Re: Proposed Treatment for Huntington's & Hereditary Ataxia

[Guest guest]

Bill,

The proposed Huntington's treatment to my knowledge would not involve

somatic cell nuclear transfer, I believe it is a drug called histone

deacetylase or HDAC. I do not know if it has specific application for MSA

patients since MSA itself is not considered a hereditary polyglutamine

disorder but I believe those with mainly ataxia symptoms would definitely

want to have genetic testing done for hereditary ataxia since we know there

is no sure diagnosis of MSA except autopsy.

Info on Huntington's and HDAC is at:

http://hdlighthouse.org/see/treat/hdac.htm

University of California, Irvine 18-Oct-01

" Pharmaceuticals currently used to treat cancer and other diseases may be

successful for treating Huntington's disease, a UC Irvine research team has

found. "

http://hdlighthouse.org/see/treat/hdac2.htm

Gillian Bates article from Nature 18-Oct-01

" Huntington's disease results from defects in the huntingtin protein, but

the exact mechanism has been unclear. Researchers now have a better idea,

and the knowledge has proved beneficial - for flies at least. "

I have this information on the treatment forwarded from the ataxia list:

----

In internaf@y..., MikeFernandes wrote:

Dear Leo and all

To many of us there seems to be no hope. But times are changing and there

is very likely a new treatment (of which there has been none) coming out for

polyglutamine diseases. I made the following announcement at our last NAF

support group meeting:

" A special long awaited announcement for all those Ataxians who have a

known later onset Ataxia. Karin Koski, a member of our group notified me of

this article and I did a bit of further research. There has been a recent

discovery that a drug (histone deacetylase - HDAC) stops Huntington's

Disease dead in its tracks in a fruit fly. Several of the SCA's are very

similar to Huntington's Disease. All are polyglutamine (CAG repeated)

diseases. They are projected to be affected by this new drug. This includes

many of the Ataxias:

- SCA1

- SCA2

- SCA3

- SCA6

- SCA7

- SCA12

- SCA17

- DRPLA

There are lots more tests to be run with mammals before this drug will

become available, but the nice thing about this new drug is that it is

currently in Phase I Trials with the FDA (for human consumption in colon

cancer patients). As many of our

members may be interested in this advancement I have copies of the actual

`Nature' journal article and some news articles that are a bit less

technical. "

The journal abstract maybe found at at PubMed:

http://www.ncbi.nlm.nih.gov:80/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list

_uids=11607033&dopt=Abstract

I have SCA3 and was very excited about this news. So I contacted Dr.

M , lead researcher of this article, who was doing further tests on

mammals with HDAC for Huntington's. She told me that it would be about a

year (this was

about a month ago) before tests were complete. She had informed me that Dr.

Huda Zoghbi at Baylor College of Medicine would be running the tests for the

rest of the polyglutamine diseases, who I also contacted.

Personally I have been trying to crystallize the mutated Ataxin-3 protein at

work (I am still able to work part-time w/walker). I work at:

--

Mike Fernandes

Lawrence Livermore National Lab

Biology & Biotechnology Research Program

Macromolecular Crystallography and Structural Genomics

7000 East Ave., L-441, Building 377, Rm 1023

Livermore, CA 94551

http://www-structure.llnl.gov

email: fernandes9@l...

One of the good things about working in research is that other researchers

are more likely to respond to your questions.

Anyway the whole upshot of this posting is to provide some hope! It may take

a year of tests to find out if this drug actually pans out, but the nice

thing is this drug already exists and is currently undergoing FDA trials.

There is hope and we

will find out in about a year.

