L to R: The late Jose Valentin and friend, The Houses
As Kevin has already related, the weeks leading up to the 2001 AMDA Patient Conference were filled with turmoil and uncertainty.
I suppose the best place to start is, as always, at the beginning. The conference was the culmination of nearly a year of planning and organizing. By early September everything was planned and we were ready to go–eager to host the first ever Pompe patient meeting in the United States. It was going to be the first time that there was a conference for Pompe patients, and devoted only to Pompe disease—and from the responses we had gotten patients were planning to turn out en-mass.
Then, the tragedy of 9/11. As you will probably all remember, this was a time of turmoil and uncertainty in our country. BUT, despite statements made by Novazyme/Genzyme the AMDA never waivered in our decision to hold the conference. Kevin has already mentioned the statement posted to the GSDnet by Julie Smith of Genzyme on behalf of John Crowley (Novazyme) and Jan van Heek (Genzyme)—can you imagine our shock when we opened that email? We swiftly, and firmly, responded by saying that the conference was NOT cancelled and that we were still expecting a very good turnout from the scientific community. The exact message that was posted is as follows:
Thank you for your response. It was a complete shock to us to check our emails and find that the AMDA conference had been cancelled. We are flabbergasted that these two companies have taken it upon themselves to decide when and where a patient conference should be held.
We want to go ahead with the meeting. We have confirmations from keynote investigators that they will definitely present information to the patients at the meeting. These dedicated scientists/physicians are truly friends of the patients and have the patients' best interests at heart. The patients have a right to gather, exchange information, and to be informed about results from the ongoing trials. They also have the right to question the corporations about timelines and patient criteria for participation in new trials.
If we let Novazyme/Genzyme dictate all phases of what patients and patient organizations have a right to know and do, then we become putty in the hands of these "CORPORATE GIANTS".
We have all been affected by the bombings in the US, and we certainly understand anyone's apprehension with boarding an airplane. But if the corporations wanted to come--there are other means of travel available in the US. They can make other arrangements as are many of the patients. Many patients have told us that they want this meeting to take place and that they want to come to this meeting in spite of many obstacles; health, travel delays, etc. If patients have this much determination, then Novazyme/ Genzyme should be willing to come also to address this gathering.
Until Novazyme/Genzyme's announcement, we had 150 participants. As recently as Friday, both of these corporations had 20 people registered to attend.
Are there other motives for not wanting to attend?????
Novazyme and Genzyme stated that they were the sole sponsors of the AMDA Patient Conference. THIS IS NOT TRUE! AMDA has funded 2/3 of the conference. Genzyme has funded 1/3 of the conference. Novazyme has contributed nothing.
The following institutions were contacted today by AMDA and have vowed their support. They will make presentations at the conference.
Duke University-presentation on the CHO Trial
Rotterdam--presentation on the Transgenic Trial
Germany--presentation on the CHO trial and the Transgenic Trial
Presentation by the FDA--approval process for orphan drug products
Australia--presentation on diagnostics
UK--presentation on patient advocacy
Patient--presentation by a patient in a current clinical trial for Pompe's Disease
Kevin has spoken as to the possible motivations for “cancelling” our conference, but you would have to ask those who made the decision to know for sure. What I do know is, despite the lingering concerns caused by 9/11 the patients and scientific community demonstrated their strength and perseverance and showed up on the first day of the conference. My mom (Marylyn House) remembers talking to Dr. Reuser and thanking him for attending. He replied that his wife said he should come–it was too important not to. I share that anecdote because I think it is important to realize that it is not just the scientists, themselves, who are dedicated to our disease — it is their families, too.
That leads us to the conference itself. There were some really great presentations. What I remember most were the presentations by Duke (made by Dr. Amalfitano) and Erasmus (made by Dr. van der Ploeg). In particular, I remember the slides showing how the infants did on the different treatments (CHO vs. transgenic). What struck me the most about these slides was that both doctors presented slides that had graphs of the progress of the patients on therapy. The Duke slide showed three patients that showed an impressive improvement. However, after this initial improvement 2 of the 3 patients (or lines) sharply dropped off and then stabilized. As I recall, the decline was to baseline (or slightly below). In contrast, the slide from Erasmus showed steady improvement for all patients–albeit different degrees of improvement (one patient had a pretty drastic improvement while another had a very slight improvement).
You may think that I am biased as I was on the transgenic product–but I’m not. What I am talking about is interpreting a black and white graph of how the patients did. I am by no means trying to degrade the accomplishments of the Duke team–the babies were alive which they would not have been without treatment. All I am saying is that, to me, it was very apparent that patients on the transgenic enzyme seemed to do better.
I remember talking to Dr. Amalfitano after his presentation about my feelings. I can’t recall his exact words but they were along the lines of: “who are we to say that stabilization isn’t good.” He’s right. I firmly believe that in a disease like Pompe the first goal that needs to be reached is stabilization. Anything else is a bonus. But, at the time, I was having a hard time reconciling the graphs I saw with Genzyme’s decision to pursue the CHO method instead of the transgenic.
Now that I am older and wiser ;-) I understand that CHO was the quickest way to bring treatment to Pompe patients around the world. Personally, though, I wouldn’t be surprised if the transgenic product rears its head again one day in the future . . .