Subtitle Forum: Advances in Treatment of ALD
ALD, or Adrenoleukodystrophy, is a rare, inherited genetic disorder that affects the brain, spinal cord, and peripheral nervous system. The disease affects approximately 1 in every 18,000 boys. Symptoms of ALD include progressive vision loss, difficulty with balance and coordination, and difficulty with speech, hearing, and motor skills. As the disease progresses, people with ALD can also experience seizures, dementia, and eventually death.
There have been many advances in the treatment of ALD over the past few decades that have improved the quality of life for those living with the disorder. Gene therapy is one of the most promising treatments for ALD. In gene therapy, a normal gene is inserted into cells to replace a defective gene responsible for a particular disease. This can be done through a variety of techniques, including viral vector delivery and stem cell transplantation. Clinical trials of gene therapy for ALD have shown promising results, and the treatment is now available to some patients.
Enzyme replacement therapy is another treatment option for ALD. In this therapy, a medication is injected that contains a form of the missing or defective enzyme that is responsible for the symptoms of ALD. This therapy has been shown to slow the progression of the disease and improve quality of life for those living with the disorder.
Stem cell transplantation is a newer treatment option for ALD that has been shown to be effective in some cases. In this procedure, stem cells from the patient's own bone marrow are transplanted into the brain in order to replace damaged or missing cells. This can help to restore some of the function that has been lost due to the disease.
Although there are no cures for ALD, these advances in treatment have made it possible for people living with the disorder to lead full and productive lives. With continued research and clinical trials, new treatments and even possible cures may be discovered in the future.