1991

First Enzyme Replacement Therapy Approved

NINDS researchers successfully develop alglucerase injection (Ceredase) for people with Type 1 Gaucher disease. This rare genetic condition disrupts the body’s ability to break down certain fats. The team created a medication that overcomes this problem by replacing a defective enzyme, improving quality of life for people with this condition. Built on decades of foundational research, this treatment (the first enzyme replacement therapy approved in the United States) shows the power of scientific research to translate discovery in the lab into effective treatments for people. The success of this treatment set the stage for developing similar treatments for related disorders. The discovery contributed to early growth in the U.S. biotechnology industry interest in targeted treatments for other rare, genetic diseases.

1996

Introduction of the First Stroke Treatment

The U.S. Food and Drug Administration (FDA) approves tissue plasminogen activator (tPA) as the first treatment for acute ischemic stroke. This type of stroke is a medical emergency in which oxygen delivery to the brain stops due to a blood clot or blocked artery. NINDS-funded lab research and clinical trials proved that when given within hours of stroke symptoms beginning, tPA dissolves blood clots, restores blood flow, and improves patient outcomes. This breakthrough revolutionizes stroke care and sets the foundation for future treatments.

1997

Deep Brain Stimulation (DBS)
Authorized to Treat Parkinson’s Disease

NINDS basic neuroscience research helps identify neural circuits involved in the body’s ability to regulate and coordinate movements and how they are disrupted in Parkinson's disease. These important early studies were critical to developing deep brain stimulation (DBS) to treat the condition. DBS delivers electrical pulses directly to the brain to improve movement symptoms. In 2009, a major clinical trial (sponsored by NINDS, the Department of Veterans Affairs, and industry partners) demonstrates that DBS works better than the standard treatment (levodopa) alone.

Brain Stimulation Emerges as a Treatment for Epilepsy

Building on findings from devices developed for Parkinson’s disease and basic research on causes of seizures in the brain, NINDS-supported researchers help develop brain stimulation devices to control seizures in people with epilepsy. Although most people with epilepsy get relief from available anti-seizure medications, about one-third do not. Brain stimulation devices, including a deep brain stimulation device and a nerve stimulator, offer alternative options for people with drug-resistant epilepsy.

2001

“Know Stroke” Campaign Kicks Off

NINDS launches the “Know Stroke” campaign to raise awareness about stroke, one of the leading causes of death. After tissue plasminogen activator (tPA) was approved in 1996 to treat ischemic stroke within the first few hours of symptoms, it became critically important to get stroke treatment as soon as possible to prevent death and disability. The Know Stroke campaign is designed to help people recognize stroke symptoms and call 911 right away.

2004

NIH Launches Blueprint for Neuroscience Research

The NIH Blueprint for Neuroscience Research is a collaborative effort to accelerate discoveries about the brain’s role in health, aging, and disease. It brings together NIH institutes, centers, and offices, pooling resources and expertise and identifying areas of shared research interest. This collaboration leads to groundbreaking research, new tools, and unique training opportunities for the neuroscience community. In 2009, the NIH Blueprint launched three large-scale research projects to transform neuroscience research: the Human Connectome Project (a major initiative to map human brain connections), the Transition from Acute to Chronic Neuropathic Pain Initiative (which helped to lay the groundwork for the NIH Common Fund Acute to Chronic Pain Signatures Initiative), and the Blueprint Neurotherapeutics Network.

2005

NINDS Launches the Annual Nonprofit Forum

NINDS hosts its first annual Nonprofit Forum to unite leaders from patient organizations, advocacy groups, and others focused on neurological health and disease. The Forum, which includes people and families with lived experience of neurological disorders, promotes collaboration, shares insights about research and advocacy, and strengthens partnerships to advance treatments for neurological disorders. “Progress through Partnership,” the theme of NINDS’s annual Nonprofit Forum, highlights the power of the public and private sectors working together to advance neurological research.

2013

NeuroBioBank Launches, Accelerating
Human Neuroscience Research

NIH establishes the NeuroBioBank to provide researchers with access to high-quality, well-characterized human brain tissue for studying brain disorders. The NeuroBioBank's goals include increasing public awareness of the importance of brain donation, facilitating brain tissue distribution for research, and sharing best practices among researchers. By providing scientists with valuable and scarce human brain tissue, the NeuroBioBank helps drive advances in understanding and treating Alzheimer’s, Parkinson’s, autism, and many other brain disorders.

2014

The Brain Research Through Advancing Innovative Neurotechnologies^® (BRAIN) Initiative Sparks New Frontiers

The Brain Research Through Advancing Innovative Neurotechnologies^® (BRAIN) Initiative, or The BRAIN Initiative^® launches, with the mission of developing and applying new technologies to answer fundamental questions about the brain. The BRAIN Initiative enables researchers to watch how cells and circuits interact within the world’s most complex and energy-efficient computer: the human brain. The Initiative is transforming neuroscience through the development of novel tools and technologies, shedding light on the links between brain function and behavior.

