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Interferon beta is a biologic immunomodulator and type I interferon that was investigated for the treatment of adrenoleukodystrophy (ALD). In a randomized, double-blind, placebo-controlled clinical trial sponsored by the FDA Office of Orphan Products Development (NCT00004450), interferon beta was evaluated in combination with thalidomide in male patients with ALD who were also receiving Lorenzo's oil therapy. The therapeutic rationale for using interferon beta in ALD was its ability to suppress the synthesis of pro-inflammatory cytokines, specifically tumor necrosis factor alpha (TNF-alpha) and interferon-gamma, which are known to be elevated in the demyelinating brain lesions of ALD patients. However, clinical studies conducted in the 1990s demonstrated that interferon beta therapy failed to prevent disease progression or halt neurological deterioration in patients with cerebral ALD.
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