What Is Cure One, Cure Many?

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.

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.