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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Wednesday, 22 August 2012

The Duke "deception": what should be done?

There is no cause more dear to me than the advancement of Pompe research. It is heartbreaking, plain heartbreaking, to see that cause tarnished in the way that it has been by the Duke paper. Science is about truth and evidence. There is very little room for short cuts and certainly none for spin.

The 2001 Duke paper in Genetics in Medicine stands exposed as containing false information. It is now clear that this was a deliberate falsehood, for which an inadequate explanation has been given.

Worse, that deliberate falsehood was used in the paper to argue against the work of another Pompe researchers, who used enzyme manufactured using a different method.

There is an established course of action in such cases: the paper must be withdrawn. It would be best if the authors, recognising the enormity of their error, asked for this to be done themselves. Failing that, the editor of Genetics in Medicine should do so to protect the integrity of the journal.

However, that is not the end of the matter. As we know, different companies were involved at this stage and the production method of alpha-glucosidase was itself a contentious subject. It is no exaggeration to say that fortunes were made or lost on that decision, on which the Duke paper may have had a bearing.

My questions now are:

Was anyone at Genzyme aware of the incorrect data reported in the Duke paper?

Was anyone within the senior management at Duke University aware that data was being misreported in this way?

Time to get digging, I guess!

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