2016

Mind Your Risks^® Campaign Raises Awareness

High blood pressure is a known risk factor for stroke and heart attack. NIH research led to the discovery that it also increases a person’s risk for cognitive decline, including dementia. In 2016, NINDS introduced Mind Your Risks^® to raise awareness about the connection between high blood pressure, stroke, and dementia. A key emphasis of the campaign is early detection of high blood pressure, since it can weaken or narrow the brain’s blood vessels and may lead to strokes, which can cause or worsen dementia. The campaign emphasizes that controlling stroke risk factors, especially high blood pressure, in middle age can help reduce the risk of cognitive decline later in life.

2016

Nusinersen (Spinraza) Approved to
Treat Spinal Muscular Atrophy

The FDA approves nusinersen (Spinraza) for spinal muscular atrophy (SMA), offering hope for individuals with the disorder, especially with early treatment. SMA is a rare genetic disorder that damages motor neurons and is the leading inherited cause of death in infants and toddlers. Nusinersen targets a gene that makes a defective protein in SMA to increase production of a more stable protein. NINDS also supported research that uncovered the genetic cause of SMA, identified a treatment strategy, and facilitated clinical trials. FDA approval of this therapy marked the first time an antisense oligonucleotide therapy, which alters genetic instructions to produce a healthy version of a missing or damaged protein, is used successfully to treat a neurological disorder.

2017

 FDA Approves Cerliponase Alfa (Brineura)
for Neuronal Ceroid Lipofuscinosis

Neuronal Ceroid Lipofuscinosis, also known as Batten disease, refers to a group of rare genetic disorders affecting young children that cause progressive vision loss, movement problems, cognitive decline, and other serious symptoms. NINDS research contributed to a new treatment for a form of Batten disease by identifying the genetic cause: an enzyme that, when functional, cleans up cell waste. Further research leads to a way to produce a replacement version of the defective protein. The treatment helps slow the loss of mobility by delivering the disorder’s missing enzyme directly to the brain. Children can now receive this treatment at birth, before any symptoms arise.

2018

NIH Helping to End Addiction
Long-term^® (HEAL) Initiative Takes Shape

Following the 2017 announcement of the opioid crisis as a national public health emergency, the NIH HEAL Initiative^® launches. This expansive public health research effort brings together researchers, community members, and organizations to find lasting solutions to the intertwined public health challenges of substance use disorder, overdose, and pain. This nationwide effort supports thousands of researchers who are focused on developing more effective treatments for pain, substance misuse, addiction, and overdose, and their associated mental health challenges. NINDS leads HEAL’s pain research portfolio, in partnership with many other NIH institutes and centers.

Brain Scans Improve Emergency Stroke Treatment

Results from a NINDS-funded clinical trial expand the window of treatment for ischemic stroke using brain imaging. The trial showed that physically removing blood clots in the brain up to 16 hours after the person first has symptoms can help people recover better than the standard medical treatment up to that point, which did not include removing blockages more than six hours after symptoms began. This finding was the culmination of decades of research on brain scanning methods, called perfusion imaging, that could identify patients with brain tissue that could still be salvaged by removing the blockage.

2023

A New Chapter for Amyotrophic
Lateral Sclerosis Treatment

In people with amyotrophic lateral sclerosis (ALS), the brain and spinal cord loses its ability to start and control voluntary movements such as walking, talking, chewing, and other functions, including breathing. ALS is a progressive condition, meaning the symptoms get worse over time. FDA approval of tofersen (Qalsody) to treat an inherited form of ALS was a breakthrough for the ALS community: This marked the first FDA-approved treatment for this form of ALS. By reducing the production of a harmful protein, this medication is the first precision medicine treatment for ALS. In 2024, NIH, the Foundation for NIH, FDA, and non-profit and industry partners launched the Accelerating Medicines Partnership^® for ALS to build on these successes and further speed development of ALS medications. People with lived experience of ALS have been important contributors to the design and implementation of this partnership.

2024

Breakthroughs in Brain Technologies to Improve Lives

Decades of NINDS research has led to recent, significant advances in brain-centered technologies, including several deep brain stimulation (DBS) therapies announced in 2024. Notably, BRAIN Initiative researchers developed brain-computer interfaces enabling people with paralysis to communicate: detecting brain patterns that represent words or sounds and translating the patterns into speech. Other advances show life-changing successes in small clinical studies involving people with severe depression; obsessive-compulsive disorder; post-traumatic stress disorder; severe opioid addiction; and other debilitating brain disorders.  Although these small proof-of-principle studies require future research and collaborations to commercialize and expand access, they offer incredible hope to people living with brain diseases.