There’s a lot happening in the adrenoleukodystrophy (ALD) world, so here’s the latest scoop.
Adrenoleukodystrophy, or ALD for short, is a genetic rare disease that destroys the protective sheath surrounding the brain’s neurons: myelin.
The disorder mostly affects boys and first takes place usually during the ages four to ten. From there, ALD progressively gets worse from there, and this gradual decline usually leads to death or permanent disability occurring within 2-5 years after diagnosis.
Due to the life-threatening nature of ALD and considering that treatment is limited and not always effective, it is extremely good news is that the biotechnology company NeuroVia currently has a $14 million backing for a new possible drug candidate for ALD, NV1205.
Presumably, this small molecule treatment could function to modify the disease itself. In clinical trials so far, it has shown to reduce levels of very long chain fatty acids (VLCFAs) in the brain, adrenal tissue, and blood of mice lacking a functioning ABCD1 gene, thus targeting the biological abnormalities at the heart of ALD.
NeuroVia is looking forward to further developing this treatment so that more ALD patients have greater, more effective options than the standard Lorenzo’s Oil and stem cell transplantation therapies.
To read more about this, click here.
Another potential ALD treatment is in the works from biopharmaceutical company SOM Biotech, who recently announced the opening of a new office in Cambridge, MA, to encourage the drug development of two candidates.
Significantly, one of these is SOM1201 for ALD.
According to the company, SOM1201 has shown to be effective in clinical trials for treating ALD and also potentially adrenomyeloneuropathy (AMN).
To learn more about this biopharmaceutical company and its pursuits, read this page.
A few patient stories and legacies can help put into perspective just how devastating this disease is to everyone involved. In local North Carolina news, a 10 year birthday celebration was recently thrown for the Roseboro Elementary School student Ryan “DaeDae” Oates.
DaeDae was diagnosed with ALD and passed away at age 9, but he was loved so much by his peers and friends that they all fundraised for medical expenses in their “Dabbin 4 DaeDae” campaign.
To learn more about DaeDae and his legacy, click this link.
In another story, a 50-year-old, Michael Conway’s life got flipped upside down when he found out he had ALD.
Although he started experiencing problems in 2010, he was not diagnosed with ALD until May 2016. In his words,
“Having ALD has made me nervous about going anywhere alone. I’m extremely forgetful and have to keep checking myself, that I’ve got money with me and things like that. […] I am always tired as well. The only time I truly feel free of ALD is when I am sleeping.”
Though the disease is harshly debilitating, to say the least, Michael tries to stay positive and maintains that the simple things in life are the ones worth celebrating.
