Re: Valproate eases symptoms of SMA

[Guest guest]

This is a very interesting study. I do know that drugs normally used to

treat one disease or disorder sometimes can work to treat another, it

happens If the trial on child patients works like this one did, we may

have another avenue for those with SMA to turn to for treatment. How about

that?

On 6/24/06, jvandyke <jvandyke@...> wrote:

>

>

> <http://mednews.wustl.edu/news/page/normal/7351.html>

>

> Epilepsy drug eases symptoms of inherited disorder that weakens muscles

>

> Media Assistance:

>

> Purdy

> Senior Medical Sciences Writer

> purdym@... <purdym%40wustl.edu>

> (314) 286-0122

> By Purdy

>

> June 23, 2006 -- An epilepsy drug that has been on the market for

> decades can ease the symptoms of adult sufferers with a genetic disorder

> that seriously weakens muscles.

>

> Scientists at Washington University School of Medicine in St. Louis

> retrospectively reviewed results from off-label use of the drug

> valproate to treat seven adult spinal muscular atrophy (SMA) patients.

> Clinicians offered the drug to patients on the basis of research

> conducted elsewhere that showed the drug increased levels of a key

> protein in cell cultures.

>

> " The treatment has been fairly successful, " says lead author Chris

> Weihl, M.D., Ph.D., a postdoctoral fellow in neurology. " The drug

> appeared to be well-tolerated and increased the strength of the patients

> who took it. "

>

> The study, now available online, will appear in the August 8 issue of

> Neurology.

>

> Weihl notes that a larger, prospective trial is needed to firmly

> establish valproate as a treatment of choice for sufferers of this type

> of SMA.

>

> Such trials are already underway elsewhere in pediatric patients who

> suffer from a different type of SMA that begins earlier in life. Weihl

> and his colleagues are concerned that valproate may not work as well in

> those patients. They wanted to make sure that researchers did not

> discard the possibility that valproate could help older sufferers even

> if the trials in pediatric patients went poorly.

>

> " Based on what we know of the unique genetics of this disease, there was

> reason to think that this drug could be more helpful to patients who

> develop SMA later in life, " Weihl says.

>

> Patients with all forms of SMA, which affects approximately one of every

> 6,000 babies born in the U.S., are missing the SMN1 gene, which makes

> the survival motor neuron (SMN) protein. This progressively weakens the

> muscles, leading to difficulty in walking, eating, clearing the air

> passageway, and other essential functions.

>

> Based on when the symptoms of SMA first manifest, physicians divide SMA

> into four subtypes. SMA I, for example, strikes very young children,

> causing weakness in the womb, preventing children from ever walking and

> typically resulting in death at an early age. Patients with SMA IV, in

> contrast, don't develop weakness until adulthood. The seven patients

> studied were either SMA III or SMA IV, and ranged in age from 17 to 54.

>

> Differences in age of SMA onset have been directly linked to a second

> human gene that also makes the SMN protein. That gene, SMN2, isn't as

> efficient at making the SMN protein as SMN1. Patients who develop SMA

> early in life have only one copy of the SMN2 gene in their DNA, leaving

> them with very low levels of the SMN protein. Patients who get the

> disorder later in life have more copies of the SMN2 gene, increasing the

> amount of SMN protein made in their cells and delaying onset.

>

> " Because we have learned so much about SMA over the last decade, there's

> been a big push at NIH to cure this disease, " Weihl says. " The search

> has been on to find a treatment that can increase the amount of SMN2

> protein synthesized by SMN2 genes. This rapid bench-to-bedside

> transition for valproate is a good example of the kind of progress that

> is encouraged both by NIH and the University's Biomed 21 initiative. "

>

> In addition to its use as an epilepsy treatment, valproate, which is

> sold under the brand name Depakote, has been used to treat bipolar

> disorder, migraine headaches and other neurological conditions. The

> drug's effects include increasing the number of times protein-building

> instructions are read from genes, which is the first step in creating

> copies of proteins like SMN.

>

> As patients took the drug, clinicians regularly gave them a series of

> strength tests. When Weihl reviewed the data from those tests, he found

> patient strength had increased significantly over the course of eight to

> 15 months of treatment with the drug.

>

> According to Weihl, simply increasing the strength of an SMA patient's

> cough might enable them to clear their lungs better and reduce incidence

> of pneumonia, the most common killer of patients with SMA III and IV.

>

> Valproate's side effects can include weight gain, hair loss and acne.

> One patient stopped taking the drug because she was concerned about

> weight gain.

>

> " Adding weight can be a problem in patients who are already weak, and

> it's certainly a legitimate reason to stop taking the drug, but overall

> we didn't see significant weight gains in patients taking the drug, "

> Weihl says.

