So many drug trials but what will be the first

[hb]

Hi... so many studies are happening and everything is so exiting & promising, but nothing is being OFFERED... those who suffer like me say from within... atleast give a chance to participate before i die, doing nothing...

anyway, keeping hopes alive is also the most important aspect and this board does it well. special thanks for david for keeping my spirits up by his untiring efforts... hb

[David Feder]

Old drug can help other disease that researcher never considered. New example:

Cheap anti-allergy drug offers hope of cure for malaria
www.guardian.co.uk/medicine/story/0,,1811349,00.html

A drug developed to treat allergies has been identified by US researchers as a potential cure for malaria. Tests in mice show that the antihistamine astemizole also kills the malaria parasite. It is already licensed for use in people, so it could be developed for use as a malaria drug in about 12 months. And because it is no longer under patent, it will be possible to manufacture it at rock-bottom prices.


Only research can give results in DMD. Only researchers worried with this generation can give results in few time. Knowledge drugs can be used in DMD in few time after trials
David

[Andy]

I currently take salbutamol (a type of albuterol) which i find quite effective in keeping me going ... it was a struggle to get a doctor to prescribe it but my doctor gave in in the end.

[hb]

hi David, Once i noticed that my cpk came down to just 400 u/l when i was suffering form typhoid... i pointed out this to my doctor who never paid attention ... while i had cpk 4000+ U/l one month before... then after my recovery it again went to 3600 levels... At the corner of my mind these findings always pinches me that it has some missing links... some drug of typhoid or my be malaria has the potential to bring down my cpk.

Anyway, I just pointed out my personal experience and has nothing to do with article above.. I shared it with you cause u never know what lies beneath... maybe a thoughtful mind can explore the possibilities...

regards
hb

[luis]

hihb
would you explain a litle more about your medical prescription for typhoid fever? was it and antibiotic? how long did you take it? best regards luis

[David Feder]

Other example that knowledge drug can help in DMD:

www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&dopt=Abstract&list_uids=16791712&query_hl=2&itool=pubmed_docsum

Blocking of Striated Muscle Degeneration by Serotonin in C. elegans.

J Muscle Res Cell Motil. 2006;27(3-4):253-8. Epub 2006 Jun 22.

Carre-Pierrat M, Mariol MC, Chambonnier L, Laugraud A, Heskia F, Giacomotto J, Segalat L. France


Prevention of muscle fiber degeneration is a key issue in the treatment of muscular dystrophies such as Duchenne Muscular Dystrophy (DMD). It is widely postulated that existing pharmaceutical compounds might potentially be beneficial to DMD patients, but tools to identify them are lacking. Here, by using a Caenorhabditis elegans model of dystrophin-dependent muscular dystrophy, we show that the neurohormone serotonin and some of its agonists are potent suppressors of muscle degeneration. Inhibitors of serotonin reuptake transporters, which prolong the action of endogenous serotonin, have a similar effect. Moreover, reduction of serotonin levels leads to degeneration of non-dystrophic muscles. Our results demonstrate that serotonin is critical to C. elegans striated muscles. These findings reveal a new fonction of serotonin in striated muscles.

Inhibitors of serotonin reuptake transporters = fluoxetine, imipramine and trimipramine. Fluoxetine = PROZAC, a knowledge antidepressive drug

[hb]

hi luis,

I will have to search my old medical records for prescription ... anyway, i shall look into and put here in coming few days. also I would like to mention that I am suffering from LGMD, age 32. and not a DMD.

thanks
hb

[Andy]

hb i have LGMD too , type 2A

[hb]

hi Andy, I too have LGMD 2A or 2B... not sure which one is correct because my "symptoms" match 2A (heels do not touch ground with stiffness and rigidity) but my muscle biopsy report says "possibility of Dysferlinopathy (2B)"... so there is confusion about which one is correct?? I consciously believe that I have 2A because (1) in 2B there is no stiffness or rigidity rather it is an opposite case of looseness of muscles (they stretch more than normal) and (2) the second reason to believe despite report of muscle biopsy is that there is no exact confirmative test available for dysferlin (2B) in Indian medical govt. hospital were I did the test.... maybe I have confused u too , but nice to know that someone with same type is also present on this board for exchange of thoughts....

have a nice day
hb

[David Feder]

