Have you ever thought about what kind of return you get from investing in medical research?
At the Vanderbilt University School of Medicine Basic Sciences, researchers are continually working to develop the next generation of therapies, train future scientists, and improve early disease detection. Below are some of the breakthroughs our researchers have achieved over the last 12 months.
Neurological disorders
Alzheimer’s disease
Alzheimer’s disease is the most common cause of dementia, affecting over seven million people in the United States alone. In a recent paper published in ACS Chemical Neuroscience, Department of Pharmacology researchers developed a tool compound that selectively inhibits a protein that is linked to Alzheimer’s but which has not been thoroughly studied due, in part, to a lack of “tool” compounds to study it with.
Read the full story: Novel compounds open new research avenues for Alzheimer’s disease therapeutics
Schizophrenia
Research from the Warren Center for Neuroscience Drug Discovery resulted in the proposal of a novel target and mechanism for treating negative and positive symptoms of schizophrenia; positive symptoms are those not typically present in people without schizophrenia, while negative symptoms are those that people with schizophrenia typically lack compared to people without the disease. Building on these results could lead to the development of novel therapeutics that treat negative schizophrenia symptoms such as cognitive deficits and positive symptoms such as delusions, hallucinations, and disorganized thinking.
Depression
Roughly 10 percent of the U.S. population is afflicted with major depressive disorder at any given time, and up to 20 percent will exhibit MDD symptoms over their lifetimes. In a recent study by the Vanderbilt Brain Institute, researchers substantially extended the efficacy of a single dose of ketamine from its current duration of up to a week to a longer period of up to two months. Improving the effective period of ketamine can help patients stay on the medicine for longer through less-frequent infusions, alleviating the burden of treatment and reducing the possibility that patients will cease treatment and relapse.
Read the full story: New ketamine study promises extended relief for depression
Addiction
For the over 50 million Americans who had a cocaine use disorder in the previous year, medical approaches to overcoming addiction are much needed. In the case of cocaine use disorder, research has typically relied on models that involve intravenous drug self-administration. A pharmacology lab has devised a new method that more closely mimics real-world cocaine use (intranasal administration or “snorting”) and overcomes the limitations of IV drug self-administration.
Read the full story: A more realistic way to study cocaine use could accelerate addiction research
Cancer
Melanoma
For patients with advanced melanoma without a BRAF mutation who no longer respond to immune checkpoint inhibitors, treatment options remain frustratingly limited. A new study from Vanderbilt researchers outlines a promising therapeutic strategy that may re-sensitize these resistant tumors to immunotherapy.
Read the full story: Targeting immune suppression to overcome melanoma resistance
Thyroid cancer
Anaplastic thyroid cancer is among the most lethal endocrine malignancies, characterized by rapid progression and limited treatment options. An SOMBS lab identified a protein that promotes tumor growth and metastasis. Understanding the molecular mechanisms driving its aggressive behavior is crucial for developing effective therapies.
A second recent paper sought to further our understanding of the effects of PFAS (“forever chemicals”) on the microscopic anatomy or histology of the thyroid. The results of the study directly tie PFAS exposure to changes in the histology and functioning of thyroid tissue, and the authors hope that the results allow people who are at risk for thyroid and other cancers to make better-informed choices, as consumers, for limiting their exposure to these chemicals.
Read the full story: PFAS—‘forever chemicals’—directly shown to alter thyroid structure and function
Visualizing and targeting cancer
A critical strategy to limiting a drug’s toxic side effects is for it to reach only its pathogenic target and nothing else. A group of biochemistry researchers paired a precisely targeted imaging agent to an anticancer agent and found that they could specifically visualize and attack cancer cells (and not normal cells) with it.
Read the full story: A package deal: Diagnosing and treating breast cancer with a single complex
Drugging the “undruggable”
MYC proteins are transcription factors pivotal for normal cellular processes, such as cell growth, division, and differentiation—and for cancer progression. Researchers are pioneering a unique strategy to make the long-undruggable MYC vulnerable, rewriting the playbook for defeating cancer.
Read the full story: The emperor of all oncogenes
Infectious diseases
Gonorrhea and uncomplicated urinary tract infections
The vast number of antibiotics produced today are just tweaks on existing drugs; the last truly novel class of antibiotics was approved by the FDA in 2003, one of only a handful developed since the 1960s.
That changed in 2025, when the U.S. Food and Drug Administration announced that it had approved the drug gepotidacin (brand name Blujepa) for the treatment of uncomplicated urogenital gonorrhea. The application was submitted by the multinational pharmaceutical and biotechnology company GSK, which developed the drug, but it was accompanied by mechanism of action data generated exclusively by researchers from the Department of Biochemistry.
