When we find the cure for one brain disease, we will find cures for many others.
The brain is a complex organ that serves many distinct, interrelated functions, from memory and thinking to coordinating complex muscle movements. And just as all parts of the brain are connected, so too are the 600+ brain diseases and disorders that impact millions of people worldwide every day.
“Cure One, Cure Many” reflects our belief that we will make a bigger impact by focusing research on these connections rather than on a single brain disease at a time. This approach gives us an edge in fighting brain disease, because it allows us to expand our understanding of how different diseases and brain functions are related, painting a broader picture of how the brain operates as a whole. Think about the difference between looking at a single point on a painting from an inch away vs. stepping back and taking in the whole canvas at once.
Just one discovery could create a ripple effect that impacts our understanding of many other related diseases, leading to better treatments, opportunities for earlier diagnosis, and cures. By investing in research across a range of diseases and disorders, we improve our chances of identifying these connections.
Types of Brain Disease
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Cerebrovascular diseases, including stroke, affect the blood vessels that supply the brain with blood flow. Most strokes are due to sudden blockage of a vessel, but in some cases blood vessels rupture, causing bleeding in or around the brain. Both blocked and ruptured vessels can lead to brain damage, disability, and in some cases, death. Age-related disease of the small blood vessels inside the brain also lead to loss of the blood-brain barrier, progressive degeneration of neurons, and dementia, including vascular dementia and Alzheimer’s dementia.
Emergency treatments for stroke include drugs that dissolve clots, catheters that open blood vessels and retrieve clots, or drugs or surgery to limit bleeding. Cerebrovascular diseases can be prevented by measuring, monitoring, and aggressively managing risk factors such as hypertension, smoking, high cholesterol, obesity, prediabetes, and diabetes. Sometimes medications, surgery, and catheter-based interventions are also used to prevent blood clotting or to open severely blocked blood vessels.
Neurodegenerative diseases progressively damage nerve cells in the brain and spinal cord, leading to a gradual decline in thinking, memory, and other cognitive functions. They may also impact movement and lead to changes in mood and behavior.
A common mechanism is accumulation of misfolded proteins in brain cells; this has created a target for exciting new therapeutic approaches. Common neurodegenerative disorders include Alzheimer’s and Parkinson’s, but progressive neurodegeneration is also a core component of diseases like Huntington’s, ALS, muscular dystrophy, and more.
Sometimes the body’s immune system mistakenly targets healthy cells, leading to chronic inflammation and damage to the brain, spinal cord, muscles, nerves, and other organ systems.
Some autoimmune diseases are part of a systemic illness like lupus, but most nervous system autoimmune diseases do not have a known cause, and include diseases and disorders like multiple sclerosis, autoimmune encephalitis, myasthenia gravis, and more. Various “disease modifying” medications targeting the immune system can improve the outcome of these diseases.
Seizures are sudden, abnormal electrical disturbances in the brain that cause a range of symptoms including loss of awareness, muscle spasms and convulsions, confusion, and sensory changes.
Epilepsy is the condition in which seizures can occur spontaneously. Epilepsy can occur at all ages, from birth to death, with increased susceptibility in the very young and the very old. Epilepsy can present with no known cause, or may be linked to genetic mutations, brain trauma, brain tumors or infection, or other brain disturbances. Seizures can be controlled with medications about 2/3 of the time and may also be treated with surgery or devices.
One of the most common causes of disability and death in adults and children is brain trauma, or traumatic brain injury (TBI). TBI is an injury to the brain caused by a sudden, external, physical force such as a violent blow or jolt to the head or body, or an object that pierces the skull and enters the brain.
Brain trauma can range from mild (like a concussion), where the brain cells are temporarily affected, to severe, where the injury can cause bleeding, bruising, or other physical damage to the brain. TBI can lead to long-term or permanent cognitive or behavioral problems.
Damage to motor pathways in the brain can disrupt a person’s ability to control voluntary movement, resulting in abnormal, involuntary movements, tremors, muscle spasms, and problems with walking and balance.
