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, 31 January 2010

Maryze's story - Part 4

In 1998, Pharming and Genzyme started to work together in a joint venture.

In 2000 Pharming started with the building of the factory in Geel, Belgium. This plant later was bought by Genzyme and now Myozyme in the 4000 Liter bioreactor is being produced. So in the end our treatment was being produced there after all.

However who would think that everyone was happy with these developments, is mistaken. The animal welfare organisations were criticizing the use of animals for medicines. For Pompe patients in the Netherlands it was quite shocking to hear this. For us the development of the production of ERT in rabbits was the only chance we got and then animal welfare organisations and animal right activists were opposed to it? Did they really prefer the life of rabbits over the life of human babies?

Greenpeace too was opposed to this development. I knew they were opposed to biotechnology anyway as their billboard posters showing their opposition of this technology had hung everyone in Leiden. Although at that time Pompe disease, wasn’t in the picture yet. At a debate on animals and biotechnology in the Dutch parliament, my father and I too were present. I just wanted to show everyone that I was serious and would fight to secure treatment for Pompe patients.

During the break of the debate, a lady came up to me and my father while we were having coffee. She introduced herself and said she was working for Greenpeace. I was honestly a bit shocked and thought by myself well here it is, now I need to use all my arguments to make clear why I am in favour of human babies instead of rabbits. Then however the lady of Greenpeace continued saying: ‘Greenpeace still is opposed to medical treatments produced in animals, but when it comes to treatments for diseases for which no other treatment yet exists and for which animals are the only solution, we will make an exception. Then we will not oppose it. I just want you to know about this’. I was relieved. I didn’t need to go into discussion with Greenpeace. They too cared for human babies who otherwise would die. The fight however wasn’t over yet.

On a normal day I was sitting behind my computer I suddenly heard a commercial on the radio. A voice was telling about a little boy who did love rabbits, but these rabbits were misused to get a treatment for Pompe disease, while another production method without animals also could be done. Listeners who also were opposed to this misuse of animals could donate money to the foundation against animal lab testing (Proefdiervrij). When I heard this I was shocked. What other production method was available? At that moment the transgenic rabbits were the only ones and 7 people were successfully treated with this. How could they say such a thing?

Animal rights lies. The Pompe community fights for the truth - and not for the last time.

I immediately called the VSN. They too heard the commercial that meanwhile had been repeated several times on several radio stations already. This foundation also had a big advertisement in one of the national newspapers in the Netherlands, giving the same statement. In that advertisement a boy of about 6 years old was shown among rabbits hopping around in his room. The advertisement text stated this boy too had Pompe disease, but was opposed to using rabbits. Even a non medical professional with knowledge on Pompe disease could see in a moment, this boy absolutely didn’t have Pompe disease. Pompe patients in the Netherlands were upset by this attack and the VSN decided to appeal at the regulatory authority to forbid this type of incorrect advertisement over the backs of Pompe patients. The director of the VSN, Marcel Timmen, and I went together and we won the case by far. The commercial and the advertisements were forbidden per direct.

1 comment:

  1. Thank you Maryze for your very interesting contribution, and for your significant actions as a very compromised activist in the fight against Pompe disease. A few more people like you and Pompe disease would be now just a bad dream of the past.

    Of all the contributions Kevin has posted here, this has been by far the most shocking to me, specially when it comes to the unpitiful actions of the animal rights group Proefdiervrij against Pompe patients. You must have gone through very hard times, suffering the injustice and the lack of empathy of such people, who have certainly demonstrated a much lower degree of humanity than the animals they claim to defend.

    Your experiences with Proefdiervrij and with Greenpeace are so amazing, so unbelievable and so extreme that it makes me to believe that something is really not working in modern societies. Our societies are rich and developed but are sick in many aspects and some social groups behave themselves on the verge of social insanity.

    Groups like Proefdiervrij nourish themselves with people who regard themselves as idealistic contributors to some superior goal. What lies behind their actions is not really idealistic, as it is clear to me that through their involvement they are just looking for social recognition, self-complacency and notoriety. However, what really worries me is that their ideology is really perverse at the bottom and is driven by a true eugenic mentality.

    Such people do not want to deal with real problems. They prefer to devote themselves to remote problems; problems which are in many aspects invented, problems they do not have to deal with face to face; problems they can turn off when they need to disconnect; problems they can forget about when they go to bed. They do not want to know anything about sickness, not to mention about children with terminal illnesses or people with extreme disabilities. In their world there is no room for them; there is only room for healthy kids playing idyllically with healthy rabbits. At the end of the day, they are not so different from the very same people who executed JC Pompe.

    As a father of a child very severely affected by Pompe disease I just not regard their commercials in the radio and the newspapers merely as an offense. This was a premeditated attempt to confuse the public opinion against Pompe patients. They were just telling lies with the only aim of harming very sick people on behalf of a group of rodents. Rats defending rabbits. A true lobby. For me is not an offense: is a genuine felony.

    And in relation to Greenpeace I can only say that even if they are not as extremists as Proefdiervrij, I cannot help detesting their sense of moral superiority. Sitting next to you to communicate you: Maryze, we the superior healthy people, who care about the right things, want to tell you that, after some deliberations, we have decided that we are going to spare your life... I know that you are a very educated person and I am certain that you had the right reaction, but I do not know what I would have answered in your place...