Genzyme up their game - into 2001

What can we tell from the previous posts on the AGSD-UK 2000 conference?

Firstly, Genzyme confirmed that they had opted for the CHO method of production, rather than the rabbit method. As far as we could tell, this was simply because the rabbit method was not feasible. In hindsight, it's hard to argue with that and the fact that they committed to continue rabbit production for those already receiving it did help to soothe some nerves.

However, things were not so straightforward. Despite assurances that the two products would be identical, some doubts remained. The emerging findings from YT Chen's Synpac trial showed an important difference from the Rotterdam trial. Chen found that the initial effectiveness of the treatment was lost when the patient began to generate antibodies in response to the enzyme. The Rotterdam team had observed the same antibody response but had not found any difference in the effect of the treatment. This led to some uneasiness that a superior product was being ditched for commercial reasons, however compelling. 

On a more positive note, it was clear that Genzyme now recognised the importance of working with the patient community. On an international level this would be through the IPA.

So, we now had a known destination - a Genzyme treatment, produced by their 'standard' CHO cell method - and an undertaking from Genzyme to keep us informed of progress on getting there.  It was clear to us that they were committed to do so.

There were monthly telephone conferences with the IPA Executive. However, much of these discussions were 'in confidence' and we were keen to let the wider community know what was going on.

Genzyme therefore agreed to an interview with Paul Kaplan, who was in charge of the overall Pompe project. I canvassed for questions via GSDNet beforehand, to try to make sure that as many of people's concerns as possible were answered. The interview took place on 29 March 2001 and was reproduced in the Pompe's Bulletin, as well as GSDNet and the IPA website.

Reading it again for the first time in years, I am impressed with how open Genzyme were willing to be and how on top of the subject Paul Kaplan was. It was a telephone interview, so he might have had a team standing by to pass answers to him.  It doubt it though - he was just good at his job.  I'm also struck by how accurate his roadmap to availability turned out to be. He reckoned on approval in 2003. He wasn't too far out.

The next day, Genzyme/Pharming announced a multi-centre clinical trial, using the CHO enzyme.

We now knew exactly what was needed before ERT became widely available as a treatment - and why. We knew it wouldn't - couldn't - happen overnight. But we knew that we were very much on our way.