2003 signaled the end of one era and the beginning of another. From now on, news of friends receiving ERT would begin to come thick and fast - every one of them a cause for rejoicing.
It would take until 29 March 2006 (Europe) and 28 April 2006 (USA) before the formal approval of Myozyme as a treatment. However even that was not the end of the road - there are still issues around funding and, above all, ERT is a treatment, not a cure.
The fight goes on, carried out by an international community of patients, scientists, doctors and companies. I hope that others will contribute the rest of the story here.
I myself 'retired' from the Pompe scene at the end of 2003. It had been 10 years since Calum died and my dream of seeing an effective treatment for Pompe disease had come true. We wanted to remember our son for his short, but wonderful, time with us and not for his disease. There were other people better suited than I to do my remaining jobs. It was time to move on.
Saturday, 6 February 2010
Heidelberg
I go to many conferences as part of my work as well as personal activities and I can honestly say that the 2003 IPA conference remains the best I have ever been to. It was held on 31 October- 2 November, in Heidelberg, Germany. Everything was superb: the location, the presentations, the cameradie, the beer...
The conference website and follow-up (including proceedings) are still available to read for yourself.
There was also a Q and A session released on the website and GSDNet.
That the conference was such a roaring success is in large part down to the organisers, Thomas Schaller, Birgit Wolf, Rita & Helmut Erny from the German patient group.
However one other reason is that, here, for the first time, much of the talk was about success. Successful trials, successful enzyme production and the prospect of many more people being treated in the coming year. We had come a long way.
There was still a long way to go, of course, but as Marilyn House put it at the time " everyone came away with new sense of enthusiasm and cooperation for the treatment of Pompe's disease."
The conference website and follow-up (including proceedings) are still available to read for yourself.
There was also a Q and A session released on the website and GSDNet.
That the conference was such a roaring success is in large part down to the organisers, Thomas Schaller, Birgit Wolf, Rita & Helmut Erny from the German patient group.
Conference participants
However one other reason is that, here, for the first time, much of the talk was about success. Successful trials, successful enzyme production and the prospect of many more people being treated in the coming year. We had come a long way.
There was still a long way to go, of course, but as Marilyn House put it at the time " everyone came away with new sense of enthusiasm and cooperation for the treatment of Pompe's disease."
2003
Before we get into the 2003 big picture, can I just step aside for a moment and mention something of huge significance for me. This was the year that, for the very first time, there was a UK clinical trial of ERT. This was at The Willink Centre in Manchester, under Dr Ed Wraith and co-workers. This was a dream come true and added to a growing feeling that my own part in the story, such as it was, was coming to a logical end.
2003 was again a year of solid progress. In retrospect, we had moved pretty clearly to a situation where we knew that ERT would be available. For many people, this was even more difficult than wondering if it would be available. It was now a waiting game, not a hoping game. In particular, many adult patients were wondering when it would be their turn to feature in a trial.
This led to occasional tensions. The IPA were in regular contact with Genzyme, yet much of this was under strict terms of confidentiality. This meant that information could not be passed on to the patients, who were - understandably - restive.
Nevertheless, a series of regular updates held things together.
Issue 10 of the Pompe's Bulletin came out in February, bringing people up to date on a whole range of things, including the Manchester trial.
There were regular Pompe Program updates (contents agreed between the IPA and Genzyme) of progress.
These were released on February 18, March 19 (Genzyme press release), September 10 (press release)
The latter brought the first news of adult onset trials and of 'Special Access' for patients who did not fit into clinical trials but who were in demonstrable need. This was a mark of Genzyme's success in ramping up production - they could not have made such promises without absolute confidence in supplies of enzyme. From now on, alpha-glucosidase would be much more widely available, in advance of formal commercialisation.
As you can imagine, having been aware of developments through regular contact with Genzyme, it was a great feeling of relief to have them out in the public domain. It was recognised though that something bigger and public was needed to signal progress to patients. That something was the biggest and best Pompe event so far - the 2003 IPA Conference at Heidelberg in Germany.