Cheers Mike

--

" You gain strength, courage, and confidence

by every experience in which you stop to look fear in the face. "

~ Eleanor Roosevelt ~

Mike Fernandes

National Ataxia Foundation

Northern California Support Group-Leader

fernande@p...

http://www.ataxia.org/

http://www.geocities.com/casupport/

--- End forwarded message ---

[Guest guest]

Pam,

I thought that at first also, but when I read all of the articles, it

seemed to be altering genes. It may be that they can alter them chemically,

but I don't think so if the cells are already dead. That is why I

wanted some clarification from Barbara as she may be able to interpret

it better than an engineer )

If it don't have gears, nuts, bolts, screws, etc. I don't always understand

it ) But then I have to go to Australia to adjust the nuts and

bolts in Aussie Anne's back ) She always says I am a pain in her

backside ) I hope I get that darn passport today! I want

to get my tickets!!!!

Take care, Bill Werre

-----------------------------------------------------

Pam Bower wrote:

Bill,

The proposed Huntington's treatment to my knowledge would not involve

somatic cell nuclear transfer, I believe it is a drug called histone

deacetylase or HDAC. I do not know if it has specific application

for MSA

patients since MSA itself is not considered a hereditary polyglutamine

disorder but I believe those with mainly ataxia symptoms would definitely

want to have genetic testing done for hereditary ataxia since we know

there

is no sure diagnosis of MSA except autopsy.

Info on Huntington's and HDAC is at:

http://hdlighthouse.org/see/treat/hdac.htm

University of California, Irvine 18-Oct-01

"Pharmaceuticals currently used to treat cancer and other diseases

may be

successful for treating Huntington's disease, a UC Irvine research

team has

found. "

http://hdlighthouse.org/see/treat/hdac2.htm

Gillian Bates article from Nature 18-Oct-01

"Huntington's disease results from defects in the huntingtin protein,

but

the exact mechanism has been unclear. Researchers now have a better

idea,

and the knowledge has proved beneficial - for flies at least."

I have this information on the treatment forwarded from the ataxia list:

----

In internaf@y..., MikeFernandes <fernande@p...> wrote:

Dear Leo and all

To many of us there seems to be no hope. But times are changing and

there

is very likely a new treatment (of which there has been none) coming

out for

polyglutamine diseases. I made the following announcement at our last

NAF

support group meeting:

" A special long awaited announcement for all those Ataxians who have

a

known later onset Ataxia. Karin Koski, a member of our group notified

me of

this article and I did a bit of further research. There has been

a recent

discovery that a drug (histone deacetylase - HDAC) stops Huntington's

Disease dead in its tracks in a fruit fly. Several of the SCA's are

very

similar to Huntington's Disease. All are polyglutamine (CAG repeated)

diseases. They are projected to be affected by this new drug. This

includes

many of the Ataxias:

- SCA1

- SCA2

- SCA3

- SCA6

- SCA7

- SCA12

- SCA17

- DRPLA

There are lots more tests to be run with mammals before this drug will

become available, but the nice thing about this new drug is that it

is

currently in Phase I Trials with the FDA (for human consumption in

colon

cancer patients). As many of our

members may be interested in this advancement I have copies of the

actual

`Nature' journal article and some news articles that are a bit less

technical. "

The journal abstract maybe found at at PubMed:

http://www.ncbi.nlm.nih.gov:80/entrez/query.fcgi?cmd=Retrieve & db=PubMed & list

_uids=11607033 & dopt=Abstract

I have SCA3 and was very excited about this news. So I contacted Dr.

M , lead researcher of this article, who was doing further

tests on

mammals with HDAC for Huntington's. She told me that it would be about

a

year (this was

about a month ago) before tests were complete. She had informed me

that Dr.

Huda Zoghbi at Baylor College of Medicine would be running the tests

for the

rest of the polyglutamine diseases, who I also contacted.

Personally I have been trying to crystallize the mutated Ataxin-3 protein

at

work (I am still able to work part-time w/walker). I work at:

--

Mike Fernandes

Lawrence Livermore National Lab

Biology & Biotechnology Research Program

Macromolecular Crystallography and Structural Genomics

7000 East Ave., L-441, Building 377, Rm 1023

Livermore, CA 94551

http://www-structure.llnl.gov

email: fernandes9@l...