Many movement disorders are “neurodegenerative disorders”, including Parkinson’s disease, Lewy body dementia (LBD), multiple systems atrophy (MSA), and progressive supranuclear palsy (PSP). Other common movement disorders include essential tremor and dystonia.
Movement disorders can be genetic, caused by head trauma or brain damage at birth, or caused by certain medications. Medications and stereotactic brain surgery are important management options for movement disorders.
Nerve and Muscle Disorders
These are conditions characterized by damage to the nerves that carry motor and sensory information from the body to the brain or from the brain to the muscles, or by disease of the muscles themselves.
Nerve and muscle disorders can result in weakness and loss of muscle mass, twitching, stiffness, pain, paralysis, and sometimes difficulty swallowing and breathing.
Neuromuscular diseases including ALS, muscular dystrophy, myasthenia gravis, and spinal muscular atrophy (SMA), and others may occur for no known reason, may be genetic, or may be secondary to autoimmune or other inflammatory conditions.
The discovery of a gene therapy that cures SMA and improves Duchenne muscular dystrophy is paving the way to cure dozens of other neuromuscular diseases.
Brain tumors can be either malignant (cancerous) or benign (non-cancerous). Malignant brain tumors may arise from the brain itself (glioma) or may arise from cancer elsewhere in the body and travel to the brain (brain metastases).
The most common malignant brain tumor is glioblastoma. Glioblastoma is a highly aggressive cancer that is often more common in adults over age 55 and is more prevalent in men than women. The treatment for glioblastoma is complex, involving surgery, radiation, and chemotherapy. Despite this aggressive therapy, life expectancy following a diagnosis of glioblastoma is often less than two years.
Meningiomas are the most common benign tumors, arising from the lining (meninges) of the brain. They are more common in women than in men, and treatment options include observation, sometimes for years before surgery or radiation.
Brain Disease Mechanisms
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Brain inflammation—also known as neuroinflammation—is present in nearly all neurologic diseases and disorders, whether as a primary cause of the condition such as with autoimmune disease, as an immune response to infection, or as a response to brain injury, stroke, seizures, or neurodegeneration.
Inflammation works to aid the body’s healing process. However, chronic inflammation can damage brain cells and disrupt normal brain function and may contribute to the development or progression of diseases like multiple sclerosis, Parkinson’s, stroke, epilepsy, Alzheimer’s disease and others.
Disorders of Metabolism
Disorders of metabolism are conditions that affect the body’s ability to convert food into energy or effectively remove buildups of toxic substances in the body. Inherited metabolic disorders appear after birth or in childhood due to genetic disturbances in cellular storage, mitochondrial function or other cellular processes. Secondary metabolic disorders are due to dietary deficiencies or toxicities or organ failure. Metabolic conditions can result in low energy, developmental delays, cognitive impairment, organ damage, and even death.
Metabolic disorders include diseases like phenylketonuria, mitochondrial diseases, adrenoleukodystrophy, and Wernicke’s encephalopathy. Evidence suggests that abnormal neuronal metabolism plays a role in many brain diseases, such as MS, migraine, ALS, and others.
Disorders of Neuronal-Glial Interaction
Glia act as support cells for neurons and form the protective myelin layer that insulates nerve fibers in the brain. When these cells are damaged, it disrupts communication between parts of the brain, resulting in symptoms like seizures, difficulty with movement, cognitive impairment, and psychiatric symptoms, among others.
Abnormal neuronal-glial interactions contribute to damage from Alzheimer’s disease, neuropathic pain, epilepsy, stroke, and other diseases and disorders.
Channelopathies are disorders of nerve cell membranes that disrupt the normal electrical signals within the nervous system and between the nervous system and various organs.
Channelopathies may contribute to a range of disorders, including epilepsy, neuromuscular diseases, migraine, peripheral pain syndromes, and others.
Excitotoxicity is a process in which unusually high levels of chemical transmitters in the brain cause neurons to be overstimulated, leading to cell damage and death. This process can contribute to the damage caused by various neurologic conditions, such as stroke, traumatic brain injury, epilepsy, neurodegenerative diseases, and more.