2003 was again a year of solid progress. In retrospect, we had moved pretty clearly to a situation where we knew that ERT would be available. For many people, this was even more difficult than wondering if it would be available. It was now a waiting game, not a hoping game. In particular, many adult patients were wondering when it would be their turn to feature in a trial.
This led to occasional tensions. The IPA were in regular contact with Genzyme, yet much of this was under strict terms of confidentiality. This meant that information could not be passed on to the patients, who were - understandably - restive.
Nevertheless, a series of regular updates held things together.
Issue 10 of the Pompe's Bulletin came out in February, bringing people up to date on a whole range of things, including the Manchester trial.
There were regular Pompe Program updates (contents agreed between the IPA and Genzyme) of progress.
These were released on February 18, March 19 (Genzyme press release), September 10 (press release)
The latter brought the first news of adult onset trials and of 'Special Access' for patients who did not fit into clinical trials but who were in demonstrable need. This was a mark of Genzyme's success in ramping up production - they could not have made such promises without absolute confidence in supplies of enzyme. From now on, alpha-glucosidase would be much more widely available, in advance of formal commercialisation.
As you can imagine, having been aware of developments through regular contact with Genzyme, it was a great feeling of relief to have them out in the public domain. It was recognised though that something bigger and public was needed to signal progress to patients. That something was the biggest and best Pompe event so far - the 2003 IPA Conference at Heidelberg in Germany.
Sunday, 31 January 2010
Maryze's story - Part 8
On March the 29th Myozyme was officially approved in Europe. That special day for Pompe patients, I was at the funeral of a Pompe patient I knew very well. It was she who called me that day on November the 14th 1996, to ask if I had heard the news and what I thought of it. It was sad that she never could benefit from the treatment she too had been waiting for so long. Shortly after the FDA approved Myozyme as well. Slowly Pompe patients all over the world did start treatment. I know of one boy in Germany who was so affected that he didn’t meet the criteria for participation in the clinical trial in Rotterdam, but he was able to start commercial treatment with Myozyme just 2 weeks after the official approval.
In spite of the approval the clinical trials on Myozyme in adults continued. The regulatory authorities had requested additional data that showed also efficacy of Myozyme in older Pompe patients. I personally admire these people who did continue going to Rotterdam and other trial sites in France and the USA to get their infusions not knowing if they received alpha-glucosidase or placebo, to do all the required tests and go through all the emotional feelings, even when they knew Myozyme was approved for the market already.
Over the years from 1996 till now, I collected a lot of documents, newspaper articles, publications etc on Pompe disease and related issues from all over the world. These 6 large files contain a lot of information on events and people not mentioned in the book ‘The Cure’. This book is a story of John Crowley and his family, but it's not the story of so many other Pompe patients in the world.
A cake to celebrate the approval of Myozyme.
In spite of the approval the clinical trials on Myozyme in adults continued. The regulatory authorities had requested additional data that showed also efficacy of Myozyme in older Pompe patients. I personally admire these people who did continue going to Rotterdam and other trial sites in France and the USA to get their infusions not knowing if they received alpha-glucosidase or placebo, to do all the required tests and go through all the emotional feelings, even when they knew Myozyme was approved for the market already.
Over the years from 1996 till now, I collected a lot of documents, newspaper articles, publications etc on Pompe disease and related issues from all over the world. These 6 large files contain a lot of information on events and people not mentioned in the book ‘The Cure’. This book is a story of John Crowley and his family, but it's not the story of so many other Pompe patients in the world.
Maryze's story - Part 7
In December 2005, Genzyme invited me to join them and Dr. Ans van der Ploeg to the official hearing at the European Medicine Agency (EMEA) in London. Genzyme had received permission from the EMEA to bring a patient to the official hearing to give her personal account. I immediately said ‘yes’, because it was important for all Pompe patients in Europe and beyond to get approval for the enzyme replacement therapy. At December 13 2005, it was the day of truth and it was one of the most exciting days in my life. We were all nervous, even after such a thorough preparation. Genzyme and Dr. Ans van der Ploeg gave an excellent and clear presentation on the effect of ERT. After their presentation it was my turn.
