1. Research

Mount Sinai: Saving Lives Through Research

At the Mount Sinai Health System, our researchers are asking questions and finding answers that save lives. From mapping the brain circuits that link stress to disease, to engineering personalized cancer vaccines, our investigators work at the intersection of breakthrough science and urgent clinical need. Our goal is to understand the mechanisms of disease so completely that we can prevent it, treat it more effectively, and ultimately cure it.

Mount Sinai’s commitment to discovery spans every stage of life and every system of the body. We are developing new therapies for childhood leukemia and creating brain-computer interfaces to restore independence to patients with paralysis. We are uncovering why aging is the single biggest risk factor for cancer—and identifying drugs that might ameleorate its effects.

Each study at Mount Sinai serves as a bridge between what is possible today and what can be standard care tomorrow. Your support, collaboration, and participation accelerate our journey to save lives.

Blocking Cells That Support Tumors

Miriam Merad, MD, PhD, and Thomas Marron, MD, PhD, have discovered that immune cells in the bone marrow produce an immune cytokine called interleukin-4, which promotes the generation of macrophages that support tumor growth. Their research demonstrates that blocking interleukin-4 responses reduces tumors in mouse models and in human patients.

How Tumors Rewire Immune Cells

Lung tumors don’t just evade the immune system. They reshape it at its source. Dr. Miriam Merad and collaborators have discovered that tumors rewire immune cells in the bone marrow before they even reach the cancer, suggesting a new target to enhance the durability of current immunotherapy.

Overcoming Immunotherapy Resistance

Researchers at the Icahn School of Medicine at Mount Sinai have discovered a way that ovarian cancer tumors manipulate their environment to resist immunotherapy and identified a drug target that could overcome that resistance.

A Personalized Vaccine for Bladder Cancer

As excitement builds around personalized cancer vaccines, researchers at the Icahn School of Medicine at Mount Sinai have reached a new milestone, showing that a custom-made vaccine, in combination with an immune checkpoint inhibitor, is safe and able to spark strong immune responses in people with bladder cancer.

Mapping the Amygdala Liver Axis

The laboratories of Sarah Stanley, MBBCh, PhD, and Paul Kenny, PhD, have collaborated to map a dedicated “amygdala-liver axis” that links social and physical stressors directly to liver glucose production. They discovered that specific neurons connecting the amygdala to the hypothalamus can trigger rapid glucose production in the liver through the nervous system alone, bypassing traditional hormonal pathways. Critically, when these neurons are repeatedly activated by chronic stress, they become less responsive, leading to weight gain, elevated blood sugar, and impaired glucose tolerance.

How Inflammation Alters the Brain

In a study published in Nature, Scott Russo, PhD, and collaborators have demonstrated that stress-induced social avoidance is mediated by a circulating molecule called MMP8 that is produced by monocytes. MMP8 remodels the extracellular space of a specific region of the brain known as the nucleus accumbens.

How Stress Affects Gut Health

Research in the laboratory of Ivan de Araujo, PhD, has uncovered an unexpected connection between brain activity, gut health, and systemic immunity via neural circuits connecting the brain to the gut. This discovery illuminates how stress-related health conditions can arise and points the way to new therapeutic approaches for conditions such as inflammatory bowel disease.

New Therapies for Childhood Leukemia

A team of researchers led by Elvin Wagenblast, PhD, has uncovered why children with the same leukemia-causing gene mutation in blood stem cells can have dramatically different outcomes: it depends on when in development the mutation first occurs. This discovery opens the door to new diagnostic tools that can identify prenatal-origin leukemias, combination therapies that more precisely target vulnerable leukemia stem cells, and clinical trials that incorporate developmental timing into risk assessment.

Identifying Acute Myeloid Leukemia Gene Mutations

Research from the laboratory of Eirini Papapetrou, MD, PhD, has provided new insights into acute myeloid leukemia (AML) and its resistance to a common treatment. Dr. Papapetrou’s group discovered that leukemia stem cells carrying mutations in the RAS gene behave differently from other leukemia stem cells and do not respond well to the widely used drug venetoclax. The findings highlight the importance of tailoring treatments to the specific genetic characteristics of each patient's cancer. Specifically, Dr. Papapetrou’s findings support testing combinations of venetoclax with RAS inhibitors in patients with AML associated with RAS mutations.

Restoring a Youthful State to Aging Blood Cells

Research in the laboratory of Saghi Ghaffari, MD, PhD, has uncovered how to reverse aging in blood-forming stem cells by correcting defects in the stem cell’s lysosomes—specialized structures that act as the cell's recycling system. This discovery opens new therapeutic avenues to prevent or reverse age-related blood disorders, improve the success of stem cell transplants in older patients, and condition blood stem cells for gene therapy.

Understanding How Age Affects Cancer Risk

A novel study by researchers at the Icahn School of Medicine at Mount Sinai addresses a critical, yet under-explored question in cancer research: Why is aging the biggest risk factor for cancer? The study reveals how an aging immune system spurs tumor growth, offering new insights into cancer prevention and treatment, especially for older adults.

Neural Mechanisms in Autism and Schizophrenia

For the first time, Mount Sinai researchers have identified the neural mechanisms in the brain that regulate both positive and negative impressions of a social encounter, as well as how an imbalance between the two could lead to common neuropsychiatric disorders like autism spectrum disorder (ASD) and schizophrenia. The researchers also found that activating a serotonin receptor in the brain of a mouse model of ASD restored positive emotional value, with encouraging implications for the development of new therapies for common neuropsychiatric disorders.

The Brain Regions Involved in Aggressive Behavior

The laboratory of Scott Russo, PhD, at Mount Sinai has identified the cortical amygdala, an olfactory cortical structure, as a key brain region in promoting aggression. This brain region is activated by olfactory cues from male mice and by aggressive behavior. Importantly, the researchers found that inhibiting the cortical amygdala and its downstream circuits reduces aggressive behavior and induces pro-social behavior.

The Power of Brain-Computer Interface Technology

Mount Sinai is leading a global shift in how humans interact with technology. Researchers are advancing a diverse portfolio of brain-computer interfaces and moving beyond laboratory experiments to create reliable tools for patients. By capturing and translating neural activity into digital commands, the researchers are restoring independence to patients with paralysis, amyotrophic lateral sclerosis, and complex neurological conditions.

A Novel Method to Deliver Therapeutics to the Brain

The blood-brain barrier serves as a protective shield, preventing many substances—including potentially beneficial therapies—from reaching the brain. Yizhou Dong, PhD, and his team at the Icahn School of Medicine at Mount Sinai have developed blood-brain barrier-crossing lipid nanoparticles that can safely and efficiently deliver mRNA into the brain. This exciting new technology paves the way for mRNA and gene therapies to treat a wide range of neurological and psychiatric diseases.

Designer Particles Accelerate Diabetic Wound Healing

Researchers in the laboratory of Dr. Dong at the Icahn School of Medicine have designed a regenerative medicine therapy to speed up diabetic wound repair. Using tiny fat particles loaded with genetic instructions to calm down inflammation, the treatment targets problem-causing cells and reduces swelling and harmful molecules in damaged skin.

A New Path for Psychedelics

Psychedelics like LSD and psilocybin (the active ingredient in "magic mushrooms") have recently moved from the counterculture into the lab, showing significant promise for treating complex mental health conditions. Traditionally, scientists believed these drugs worked primarily by activating one specific brain sensor called the 5-HT2A serotonin receptor. However, new research suggests this is only part of the story.

Better Science. Better Lives.

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