Re: Any hope of finding a treatment for CMT in the next 20-30 years

[Guest guest]

I believe the CMTA thinks they will have at least a treatment if not a cure

within 5 years for some types of CMT. There are people actively working on

CMT research to find a cure and /or treatment. Hall, president of CMTA

is quite confident the money is being raised and research done and we should

have positive news way before 20 years.

Lori

[Guest guest]

Are you new to this group? I am sure Gretchen will direct you, but this is

probably the best place to get the most up to date information in an easy format

along with support from members. Gretchen is constantly posting the latest in

medical technology and it seems to me that the treatment/cure is truly making

huge progress and maybe in our lifetime we will see it.

Jackie

Any hope of finding a treatment for CMT in the next 20-30 years

 

Hello all,

I posted this question on CMTA forum but I decided to post it here. I know this

is kind of stupid question to ask but I would like to know if there is any hope

of finding a treatment of CMT in the next 20 years or so? Can anyone who has the

latest info share it with me? I know that during the last couple years, we know

more about CMT than ever before and there are several trials will end by this

year. However the one thing that worry me is that it seems like CMT is not a a

priority decease to research and there are not many doctors spend time to find a

cure for it. Please correct me if I am wrong.

[Guest guest]

Dear Gretchen,

Thank you so much for helping me out. I am new this group since I only heard

about CMT more than 1 week ago after a doctor said to me that he thought that I

have had CMT.

Can I ask 3 more questions:

1. As mentioned on CMTA, this decease is a rare one, so it seems to me that it

is not really a priority for researchers. Therefore, can you please tell me if

the money has been raised is enough to achieve the goal from CMTA?

2. I know that in order to get a treatment, it needs to be approved by the

government. Could you please tell me how long it will take to get through this?

3. I know there are couple trials will finish at the end of this year, could you

please share with me if there is any progress in the trials?

Again, thank you so much for spending your time to help me.

Best regards,

>

> There sure is hope for treatment of CMT 1A in 3-5 years. Are you not familiar

with STAR?

>

> http://www.charcot-marie-tooth.org/STAR.php Project 1 has been completed

http://www.charcot-marie-tooth.org/STAR_update2.php

>

>

http://www.charcot-marie-tooth.org/headlines/117/Latest-Research-Presented-at--C\

MT-Consortium-Abstracts-Now-Available/

>

> Lonaprisan research is ongoing in Germany visit this site to read about it

from the Consortium papers

http://www.charcot-marie-tooth.org/headlines/117/Latest-Research-Presented-at--C\

MT-Consortium-Abstracts-Now-Available/arch

>

> Because CMT 1A affects 70% of people with CMT, that type is being tested

first, followed by Types X and 2. With high thoroughput screening and

partnerning with pharmaceuticals, we will be seeing much more research soon.

Nerve growth factors and gene silencing are but two other projects ongoing

worldwide.

>

> Gretchen

>

[Guest guest]

To answer your questions:

While CMT is considered 'rare', many researchers specifically interested in CMT

are working around the world. Their research is a priority. Grant funding is

increasingly competitive, so other sources of funding come from personal

donations, memberships, The NIH, special events like a golf tournament, and

more. You may wish to join the CMTA and learn about STAR and all of the

fundraising efforts.

I do not know how long a treatment approval from the FDA would take. Supposing

that Ascorbic Acid (Vitamin C) does prove to be a treatment for CMT 1A, it

should be short, since the Vitamin is already approved.

I will post all research news on trials as soon as I receive it. I think one in

Belgium is in either Phase 2 or 3. And the one in Italy ended in August. I

haven't heard of the results yet. The one for Effects of Coenzyme Q10 on CMT

will conclude in 2012.

http://clinicaltrials.gov/ct2/show/NCT00541164?term=charcot+marie+tooth & rank=1

Gretchen

[Guest guest]

Thank you so much for answering my questions. I have 3 more questions, hopefully

you don't mind it at all.

1. It seems to me that all the trials now is just looking for away to make the

progress of CMT becoming much more slower rather than stop it, is it a correct

statement?

2. I don't want to be a jerk here, but can you please let me know based on what

facts STAR projects hope to have some type of treatment or cure for CMT Type 1A

in the next 3 to 5 years. I guess it must back up by something but I could not

find it over the internet.

3. Due to the financial crisis, I guess so, the 350K Board Challenge from CMTA

is only raised about 1/5 of the amount of money they wish to raise. So what

happens if they could not raise the amount they are looking for? Does it mean

that all the projects they are funding now will get affected or even abandoned?

>

> To answer your questions:

>

> While CMT is considered 'rare', many researchers specifically interested in

CMT are working around the world. Their research is a priority. Grant funding is

increasingly competitive, so other sources of funding come from personal

donations, memberships, The NIH, special events like a golf tournament, and

more. You may wish to join the CMTA and learn about STAR and all of the

fundraising efforts.

>

> I do not know how long a treatment approval from the FDA would take. Supposing

that Ascorbic Acid (Vitamin C) does prove to be a treatment for CMT 1A, it

should be short, since the Vitamin is already approved.

>

> I will post all research news on trials as soon as I receive it. I think one

in Belgium is in either Phase 2 or 3. And the one in Italy ended in August. I

haven't heard of the results yet. The one for Effects of Coenzyme Q10 on CMT

will conclude in 2012.

>

> http://clinicaltrials.gov/ct2/show/NCT00541164?term=charcot+marie+tooth & rank=1

>

>

> Gretchen

>

[Guest guest]

After more reading and research, I still have something that is confused me

about CMT, could you please help me to answer the following questions:

1. In term of NCV and EMG, what are the main differences between type 1 and type

2?

2. CMT1A is the most popular, which is the second one?

3. I know that trial and research for finding a treatment of CMT1A is now

undergoing many places around the world, but apart from it, is there any

research to find treatment for type 2 is performing?

>

> Hello all,

>

> I posted this question on CMTA forum but I decided to post it here. I know

this is kind of stupid question to ask but I would like to know if there is any

hope of finding a treatment of CMT in the next 20 years or so? Can anyone who

has the latest info share it with me? I know that during the last couple years,

we know more about CMT than ever before and there are several trials will end by

this year. However the one thing that worry me is that it seems like CMT is not

a a priority decease to research and there are not many doctors spend time to

find a cure for it. Please correct me if I am wrong.

>