I had 5 minutes to tell about my personal experience with enzyme replacement therapy. For me these 5 minutes was worse than doing 3 school exams. I felt as if all the European patients were sitting on my shoulder, depending on my performance and ability to explain the impact of ERT on my life. All the experts in the room were silent and listened carefully. I was able to tell them the story behind the data Genzyme and my physician presented. This mixture of hard scientific data and a personal account was good and clear. After the official hearing we received a debriefing of the French and Belgium representatives who were leading this hearing. The French representative told us that apart from the data, my personal story was what they wanted to hear. Sometimes hard data can’t tell what one story can explain…the real impact of a treatment on a life.
On January the 27th 2006 we were told in a press release that Myozyme™ Receives Positive Opinion from European Regulatory Committee (London, 27 January 2006, Doc. Ref. EMEA/32796/2006). This meant that the regulatory committee would advise the European commission to approve Myozyme for Pompe disease. Just before this press release I was called by Genzyme by someone I know very well. He just asked me: ‘Do you have Champagne?’ Tears ran below my cheeks, as I felt so relieved that now all Pompe patients in Europe could start treatment and I knew other countries would follow Europe. We were both emotional and happy. It was as if we won a battle together and survived. One day after this press release, on January the 28th 2006, the VSN had organised a Pompe patient meeting. It couldn’t have be timed better and in a movie one wouldn’t belief it as it would be too good to be true. At that meeting Pompe patients from all over the Netherlands gathered and prepared to hear another delay in treatment as we had been hearing for so many years already. Someone of Genzyme did tell everyone about the press release, that most of the people didn’t know about yet as it was so fresh, but the message wasn’t understood. Then my mother decided to tell everyone this good news in a symbolic way.
She left the room and came back with a bottle of Champagne and some glasses. She entered the stage and called several people to get a glass of Champagne. Dr. Arnold Reuser, Dr. Ans van der Ploeg, Willem van Weperen (Genzyme), Gezinus Wolters, my father, and I. We all represented those who were so closely involved in the process of getting a treatment: scientists, physicians, industry, patient organisation, patients, parents and partners.
When the Champagne was poured in the glasses, everyone started slowly to understand. It took a while as no one really could belief it. After all those years of waiting since 1996, there finally a treatment would be available. This was the moment everyone was waiting for so long. Some people got emotional and hardly could express their feelings. Later I received an email from a patient who thanked me as she couldn’t say it personally, because she was afraid to cry. In the afternoon of Monday the 30th of January 2006, our doorbell rang. I was surprised to see a fellow Pompe patient and his wife at my door. They live not very far from me and decided to give me a bouquet of flowers for the work that I had done. I was deeply touched, because it was for people like him that I hoped the treatment would help. From now on when a new Pompe patient would be diagnosed, they would be told: ‘I am sorry to tell you that you have Pompe disease, but there is a treatment available’.
I had 5 minutes to tell about my personal experience with enzyme replacement therapy. For me these 5 minutes was worse than doing 3 school exams. I felt as if all the European patients were sitting on my shoulder, depending on my performance and ability to explain the impact of ERT on my life. All the experts in the room were silent and listened carefully. I was able to tell them the story behind the data Genzyme and my physician presented. This mixture of hard scientific data and a personal account was good and clear. After the official hearing we received a debriefing of the French and Belgium representatives who were leading this hearing. The French representative told us that apart from the data, my personal story was what they wanted to hear. Sometimes hard data can’t tell what one story can explain…the real impact of a treatment on a life.
On January the 27th 2006 we were told in a press release that Myozyme™ Receives Positive Opinion from European Regulatory Committee (London, 27 January 2006, Doc. Ref. EMEA/32796/2006). This meant that the regulatory committee would advise the European commission to approve Myozyme for Pompe disease. Just before this press release I was called by Genzyme by someone I know very well. He just asked me: ‘Do you have Champagne?’ Tears ran below my cheeks, as I felt so relieved that now all Pompe patients in Europe could start treatment and I knew other countries would follow Europe. We were both emotional and happy. It was as if we won a battle together and survived. One day after this press release, on January the 28th 2006, the VSN had organised a Pompe patient meeting. It couldn’t have be timed better and in a movie one wouldn’t belief it as it would be too good to be true. At that meeting Pompe patients from all over the Netherlands gathered and prepared to hear another delay in treatment as we had been hearing for so many years already. Someone of Genzyme did tell everyone about the press release, that most of the people didn’t know about yet as it was so fresh, but the message wasn’t understood. Then my mother decided to tell everyone this good news in a symbolic way.