One of the good things about working in research is that other researchers

are more likely to respond to your questions.

Anyway the whole upshot of this posting is to provide some hope! It

may take

a year of tests to find out if this drug actually pans out, but the

nice

thing is this drug already exists and is currently undergoing FDA trials.

There is hope and we

will find out in about a year.

Cheers Mike

--

"You gain strength, courage, and confidence

by every experience in which you stop to look fear in the face."

~ Eleanor

Roosevelt ~

Mike Fernandes

National Ataxia Foundation

Northern California Support Group-Leader

fernande@p...

http://www.ataxia.org/

http://www.geocities.com/casupport/

--- End forwarded message ---

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[Guest guest]

I still think it's a drug --- it's not a cure or a reversal of

symptoms ---it does NOT fix the Huntington gene --- it's intent would be to

slow down further loss of brain cell function. My interpretation is

below... I could be wrong, so please if anyone understands this better,

chime in.

---

" The researchers found that the mutant form of the protein called Htt, or

" Huntingtin, " that results from genetic changes causing Huntington's disease

inhibits the actions of several other proteins whose normal function is

essential for brain cells. The mutated form of Huntingtin stifles the

activity of key enzymes called acetyltransferases. Reducing the levels of

these acetyltransferases and related proteins results in the nerve damage

seen in the diseased brain. "

---> I'm understanding from this that low levels of acetyltransferases and

other related proteins result in nerve cell damage in Huntington's

" Cells in the body normally maintain a balance between the opposing forces

of acetyltransferase enzymes that modify proteins to increase genetic

activity and HDAC enzymes that reverse these modifications to reduce gene

activity. Since the mutated Huntingtin protein destroys this balance, the

researchers sought to restore the balance by reducing HDAC activities to

compensate. The HDAC inhibitors were able to prevent neuron damage in fruit

flies that were engineered to carry a disease-producing form of human

Huntingtin. "

---> HDAC inhibitors would not restore the huntingtin protein but would

maintain a balance between HDAC enzymes and aceyltransferase enzymes ...

when the balance is off that's when nerve cell damage occurs.

" While presently, we can't eliminate the genetic mutation that ultimately

causes Huntington's disease, this study indicates that we may be able to

significantly reduce the effects of that mutation, " Marsh said. " This study

also points the finger at a complex of genes and tells us what we need to do

next. Perhaps we can find even better and more precisely targeted strategies

to help this disease. If this strategy proves effective in other animals

such as mice, we will still need to know when to administer such a drug, how

to administer it and what the long-term side effects may be. "

--> They can not fix the gene that causes Huntington's but believe that if

people were given drugs known as HDAC inhibitors it would result in less

damage to brain cells.. so far they have tested this theory in fruit flies

with very positive results... still work to do in mice and other animals

before it is tried in humans and they'd need to figure out the appropriate

dosages. Some HDAC inhibitor drugs are in the FDA approval process now for

use in treating cancer.

Re: Proposed Treatment for Huntington's & Hereditary Ataxia

> Pam,

>

> I thought that at first also, but when I read all of the articles, it

seemed to

> be altering genes. It may be that they can alter them chemically, but I

don't

> think so if the cells are already dead. That is why I wanted some

clarification

> from Barbara as she may be able to interpret it better than an engineer

)

>

> If it don't have gears, nuts, bolts, screws, etc. I don't always

understand it

> ) But then I have to go to Australia to adjust the nuts and bolts in

Aussie

> Anne's back ) She always says I am a pain in her backside ) I hope I

get

> that darn passport today! I want to get my tickets!!!!

