Dr J C Pompe

Dr J C Pompe
Discoverer of Pompe disease

About this blog

What you can read here is the story of the development of enzyme replacement therapy (ERT), the first effective treatment for Pompe disease. It is an incredible story, rich with events, characters and science. Above all, it is the story of an international community of scientists, doctors, patients and companies, working together towards a common goal.

It is not a story that features in Geeta Anand's book, The Cure , or the film based on it, Extraordinary Measures despite the fact that they are ostensibly about the development of ERT for Pompe ( you can link straight to the relevant articles covering the events described in the book and film here, here and here).

This blog represents my small attempt to set the record straight and to give the story back to its rightful owners - the international Pompe community. It is written here in roughly chronological order i.e. you'll need to start at the bottom of the April 2009 archive page and work your way up.

It is also a personal account and, although I've tried to make it as objective as possible, there is an inevitable degree of subjectivity. For that reason I have included contributions from other members of the worldwide Pompe community and would be delighted to receive more. Feedback is also welcome.

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Sunday, 3 January 2010

Novazyme visit

Gosh, I was excited by that Novazyme visit. All of my scepticism was blown away. I found them to be open, engaged and committed. Their approach was refreshingly patient-centred, compared to that of Genzyme. I came away from that visit on a real high - Novazyme had the right people and the right product! I remember saying to Randall House that I didn't understand why their product worked but that the evidence we were shown was so compelling that it obviously did.

Here's what I wrote on my return:

"The major impression I have of Novazyme is movement.Have you ever seen one
of those stop-motion films of a flower bursting into bloom? It was just like
that. It is a place where things are happening, not one at a time but in
parallel and at great speed. Everywhere, people are working away. Every
conceivable nook and cranny is put to use to store equipment or consumables
or has been commandeered for lab space. Here, a high grade facility - there,
an office - there again, walls are being knocked down and a new facility

I have never seen so many people all working on Pompe's disease. There were
more here, I think, in one place than were at the AMDA conference for
researchers in Bethesda, which pulled in people from all over the world.

Like Trae a few weeks back, Randall and I spoke at one of their regular
'Lunch and Learn' meetings, where they invite a speaker to talk about an
aspect of Pompe's. This struck me as a very good idea and illustrative of
the Novazyme approach and corporate culture. But I'm getting ahead of
myself - the detailed stuff can wait for the interview transcript.

As you know, some work has already been presented regarding Novazyme's
product. A question I had when I went over was how solid was the scientific
base for the company. This is dealt with in the interview but I'll give my
opinion here. From the evidence I saw, yes, they have something special -
astonishing, in fact. Obviously, what we saw was a company presentation and
not a peer reviewed paper, so caveat emptor, to an extent. However, I think
it is unlikely that the wool was being pulled over our eyes. Two reasons for

Firstly, they were incredibly open - to the extent of showing us information
on their costs (yes, we signed a non-disclosure agreement but all the same,
it's a gesture of trust).

Secondly, and most importantly: John Crowley. He's one of us, remember, and
his motivation is certainly not to make money.

So, interesting times ahead.

I hope that little taster will last you until the interview transcript:-)

Lastly, a few riders on all of the above:

Those are my own views - I'm not presuming to speak for Randall.

I'm making no judgements about which company has the best ERT product - just
noting that it is now very much a two horse race.

The transcript of the interview referred to was published on the IPA, AGSD-UK and AMDA websites and can be found in the AGSD-UK's Pompe's Bulletin (now a glossy full-colour production).

In retrospect this was astonishingly naive. Particularly so when the above is read in conjunction with The Cure. From that book we learn that what we were told about the intention to hold a clinical trial was false; John Crowley had already decided to sell Novazyme. In fact discussions had already taken place at Genzyme HQ. So what the heck was the point of it all? 

What makes me particularly uncomfortable is the thought that my enthusiastic report and the associated interview might have, in however small a way, been have responsible for nudging Genzyme into the disastrous decision to buy out Novazyme. Note the section below, in particular:

IPA: Gaucher disease? You’re really planning to compete with Genzyme’s big product?

Novazyme: the decision isn’t driven by a desire to go head to head with Genzyme. We believe that we have a product which will be an improvement on Cerezyme (the Genzyme ERT for Gaucher) and which may help patients with difficulties that product does not help.
In retrospect, it kind of looks like a signal doesn't it?

Hindsight is a great thing. Before we move on though, it is worth taking a look at the evidence presented to us that we found so convincing at the time.

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