Minoryx Therapeutics is on their way to making strides for patients with adrenomyeloneuropathy, also known as AMN, reports Labiotech.
They just started their phase II initiative to test patients for a new drug that could potentially help those with AMN. Minoryx just treated their first patient and they are hoping to treat 100 more soon, to develop answers come 2020. Trials will continue to treat male adults.
Adrenomyeloneuropathy is a rare neurological genetic disorder that affects one’s motor function. A more frequent form of the disease is X-linked adrenoleukodystrophy, or X-ALD. This is then caused by disfunction of one’s peroxisomes, which duties are contributed to breaking down long fatty acids. This happens with a mutation of the ABCD1 gene and often affects men, which is why the trial is currently focused on them.
X-ALD is quite aggressive, leading to potentially severe neurological inflammation that can lead to death just 4 years after diagnosis. Unfortunately, there is no current treatment or medication on the market to help aid those with AMN or X-ALD.
There is a hematopoietic stem cell transplant that is used, but needs to be performed right when the disease onsets. So these patients are left with little to no options. Gene therapy is known to be the hot ticket right now for rare genetic disease treatment, and fortunately another organization, Bluebird, is developing an approach for adrenomyeloneuropathy. Bluebird continues to focus and develop their gene therapy, while Minoryx continues trials for their treatment.
There is still much more to be done for a rare disease with little treatment options to patients, but that doesn’t mean we aren’t far from success. As both companies continue to build upon their initial developments, we could be near a good time for patients with adrenomyeloneuropathy and hopefully soon they will have multiple options for treatment, and maybe even a cure.
