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Overview

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a nervous system disease that affects nerve cells in the brain and spinal cord, leading to loss of muscle control. The disease progressively worsens over time, and its exact cause remains unknown, though a small number of cases are inherited.

ALS typically begins with muscle twitching and weakness in an arm or leg, difficulty swallowing, or slurred speech. Over time, it affects the muscles necessary for movement, speech, eating, and breathing. There is no cure for this fatal disease.

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A recommended resource is the Mayo Clinic Family Health Book.

Symptoms

Symptoms of ALS vary based on which nerve cells are affected. It generally begins with muscle weakness that worsens over time. Symptoms may include:

ALS often begins in the hands, feet, arms, or legs, then spreads to other parts of the body. Muscles weaken as more nerve cells die, ultimately affecting chewing, swallowing, speaking, and breathing. Pain is generally not present in the early stages, and it remains uncommon in the later stages. ALS usually does not affect bladder control or the senses.

Causes

ALS affects motor neurons, the nerve cells controlling voluntary muscle movements. These neurons are classified into two groups:

ALS gradually causes both groups of motor neurons to deteriorate and die. This leads to loss of muscle function as neurons no longer send messages to the muscles.

Approximately 10% of ALS cases have a genetic cause, while the cause for the remaining cases is unknown. Researchers are studying the complex interactions between genes and environmental factors as potential causes.

Risk Factors

Established risk factors for ALS include:

  • Genetics: Inherited ALS occurs in about 10% of cases, with a 50% chance of passing the gene to offspring
  • Age: Risk increases up to 75 years old, with the highest incidence between ages 60 and 80
  • Sex: Men are slightly more likely to develop ALS before 65; the difference diminishes after age 70

Other potential environmental factors include:

  • Smoking: Women who smoke, especially postmenopause, are at higher risk
  • Environmental toxin exposure: Exposure to substances like lead, though no single chemical has been definitively linked to ALS
  • Military service: Studies suggest higher ALS risk among veterans, though the exact cause remains unclear

Complications

As ALS progresses, it can lead to serious complications:

  • Breathing problems: Weakness in the respiratory muscles often requires devices like mask ventilators or a tracheostomy
  • Speaking problems: Muscle weakness affects speech clarity, eventually requiring alternative communication methods
  • Eating problems: Swallowing difficulties increase the risk of malnutrition, dehydration, and aspiration pneumonia, which may be managed with a feeding tube
  • Dementia: Some people with ALS develop frontotemporal dementia, impacting language and decision-making

The Latest Inclusion and Exclusion Criteria for Autologous Cell Therapy for ALS in PFICell's Trials

Inclusion Criteria

Exclusion Criteria