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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Saturday, 12 December 2009

A two-horse race

In September 1997 one of those twists took place. Duke University announced that YT Chen's group would also be conducting clinical trials of ERT, in collaboration with a company called Synpac Pharmaceuticals. They would be using the more 'traditional' method of producing the enzyme in Chinese hamster ovary (CHO) cells engineered to produce the human enzyme.

This just seemed incredible - not one but two pharmaceutical companies, competing to conduct a clinical trial and bring a product to market.

In a pretty strange and complicated saga, Synpac were one of the more mysterious participants. They were (probably still are) a Taiwanese company, very private, whose main line of business was the bulk production of generic pharmaceuticals like penicillin.  How they became involved with Pompe disease is a story that I suspect only YT Chen knows.

Anyhow, by the end of 1997 we had two teams racing to stage a clinical trial of ERT, something which seemed impossible just a few years before.

However, this was just a taste of what was to come...

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