Deacetylase inhibitors - Trichostatin A

[Juse]

Dear Dr. Feder,
I've just found out this post on DMD, and it is really interesting, and active.

As I guess many others, I am both surprised and sad, of the large number of news and new research that come out on the internet and other media.

It sounds like a thousand things alltogether could be able to fight in some way DMD, but nothing ever really comes out to benefit and treat the patients.

I see commercial purposes beyond this, but also a great lack of sensitivity and respect for parents and all those children.

I can't really believe there are so many many years ahead to even get some medicine that stops or reliefs DMD.

I then wished to ask you some question on a research on deacetylase inhibitors (Trichostatin A), published on Nature [Nature Medicine - 12, 1147 - 1150 (2006) - doi:10.1038/nm1479].

I attended a conference in Milan of Parent Project Italy, last October 2006, where they showed some very encouraging results, and said that this sort of drug is already in use, for breast cancer, but they are doing further studies to see if there are side effects for long-term use, and as long as this would apply to children and not to adults.

Finally he said there are good chance that this drug could obtain to be declared as "orphan drug", and then have a quicker trial process.

I haven't heard of other updates since then.

My questions are:
do you know something about this study? How do you consider it?

Again, as for other drug in commerce such as Losartan etc., why it should ever take so long (years) to get to an application on humans?

Would you suggest to use Losartan? Here in Italy I don't think anyone is yet starting using it. Why?

Thank you, best regards.

[David Feder]

Hi Juse:
I see and have the full article about trichostatin. I can send by e-mail to all. The mechanism of this drug is very interesting. Since this publication I look for drugs that have the same mechanism of action. I think that valproic acid can give similar effect of trichostatin with more advantage: valproic acid is a well-know drugs to pediatricians and neurologists. But I need prove this effects and until this time I can't. I agree that commercial purposes delay the evolution of therapy but we have other problems: very few trials;I think that we need more clinicial trials and doctors with more courage to start new therapies.
My wife is a pediatrician and she had several patients with DMD. She use losaran to treat cardiac dysfunction in DMD and she have intention to start a trial with losartan in few months. Until this moment is very difficult to say anything about losartan. We must try
David

[Juse]

Thank you indeed for your reply.

Let's hope some good news from this kind of research might come soon.

Juse