Dad's campaign to save daughter's life (From Basingstoke Gazette)

I can empathize with Mr. Massart and his family. MLD is a very cruel disease. We, the MLD Foundation, know hundreds of families like the Massarts that are having to watch their little ones suffer, slowly lose functions and slip away. Unfortunately, there is NO PROVEN DRUG TREATMENT for MLD as yet. The drug Metazym, now known as HGT-1111, has only undergone safety trials to date. This means it has been proven safe to give to humans BUT it has NOT BEEN PROVEN to do anything for MLD affected persons. Efficacy will be proven in the next phase of clinical trials. You can find the most recent information on this at http://mldfoundation
.org/research-shire.
html
We know first hand that a day is like a lifetime to a MLD affected person and time is running out not only for Sandra but for many of the children and adults affected with MLD. For now there is NO DRUG that can be bought at any price to help them. We are all hopeful that the next phase of clinical trials will show the HGT-1111 is effective in stopping the progression of MLD. I am so sorry that Mr. Massart has misconceptions about the drug (Metazym, HGT-1111). I know his heart is in the right place. He not unlike other MLD parents who would go to any length to save their child. We are all praying for a miracle as the research continues on HGT-1111.
The MLD Foundation is a US based non-profit. Our We C.A.R.E. mission is dedicated to facilitating Compassion for MLD affected families, increasing Awareness, influencing Research, and promoting Education for Metachromatic Leukodystrophy. For more information go to www.MLDfoundation.or
g

Teryn Suhr
Executive Director
MLD Foundation

I found it very strange that Teryn Suhr from an American patient foundation would find the time to comment on a story in a Basingstoke newspaper - until I was told that this foundation is sponsored by Shire. He will be aware that Metazym achieved orphan drug status over a year ago and is about to enter stage three trials in EU

Greetings Southampton and European colleagues,

The MLD Foundation is not sponsored by Shire. We were formed in 2001 to serve families around the world. We are actively involved with researchers and industry advocating on behalf of families and patients.

As to why we responded to the Basingstroke article ... we serve families throughout the world and feel that the information being printed and broadcast by the media is emotionally driven, woefully one sided, incomplete, and inaccurate. We want all families (and readers) worldwide to have accurate and complete information.

You may have misinterpreted overall sponsorship of the foundation with some specific event funding for our recent MLD Family Conference which was partially sponsored via an educational grant from Shire, along with funds from the participating families, the Stennis Foundation, Mac's Team, Boinx Software, and in the case of our Munich conference in March, the Myelin Project. We work closely with many other organizations but are not controlled by any of them. We are the world's largest family focused MLD specific organization. No one here at the MLD Foundation is paid a salary. The dollar does not drive us.

We have had numerous conversations with Shire advocating on behalf of families as recent as this last Sunday where were were suggesting changes in some aspects of the Phase II/III trial to better protect trial participants.

With all due respect to the Massart family, who we have communicated with and with whom we empathize as fellow parents of two MLD affected children, we could very well list dozens if not hundreds of equally worthy families to receive this enzyme if it was available. But as stated above, the enzyme is in early clinical trials and has not yet been proven effective nor are the manufacturing issues resolved to create "excess" supplies of the enzyme. We all hope this and other therapies will work but EMEA and FDA approvals are some time away. In addition, we have personally verified that there is not yet adequate supply of the enzyme to supply the upcoming trial participants - this supply could become a pacing item in ramping up the trial to benefit all patients is an expeditious a manner as possible.

And finally, it should be made clear that any commitments Mr. Massart had with Zymenex, the original developer developer of the enzyme, were with a start up company anxious for operational cash. Shire inherited the Phase I/II clinical trial, the enzyme, and the manufacturing challenges from the initial developer of the enzyme. HGT-1111 (formerly Metazyme) was purchased by Shire last April in a very public transaction - many months before Mr. Massart raised the funds he is currently waving about.

As you can tell we are intimate in the intricacies of this program because it affects hundreds of families with MLD. Please feel free to call or email with further questions. You can see full details about the status of the enzyme and the upcoming international Phase II/III trial at MLDfoundation.org

Best Regards,

Dean Suhr
President
MLD Foundation
www.MLDfoundation.or
g
+1 503-656-4808

I think you have forgot to mention that the drug was sold to Shire for 130 million !! So I think somethink is driven by the dollar

Of course something is driven by the dollar: two pharma companies. But what has this to do with the MLD Foundation? I know them personally, and I can assure you that they are driven by the most noble intentions. They are the ones connecting afflicted families worldwide, and if they receive modest funding from the industry for their conferences, then so be it. I also know personally a family who had their child in the early Metazym trials and can assure you that the "drug" indeed appears to be years away from the market- a fact that Shire HGT tries to get across ever since. Most of all, I am piqued by "newsdesk22" who enters into a cheap
p...ing contest because of a poorly researched news item. Why don't you let the Suhr´s and us families and the experts do their job, which is finding a way to stop this most terrible of all diseases. H.W., Germany

Shire issued a detailed public statement regarding HGT-1111 and the Massart situation. We at the MLD Foundation very strongly empathize with the Massart family ... and the hundreds of other families just like them seeking a cure for their MLD-ravaged children. You can see the Shire statement here:
http://mldfoundation
.org/pdfs/Shire_2009
-07-16.pdf

Dean Suhr
President
MLD Foundation
http://mldfoundation
.org