The “Big Ideas in Medicine”: Mass General Brigham Leaders Identify Top Opportunities to Impact Health Care

Based on the insight of more than 100 of the system’s clinicians, researchers, scientists and administrators, Mass General Brigham, one of the leading integrated academic healthcare systems in the U.S., has identified the “Big Ideas in Medicine,” a collection of healthcare advances to influence the future of medicine and help reshape the landscape of patient care. The list was announced at the annual World Medical Innovation Forum.

In a forward-thinking approach, Mass General Brigham convened a diverse group of its experts to envision implementation opportunities and challenges. The resulting collection of ideas centers on next-generation gene editing technologies, modulating the immune system to address brain health and other diseases, new funding models for biomedical research and innovation, and leveraging AI as a workforce multiplier to better understand and anticipate nuances in patient care, among others. The ideas coalesced around emerging scientific breakthroughs, transformative technologies and bold strategies with the potential to fundamentally change diagnosis, treatment and prevention of disease.

The “Big Ideas in Medicine” are:

1. Blazing new trails in the clinic with next-generation genome editing technologies to enable safe, effective, and scalable genetic medicines
   Pursue a new generation of treatments that harness base editing or prime editing technologies to correct and prevent the manifestations of devastating genetic diseases. The continued development and deployment of these novel treatments to a broader patient base, including for diseases that emerge early in life, will require innovations touching the understanding of disease histories, regulatory frameworks for clinical trial applications, manufacturing, delivery methods, and others — all towards the goal of enabling safe, effective and scalable genetic medicines.
2. Reinventing biomedical research funding
   Create new models to fund biomedical research and innovation, both in academia and industry, that support a diverse array of programs and streamline the funding process, including prioritization, program selection and clinical validation.
3. Mapping new frontiers of the immune system in the brain
   Decipher pathways that modulate the immune system both within and outside the brain that help maintain neurological health. This includes potential strategies for blocking T-cells from crossing the blood-brain barrier (BBB) and inducing monocytes to cross the BBB to help clear amyloid in Alzheimer’s disease. In addition, develop a deeper understanding of the biology of T-cell exhaustion and how it applies to diseases beyond cancer, and in organs beyond the brain.
4. An AI-native, agentic care operating system
   Develop the next generation of electronic health records by rethinking how data is organized and how relevant data is extracted and presented to the clinician. This includes the use of AI agents that can harness all relevant patient and external data and suggest subsequent steps of patient care, thereby acting as a workforce multiplier.
5. A new paradigm for organ transplantation
   Build a new paradigm in transplantation medicine through novel approaches for bridging and permanent usage (such as xenotransplantation) and new biological insights that reduce the need for immunosuppressive drugs. This includes technologies for gene editing whole organs as well as for organ preservation and resuscitation — both for human organs and those engineered from animal sources.
6. Living health “mirrors” for prediction and prevention
   Create AI-based, longitudinal digital twins that continuously collect multimodal data from patients, such as through genomic tests, wearables and other sources, to help predict future outcomes and guide patient care strategy and healthcare decisions. This approach includes capabilities for future cost forecasting derived from predicted care paths.
7. Reimagining the future of healthcare delivery
   Explore the future of healthcare through the lens of costs and revenue — where pricing can no longer be a solution. New approaches may lie in massive labor technology advances, rooted in generative AI that can augment the work of clinicians. Another lies in measuring the value of medical innovation, and intersecting with non-traditional models of health care management involving community-based outreach and food and housing support.
8. Turning the tide of antimicrobial resistance
   Commit to addressing the persistent and growing problem of antimicrobial resistance (more than 2.8 million cases per year in the U.S), by more accurately diagnosing and treating infections using rapid diagnostics of common pathogens that work within the timespan of a typical office visit.
9. A new momentum for the menopausal transition in women’s health and wellness
   Develop a more complete understanding of the effects of hormone reduction following menopause and the potential role of hormone therapy during and after the menopausal transition in tissues and systems beyond the reproductive system (e.g., brain, heart, etc.) This includes more complete phenotyping of patients, as well as improved research models that more closely mimic the human condition.
10. Tackling the youth mental health crisis
    Create an early detection and staging system to identify mental health and substance use disorders in children and adolescents. It should include deep partnerships with schools and other community organizations, prepare parents and guardians to recognize early signs, and launch a concerted education effort that de-stigmatizes these conditions. 
11. In cancer, understanding and targeting the soil, not just the seed
    Drive a paradigm shift in cancer treatment through a multi-pronged effort that includes a deeper understanding of the tumor microenvironment — the “soil” a cancer grows in — and its role in tumor initiation, growth, and progression. Additionally, there needs to be a parallel effort aimed at developing drugs that target its components, including blood vessels, peripheral nerves, and the microbiome.
12. An AI-fueled discovery-to-bedside loop for precision medicine
    Design a new, AI-based framework for bringing precision medicine to patients with severe disease that involves the collection of patient-specific parameters, including the disease pathways and targets that are activated within a specific patient. This AI-driven loop can help select available treatment modalities or highlight the need for the development of novel agents to optimize therapeutic outcomes at the individual patient level, including designing n-of-1 or n-of-few clinical trials.

According to Niyum Gandhi, CFO, Mass General Brigham, the “Big Ideas in Medicine” are intended to catalyze new thinking and innovation. “Mapping the future of health care is one thing, but implementation is another,” explains Gandhi. “Our goal is to inspire and encourage clinicians, industry, investors and policymakers to consider these ideas as they collaboratively reimagine what healthcare could be in years ahead.”

The World Medical Innovation Forum brings together leading healthcare experts, investors and industry to accelerate the translation of innovation into real-world impact. Mass General Brigham and presenting sponsor Bank of America host the conference, which is also supported by industry sponsors including biotech pioneer Amgen. The company helped establish the biotechnology industry more than 40 years ago and discovers, develops, manufactures and delivers innovative medicines to help millions of patients in their fight against some of the world's toughest diseases.