Adrenoleukodystrophy (ALD) is a rare hereditary mutation and neurodegenerative disease that affects about 0.005% of young boys, and has very few and risky treatment options. Typically, the only option is an allogeneic hematopoietic stem cell transplant, done through a bone marrow transplant, and the procedure needs to be done at an early stage of the disease. This procedure is the only treat able to halt hallmark demyelination seen in the disease, but can also worsen the patient's condition if done when the disease has progressed too far.
In a small study recently published, doctors tried using gene therapy in ALD for the first time, using disabled HIV to deliver bone marrow stem cells with the non-mutated ALD gene that would eventually become glial cells around the patient's neurons.
Though this was just a first step and was a very small study and gene therapy has seen complication in the past, its success has made the case for additional research in this area and given a glimmer of hope for those affected by this debilitating disease.
For the first time, doctors have used gene therapy to stave off a fatal degenerative brain disease, an achievement that some experts had thought impossible. The key to making the therapy work? One of medicine’s greatest villains: HIV.