. Siddhartha Mukherjee (Assistant Professor of Medicine, Columbia University), Dr. Ursula Matulonis (Chief of Gynecologic Oncology, Dana-Farber Cancer Institute), Dr. Michael Birrer (Professor of Medicine, the University of Alabama at Birmingham), Dr. Laura Andres-Martin (NYSCF Research Investigator, Oncology)

“The biology of the disease is so complex, and not much has been done to understand it, in part, because there hasn’t been much funding,” remarked Dr. Andres-Martin. “We realized this had to be addressed on a large scale, so we’ve brought together all these minds from around the world, and are using advanced technologies such as the organoids that Hans has developed to find new ways to help these patients.”

Organoids are 3D aggregates of human tissue made from stem cells that recapitulate how cells develop and interact, just as they would in the body. Dr. Clevers pioneered the development of organoids, which are now being used in labs around the world to study disease and development. At NYSCF, scientists are creating organoids from tumor samples, creating a living biobank that can be used to examine disease progression and test drugs.

“By definition, there are no animal models that are especially predictive. The only way to truly test is in a clinical trial, and going from an animal model to a trial is not very efficient. There’s a dire need for building new models like organoids,” said Dr. Clevers, who also delivered a keynote on how he is using organoids to understand the biology of cancer and screen drugs in his own lab.

The Future of Stem Cell Research

While progress is moving quickly, we cannot realize the full potential of stem cell research if we don’t have 100% of the available brainpower working toward cures. Gender equity in science continues to be a major issue— one that NYSCF is committed to addressing through its Initiative on Women in Science and Engineering (IWISE). Recently, members of IWISE took a closer look at the landscape of gender balance in science, and NYSCF Associate Vice President of External Programs Kristin Smith-Doody presented their findings.

“Our results across hundreds of institutions show that women continue to be systematically underrepresented in STEM leadership, making up under one quarter of full professors,” she explained. “We still have a lot of work to do, and it will take a concerted effort across many groups to reach equality.”

NYSCF Associate Vice President, External Programs

In addition to ensuring women have the resources they need to succeed in STEM, it is also important to foster the field’s young minds, seeding the pipeline for future discoveries. A poster session held on the first day of the conference featured 68 posters from talented young scientists around the world. This year’s winner was Jian Shu of the Broad Institute and Whitehead Institute, and runner-up was Fadi Jacob of Johns Hopkins University and the University of Pennsylvania.

Thank you to everyone who joined us for this year’s conference, and we hope to see you at next year’s meeting, which will take place October 20-21.

More Highlights

  • Core Member of the Broad Institute of MIT and Harvard, Aviv Regev, PhD, is a leader in single-cell biology, using machine learning to create ‘cell atlases’— maps of cell fate that can precisely detail how molecular processes and genetic differences drive development and diseases like autism spectrum disorder.
  • Jun Takahashi, MD, PhD, Professor at Kyoto University, is running a stem-cell-based clinical trial for Parkinson’s. Following positive results in animal models, this therapy is now being tested in 7 patients over 2 years.
  • Graziella Pellegrini, PhD, a Professor at The University of Modena and Reggio Emilia, is currently involved in several clinical trials, including those testing stem-cell-based therapies for ocular burn and the rare genetic disease epidermolysis bullosa. Her update on this work demonstrated that both have promoted healing in patients.
  • Brian Wainger, MD, PhD, a NYSCF – Robertson Stem Cell Investigator, Assistant Professor of Neurology and Anesthesiology at Harvard Medical School, and an attending physician at Massachusetts General Hospital, described how stem cells helped him and his colleague Kevin Eggan, PhD, discover that an epilepsy drug could be used to reduce neuron overactivity in ALS, a therapy that has shown promise in a phase II clinical trial of 65 participants.
  • NYSCF – Robertson Investigator and Assistant Professor of Psychiatry and Behavioral Sciences at Stanford University Sergiu Pasca, MD, is taking advantage of organoids to investigate conditions of the brain. His team identified a group of brain cells severely affected by reduced oxygen in premature birth as well as a compound that could reverse this damage.
  • Tracy Young-Pearse, PhD, Associate Professor in the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital and Harvard Medical School in the Department of Neurology, focuses her research on Alzheimer’s. In collaboration with NYSCF, Dr. Young-Pearse is taking a closer look at what drives the disease and what protects from it, using a cohort of 3,000 people to identify which genetic variants put people at risk and which promote cognitive resilience.
  • NYSCF – Robertson Stem Cell Investigator and the Carolyn Walch Slayman Professor of Genetics at Yale University Valentina Greco, PhD, has made an important discovery about how our hair follicles protect us from the spread of skin cancer. Cells carrying the common HRAS cancer mutation actually spark regeneration in hair follicles, which helps contain cancerous cells.

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