>

> Take care, Bill Werre

>

> -----------------------------------------------------

>

> Pam Bower wrote:

>

> > Bill,

> >

> > The proposed Huntington's treatment to my knowledge would not involve

> > somatic cell nuclear transfer, I believe it is a drug called histone

> > deacetylase or HDAC. I do not know if it has specific application for

MSA

> > patients since MSA itself is not considered a hereditary polyglutamine

> > disorder but I believe those with mainly ataxia symptoms would

definitely

> > want to have genetic testing done for hereditary ataxia since we know

there

> > is no sure diagnosis of MSA except autopsy.

> >

> > Info on Huntington's and HDAC is at:

> >

> > http://hdlighthouse.org/see/treat/hdac.htm

> > University of California, Irvine 18-Oct-01

> > " Pharmaceuticals currently used to treat cancer and other diseases may

be

> > successful for treating Huntington's disease, a UC Irvine research team

has

> > found. "

> >

> > http://hdlighthouse.org/see/treat/hdac2.htm

> > Gillian Bates article from Nature 18-Oct-01

> > " Huntington's disease results from defects in the huntingtin protein,

but

> > the exact mechanism has been unclear. Researchers now have a better

idea,

> > and the knowledge has proved beneficial - for flies at least. "

> >

> > I have this information on the treatment forwarded from the ataxia list:

> >

> > ----

> >

> > In internaf@y..., MikeFernandes wrote:

> > Dear Leo and all

> >

> > To many of us there seems to be no hope. But times are changing and

there

> > is very likely a new treatment (of which there has been none) coming out

for

> > polyglutamine diseases. I made the following announcement at our last

NAF

> > support group meeting:

> >

> > " A special long awaited announcement for all those Ataxians who have a

> > known later onset Ataxia. Karin Koski, a member of our group notified me

of

> > this article and I did a bit of further research. There has been a

recent

> > discovery that a drug (histone deacetylase - HDAC) stops Huntington's

> > Disease dead in its tracks in a fruit fly. Several of the SCA's are very

> > similar to Huntington's Disease. All are polyglutamine (CAG repeated)

> > diseases. They are projected to be affected by this new drug. This

includes

> > many of the Ataxias:

> > - SCA1

> > - SCA2

> > - SCA3

> > - SCA6

> > - SCA7

> > - SCA12

> > - SCA17

> > - DRPLA

> > There are lots more tests to be run with mammals before this drug will

> > become available, but the nice thing about this new drug is that it is

> > currently in Phase I Trials with the FDA (for human consumption in colon

> > cancer patients). As many of our

> > members may be interested in this advancement I have copies of the

actual

> > `Nature' journal article and some news articles that are a bit less

> > technical. "

> >

> > The journal abstract maybe found at at PubMed:

> >

http://www.ncbi.nlm.nih.gov:80/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list

> > _uids=11607033&dopt=Abstract

> >

> > I have SCA3 and was very excited about this news. So I contacted Dr.

> > M , lead researcher of this article, who was doing further tests

on

> > mammals with HDAC for Huntington's. She told me that it would be about a

> > year (this was

> > about a month ago) before tests were complete. She had informed me that

Dr.

> > Huda Zoghbi at Baylor College of Medicine would be running the tests for

the

> > rest of the polyglutamine diseases, who I also contacted.

> >

> > Personally I have been trying to crystallize the mutated Ataxin-3

protein at

> > work (I am still able to work part-time w/walker). I work at:

> > --

> > Mike Fernandes

> > Lawrence Livermore National Lab

> > Biology & Biotechnology Research Program

> > Macromolecular Crystallography and Structural Genomics

> > 7000 East Ave., L-441, Building 377, Rm 1023

> > Livermore, CA 94551

> > http://www-structure.llnl.gov

> > email: fernandes9@l...

> > One of the good things about working in research is that other

researchers

> > are more likely to respond to your questions.

> >

> > Anyway the whole upshot of this posting is to provide some hope! It may

take

> > a year of tests to find out if this drug actually pans out, but the nice

> > thing is this drug already exists and is currently undergoing FDA

trials.

