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.
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’.