Aging and metabolic diseases and disorders
Obesity
The treatment landscape for obesity and type 2 diabetes has been transformed by GLP-1 receptor agonists. Key questions remain about how these drugs reshape patient behavior around food, their metabolism over time, and why individual responses to the drug vary so widely. Vanderbilt researchers reported that prolonged treatment with the GLP-1R agonist semaglutide induces changes in feeding behavior and fuel use that correspond to three distinct stages.
Diabetes
Type 2 diabetes occurs when your body develops a resistance to insulin, the hormone that helps regulate glucose levels in your blood. Insulin is secreted by pancreatic cells called β-cells. In T2D, β-cells ramp up insulin production to try to regulate blood glucose levels, but even that is insufficient and the β-cells eventually become exhausted over time. New research by Vanderbilt faculty members is moving the needle toward determining whether it is possible to enhance functional β-cell mass to reduce the risk of T2D.
Read the full story: New research points to cell subtypes that increase risk of diabetes
Age-related macular degeneration
According to the National Eye Institute, most older adults who experience vision loss can blame it on age-related macular degeneration, and approximately 11 million people in the U.S. have the disease. One contributing factor in diagnosing AMD is the presence of “hyperreflective foci” in the retina—the light-sensing part of the eye. Department of Biochemistry researchers set out to dig into the molecular and cellular nature of hyperreflective foci in and found a role for lipids in age-related vision loss, paving the way for potential new therapies.
Read the full story: New research points to lipids as possible culprit in age-related vision loss
Aging
Aging is a natural part of life, but it is associated with a greatly increased incidence of most chronic diseases, including various cancers, diabetes, and Alzheimer’s disease. A cell and development lab wants to determine if there is a way to break the links between the aging process and disease so that we can stay healthy longer, allowing us to better enjoy our later years.
Read the full story: Aging researchers find new puzzle piece in the game of longevity
Development and congenital disorders
Kidney
The kidney, a critical organ for waste filtration and fluid regulation, is the subject of a groundbreaking molecular mapping project that could reshape our understanding of renal health thanks to cell and developmental biologists.
Tissue repair
A study by Vanderbilt investigators uncovered a previously unknown biological mechanism: how tissues detect and respond to damage in basement membranes, the thin layers of extracellular matrix that surround and support nearly every organ in the body. The results of this study have far-reaching implications, as basement membrane damage plays a role in numerous diseases, including diabetes, dementia, and cancer.
Read the full story: How tissues detect and repair damage to the body’s hidden support system
Irregular heartbeat
Medicine often takes a one-size-fits-all approach, but a disorder’s root cause can vary. Vanderbilt researchers have found that, for people with long QT syndrome, a heart condition that causes an irregular heartbeat, a more tailored approach could be beneficial. The study is a step toward the development of treatments tailored to a patient’s genetic make-up, part of the growing field of personalized medicine.
Gut health
Crohn’s disease
New research challenges a longstanding assumption about Crohn’s disease: that all regions of the gut respond similarly to inflammation and treatment. The study reveals that microbial interactions and genetic factors contribute to region-specific inflammation, particularly in the ascending colon—an often-overlooked site of disease.
Intestinal disorders
Recent research offers insights that may advance treatments for gastrointestinal motility ailments like Hirschsprung disease (a birth condition that prevents normal movement of stool through the colon), irritable bowel syndrome, and chronic constipation.
AI-enabled research
Optimizing drug discovery
An SOMBS pharmacologist and computational biologist is improving the way the field of drug discovery creates machine learning algorithms to predict a protein’s interactions with a small molecule. These improvements bring ML closer to fulfilling its potential in the field—something that has not been realized after more than a decade of work.
Read the full story: Vanderbilt scientist tackles key roadblock for AI in drug discovery
Targeting RNAs
Noncoding RNAs have critical roles in many biological processes—such as gene expression—making them ideal targets for a variety of ailments, including cancers. Researchers in the Department of Molecular Physiology and Biophysics are working with collaborators to unlock the untapped potential of ncRNAs, as they are a promising family of targets for the development of novel small-molecule therapeutics.
Read the full story: Vanderbilt scientists pioneer an AI-driven drug discovery tool targeting RNAs
Advances emerging from the SOMBS underscore how curiosity-driven discovery fuels tangible impact. With technological momentum building and partnerships spanning campus, clinics, and industry, we enter 2026 poised not only to ask deeper questions, but to deliver more answers that improve lives—proof that foundational science remains the engine of innovation and the compass guiding medicine forward.