Dr. Pujol from Spain published her latest findings last month in "Brain" (Brain 2013: 136; 2432–2443) that Pioglitazone "halts axonal degeneration in a mouse model of X-linked adrenoleukodystrophy". Pioglitazone is a medicine originally for type II diabetes and was approved for human patients. Dr. Pujol is conducting clinical trial (or planning). But it may take years to get finally approved by FDA to treat AMN. In the meantime, I think we all should ask our physician to prescribe it to us to try it out.
It is quite obvious that all the research on ALD or AMN is focusing on reducing the very long chain fatty acid level. This new approach addressed the symptom from a different angle. I believe there are at least two beneficial effects with this treatment. 1. The anti-oxidative stress effect might reduce the damage to the myelin in human AMN. 2. Increase the biogenesis of mitochondria to generate more energy for the patients as the most difficult part of our life is the strength of our legs.
Even Pioglitazone may lower our blood suger level, but our blood suger level can be easily monitored by device for less than 50 dollars.
We all understand even the drug is effective in mouse model, it may not be effective in AMN patients. I am doing medical research myself as a career. I believe the potential benefit of this medicine exceedingly overweighs the possible side effect (Lowering blood suger, etc.) of it. I also feel that at this point, we AMN patients actually have nothing to lose by trying this medicine. It is almost like we see some light by the end of the tunnel. I myself will try it as soon as I get my hand on it. Hope you guy have better luck to get it soon and we can share our experience with it in this forum. Good luck!
As somebody said it the best in this forum "we all in this together".