Synaptopathy refers to disruption or damage to synapses—the connections between neurons found throughout the brain and the body. This makes it difficult for the brain to send signals to the rest of the body and can contribute to the development of diseases like epilepsy, Parkinson’s, dementias, and more.
Tau-, amyloid-, and synucleinopathy are three types of protein abnormalities that are associated with several neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and Lewy body dementia. When normal proteins like tau, amyloid and alpha-synuclein become abnormally folded, they interfere with the normal functioning of neurons and contribute to cell death and cognitive decline.
That Can Lead
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Migraine and Epilepsy
Both diseases involve abnormal electrical signals in the brain and may be preceded by a phase known as “aura.” Additionally, people with migraine are more than twice as likely to have epilepsy and vice versa. Researchers continue to investigate whether the diseases may share an underlying cause.
Alzheimer’s Disease, Parkinson’s Disease, and Lewy Body Dementia (LBD)
Researchers are uncovering more and more evidence that these diseases all share common factors like abnormal protein deposits, brain metabolism, and inflammation. Better understanding how even one of these factors relates to loss of cognitive abilities and neurodegeneration could be the key to breakthroughs in all of these diseases. For example, learning how to identify and diagnose Lewy body deposits in the brain could help us understand how to stop the accumulation of harmful protein deposits in Alzheimer’s disease.
Neuroinflammation and Neurodegenerative Diseases
Scientists are currently investigating the role of neuroinflammation in the progression of Parkinson’s disease, multiple sclerosis, and Alzheimer’s. Researchers believe that in some cases inflammation can lead to brain cell death (Parkinson’s disease), while in others the brain’s own immune response may trigger inflammation that damages nerve cells (multiple sclerosis). Understanding how neuroinflammation impacts cognitive aging and brain function could shed light on key processes involved in neurodegenerative diseases, leading to earlier diagnosis methods and treatments.
“One of the things that really resonates for me is this idea of Cure One, Cure Many. Something somebody learns in migraine might inform epilepsy. Something somebody learns in dementia might inform Parkinson’s disease.”
Frances Jensen, MD, FACP, FAAN
Professor of Neurology, Perelman School of Medicine, University of Pennsylvania
“Shared pathology across Parkinson’s disease and Lewy body dementia means that better understanding the biology of one can help advance research into the other.”
Todd Sherer, PhD
Chief Mission Officer, The Michael J. Fox Foundation
Jerry Mendell, MD
We gave Dr. Mendell our Scientific Breakthrough Award in 2019 for his discovery of a one-time gene-therapy treatment for spinal muscular atrophy (SMA). Similar treatments are now being developed for Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy.
“The cure developed for [spinal muscular atrophy] is now being applied to other diseases, and is therefore really a beautiful example of why the vision of the American Brain Foundation is Cure One, Cure Many.”
– David Dodick, MD, FAAN, Professor of Neurology at Mayo Clinic Arizona and Chair of the American Brain Foundation
Shafali Jeste, MD, FAAN
Dr. Jeste received a Next Generation Research Grant to better understand early brain development in babies with tuberous sclerosis complex (TSC). At the time, TSC was associated with a higher risk of developing autism, but researchers did not understand exactly how the two conditions were linked. Through this early research, Dr. Jeste gained insights that helped successfully identify an early predictor of autism in babies with TSC. By uncovering this critical link, Dr. Jeste’s research has not only enabled earlier diagnosis for both conditions, but has also advanced research into early intervention options for children with autism.
Cure One, Cure Many Award for the Early Diagnosis of Lewy Body Dementia (LBD)
We partnered with the Alzheimer’s Association, The Michael J. Fox Foundation for Parkinson’s Research, and the American Academy of Neurology to establish this research award for early diagnosis of Lewy body dementia. We awarded $3 million to a team of researchers from the Mayo Clinic, the University of Pennsylvania, and the University of North Texas Health Science Center to work to identify a blood-based biomarker that will aid in the diagnosis of LBD.
Cure One, Cure Many
Look Like in Action?
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