Ascorbic Acid French correspondence

[Guest guest]

(note: this is a brief summary from the head of the French research team

- I have more information coming. ~ Gretchen)

Summary of Correspondence from French Researcher Prof. Michel Fontes of

the Genetique Medicale et Developpement regarding Ascorbic Acid and CMT:

1) - Regarding clinical trials, it is obvious that a molecule is not a

medicine. We do not know if ascorbic acid works in humans and what the

optimal dose is. We have an idea that Ascorbic Acid at low doses is

inefficient. Mice are synthesizing Ascorbic Acid, and have thus a

continous level of circulation of it but our CMT mice suffer from a

neuropathy. Moreover, we have indications that only very high doses are

active. Thus, only the development of a true medicine will be efficient

and this needs clinical trials. A protocol of clinical trial has been

written (Professor Olivier Blin is the coordinator). We already have

regular approval of the trial through the competent governmental agency.

We are thus trying to raise funds to do it. I hope that trials will

start in France soon. We have also contacts in the USA to go on as quick

as possible in this country. It will certainly be interesting to do the

same in Europe, and we are open to any suggestions.

2 - For the moment, we have only tested CMT 1A mice and the action of

Ascorbic Acid on PMP22 expression. However, several others targets are

possible (we have another good candidate). My feeling is thus to try to

have an answer on CMT 1A patients as soon as possible (if it did not

work on CMT 1A patients, it will not work on other forms of CMT, meaning

that Ascorbic Acid is only active in the animal model). If it works on

CMT 1A, then other CMT patients with other CMT forms should be tested

(may be the dose will not be the same?).

3 - Regarding doses, just a word. I perfectly understand that you are

impatient to begin the testing the potential efficiency of Ascorbic

Acid, but if CMT patients are doing " self medication " , we will never

know if the molecule really works (because it should be tested in double

blinded tests). This will be a pity, because Ascorbic Acid will never be

recognized as a real medicine for CMT. You will thus never have toxic

monitoring (who can say that Ascorbic Acid at a high dose adminstered

during 10, 20, 30 years has no toxicity?). Moreover, this drug will

never have the approval of the European (or national agency). If you

make a brief calculation, a high dose every day, during the year, is not

totally insignificant.

A last suggestion, we are working in close relationship with CMT France,

the French CMT association). The annual meeeting was yesterday in

Marseille. They are very interested in contacts with CMT patients in

other European countries.