She left the room and came back with a bottle of Champagne and some glasses. She entered the stage and called several people to get a glass of Champagne. Dr. Arnold Reuser, Dr. Ans van der Ploeg, Willem van Weperen (Genzyme), Gezinus Wolters, my father, and I. We all represented those who were so closely involved in the process of getting a treatment: scientists, physicians, industry, patient organisation, patients, parents and partners.
My mother, Tanneke van der Linde, and Dr Arnold Reuser opening a bottle of champagne.
Maryze's story - Part 6
In April 2002, I met John Crowley, former CEO of Novazyme and at that time working for Genzyme at a meeting. I also knew he had two children with Pompe disease and both were severely affected. John and my mother sat next to eachother and talked about their children: Megan, Patrick and me. I saw two parents who were both very worried about their children. They cried together. This is how I remember John, crying with my mother as they both saw their children deteriorating and didn’t know if treatment would come in time.
Both his children, Megan and Patrick and I finally received our treatment on time. The opportunity to receive treatment was a gift of life. I told my physician, Dr. Ans van der Ploeg that I wanted to contribute to the knowledge of enzyme replacement therapy like the other patients were doing in the clinical trial. A muscle biopsy was taken and all neurological, lung and blood tests were done. These outcomes were also used in the data gathering to get approval for enzyme replacement therapy at the regulatory authorities. It was good to see that also other patients were able to start treatment. It was a time in which Anton and I regularly drank German Sekt to celebrate the start of treatment from a friend somewhere in the world.
Through the contacts with patients world wide, the IPA learned that it was important to inform Pompe patients about the issues involved in Pompe disease such as breathing problems, common health problems, exercises and physical therapy, pregnancy, genetics, medical developments etc. We felt that people should know how to stay in a as good physical condition as possible. Especially regarding the breathing problems that can occur we felt Pompe patients should know how to treat it and what to do and especially what not to do, like for example using oxygen or using a C-pap. To inform as many Pompe patients as possible, The Pompe Connections were written and reviewed by medical experts from several countries. Later it was translated in several languages such as Japanese, Turkish, German, Spanish, French and Dutch.
Later I heard that people around the world were very happy with this information, especially when it was in their own language. It was a tremendous task, but absolutely worth all the energy and effort.
In November 2003, the International Pompe Association organised an international conference in Heidelberg, Germany. Scientists, physicians, patients and industry were present and shared the latest information on research. It was at that conference a mystery was solved. The mystery of the ‘Holy Grail’, the treatment Novazyme had been working on, but that suddenly disappeared. This statement of ‘Holy Grail’ is not mine, but Novazyme used this terminology in one of their press releases.
In answer to a question, Dr. William Canfield told everyone at the conference that during a test with muscle cells stored with glycogen in a Petri dish where they added the ERT of Novazyme, a huge mistake was made. At first it was claimed that the Novazyme treatment was working so well that in just a couple of minutes the glycogen in these muscle cells was decreased to almost zero. Based partly on this result the Pharming ERT was wiped from the Pompe development program, as according to the results of that famous test it was the most inefficient treatment. Later it was admitted that the Pharming ERT should have been tested in vivo (experimentation using a whole, living organism as opposed to a partial or dead organism, or an in vitro controlled environment) as it would have been more fair and probably would have revealed that it was more effective in vivo than in vitro (experimentation done in live isolated cells). Genzyme was presented the great results of Novazyme and convinced that the treatment Novazyme was developing was indeed very promising.
Genzyme bought Novazyme in 2001, the company of John Crowley, for millions of dollars. John Crowley was offered the position of senior vice president at Genzyme Therapeutics after the sale. The take over from Novazyme by Genzyme was investigated thoroughly by the Federal Trade Commission. While this investigation was still going on, it became clear that the Novazyme treatment wasn’t promising at all, but that its results were based of a mistake. While doing the test, the in vitro muscle cells weren’t fixed, so when the Novazyme treatment was added, the glycogen simply was washed away. In such a situation even plain water would have been able to remove the glycogen from muscle cells.