> > There is hope and we

> > will find out in about a year.

> >

> > Cheers Mike

> > --

> > " You gain strength, courage, and confidence

> > by every experience in which you stop to look fear in the face. "

> >

> > ~ Eleanor Roosevelt ~

> >

> > Mike Fernandes

> > National Ataxia Foundation

> > Northern California Support Group-Leader

> >

> > fernande@p...

> > http://www.ataxia.org/

> > http://www.geocities.com/casupport/

> > --- End forwarded message ---

[Guest guest]

I still think it's a drug --- it's not a cure or a reversal of

symptoms ---it does NOT fix the Huntington gene --- it's intent would be to

slow down further loss of brain cell function. My interpretation is

below... I could be wrong, so please if anyone understands this better,

chime in.

---

" The researchers found that the mutant form of the protein called Htt, or

" Huntingtin, " that results from genetic changes causing Huntington's disease

inhibits the actions of several other proteins whose normal function is

essential for brain cells. The mutated form of Huntingtin stifles the

activity of key enzymes called acetyltransferases. Reducing the levels of

these acetyltransferases and related proteins results in the nerve damage

seen in the diseased brain. "

---> I'm understanding from this that low levels of acetyltransferases and

other related proteins result in nerve cell damage in Huntington's

" Cells in the body normally maintain a balance between the opposing forces

of acetyltransferase enzymes that modify proteins to increase genetic

activity and HDAC enzymes that reverse these modifications to reduce gene

activity. Since the mutated Huntingtin protein destroys this balance, the

researchers sought to restore the balance by reducing HDAC activities to

compensate. The HDAC inhibitors were able to prevent neuron damage in fruit

flies that were engineered to carry a disease-producing form of human

Huntingtin. "

---> HDAC inhibitors would not restore the huntingtin protein but would

maintain a balance between HDAC enzymes and aceyltransferase enzymes ...

when the balance is off that's when nerve cell damage occurs.

" While presently, we can't eliminate the genetic mutation that ultimately

causes Huntington's disease, this study indicates that we may be able to

significantly reduce the effects of that mutation, " Marsh said. " This study

also points the finger at a complex of genes and tells us what we need to do

next. Perhaps we can find even better and more precisely targeted strategies

to help this disease. If this strategy proves effective in other animals

such as mice, we will still need to know when to administer such a drug, how

to administer it and what the long-term side effects may be. "

--> They can not fix the gene that causes Huntington's but believe that if

people were given drugs known as HDAC inhibitors it would result in less

damage to brain cells.. so far they have tested this theory in fruit flies

with very positive results... still work to do in mice and other animals

before it is tried in humans and they'd need to figure out the appropriate

dosages. Some HDAC inhibitor drugs are in the FDA approval process now for

use in treating cancer.

Re: Proposed Treatment for Huntington's & Hereditary Ataxia

> Pam,

>

> I thought that at first also, but when I read all of the articles, it

seemed to

> be altering genes. It may be that they can alter them chemically, but I

don't

> think so if the cells are already dead. That is why I wanted some

clarification

> from Barbara as she may be able to interpret it better than an engineer

)

>

> If it don't have gears, nuts, bolts, screws, etc. I don't always

understand it

> ) But then I have to go to Australia to adjust the nuts and bolts in

Aussie

> Anne's back ) She always says I am a pain in her backside ) I hope I

get

> that darn passport today! I want to get my tickets!!!!

>

> Take care, Bill Werre

>

> -----------------------------------------------------

>

> Pam Bower wrote:

>

> > Bill,

> >

> > The proposed Huntington's treatment to my knowledge would not involve

> > somatic cell nuclear transfer, I believe it is a drug called histone

> > deacetylase or HDAC. I do not know if it has specific application for

MSA

> > patients since MSA itself is not considered a hereditary polyglutamine

> > disorder but I believe those with mainly ataxia symptoms would

definitely

> > want to have genetic testing done for hereditary ataxia since we know

there

> > is no sure diagnosis of MSA except autopsy.

