Adrenoleukodystrophy is a rare inherited disorder identified in patients with a defective gene that produces a protein called ALD; which leads to progressive brain damage. ALD is a transporter required for the break down fats. Defects in ALD lead to the build up of fats and the eventual damage of the myelin sheath that protects nerves. For many years the only treatment for this has been bone marrow transplants, which is limited in the availability of donors and let us not forget the number of complications that could arise.

A new treatment has shown itself in the form of gene therapy. Bone marrow cells can be treated with a modified and inactivated HIV virus carrying the correct message for the ALD gene and infused back into patients with adrenoleukodystrophy. The blood cells with the correct message would then be carried to the brain where ALD will be produced and utilized to improve the disease. At present HIV is the only virus that can deliver a therapeutic gene into the nucleus of non-dividing cells. Who would have thought that HIV virus could have a positive side?

This is a great news piece and I read about it too. They do say that it is an ill wind that blows nobody any good and I suppose you could say that this is the proof of that old saying. Medical science is tireless in its efforts to find cures and to end disease and it is remarkable how much progress has been made over the years. We may not hear of all the break throughs as some may not be headline grabbers like this one, and we may be frustrated as the testing periods for new drugs seems endless, but all the same, we all owe a debt of gratitude to the men and women who devote their lives to trying to make ours better!