You are an artist, repairing a fresco. Lime accidentally falls into one eye. Slowly but surely, the light dims in the affected eye. It is easy to imagine the despair. What will it mean for your work, for travel, for your quality of life? It happened to Anne-Mieke. A stem cell transplant provided the solution. Her eyesight has improved and she can lead a normal life.
Thankfully, this kind of treatment is no longer science fiction. The European Medicines Agency EMA has already approved fourteen cell and gene therapies. For example CAR T cells. A treatment in which the patient’s immune cells are processed so that they can defeat cancer. Such therapies are now available for blood conditions, blindness and joint com-plaints.
Another example. Little eight month old Louisa has a rare form of leukaemia. She is being treated with chemotherapy, but the side-effects are severe. The disease is briefly defeated, but comes back again. Louisa is given an experimental treatment with CAR T cells, which is successful. Louisa is now almost four years old. She has recovered and is going to school.
The stories of Anne-Mieke and Louisa can be found in this brochure. They illustrate the exceptional possibilities offered by cell and gene therapy. However, these treatments are – unfortunately – only rarely used in our country.
Why is that? Current legislation is not designed to deal with them. There is a great deal of discussion about the relationship between costs and benefits. Practical adjustments are also required, for example in manufacturing, transportation and training. Of course, there are also ethical questions. What interventions in the human body do we consider justified?
It is high time to consider the matter. There are already over one thousand cell and gene therapies under development, so the future is bright. The revolutionary aspect for many of these treatments is that they truly can provide a cure, or at least improve quality of life significantly and enduringly. They are game changers.
In order to achieve breakthroughs, the government, doctors, insurers, patients and pharmaceutical companies must work together. Only then can we quickly and responsibly bring the innovations of the twenty first century to the patient. Let’s see how far we can go together! That’s the challenge this brochure wants to promote.
Director, Dutch Association Innovative Medicines
Photo: Ruud van der Graaf, Amersfoort
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