> >

> > Info on Huntington's and HDAC is at:

> >

> > http://hdlighthouse.org/see/treat/hdac.htm

> > University of California, Irvine 18-Oct-01

> > " Pharmaceuticals currently used to treat cancer and other diseases may

be

> > successful for treating Huntington's disease, a UC Irvine research team

has

> > found. "

> >

> > http://hdlighthouse.org/see/treat/hdac2.htm

> > Gillian Bates article from Nature 18-Oct-01

> > " Huntington's disease results from defects in the huntingtin protein,

but

> > the exact mechanism has been unclear. Researchers now have a better

idea,

> > and the knowledge has proved beneficial - for flies at least. "

> >

> > I have this information on the treatment forwarded from the ataxia list:

> >

> > ----

> >

> > In internaf@y..., MikeFernandes wrote:

> > Dear Leo and all

> >

> > To many of us there seems to be no hope. But times are changing and

there

> > is very likely a new treatment (of which there has been none) coming out

for

> > polyglutamine diseases. I made the following announcement at our last

NAF

> > support group meeting:

> >

> > " A special long awaited announcement for all those Ataxians who have a

> > known later onset Ataxia. Karin Koski, a member of our group notified me

of

> > this article and I did a bit of further research. There has been a

recent

> > discovery that a drug (histone deacetylase - HDAC) stops Huntington's

> > Disease dead in its tracks in a fruit fly. Several of the SCA's are very

> > similar to Huntington's Disease. All are polyglutamine (CAG repeated)

> > diseases. They are projected to be affected by this new drug. This

includes

> > many of the Ataxias:

> > - SCA1

> > - SCA2

> > - SCA3

> > - SCA6

> > - SCA7

> > - SCA12

> > - SCA17

> > - DRPLA

> > There are lots more tests to be run with mammals before this drug will

> > become available, but the nice thing about this new drug is that it is

> > currently in Phase I Trials with the FDA (for human consumption in colon

> > cancer patients). As many of our

> > members may be interested in this advancement I have copies of the

actual

> > `Nature' journal article and some news articles that are a bit less

> > technical. "

> >

> > The journal abstract maybe found at at PubMed:

> >

http://www.ncbi.nlm.nih.gov:80/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list

> > _uids=11607033&dopt=Abstract

> >

> > I have SCA3 and was very excited about this news. So I contacted Dr.

> > M , lead researcher of this article, who was doing further tests

on

> > mammals with HDAC for Huntington's. She told me that it would be about a

> > year (this was

> > about a month ago) before tests were complete. She had informed me that

Dr.

> > Huda Zoghbi at Baylor College of Medicine would be running the tests for

the

> > rest of the polyglutamine diseases, who I also contacted.

> >

> > Personally I have been trying to crystallize the mutated Ataxin-3

protein at

> > work (I am still able to work part-time w/walker). I work at:

> > --

> > Mike Fernandes

> > Lawrence Livermore National Lab

> > Biology & Biotechnology Research Program

> > Macromolecular Crystallography and Structural Genomics

> > 7000 East Ave., L-441, Building 377, Rm 1023

> > Livermore, CA 94551

> > http://www-structure.llnl.gov

> > email: fernandes9@l...

> > One of the good things about working in research is that other

researchers

> > are more likely to respond to your questions.

> >

> > Anyway the whole upshot of this posting is to provide some hope! It may

take

> > a year of tests to find out if this drug actually pans out, but the nice

> > thing is this drug already exists and is currently undergoing FDA

trials.

> > There is hope and we

> > will find out in about a year.

> >

> > Cheers Mike

> > --

> > " You gain strength, courage, and confidence

> > by every experience in which you stop to look fear in the face. "

> >

> > ~ Eleanor Roosevelt ~

> >

> > Mike Fernandes

> > National Ataxia Foundation

> > Northern California Support Group-Leader

> >

> > fernande@p...

