Diseases that affect the brain or other parts of the central nervous system raise fascinating questions in neuroscience and are among the most disabling and complex conditions plaguing humankind. As populations around the world are living longer, neurodegenerative disorders have been rising in prevalence at an unprecedented pace. Effective treatments for these conditions are lacking and urgently needed.

Consequently, scientists at the Gladstone Institute of Neurological Disease maintain a strong focus on neurodegenerative and neuroinflammatory diseases, including Alzheimer’s disease and other dementias, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and multiple sclerosis. Because of intriguing overlaps between these conditions and other challenging brain diseases, they also study epilepsy and neuropsychiatric disorders, such as autism and obsessive compulsive disorders.

The investigators are searching for common threads that will allow them to identify therapeutic interventions that might benefit multiple conditions. They are using unconventional approaches to identify such multi-faceted solutions, while also leveraging capabilities in regenerative and personalized medicine.

To prepare tomorrow’s scientists for this mission, they are strongly committed to training and mentoring at all levels of education. Learn more about Gladstone’s training programs.

“Our goal is to discover the most impactful disease mechanisms and translate basic scientific discoveries into better treatments for patients.” 

Lennart Mucke, MD
Director, Gladstone Institute of Neurological Disease

Major Scientific Achievements

Discovered Ways to Counteract Genetic Risk Factors of Alzheimer’s Disease

Gladstone scientists used disease models to identify molecular, cellular, and network mechanisms by which amyloid proteins and apolipoprotein E4 (apoE4), the most important genetic risk factor for the disease, may contribute to the development of Alzheimer’s disease. They also identified drugs that can counteract the detrimental effects of amyloid-β and apoE4.

Identified New Functions of Tau and Ways to Block Its Adverse Effects on the Brain

Investigators at Gladstone discovered that reducing brain levels of the protein tau prevents neural network dysfunction, cognitive decline, and behavioral abnormalities in models of diverse disorders, including Alzheimer’s disease, autism, and epilepsy. Efforts to translate these discoveries into better treatments for these conditions are in progress, including the development of small-molecule tau reducers.

Revealed How Blood-Brain Barrier Disruptions and Abnormal Immune Responses Lead to Neurological Disease

Gladstone researchers showed that leakage of blood proteins through the damaged blood-brain barrier triggers abnormal immune responses that may promote the degeneration of brain cells and their connections in Alzheimer’s disease, multiple sclerosis, and other conditions. They also identified alterations in brain cells called glia that could promote memory loss and neurodegeneration, and developed new strategies to block the disease-promoting activities of these cells.

Demonstrated How Neuronal Dysfunctions Can Contribute to Neurological and Psychiatric Disorders

Investigators at Gladstone unraveled how alterations in the activity of specific types of neurons in the brain can cause memory loss, obsessive compulsive behaviors, or movement disorders, such as Parkinson’s disease and dystonia. They also developed investigational strategies to overcome these abnormal brain activities through the transplantation of molecularly engineered precursor cells, deep brain stimulation, and drug treatments.

Detected Subclinical Epileptic Activity and Discovered New Ways to Stop Seizures

Gladstone scientists demonstrated that hard-to-detect, non-convulsive epileptic activity is a key feature of Alzheimer’s disease and that it may promote cognitive decline. They have developed innovative approaches to prevent brain networks from becoming overactive, to stop seizures, and to protect against cognitive impairments. They are pursuing novel therapies to prevent and treat epileptic activity resulting from traumatic brain injury, stroke, or Alzheimer’s disease.

Neurological Experts

Director’s Message

We recognized early on that many brain disorders are multi-causal in nature and, therefore, will likely require a multi-pronged therapeutic approach. To meet this challenge, we assembled a highly interactive team of investigators with complementary areas of expertise who are tackling important unresolved problems in neuroscience in unorthodox ways and with unusual levels of creativity and courage. 

“We are on the cusp of several important advancements to better understand, prevent, treat, and—ultimately—cure some of the most devastating diseases.”

Our goal is to discover the most impactful disease mechanisms and translate basic scientific discoveries into better treatments for patients. We are on the cusp of several important advancements to better understand, prevent, treat, and—ultimately—cure some of the most devastating diseases affecting the central nervous system.

Scientific Advisory Board

Carol A. Barnes, PhD
Evelyn F. McKnight Brain Institute
Columbia University College of Physicians & Surgeons

Fred H. Gage, PhD
Salk Institute for Biological Sciences

Joseph B. Martin, MD, PhD
Department of Neurobiology
Harvard Medical School

Richard Ransohoff, MD
Third Rock Ventures

Dennis J. Selkoe, MD
Ann Romney Center for Neurologic Diseases
Brigham and Women’s Hospital
Harvard Medical School

D. James Surmeier, PhD
Feinberg School of Medicine
Northwestern University

Li-Huei Tsai, PhD
Picower Institute for Learning and Memory
Department of Brain and Cognitive Sciences
Massachusetts Institute of Technology

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