According to a study in The Lancet Neurology, researchers at the Paris Brain Institute have unveiled a new drug candidate that may help slow the progression of X-linked adrenoleukodystrophy (X-ALD).
X-ALD is an inheritable condition that involves an excessive accumulation of very-long-chain fatty acids in tissues within the brain, spinal cord, and adrenal glands.
The research was a 96-week randomized, placebo-controlled phase 2-3 trial conducted in eight American and European countries for the candidate drug leriglitazone.
“Between Dec 8, 2017, and Oct 16, 2018, of 136 patients screened, 116 were randomly assigned; 62 [81%] of 77 patients receiving leriglitazone and 34 [87%] of 39 receiving placebo completed treatment,” the journal article explains.
“The primary endpoint was not met, but leriglitazone was generally well tolerated and rates of adverse events were in line with the expected safety profile for this drug class.”
“The finding that cerebral adrenoleukodystrophy, a life-threatening event for patients with adrenomyeloneuropathy, occurred only in patients in the placebo group supports further investigation of whether leriglitazone might slow the progression of cerebral adrenoleukodystrophy.”