> > http://www.ataxia.org/

> > http://www.geocities.com/casupport/

> > --- End forwarded message ---

[Guest guest]

I still think it's a drug --- it's not a cure or a reversal of

symptoms ---it does NOT fix the Huntington gene --- it's intent would be to

slow down further loss of brain cell function. My interpretation is

below... I could be wrong, so please if anyone understands this better,

chime in.

---

" The researchers found that the mutant form of the protein called Htt, or

" Huntingtin, " that results from genetic changes causing Huntington's disease

inhibits the actions of several other proteins whose normal function is

essential for brain cells. The mutated form of Huntingtin stifles the

activity of key enzymes called acetyltransferases. Reducing the levels of

these acetyltransferases and related proteins results in the nerve damage

seen in the diseased brain. "

---> I'm understanding from this that low levels of acetyltransferases and

other related proteins result in nerve cell damage in Huntington's

" Cells in the body normally maintain a balance between the opposing forces

of acetyltransferase enzymes that modify proteins to increase genetic

activity and HDAC enzymes that reverse these modifications to reduce gene

activity. Since the mutated Huntingtin protein destroys this balance, the

researchers sought to restore the balance by reducing HDAC activities to

compensate. The HDAC inhibitors were able to prevent neuron damage in fruit

flies that were engineered to carry a disease-producing form of human

Huntingtin. "

---> HDAC inhibitors would not restore the huntingtin protein but would

maintain a balance between HDAC enzymes and aceyltransferase enzymes ...

when the balance is off that's when nerve cell damage occurs.

" While presently, we can't eliminate the genetic mutation that ultimately

causes Huntington's disease, this study indicates that we may be able to

significantly reduce the effects of that mutation, " Marsh said. " This study

also points the finger at a complex of genes and tells us what we need to do

next. Perhaps we can find even better and more precisely targeted strategies

to help this disease. If this strategy proves effective in other animals

such as mice, we will still need to know when to administer such a drug, how

to administer it and what the long-term side effects may be. "

--> They can not fix the gene that causes Huntington's but believe that if

people were given drugs known as HDAC inhibitors it would result in less

damage to brain cells.. so far they have tested this theory in fruit flies

with very positive results... still work to do in mice and other animals

before it is tried in humans and they'd need to figure out the appropriate

dosages. Some HDAC inhibitor drugs are in the FDA approval process now for

use in treating cancer.

Re: Proposed Treatment for Huntington's & Hereditary Ataxia

> Pam,

>

> I thought that at first also, but when I read all of the articles, it

seemed to

> be altering genes. It may be that they can alter them chemically, but I

don't

> think so if the cells are already dead. That is why I wanted some

clarification

> from Barbara as she may be able to interpret it better than an engineer

)

>

> If it don't have gears, nuts, bolts, screws, etc. I don't always

understand it

> ) But then I have to go to Australia to adjust the nuts and bolts in

Aussie

> Anne's back ) She always says I am a pain in her backside ) I hope I

get

> that darn passport today! I want to get my tickets!!!!

>

> Take care, Bill Werre

>

> -----------------------------------------------------

>

> Pam Bower wrote:

>

> > Bill,

> >

> > The proposed Huntington's treatment to my knowledge would not involve

> > somatic cell nuclear transfer, I believe it is a drug called histone

> > deacetylase or HDAC. I do not know if it has specific application for

MSA

> > patients since MSA itself is not considered a hereditary polyglutamine

> > disorder but I believe those with mainly ataxia symptoms would

definitely

> > want to have genetic testing done for hereditary ataxia since we know

there

> > is no sure diagnosis of MSA except autopsy.

