Adrenoleukodystrophy (ALD) is a genetic disorder that is very rare and usually occurs in childhood.
People with ALD have two options for treatment, both of them less than favorable and neither one a cure. However, according to the Myelin Project, a new drug may soon rival these other two treatment competitors.
The first option for those with ALD is Lorenzo’s oil, a combination of oil extracts that slows down the effects of the disease. While popular, research is inconclusive on whether or not this treatment can actually prevent the development of disease. The FDA has deemed Lorenzo’s oil experimental.
The second option is a stem cell transplant. This transplant can slow down the recession of brain and muscle damage but cannot repair any damage already done. Furthermore, there are potentially deadly complications to the process.
However, a startup called NeuroVia has a medicine that the company believes will beat out competitors in the race to treat and potentially cure ALD.
ALD is a debilitating and eventually fatal disease that results in gradual loss of brain and muscle function. It affects boys mostly, who start developing behavioral problems from a young age. This progresses to issues that are more serious as brain and muscle function are impaired, eventually leading to paralysis and death. Following diagnosis, this disease usually proves fatal in three to five years.
People with this disease are lacking an essential gene that breaks down fatty acids. This fatty acid buildup damages a coating, called myelin, that protects a person’s nerves.
NeuroVia is just one of several companies trying to develop a cure. One treatment, by Cambridge-based BlueBird Bio has a gene therapy that may offer a treatment for ALD that will not involve a stem cell transplant. This treatment is currently in the clinical trial phase.
The NeuroVia founder is a man called Thomas Scanlan, and he is confident this new treatment will be the answer ALD patients and healthcare providers have been searching for. The NeuroVia drug is a synthetic thyroid hormone that promises to reduce fatty acid levels in ALD patients.