Both his children, Megan and Patrick and I finally received our treatment on time. The opportunity to receive treatment was a gift of life. I told my physician, Dr. Ans van der Ploeg that I wanted to contribute to the knowledge of enzyme replacement therapy like the other patients were doing in the clinical trial. A muscle biopsy was taken and all neurological, lung and blood tests were done. These outcomes were also used in the data gathering to get approval for enzyme replacement therapy at the regulatory authorities. It was good to see that also other patients were able to start treatment. It was a time in which Anton and I regularly drank German Sekt to celebrate the start of treatment from a friend somewhere in the world.
Through the contacts with patients world wide, the IPA learned that it was important to inform Pompe patients about the issues involved in Pompe disease such as breathing problems, common health problems, exercises and physical therapy, pregnancy, genetics, medical developments etc. We felt that people should know how to stay in a as good physical condition as possible. Especially regarding the breathing problems that can occur we felt Pompe patients should know how to treat it and what to do and especially what not to do, like for example using oxygen or using a C-pap. To inform as many Pompe patients as possible, The Pompe Connections were written and reviewed by medical experts from several countries. Later it was translated in several languages such as Japanese, Turkish, German, Spanish, French and Dutch.
In November 2003, the International Pompe Association organised an international conference in Heidelberg, Germany. Scientists, physicians, patients and industry were present and shared the latest information on research. It was at that conference a mystery was solved. The mystery of the ‘Holy Grail’, the treatment Novazyme had been working on, but that suddenly disappeared. This statement of ‘Holy Grail’ is not mine, but Novazyme used this terminology in one of their press releases.
In answer to a question, Dr. William Canfield told everyone at the conference that during a test with muscle cells stored with glycogen in a Petri dish where they added the ERT of Novazyme, a huge mistake was made. At first it was claimed that the Novazyme treatment was working so well that in just a couple of minutes the glycogen in these muscle cells was decreased to almost zero. Based partly on this result the Pharming ERT was wiped from the Pompe development program, as according to the results of that famous test it was the most inefficient treatment. Later it was admitted that the Pharming ERT should have been tested in vivo (experimentation using a whole, living organism as opposed to a partial or dead organism, or an in vitro controlled environment) as it would have been more fair and probably would have revealed that it was more effective in vivo than in vitro (experimentation done in live isolated cells). Genzyme was presented the great results of Novazyme and convinced that the treatment Novazyme was developing was indeed very promising.
Novazyme's mistake
Genzyme bought Novazyme in 2001, the company of John Crowley, for millions of dollars. John Crowley was offered the position of senior vice president at Genzyme Therapeutics after the sale. The take over from Novazyme by Genzyme was investigated thoroughly by the Federal Trade Commission. While this investigation was still going on, it became clear that the Novazyme treatment wasn’t promising at all, but that its results were based of a mistake. While doing the test, the in vitro muscle cells weren’t fixed, so when the Novazyme treatment was added, the glycogen simply was washed away. In such a situation even plain water would have been able to remove the glycogen from muscle cells.
Maryze's story - Part 5
Meanwhile in 1999 the International Pompe Association was founded. One year before, in 1998 at the annual patient meeting of the VSN in the Netherlands, also some parents from other countries such as Germany, UK, USA and the Netherlands, were present. After the annual meeting we came together in a separate meeting and it was discussed how we could work together on behalf of all Pompe patients in the world. Also for those who didn’t have a strong patient organisation in their country. It was at that particular meeting the name International Pompe Association was mentioned. One year later the IPA was officially established and registered. The first board members of IPA were: Kevin O’ Donnell (UK), Ria Broekgaarden (the Netherlands), Bob Morrisson (Australia), Randall House (USA), Thomas Schaller (Germany) and Maryze Schoneveld van der Linde (the Netherlands).
