During the corona news storm of recent months, an important development in the medical world has gone virtually unnoticed: after more than twenty years of studies, there are for the first time treatments in the Netherlands that can help patients with very serious genetic diseases. These are therapies that repair faulty human genes or replace them with good ones.
Two weeks ago, the Minister of Medical Care, Tamara van Ark, decided that treatment against a hereditary retinal disease can be reimbursed through the basic package. People with this genetic abnormality are born very visually impaired and are usually blind before the age of 40.
That fate also threatens for two friends from Urk, coincidentally with the same eye condition, Meindert van de Berg and Michiel van den Berg. They hope for the newly developed gene therapy.
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Do Michiel and Meindert have a chance of new gene therapy?
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Ingeborgh van den Born, of Het Oogziekenhuis Rotterdam, explains: “Because of the wrong gene, the retina cannot function properly. Now it is possible to bring a healthy gene under the retina. That healthy gene was developed in a laboratory. But there must still be a vital retina. Otherwise gene therapy is not a solution. “
The gene therapies cost tons per patient. For the eye defect, one treatment is sufficient, but it does have a price tag of around 600,000 euros. In May, the very first gene therapy in the Netherlands was already approved: a treatment against lymph node cancer. This treatment costs about 400,000 euros.
Henk Mondeel, team leader at Ambulance Oost, had lymph node cancer and was treated at one point. “I had already arranged everything, including my funeral. Until I was allowed to participate in an experimental study in Groningen.”
Mondeel was in bad shape, says hematologist Tom van Meerten of the UMC Groningen. Until therapy came. “His immune cells were genetically altered in a special lab in the US. After he received the genetically altered immune cells, he was fine again. He has been disease free for a year now.”
A miracle
The treatment of Mondeel consists of a single administration of the cells, which heals half of the treated people. “The other half does not recover, the prognosis is very bleak for that, they die,” says Van Meerten.
For Mondeel it is as if a miracle has happened. “With that thought I still get up every day. A last resort what people have found and what I have received. I am grateful for that every day.” If the disease stays away for five years, Mondeel can be officially cured, according to his doctor.
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In the Netherlands there are now two available gene therapies, but many hundreds more will follow. Positive for some patients, but there is also a negative consequence: drug costs will rise. Expensive medicines in the Netherlands must first be assessed by the Zorginstituut, an advisory body of the government. The Minister of Medical Care then decides whether the drug is included in the basic package.
Eye patient Michiel van den Berg: “It is of course an astronomical amount, but at the same time, if you look at the results, I think it’s worth it.”
Doctor Van Meerten says about the costs. “I think it is important that we can now give this to a patient, who will really benefit from it. If more patients can be treated with this, we will of course have to consider whether these prices will also be sustainable.”
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