> >

> > Info on Huntington's and HDAC is at:

> >

> > http://hdlighthouse.org/see/treat/hdac.htm

> > University of California, Irvine 18-Oct-01

> > " Pharmaceuticals currently used to treat cancer and other diseases may

be

> > successful for treating Huntington's disease, a UC Irvine research team

has

> > found. "

> >

> > http://hdlighthouse.org/see/treat/hdac2.htm

> > Gillian Bates article from Nature 18-Oct-01

> > " Huntington's disease results from defects in the huntingtin protein,

but

> > the exact mechanism has been unclear. Researchers now have a better

idea,

> > and the knowledge has proved beneficial - for flies at least. "

> >

> > I have this information on the treatment forwarded from the ataxia list:

> >

> > ----

> >

> > In internaf@y..., MikeFernandes wrote:

> > Dear Leo and all

> >

> > To many of us there seems to be no hope. But times are changing and

there

> > is very likely a new treatment (of which there has been none) coming out

for

> > polyglutamine diseases. I made the following announcement at our last

NAF

> > support group meeting:

> >

> > " A special long awaited announcement for all those Ataxians who have a

> > known later onset Ataxia. Karin Koski, a member of our group notified me

of

> > this article and I did a bit of further research. There has been a

recent

> > discovery that a drug (histone deacetylase - HDAC) stops Huntington's

> > Disease dead in its tracks in a fruit fly. Several of the SCA's are very

> > similar to Huntington's Disease. All are polyglutamine (CAG repeated)

> > diseases. They are projected to be affected by this new drug. This

includes

> > many of the Ataxias:

> > - SCA1

> > - SCA2

> > - SCA3

> > - SCA6

> > - SCA7

> > - SCA12

> > - SCA17

> > - DRPLA

> > There are lots more tests to be run with mammals before this drug will

> > become available, but the nice thing about this new drug is that it is

> > currently in Phase I Trials with the FDA (for human consumption in colon

> > cancer patients). As many of our

> > members may be interested in this advancement I have copies of the

actual

> > `Nature' journal article and some news articles that are a bit less

> > technical. "

> >

> > The journal abstract maybe found at at PubMed:

> >

http://www.ncbi.nlm.nih.gov:80/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list

> > _uids=11607033&dopt=Abstract

> >

> > I have SCA3 and was very excited about this news. So I contacted Dr.

> > M , lead researcher of this article, who was doing further tests

on

> > mammals with HDAC for Huntington's. She told me that it would be about a

> > year (this was

> > about a month ago) before tests were complete. She had informed me that

Dr.

> > Huda Zoghbi at Baylor College of Medicine would be running the tests for

the

> > rest of the polyglutamine diseases, who I also contacted.

> >

> > Personally I have been trying to crystallize the mutated Ataxin-3

protein at

> > work (I am still able to work part-time w/walker). I work at:

> > --

> > Mike Fernandes

> > Lawrence Livermore National Lab

> > Biology & Biotechnology Research Program

> > Macromolecular Crystallography and Structural Genomics

> > 7000 East Ave., L-441, Building 377, Rm 1023

> > Livermore, CA 94551

> > http://www-structure.llnl.gov

> > email: fernandes9@l...

> > One of the good things about working in research is that other

researchers

> > are more likely to respond to your questions.

> >

> > Anyway the whole upshot of this posting is to provide some hope! It may

take

> > a year of tests to find out if this drug actually pans out, but the nice

> > thing is this drug already exists and is currently undergoing FDA

trials.

> > There is hope and we

> > will find out in about a year.

> >

> > Cheers Mike

> > --

> > " You gain strength, courage, and confidence

> > by every experience in which you stop to look fear in the face. "

> >

> > ~ Eleanor Roosevelt ~

> >

> > Mike Fernandes

> > National Ataxia Foundation

> > Northern California Support Group-Leader

> >

> > fernande@p...

> > http://www.ataxia.org/

> > http://www.geocities.com/casupport/

> > --- End forwarded message ---