In April 2000, at Easter, Anton and I went on holiday in Andalusia, Spain. I always wanted to see the beautiful Moorish architecture of the Alhambra near Granada and the Mesquita in Cordoba. When we returned home, my parents told us that Genzyme would stop the development of human alpha-glucosidasis in rabbits. For us this news was a complete shock as the Lancet had just published a convincing article that showed that enzyme replacement therapy was safe and effective in children with infantile onset Pompe disease (Van den Hout H, Reuser AJ, Vulto AG, Loonen MC, Cromme-Dijkhuis A, Van der Ploeg AT. Recombinant human α-glucosidase from rabbit milk in Pompe patients. Lancet 2000; 356:397-398). Many Dutch Pompe patients were emotional about this decision. The dutch company Pharming that we got to know so well from the beginning and that had proved to have a treatment that was effective, was now put aside without getting a real treatment in return. The treatment that would be further developed had still to be proven effective at that time
On September 28, 2001 Genzyme completed the take over of Oklahoma City-based Novazyme.
On October 30, 2001 Genzyme acquired the manufacturing facility in Geel, Belgium. In a pressrelease the following was said: ‘CAMBRIDGE, Mass.-Genzyme Corp. announced today that it has acquired certain assets of Pharming N.V., the Belgian subsidiary of the Pharming Group currently operating under a court-supervised receivership. These assets include a 70,000 square foot cGMP protein manufacturing facility currently under construction and a pilot plant that is currently used to produce transgenic human alpha-Glucosidase, both located in Geel, Belgium. The acquisition has been approved by the Commercial Court in Turnhout, the Province of Antwerp, and Genzyme's board of directors. In the near term, the acquisition of the Geel facility is intended to allow Genzyme to assume control over the production of the transgenic enzyme and secure its supply to nine patients with Pompe disease participating in the extension of a clinical trial. Genzyme has been solely funding the production of the enzyme since Pharming Group sought receivership’.
When we heard about the financial problems of Pharming. It was a shock to all of us. Who would have ever thought that the company that was developing and producing our treatment was not able to manage and continue the production? It was a real nightmare and it made clear that even when a treatment is successful, the race isn’t finalised. Patients always will be in a vulnerable position as they always will be dependent on markets, CEOs, politics, researchers, physicians, governments etc.
When Pharming had financial problems it became a difficult period for patients, physicians and the employees of Pharming. At a certain moment there wasn’t even money to pay salaries of those people who were producing the ERT in Geel, Belgium. Most of these people were young, just starting their carreer. I really was impressed with their persistence and loyalty to continue working for those 9 Pompe patients in the Netherlands and Germany who were depended on their ERT. They even continued working when they didn’t know if they would receive a salary for it. The problems of Pharming and especially the threads of the financial problems for the Pompe patients were discussed in the news.
When Genzyme finally took over the Pompe program from Pharming. The employees in Geel were taken over as well. Most of these employees stayed at Genzyme to continue producing the ERT with the genetically modified rabbits. Many of them still work there and are closely involved in the Myozyme production in the 4000 Liter bioreactors in Geel.
In April 2000, at Easter, Anton and I went on holiday in Andalusia, Spain. I always wanted to see the beautiful Moorish architecture of the Alhambra near Granada and the Mesquita in Cordoba. When we returned home, my parents told us that Genzyme would stop the development of human alpha-glucosidasis in rabbits. For us this news was a complete shock as the Lancet had just published a convincing article that showed that enzyme replacement therapy was safe and effective in children with infantile onset Pompe disease (Van den Hout H, Reuser AJ, Vulto AG, Loonen MC, Cromme-Dijkhuis A, Van der Ploeg AT. Recombinant human α-glucosidase from rabbit milk in Pompe patients. Lancet 2000; 356:397-398). Many Dutch Pompe patients were emotional about this decision. The dutch company Pharming that we got to know so well from the beginning and that had proved to have a treatment that was effective, was now put aside without getting a real treatment in return. The treatment that would be further developed had still to be proven effective at that time
The decision to stop production of the rabbit enzyme received extensive coverage in the Dutch press
On October 30, 2001 Genzyme acquired the manufacturing facility in Geel, Belgium. In a pressrelease the following was said: ‘CAMBRIDGE, Mass.-Genzyme Corp. announced today that it has acquired certain assets of Pharming N.V., the Belgian subsidiary of the Pharming Group currently operating under a court-supervised receivership. These assets include a 70,000 square foot cGMP protein manufacturing facility currently under construction and a pilot plant that is currently used to produce transgenic human alpha-Glucosidase, both located in Geel, Belgium. The acquisition has been approved by the Commercial Court in Turnhout, the Province of Antwerp, and Genzyme's board of directors. In the near term, the acquisition of the Geel facility is intended to allow Genzyme to assume control over the production of the transgenic enzyme and secure its supply to nine patients with Pompe disease participating in the extension of a clinical trial. Genzyme has been solely funding the production of the enzyme since Pharming Group sought receivership’.