>

> Weihl and his colleagues are continuing to follow the seven patients

> reviewed in the study, who are still taking a daily maintenance dose of

> the drug.

>

> Weihl CC, Connolly AM and Pestronk A. Valproate may improve strength and

> function in patients with type III/IV spinal muscle atrophy. Neurology,

> August 8, 2006.

>

> Washington University School of Medicine's full-time and volunteer

> faculty physicians also are the medical staff of -Jewish and St.

> Louis Children's hospitals. The School of Medicine is one of the leading

> medical research, teaching and patient care institutions in the nation,

> currently ranked fourth in the nation by U.S. News & World Report.

> Through its affiliations with -Jewish and St. Louis Children's

> hospitals, the School of Medicine is linked to BJC HealthCare.

>

>

>

[Guest guest]

It's interesting to me, too... but the numbers in the study were so

small. It does make you wonder if it'd help, though.

jv

Buck wrote:

> This is a very interesting study. I do know that drugs normally used

> to treat one disease or disorder sometimes can work to treat another,

> it happens If the trial on child patients works like this one

> did, we may have another avenue for those with SMA to turn to for

> treatment. How about that?

>

>

>

> On 6/24/06, jvandyke <jvandyke@...> wrote:

>

>>

>> <http://mednews.wustl.edu/news/page/normal/7351.html>

>>

>> Epilepsy drug eases symptoms of inherited disorder that weakens

>> muscles

[Guest guest]

That's true. Small sample size so it's not really enough to make a

judgement. I guess the jury is still out on it right now. But if the tests

do prove that it works, it will be good news.

On 6/25/06, jvandyke <jvandyke@...> wrote:

>

> It's interesting to me, too... but the numbers in the study were so

> small. It does make you wonder if it'd help, though.

> jv

>

>

> Buck wrote:

>

> > This is a very interesting study. I do know that drugs normally used

> > to treat one disease or disorder sometimes can work to treat another,

> > it happens If the trial on child patients works like this one

> > did, we may have another avenue for those with SMA to turn to for

> > treatment. How about that?

> >

> >

> >

> > On 6/24/06, jvandyke <jvandyke@... <jvandyke%40outdrs.net>>

> wrote:

> >

> >>

> >> <http://mednews.wustl.edu/news/page/normal/7351.html>

> >>

> >> Epilepsy drug eases symptoms of inherited disorder that weakens

> >> muscles

>

>

>

[Guest guest]

I'm currently trying the drug-it's been a bit over 3 months. I've read in

several articles the biggest signs show in 8 months or more;however, I do feel

like it is helping somewhat. I totally think it is worth a try!

Buck <jared.buck@...> wrote:

That's true. Small sample size so it's not really enough to make a

judgement. I guess the jury is still out on it right now. But if the tests

do prove that it works, it will be good news.

On 6/25/06, jvandyke wrote:

>

> It's interesting to me, too... but the numbers in the study were so

> small. It does make you wonder if it'd help, though.

> jv

>

>

> Buck wrote:

>

> > This is a very interesting study. I do know that drugs normally used

> > to treat one disease or disorder sometimes can work to treat another,

> > it happens If the trial on child patients works like this one

> > did, we may have another avenue for those with SMA to turn to for

> > treatment. How about that?

> >

> >

> >

> > On 6/24/06, jvandyke >

> wrote:

> >

> >>

> >>

> >>

> >> Epilepsy drug eases symptoms of inherited disorder that weakens

> >> muscles

>

>

>

[Guest guest]

As anyone been able to get this drug outside of a trial? My

neurologist wouldn't prescribe it last year. I'm thinking about

asking again this year.

I'm borderline losing functional ability in a number of areas. If

this drug benefited me by either slowing or stopping strength lose.

Or even improving, I would be able to tell immediately.

It's already approved and supposedly, it's safe. My point is that

what do I have

> > >

> > >>

> > >>

> > >>

> > >> Epilepsy drug eases symptoms of inherited disorder that

weakens

> > >> muscles

> >

> >

> >

>

>

>

[Guest guest]

Hi Joy, Can I ask the dosage that you are taking? I tried it last year, but

probably not for long enough and , possibly the dosage wasn't strong enough

either. Thanks, Robin

Re: Valproate eases symptoms of SMA

I'm currently trying the drug-it's been a bit over 3 months. I've read in

several articles the biggest signs show in 8 months or more;however, I do feel

like it is helping somewhat. I totally think it is worth a try!

Buck <jared.buck@...> wrote:

That's true. Small sample size so it's not really enough to make a

judgement. I guess the jury is still out on it right now. But if the tests

do prove that it works, it will be good news.

On 6/25/06, jvandyke wrote:

>

> It's interesting to me, too... but the numbers in the study were so

> small. It does make you wonder if it'd help, though.

