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Drugs specially made for sick girls: "Finally it's proven that this is possible"

This breakthrough was also closely monitored in the Netherlands. "This is proof that it's possible," said clinical pharmacology professor Noortje Swart from Amsterdam UMC.

Mila is suffering from a very rare Batten disease. The first symptoms appeared when he was three years old. Within a few years he experienced an extraordinary setback and even had to use tube food. The girl has a defect in her DNA, a comprehensive analysis shows. The drug that is specially developed now includes that, allowing his parents to see the future again.


This is an extraordinary case, experts say soon, but in the future the drug will be able to be adjusted more accurately to the patient's DNA.

When professor Noortje Swart heard about the breakthrough, his heart pounded. He conducted research on personal medicine. "Developments are progressing smoothly. I know it will happen, but it is fun to see when the time comes."

Nowadays, drugs are increasingly made up. Cancer sufferers or metabolic diseases especially benefit from this. But it still focuses on patient groups and not on individuals like American girls. "Because we know better and better what causes a disease, we can overcome it by specifically developing drugs for it."

Better estimate

"As a result, people with the same disease may need different drugs," Swart said. "We have been able to adjust the dose based on the patient's DNA profile and we can also analyze tumors more accurately so we use certain drugs for control. The development continues."

He expects more breakthroughs to follow in a few years. For example, there are a few dozen Dutch people with DNA abnormalities, which means they have rare metabolic disorders such as Pompe's disease or Fabry's disease. They are now receiving drugs to inhibit disease and fight symptoms. Swart believes that in ten years there will be drugs that can overcome the defects in their DNA.

Get the benefits of treatment

That does not mean that every patient with DNA defects can be helped. But this is good news that the development of personal medicines is going fast, said Ruud Coolen van Brakel, director of the Institute for Responsible Drug Use. "We welcome it. We only want patients who benefit from treatment."

However, it also leads to a different discussion: the more accurate the drugs, the more expensive they are. For the development of medicine for Makovec America, his parents collected no less than three million dollars. According to Swart, this raises an ethical question: how many health benefits are there for patients? "If we proceed on this basis and medicines are increasingly made on individuals, you will end up in an untenable situation."

Throw less

But according to Coolen van Brakel, things don't have to go that fast. "Now we are prescribing drugs for people who will only succeed in 1 in 10 patients, but we don't know who. They cost millions per year. If we know who will succeed based on DNA, you end up throwing fewer drugs and you save money . "

8-year-old Mila is much better now, writes The New York Times after an interview with her parents. He can't stand alone yet, but it's better to stay upright after he stands. And he rarely needs more tube food. "He started responding to things that made him smile," his mother said.

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