Scientists reveal the results of a controlled clinical trial for a new drug to treat X-linked adrenoleukodystrophy. X-ALD is the most prevalent of the approximately 50 rare diseases that affect the white matter of the brain, referred to as leukodystrophies. The genetic damage in X-ALD is due to a d
X-linked adrenoleukodystrophy is a rare genetic disorder that affects the nervous system and adrenal glands. It is caused by a mutation in the ABCD1 gene on the X chromosome and is characterized by progressive damage to the myelin sheath surrounding the nerve fibers in the brain and spinal cord. Symptoms may include behavioral changes, difficulty with coordination and movement, and vision and hearing loss.
X-ALD is the most prevalent of the approximately 50 rare diseases that affect the white matter of the brain, referred to as leukodystrophies. The genetic damage in X-ALD is due to a defect in the X chromosome. Men who are affected by X-ALD experience a progressive deterioration of their mobility, balance, and sensory abilities, leading to issues such as incontinence and sexual dysfunction.
Now, for the first time, scientists from all relevant leukodystrophy centers in Europe and the US have jointly succeeded in obtaining controlled trial data for X-linked adrenoleukodystrophy. Of the 116 patients, 77 received the drug leriglitazone and 39 a placebo. The drug had already shown in preclinical studies that it can prevent neurodegeneration and offer protection against the life-threatening inflammation of the brain.
The actual aim of the study had been to show that the drug would prevent gait disorders in X-ALD patients from worsening over the course of two years. “This worked particularly well in those who were treated early. The more advanced the gait disorder, the less apparent the effect. Overall, there was no significant difference, so the actual aim of the study was not achieved,” explains Dr Köhler.
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