> jv

>

>

> Buck wrote:

>

> > This is a very interesting study. I do know that drugs normally used

> > to treat one disease or disorder sometimes can work to treat another,

> > it happens If the trial on child patients works like this one

> > did, we may have another avenue for those with SMA to turn to for

> > treatment. How about that?

> >

> >

> >

> > On 6/24/06, jvandyke >

> wrote:

> >

> >>

> >>

> >>

> >> Epilepsy drug eases symptoms of inherited disorder that weakens

> >> muscles

>

>

>

[Guest guest]

Some doctors are very relunctant to prescribe drugs that are used for other

purposes other than what you have. Why don't you show the doc the article

on this trial? Maybe that will convince him to prescribe it for you.

And Joy, that's great that it seems to be helping you a little bit right

now. Some drugs can take a while to take effect fully, just be patient and

I bet you will feel a whole lot better once it starts working completely.

On 6/26/06, <boojiboy@...> wrote:

>

> As anyone been able to get this drug outside of a trial? My

> neurologist wouldn't prescribe it last year. I'm thinking about

> asking again this year.

>

> I'm borderline losing functional ability in a number of areas. If

> this drug benefited me by either slowing or stopping strength lose.

> Or even improving, I would be able to tell immediately.

>

> It's already approved and supposedly, it's safe. My point is that

> what do I have

>

>

>

>

>

> > > >

> > > >>

> > > >>

> > > >>

> > > >> Epilepsy drug eases symptoms of inherited disorder that

> weakens

> > > >> muscles

> > >

> > >

> > >

> >

> >

> >

[Guest guest]

It's beginning to sound like it...

jv

Joy wrote:

> more;however, I do feel like it is helping somewhat. I totally think

> it is worth a try!

[Guest guest]

I hope this medicine works for you [and all of those who are trialing

them.]

I give you credit for taking new meds.

I trialed neurontin years ago and had pancreatitis on it- so I'm

chicken about trying brand new meds right away.

> > >

> > >>

> > >>

> > >>

> > >> Epilepsy drug eases symptoms of inherited disorder that weakens

> > >> muscles

> >

> >

> >

>

>

>

[Guest guest]

I wish these meds worked for me. I had severe joint and muscle pain as a

side effect to the valproate. I hope it helps others. But I am

disappointed that I can’t even try it long enough to see if it could help

me. And my MDA doctor had no problem prescribing it for me. I guess some

doctors aren’t as strict as others.

Blessed Be,

Meg

( <http://www.myspace.com/sexytear> http://www.myspace.com/sexytear)

( <http://blog.myspace.com/sexytear> http://blog.myspace.com/sexytear)

_____

From: [mailto: ] On

Behalf Of charmedcripchic

Sent: Tuesday, June 27, 2006 2:45 PM

Subject: Re: Valproate eases symptoms of SMA

I hope this medicine works for you [and all of those who are trialing

them.]

I give you credit for taking new meds.

I trialed neurontin years ago and had pancreatitis on it- so I'm

chicken about trying brand new meds right away.

> > >

> > >>

> > >>

> > >>

> > >> Epilepsy drug eases symptoms of inherited disorder that weakens

> > >> muscles

> >

> >

> >

>

>

>

[Guest guest]

I wonder how significantly it increases strength? Because I read in an

artical sent to me by MDA...(their new's letter) that Folic Acid

helps...I started taking even just one dose a day...over my already

Daily Multiple Vitamin (which also has Folic Acid in it) and I feel so

much better. I didn't think so much about strength altho I am sure that

has improved but my energy level has increased. I am already very

active but now I out last even my " normal " friends it seems. I've

always taken a multi vitamin but adding that extra does of Folic Acid

makes me feel even better. And I have been a big Whole Milk drinker for

43 years which I think also helps. Oh and for pain and swelling...I

swear by " Butchers Broom " . If any of you take it or start to try it let

me know how either the Folic Acid and or Butcher's Broom make you feel.

Have to get the Butcher's Broom at like a GNC or Pilgrams or a place

like them who sell natural herb suppliments.

[Guest guest]

I find all this fascinating and I really hope that it works for those

of you who are able to tolerate the side effects.

Many years ago I had a trial of it for a completely different reason -

pain - but I had an allegic reaction and was taken off it. The other

drug that was trialed before sodium valporate (UK name) for SMA was of

a similar kind named Gabapentin. I use this drug to-date for pain

management but I have not had any muscular benefit except pain relief.

So at least we all know that there are drugs out there being trialed

and hopefully something for everyone will materialise.

Wouldn't it be fantastic to hope that in years to come we can all look

too having our strength maintained for our lifetimes.