Discoveries made at Harvard Medical School have improved the health and lives of countless people. Today, we continue our progress toward the treatments of tomorrow.
Research Under Threat
Research at HMS now faces an existential threat from sweeping federal cuts, with more than 350 HMS grants worth $230 million having been terminated.
Sections
- Artificial Intelligence
- Autoimmune Disease and Inflammation
- Brain Health and Neurodegenerative Disease
- Cancer
- Heart Disease and Related Vascular Diseases
- Infectious Diseases and Pandemics
- Microbiome
- Therapeutic Discovery
Artificial Intelligence
HMS researchers are using artificial intelligence (AI) to powerfully augment and accelerate their discoveries.
- In cancer, AI is optimizing diagnosis, forecasting treatment response, and predicting survival
- In infectious disease, AI is illuminating viral maneuvers and pandemic risk
- In genetics, AI is helping to distinguish benign from disease-causing gene variants
- AI is redefining drug development, with the goal of optimizing what’s been a long, costly bench-to-bedside journey
- In rare disease research, AI is helping to repurpose old drugs for new uses
- AI is helping scientists untangle the exposome to understand how various environmental exposures — diet, pollution, and stress — interact with our genes to affect disease risk and influence our health
- In structural biology, AI is predicting protein folding — which is key to understanding protein behavior in disease and health
- AI is analyzing how proteins behave in context, illuminating how surrounding cells and tissues influence protein behaviors involved in maintaining health and fueling disease
Beyond discovery, AI is empowering physicians to become more astute diagnosticians, it’s reshaping medical education, and it could even help make medicine more human.
Autoimmune Disease and Inflammation
HMS scientists are working to understand how the immune system protects the body – and how it sometimes turns against it. This knowledge can lead to new therapies for autoimmune disease, inflammatory conditions, and cancer.
We’ve made major strides in the following areas:
- Decoding how the immune system recognizes threats – and what happens when that recognition fails
- Elucidating mysteries about the immune system and why sometimes symptoms linger long after infection
- Understanding the crosstalk between brain and immune cells to explore how inflammation may influence mood and behavior — insights that could lead to immune-modulating treatments for anxiety and autism
- Discovering a population of highly specialized regulatory T cells that act as inflammation gatekeepers in the brain. These findings could open new paths for treating neurodegenerative diseases driven by inflammation in the brain
- Understanding how the immune and nervous systems interact to defend the brain. Researchers have mapped how bacteria breach the brain’s protective barrier in meningitis, a highly fatal disease. They have also identified a sensor protein that controls gut movement in response to pressure, exercise, and inflammation, which could guide the design of new treatments for gut disorders
Brain Health and Neurodegenerative Disease
Scientists at HMS are uncovering new insights on the brain in remarkable detail, including:
- How the brain develops and wires itself
- How it forms memories and maintains its plasticity
- How autism, Huntington’s disease, and frontotemporal dementia arise
Using genetics and artificial intelligence, our researchers are decoding how neurons communicate with the immune system in search of new treatments for neuropsychiatric disorders such as schizophrenia. This exploration extends to the senses, such as interoception, touch, smell, pain and the brain-gut connection.
We’re also working to understand the mechanisms that regulate the function and integrity of the blood-brain barrier, a network of interlaced cells that line the blood vessels to guard the central nervous system against harmful bacteria, toxins, and other blood-borne pathogens. Understanding how the barrier works could help scientists develop medicines that selectively cross it to deliver much needed therapies into hard-to-reach regions of the brain.
Our progress in neurobiology and gene-editing techniques is fueling clinical trials of new therapies for rare disorders that affect vision and hearing such as Usher syndrome and retinitis pigmentosa.
Cancer
Since 2020, 56% of all new cancer drugs and cell therapies approved by the FDA have come from scientific and clinical contributions from Harvard Medical School and its affiliated hospitals.
Today, our researchers are elucidating some of cancer’s most confounding maneuvers that continue to thwart prevention and treatment, including:
- How tumors arise and evolve
- How tumors develop and why they come back
- How tumors evade detection and disarm our defenses against them
- What renders tumors vulnerable
These insights can help to improve existing therapies and inform the design of new treatments, such as individualized kidney cancer vaccines and novel forms of CAR-T therapy for glioblastoma, an aggressive and often-fatal brain cancer.
Heart Disease and Related Vascular Diseases
HMS scientists are making discoveries to advance the care and treatment of cardiovascular conditions, including increased understanding of:
- Molecular mechanics that drive the heart to contract and relax, uncovering how subtle changes can lead to hypertrophic cardiomyopathy, a condition tied to sudden cardiac death
- How genetic alterations in the cells that line blood vessels can amplify heart disease risk and cause brain-vessel anomalies
- How molecular pathways can disrupt heart muscle function and lead to different forms of heart failure
To stay ahead of heart disease, they’re developing new ways to more accurately gauge heart disease risk decades before symptoms appear and using computational tools to refine existing heart-risk calculators to help tailor treatments to each individual
Infectious Diseases and Pandemics
In ongoing efforts to contain current outbreaks and prepare for future threats, HMS scientists are making critical discoveries across the infectious disease landscape. Recent advances include:
- Identifying a key component of the deadly Nipah virus — a discovery that can guide the design of new treatments
- Mapping the structure of a receptor on antibody-producing immune cells, offering insights that could can inform vaccine design and new therapies for COVID, cancer, and beyond
- Uncovering how alphaviruses, including the deadly eastern equine encephalitis virus, infect host cells. This work lays the groundwork for targeted therapies and preventive measures against these highly fatal viruses
- Identifying three new safe and effective oral regimens to treat drug resistant forms of tuberculosis, offering shorter, more accessible options for patients worldwide
- Finding a major clue in the long-standing mystery of how dormant bacteria spring back to life, informing new strategies to prevent infections and food spoilage
- Developing an imaging technique that captures gene activity at the single-cell level within individual bacteria, allowing unprecedented insight into bacterial behavior, virulence, and drug resistance
Harvard Medical School is also home to a global consortium for pathogen readiness focused on infectious threats — both old and new.
Microbiome
Once dismissed as passive bystanders, the trillions of microbes living in and on our bodies are increasingly seen as powerful players in health — so much so that many scientists consider the microbiome an organ in its own right.
HMS scientists have made critical discoveries on the microbiome, including:
- How it shapes everything from the immune system, to mental health and postpartum depression, to metabolic disorders like diabetes, to autoimmune neurologic diseases like multiple sclerosis and Parkinson’s disease
- Uncovering causal links between the microbiome and disease using advances in computational biology, AI, and multi-omic tools
- How microbes may be used to boost the efficacy of cancer immunotherapy
Therapeutic Discovery
Our ongoing efforts to understand how diseases arise at the molecular and cellular level are animated by a singular focus — our quest to transform these insights into frontline therapies used at the patient bedside to change outcomes and transform lives.
In 2020, Harvard Medical School launched its Therapeutics Initiative, a suite of programs aimed at accelerating the transition from basic insights made in the lab into real-world treatments.
Key elements of this effort include:
- The Blavatnik Harvard Life Lab Longwood, a 10,000-square-foot incubator designed to support early-stage biotech and life sciences startups founded by Harvard scientists
- Funding for early-stage research with therapeutic promise as well as for more mature science on the cusp of therapeutic translation
- Education in therapeutic science: Training the inventors of tomorrow’s medicines through programs like the Therapeutics Graduate Program and a new master’s degree in therapeutic discovery