In Parent Project Website:
VASTOX RECEIVES POSITIVE ORPHAN DRUG DESIGNATION OPINION FROM
THE EMEA FOR ITS DUCHENNE MUSCULAR DYSTROPHY THERAPY
www.parentprojectmd.org/site/DocServer/EMEA_opinion_220606.pdf?docID=1121

In vastox website: www.vastox.com/research/duchenne_muscular_dystrophy.html
We have demonstrated that several structural classes of proprietary small molecules have the ability to upregulate utrophin in vivo when delivered systemically. These compounds are now being optimised by medicinal chemistry methods to make them more effective. VASTox is planning to begin phase I clinical trials within 2 years..

It's possible to believe that they really are troubled with this generation of patients?

[sonrise]

What do you mean when you say they are troubled with this generation of patients?

[David Feder]

1) They have tecnology to develop several potential drugs to treat DMD. They will study the most promising drug and don't give the others to others companies to study.
2) They publish very early informations to sharehold and parents to stimulate parents and sharehold to give money to this research
3) Parents give the money but they receive the gain: "VASTox holds worldwide exclusive licences to patented cell lines capable of monitoring the small molecule mediated up-regulation of utrophin".
4) They don't complete the early studies until this moment: "These compounds are now being optimised by medicinal chemistry methods to make them more effective" . Who can give confidence that they complete this studies in 2 years? Who can give confidence that this drug can work? With some optimist view they need more 10 years to receive FDA approvation for this drug.
5) This generation need solution in few time. AIDS is the best example of research effort: first diagnosis in 1980 and 26 years after we have more than 20 drugs to delay the evolution of AIDS. We know all steps of virus in human body, the source of HIV to human, and other information. Pathophysiology of muscular dystrophy is discussable and researchers don't have the same opinion about the point that it's more important to muscle degeneration.
6) I see early information about several drugs but after some years I don't see results. An example: poloxamer 188. In 2001 I post an information about poloxamer in my Brazilian website; they report that they will start trial in DMD patients; after 5 year any human publication.
David

[bhopeful]

Hi David: What can we do to change the way the world does research. We need results now or at least as soon as possible. If they can do it for Aids, they can surely do it for Muscular Dystrophy. I guess the MD community does not have a loud enough voice. I believe that nobody wants to "rock the boat".
I get really angry when we are enticed to give more money for research and then later we are told all this will take years and years before anything goes to trial. There must be a better way. WHAT CAN WE DO?

[Andy]

I heard from a researcher once that type 2A LGMD might be different from all other types of MD and might be more of a muscle growth problem than muscle wasting and that in the mouse model mice responded well to light exercise ....

how have you found exercising ??

[David Feder]

BHopeful: I don't want use hard words or generalize my critiques but in this way we don't will have results in few time:
1) Very few research centers in the world; some research receive money for the same project from PP, PPUK, NIH, MDA, MDA Australia and others. Some research center survive only with donations from parents.
2) We must invert the equation: now researchers ask money to several organizations and after valuation he receive money for 1 to 3 years. They don't have obligation with results. How should be: parents organizations decide types of research that will receive money. An hypothetic example: "David organization" will select researchers to study knowledge drugs that could help in DMD - I will pay for example US$ 10,000 for each drug studied. The research will receive the money when he complete the study with all analysis and publication of results. "David organization" will select researchers that could study more drugs in few time. "david organization" will select the most important drugs. This new way can reduce the spend of money and permit more studies in few time.
4) Other example of waste: An hypothetic research want money to develop a new viral vector to treat DMD; he ask and receive a big money from organizations. We have now at least 5 vectors, micro, mini, full dystrophin and other. There are at least 5 big teams working with oligonucleotides. We need more vectors? We need more oligonucleotides patents? How should be: parent organization join the teams to working in the same project. They will do the same trial in several countries to have results in few time as I see in AIDS medication. All knowledge of vector, oligonucleotides, mini, micro dystrophin must be available to all researchers. Parents organization pay for studies and the companies receive the gain; for example some informations about myodur, poloxamer 188, PTC 124 are secrets and not published despite supported by parents organizations.
5) It's not easy to change anything now because the majority of parents are satisfied with the velocity of research. It's not easy to develop treatments to this generation of patients but we must try. Only a worldwide effort can change the actual situation.