When we heard about the financial problems of Pharming. It was a shock to all of us. Who would have ever thought that the company that was developing and producing our treatment was not able to manage and continue the production? It was a real nightmare and it made clear that even when a treatment is successful, the race isn’t finalised. Patients always will be in a vulnerable position as they always will be dependent on markets, CEOs, politics, researchers, physicians, governments etc.
When Pharming had financial problems it became a difficult period for patients, physicians and the employees of Pharming. At a certain moment there wasn’t even money to pay salaries of those people who were producing the ERT in Geel, Belgium. Most of these people were young, just starting their carreer. I really was impressed with their persistence and loyalty to continue working for those 9 Pompe patients in the Netherlands and Germany who were depended on their ERT. They even continued working when they didn’t know if they would receive a salary for it. The problems of Pharming and especially the threads of the financial problems for the Pompe patients were discussed in the news.
When Genzyme finally took over the Pompe program from Pharming. The employees in Geel were taken over as well. Most of these employees stayed at Genzyme to continue producing the ERT with the genetically modified rabbits. Many of them still work there and are closely involved in the Myozyme production in the 4000 Liter bioreactors in Geel.
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?
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.
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.
Maryze's story - Part 3
In February 1997 my brother, Onard, returned from his 6 month exchange program at Trinity
College in Dublin, Ireland. To hear the latest developments in Pompe disease,
he came with us to the patient meeting of VSN. At that meeting I introduced him
to Dr. Arnold Reuser. They talked and a little while later Onard applied for a
training opportunity at the Department of Clinical Genetics of the Erasmus
Medical Center in Rotterdam. He worked there from March 1997 till March 1998
and participated actively in the research in Pompe knock out mice and the
effect of enzyme replacement therapy derived from genetic modified rabbits.
He was not allowed to tell us anything, but my father always asked him if the sun
was shining in Rotterdam or if it was cloudy. Often the answer was that it was
shining. Later we heard from Dr. Arnold Reuser that it was sometimes tough, as
some mice got into an anaphylactic shock during the treatment with
alpha-glucosidase. This was a serious adverse effect, and then it is even more
serious when it concerns a disease your sister is suffering from. Luckily these
adverse effects could be managed well (Human Molecular Genetics, 1999, Vol. 8,
No. 12 2145-2153).
College in Dublin, Ireland. To hear the latest developments in Pompe disease,
he came with us to the patient meeting of VSN. At that meeting I introduced him
to Dr. Arnold Reuser. They talked and a little while later Onard applied for a
training opportunity at the Department of Clinical Genetics of the Erasmus
Medical Center in Rotterdam. He worked there from March 1997 till March 1998
and participated actively in the research in Pompe knock out mice and the
effect of enzyme replacement therapy derived from genetic modified rabbits.
He was not allowed to tell us anything, but my father always asked him if the sun
was shining in Rotterdam or if it was cloudy. Often the answer was that it was
shining. Later we heard from Dr. Arnold Reuser that it was sometimes tough, as
some mice got into an anaphylactic shock during the treatment with
alpha-glucosidase. This was a serious adverse effect, and then it is even more
serious when it concerns a disease your sister is suffering from. Luckily these
adverse effects could be managed well (Human Molecular Genetics, 1999, Vol. 8,
No. 12 2145-2153).
Thesis of Dr Anges Bijvoet, with a contribution from my brother, Onard Schoneveld.
