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Breakthrough Leukemia Treatment – Methotrexate’s Impact 70 Years Later

1954 was a memorable year. Then methotrexate came onto the international market. That was a major breakthrough for children with leukemia, because previously it was almost untreatable. It’s no fun, that medicine. “The list of side effects may make it seem that the drug is worse than the disease,” information websites say today. But for the first time it gave patients a greater chance of survival.

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Seventy years old

Those who were the first to survive thanks to that medicine are now over seventy. And new leukemia patients are still treated with that drug. Despite the side effects, despite the toxicity.

“Unfortunately, there is little research into specific medicines for children,” says Laure Konreich of the Queen Fabiola University Children’s Hospital in Laeken. “There are only a small number of patients and investments from pharmaceutical companies often fail to materialize. We do our best with the best options available, even if that means using older medications. They have proven their effectiveness and safety, but unfortunately have a very high toxicity.”

Too few successes

In the 1970s, 1980s and 1990s, new therapies abounded and the survival rate in children increased significantly. But little progress has been made in the past fifteen years. It is encouraging that treatment against the rare brain stem cancer DIPG has led to survival for the first time. But there should be many more such successes, says the Belgian organization KickCancer.

In the meantime, adult cancer is progressing very quickly. The survival rate after 5 years has risen to just under 70 percent, compared to 61 percent fifteen years ago. In children, the chance of survival increased by barely 1.5 percentage points in the same period.

Serious, long-lasting side effects

Innovations in medication would not only have an effect on the chances of survival themselves. “Two out of three patients suffer from serious, long-term side effects due to the use of old, toxic medication,” says KickCancer, which collected figures. This concerns, for example, reduced vision, heart disease or an increased risk of secondary cancers.

“It is essential to pay attention to the long-term side effects and strive for less toxic treatments,” says pediatric hemato-oncologist Sandra Jacobs (UZ Leuven). “But childhood cancer is rare, which makes organizing large-scale studies complex. Older medicines therefore remain invaluable for pediatric oncology.”

In the past seventeen years, seventeen new drugs have been approved for pediatric cancers, compared to two hundred for other cancers. “The introduction of new medication for children is very slow, leaving patients for whom the initial treatment does not work with limited options,” says Delphine Heenen of KickCancer. “This situation must change, because children with cancer have the right to access more innovation.”

2024-02-16 02:00:00


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