The birth of the International Pompe Association

The birth of the IPA was an important event in the Pompe story. In fact, I think it was almost - but not quite - as important a development as ERT itself. Certainly the two stories are very intertwined. It represented the maturing of an international patient community into a representative organisation that could talk on something like equal terms with industry and scientists.

The roots of the IPA are as follows. I should say that my picture here is incomplete, for reasons that will become clear. Where I have gaps, I will follow my usual course of guessing (i.e. making stuff up) to fill them in. I will point out when I am doing that though.

I knew that Pompe groups were beginning to coalesce in different parts of the world. Helen Walker, Bet Cook and Bob Morrison were organising up a storm in Australia; Thomas Schaller and Gerd Hassler were bringing together the German patients within the glycogen storage disease group there. The UK and US have been mentioned. However there is a gaping hole in the canvass that I will now try to fill - The Netherlands.

The Dutch patients were the largest group of all and were organised within the neuro-muscular diseases charity, the VSN (Vereniging Spierziekten Nederland).  This was a very different group from the others. The VSN was a professional organisation, well-funded, with a critical mass that allowed it to sustain full time employees and a national head-quarters. They would bring those resources to bear on helping to organise the international Pompe community. This was, I think, a strategy formulated by the VSN head, Ysbrand Poortman, who had the far-sighted idea that the interaction of the Pompe patient community with scientists/doctors and industry could be a model for the development of treatments for other diseases.

The VSN were obviously closely involved with Pharming. This, in turn, brought them into contact with Randall and Marylyn House. The Houses took a close interest in Pharming, for obvious reasons, and may (I don't know; none of my business) have invested in the company. Randall, I think, viewed this fledgling company with a businessman's eye.

Ysbrand therefore suggested that, with the prospect of clinical trials, now was the time for an international patient organisation. I remember that Randall called me about this is obviously had some doubts. I think he had a concern that this would be a distraction and would there actually be a benefit? I was immediately struck by the thought that this was an idea whose time had come and hopefully my enthusiasm went some way to bringing the AMDA on board. The IPA would not have been the success it became without them; together we were all stronger.

The upshot of that was a letter from Ysbrand Poortman, inviting me to a meeting on 21 March, to discuss the organisation of an international Pompe conference.  The meeting was to be held after a meeting of the Dutch Pompe group, which I also attended. The international conference was original planned for October 1998 but was held over until 1999, both to accommodate some clinical trial results and to avoid a clash with a US conference being held at the National Institutes of Health, Bethesda, Maryland (of which more later).

I went along to the Dutch patient meeting.  I remember being impressed at the size of the meeting (though, of course, the actual proceedings were in Dutch). I met, for the first time, Maryze Schoneveld van der Linde, who also took part in the organising meeting and this was also the first time that I met Marylyn House. As you can imagine, both of them made a strong impression on me.  But more of that later.

The Rotterdam team were present, as were Pharming and their PR company. Actually, I've just remembered an amusing side track. Before we left for the IPA meeting, there was discussion where the Pharming PR man (Rob Meines?) invited the Houses to a lunch meeting the next day, with some politicians and Pharming senior staff. I was clearly not part of the plan, however Arnold Reuser interjected with "And of course, Kevin will be there too, yes?". They could hardly say no, so I ended up sitting in on a lunch at a rather grand hotel in The Hague (the Indies perhaps?), where I was the only person in the building without a tie. Arnold was making sure the AGSD-UK was in the loop and was at the same time entertained by 'the game'.

Anyways, back to the  founding IPA meeting. It took place at the Tulip Inn in Naarden. Present were Randall and Marylyn, Ysbrand Poortman, Helmut Erny, Maryze (and Anton), Miriam Bonink and Nelleke van den Berg (both VSN) and myself.  We agreed to some important actions:

• To contact individuals in different countries and encourage them to form their own Pompe groups
• To found an International Pompe Association at an international conference, meantime to be run by an Interim Executive Committee consisting of the above people (but with Ria Broekgaarden representing the VSN)
• To organise an international Pompe conference for October 1998 (later changed as above)
• To continue to meet via telephone conferences
  

And we were off. From now on the Pompe community was a force to be reckoned with.