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qianyang Message
13 Sep 2013, 04:01 AM

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".
fdemiranda Message
13 Sep 2013, 01:01 PM

Interesting... Thanks!
qianyang Message
14 Sep 2013, 02:36 PM

Thanks. fdemiranda. I have been fighting AMN for 20 years and understand every bit of suffering a AMN patient has to endure. I regard any AMN patient who has an input in this forum is a "surviver" and must be optimistic. As a professor who is doing medical research in a university, I have a better access to the latest publications on AMN and good knowledge about the potential therapeutic effect of any findings on AMN. I found Dr. Pujol's paper and believed it's my duty to inform my fellow AMN sufferer about this good news. Never even thought any of our AMN patient would think it is a Ad for a drug company. BTW, pioglitazone is a generic drug now. Plus no drug company would spend a dime on a Ad targetting less than a few hundred people (such as AMN patients in this forum). My doctor is still doing his "research" on this medicine and has not prescribe it to me yet. I hope I can get it soon and inform you guys about possible effect it may has on my condition. May God bless us.
nikolahn Message
15 Sep 2013, 07:51 AM

keep fingers crossed!
mgiuseff Message
22 Jan 2014, 02:39 AM

Thanks all the above for interest in this experimental drug. In a few years, this may be recommended. I will print out this article and take it to my general practitioner. I feel pretty well, although my leg weakness keeps my from much activity. I'd do anything to get the strength back in my legs. Good luck to you all. There's too much medical research on this disease and it's just a matter of time before meaningful therapies are available.
mgiuseff Message
8 Apr 2014, 04:34 AM

The piliatazone you mention is for diabetics. Side effects can mess with other things. My doc told me to go to a research hospital, so I won't be doing that anytime soon. Still playing golf so things are good. Wishing you all well. In heaven we'll all be healthy.