Re: Office of the Prime Minister / Cabinet du Premier ministre

December 13, 2010

Brillant !

Those of us with CMT in the US should share this news with all our elected

officials, those at the CDC, at the FDA, etc. Canada, like Europe is so much

more progressive. Very interesting to know the Canadian statistics.

Gretchen

>

> I thought my friends would like to see the following.

>

> It is great to get a response from our Prime Minister, Harper.

> Sometimes it pays to know people in high places.

>

> Office of the Prime Minister / Cabinet du Premier ministre

>

> Dear Mr. Beckingham:

>

> On behalf of the Right Honourable Harper, I would like to

> acknowledge receipt of your e-mail correspondence regarding

> Charcot-Marie-Tooth Disease.

>

> Please be assured that your comments have been carefully reviewed. I have

> taken the liberty of forwrading your e-mail to the Honourable Leona

> Aglukkaq, Minister of Health, in order that she may be made aware of your

> interest in this matter.

>

> Thank you for writing to the Prime Minister.

>

> A. Opalick

> Executive Correspondence Officer

> for the Prime Minister's Office

> Agent de correspondance

> de la haute direction

> pour le Cabinet du Premier ministre

>

> >>> From : Dave Beckingham dgbeckingham@... Received : 03 Dec

> >>> 2010 04:13:17 PM >>>

>

> >>> Subject : CMT 1A: Pharnext Pleodrug PXT3003 Phase 2 Trials

> >>> BEGIN >>>>

>

> Good Day:

>

> As a Conservative Member and supporter (very little financial/CPP

> Disability), I am sending you the following important and encouraging

> article.

>

> I do indeed have CMT1A, a neuromuscular disease. CMT1A is the most common

> form of hereditary neuropathy and it affects 1 in 2,500 people. This equates

> to 13,600 Canadians. CMT (Charcot-Marie-Tooth) is named after the 3 doctors

> who discovered the disease in 1885. There were 2 French doctors, Jean

> Charcot & Pierre Marie along with an English doctor named Tooth. Jean

> Charcot also discovered ALS (Lou Gehrig's disease). It is interesting that

> these trials are being done in France.

>

> It would be a blessing if Health Canada could get involved in these clinical

> trials, or at least keep abreast of these trials. I am presently 61 years

> old, so this may not happen in my time, but perhaps my son's time who is

> only 21 years old. Your assistance would be greatly appreciated.

>

> Kindest regards,

>

> G. Beckingham......Port Elgin, ON.

> 519-832-3225

> dgbeckingham@...,

>

> From: -owner

> Sent: Friday, December 03, 2010 12:14 PM

> ] CMT 1A: Pharnext Pleodrug PXT3003 Phase 2 Trials BEGIN

>

> PHARNEXT Receives Clinical Trial Authorization for a Phase II Study with the

> First Drug Generated by Its PleotherapyTM Technology

>

http://www.benzinga.com/press-releases/10/12/b665794/pharnext-receives-clinical-\

trial-authorization-for-a-phase-ii-study

>

> Pharnext SAS, a biopharmaceutical company specializing in the development of

> innovative treatments for severe neurological diseases and other major unmet

> medical needs, today announced that the French Agency for Healthcare Product

> Safety (AFSSAPS) has authorized a Phase II clinical trial with the first

> drug (a Pleodrug?) generated by the company's Pleotherapy? technology

> platform in patients suffering from Charcot-Marie Tooth disease type 1A

> (CMT1A) - a chronic, severe, invalidating and currently incurable

> neuromuscular disease which affects 3 million people worldwide.

>

> Pharnext's first Pleodrug? is a composed of three generic drugs in a new

> formulation (a syrup) and which have already received marketing approval for

> therapeutic indications other than CMT1A. Given that the three compounds are

> present in the Pleodrug? at 10- to 100-fold lower doses than in their usual

> indication, the clinical development programme in humans has not

> necessitated the performance of a Phase I safety trial and has enabled

> Pharnext to obtain a Phase II clinical trial authorization directly after

> just 3 years (rather than the 8 years required on average for most drugs).

>

> The forthcoming Phase II randomized, comparative, placebo-controlled

> clinical trial will evaluate the safety, efficacy, pharmacodynamics and

> pharmacokinetics of PXT3003 in patients suffering from CMT1A. The trial will

> be performed in 5 investigating centres (Timone University Hospital in

> Marseilles, Salpêtrière Hospital in Paris, Limoges University Hospital,

> Lyons Sud University Hospital and Pierre Swynghedauw Hospital in Lille) and

> will be initiated first at Timone University Hospital (coordinating

> investigator: Shahram Attarian MD) in December.

>

> 'This first clinical trial authorization constitutes key proof of the

> efficiency of our Pleotherapy? technology. We are continuing to screen for

> Pleodrugs? for six other serious diseases (including Alzheimer's disease);

> our model means that we can hope to make Pleodrugs? available to patient

> more rapidly', emphasized Pharnext founder and CEO Professor Cohen.

> 'We are delighted to be able to start the first clinical trial of our first

> Pleodrug? for the treatment of Charcot-Marie-Tooth disease', he concluded.

>

> Pharnext has designed a novel, proprietary platform (Pleotherapy?) based on

> network pharmacology. The company's discovery engine identifies rational

> drug combinations (Pleodrugs?) made up of individual Pleocompounds?. This

> innovative approach could provide a solution to the ongoing and worrying

> decline in pharmaceutical pipeline productivity.

>

> About Pharnext

> Pharnext is a biopharmaceutical company focused on discovering, developing

> and licensing new pharmaceutical treatments based on intelligent

> Pleocompounds? that form the basis of Pleodrugs?. Pharnext was founded in

> Paris, France in April, 2007, by Professor Cohen, MD, PhD, his

> research group (composed of pioneers in genome science and technology) and

> Philippe Pouletty, MD (General Partner at Truffle Capital). Thanks to

> Truffle Capital's founding investment, strong support from OSEO, a grant

> from the French Muscular Dystrophy Association (AFM) and the benefits of

> France's research tax credit scheme, Pharnext has developed a novel,

> proprietary platform (PleotherapyTM) based on network pharmacology and which

> may provide a solution for the current decline in pharmaceutical pipeline

> productivity. Pharnext is advised by La Compagnie Financière Edmond de

> Rothschild.

>

December 13, 2010

Well done !

I would be interested to know where the stats for Canadians affected with CMT

originate and how recently they were gathered. (I believe I read these same

numbers around 15 years ago). I wouldn't be surprised if this number is much

larger. I, along with 2 of my children, have CMT and I have no knowledge of

being included in any official poll.

Because of the diverse levels of severity of CMT I âm sure many people are not

even diagnosed. It would be great if we could have some Health Canada

involvement to reach everyone facing the challenges of living with CMT in an

effort to discover the true magnitude of this disease. Maybe then there would

be more motivation to find a cure!

I hate to think that not enough people have CMT (as even one is too many) but

this is probably the case when it comes to getting a financial commitment from

any government.

Warmest wishes,

From: gfijig

Sent: Monday, December 13, 2010 12:34 PM

Subject: Re: Office of the Prime Minister / Cabinet du Premier ministre