Maryze's story - Part 2
In the morning of the 14th of November 1996 I was still in bed and felt not that well. My parents were at work and Anton too was busy with a project. I was thinking about what to do, while at the same time I couldn’t do much physical activity as my body had deteriorated. Walking was very difficult and also breathing became such an effort that I often just sat on my bed or desk using the ventilator.
That morning I turned on the radio while laying still in my bed. The news was on an I heard a male voice saying: ‘A dutch biotechnological company had announced to develop a treatment for a rare disease. It would be the first time ever in human history that genetically modified rabbits were used to produce a medicine’. I was somehow shocked and felt awake right away. I sat still in my bed awaiting for the next bulletin to be sure. Then again it was said, but this news reporter added one little important detail: He mentioned it was about Pompe disease. I somehow still couldn’t believe it and walked to the living room to turn on the television and to watch video text. There it was in black and white: Pompe disease, treatment, rabbits.
I called my father and Anton. They too heard this news and were like me excited, but also with a bit of restriction as this news was so overwhelming that we couldn’t fully understand it yet. This was what my brother told me about. Then the phone rang and a Pompe patient I knew very well, asked if I heard the news. We talked about it and she was like me very excited. We promised eachother to keep eachother informed. I dressed myself and kept listening to the radio that continued bringing the news out. Then I was called again. It was Ysbrand Poortman of the VSN who had attended the press conference in Geel, Belgium, where Pharming had a small pilot plant to produce the enzyme. Ysbrand Poortman too asked me if I was informed about the latest developments and I told him that I indeed was. He told me he was approached by the Dutch News Broadcasting Service (NOS) and they wanted to interview a Pompe patient. ‘Are you interested?’, he asked. I said yes, without understanding the impact of this answer.
One and a half hour later the camera crew and reporter were at my home. Luckily my sister and a good friend of hers also were home from school already, so they had tidied up my room a bit. Three hours later the news item on Pompe disease, treatment with rabbit milk and Pharming was on prime time news in the Netherlands. This was my first television appearance and many more would follow on broadcastings in the Netherlands, Germany, Belgium, BBC world and of course interviews with magazines and newspapers in The Netherlands, Germany, Belgium, Norway and Finland. Pompe disease was brought into the world.
That morning I turned on the radio while laying still in my bed. The news was on an I heard a male voice saying: ‘A dutch biotechnological company had announced to develop a treatment for a rare disease. It would be the first time ever in human history that genetically modified rabbits were used to produce a medicine’. I was somehow shocked and felt awake right away. I sat still in my bed awaiting for the next bulletin to be sure. Then again it was said, but this news reporter added one little important detail: He mentioned it was about Pompe disease. I somehow still couldn’t believe it and walked to the living room to turn on the television and to watch video text. There it was in black and white: Pompe disease, treatment, rabbits.
I called my father and Anton. They too heard this news and were like me excited, but also with a bit of restriction as this news was so overwhelming that we couldn’t fully understand it yet. This was what my brother told me about. Then the phone rang and a Pompe patient I knew very well, asked if I heard the news. We talked about it and she was like me very excited. We promised eachother to keep eachother informed. I dressed myself and kept listening to the radio that continued bringing the news out. Then I was called again. It was Ysbrand Poortman of the VSN who had attended the press conference in Geel, Belgium, where Pharming had a small pilot plant to produce the enzyme. Ysbrand Poortman too asked me if I was informed about the latest developments and I told him that I indeed was. He told me he was approached by the Dutch News Broadcasting Service (NOS) and they wanted to interview a Pompe patient. ‘Are you interested?’, he asked. I said yes, without understanding the impact of this answer.
Articles in the international media on Pompe diseaseand its upcoming treatment derived from genetically modified rabbits.
One and a half hour later the camera crew and reporter were at my home. Luckily my sister and a good friend of hers also were home from school already, so they had tidied up my room a bit. Three hours later the news item on Pompe disease, treatment with rabbit milk and Pharming was on prime time news in the Netherlands. This was my first television appearance and many more would follow on broadcastings in the Netherlands, Germany, Belgium, BBC world and of course interviews with magazines and newspapers in The Netherlands, Germany, Belgium, Norway and Finland. Pompe disease was brought into the world.
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