VK0214 is considered as one of the most promising cures for X-linked adrenoleukodystrophy (X-ALD) these days after positive results following a 25-week proof-of-concept in vivo study conducted at the Kennedy Krieger Institute were recently made public.

X-ALD Damages the White Matter of the Brain

Adrenoleukodystrophy or X-ALD damages the white matter of the brain and causes adrenal gland impairment

According to its makers, Viking Therapeutics, Inc., VK0214 was very promising in treating X-ALD by considerably bringing down plasma levels of multiple very long chain fatty acids (VLCFAs) as well as the benchmark highly toxic C26 fatty acid in animals.

The research data was unveiled during the 87th Annual Meeting of the American Thyroid Association (ATA) in Victoria, British Columbia. A clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, Viking Therapeutics, Inc. is based in California, United States.

Representatives from Viking Therapeutics, Inc. emphasized that the most significant test results include the robust and durable reductions in plasma levels of the lysophosphatidylcholine (LPC), reductions in plasma VLCFAs, significant reductions in C26:0, C24:0, C22:0 and C20:0 VLCFA levels in liver, spinal cord and brain tissue, and significant increases in ABCD2 transporter expression.

An illustration of how VK0214 Works

An illustration of how VK0214 works

X-ALD is basically a rare, genetic disorder that affects 1 in 18,000 people. It is indicated by a plunge in myelin, the fatty covering that surrounds the brain’s nerve cells, and can prevent the adrenal gland from functioning like it should result in seizures and hyperactivity. Scientists point out that ABCD1 is the gene that causes this illness.