The US approves gene therapy worth 2.75 million euros

Gene therapy is developing at breakneck speed, and the possibilities now seem endless.

The United States Food and Consumer Product Safety Authority (FDA) has approved the first gene therapy based on stem cells. This means that it is the first official treatment where stem cells are removed, genetically modified and then put back into a patient. With a price of 2.8 million dollars (about 2.75 million euros) per person, Zynteglo, as the medical treatment is called, is also the most expensive ever.

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Why gene therapy?

Our body is made up of around 100,000 billion cells that together ensure that everything works properly. To do that, they need protein. If you think of a cell as a factory, proteins are the workers that keep this factory going. They know what to do because they have a manual at their disposal that can be found in the cell nucleus: our DNA.

The genes that make up our DNA explain exactly what is expected of the proteins. And then there is also such a thing as RNA. Think of it as an intermediary: RNA copies the DNA, leaves the cell nucleus and makes the proteins there, exactly as the DNA prescribes. But sometimes there are errors in one or more genes. And if the manual is not correct, things also go wrong in the implementation. In that case, you are talking about a genetic disorder. Gene therapy can correct these errors.

beta thalassemia

The gene therapy has been developed for people with the rare blood disease beta-thalassemia. Patients with the inherited condition suffer from chronic anemia because the body does not make the protein hemoglobin properly. Hemoglobin carries oxygen from your lungs to the rest of your body and removes carbon dioxide.

Currently, people with beta thalassemia receive many blood transfusions to replenish the amount of red blood cells – which are largely made up of hemoglobin. But the danger of this is that the patients consume too much iron, so that it accumulates in the body – and that is also dangerous.

gene therapy

Gene therapy is a promising alternative. In this case, a correctly functioning variant of the defective gene is introduced into the patient’s stem cells, which takes over the functions of the defective gene. A doctor takes the cells from the patient and takes them to the laboratory. The correct genes are then added there using a virus, after which the patient ‘gets them back’.

© LOOK Summer issue 2020

Thanks to this adaptation, the genetically modified stem cells can change into red blood cells that contain the protein hemoglobin. The effects of the individual treatment are expected to last a lifetime.

Disadvantages

Zynteglo could help many beta thalassemia patients get rid of blood transfusions. However, there are also risks associated with the treatment. For example, cases of leukemia have been reported in clinical trials testing similar gene therapies. Therefore, the FDA recommends that patients have regular blood tests for 15 years after treatment. However, no cases of cancer have been observed in studies of Zynteglo, the FDA reports.

Then there is the price of 2.75 million euros per treatment. Beta thalassemia and many other inherited conditions such as SCID, where babies are born without an immune system, are rare. In addition, the treatment is only a one-time treatment. This means that there is not much profit to be made for the producer, and so the price is significantly inflated.

crooked

“In itself, this is an important milestone for stem cell-based gene therapy and for thalassemia patients,” says Professor of Molecular Stem Cell Biology Frank Staal. “It’s an expensive therapy, but it also has a high price, and it’s a life-saving one-time treatment that’s fundamentally safer and appears to be better than current treatments.” Still, Staal admits that almost 3 million are very significant.

“I find it particularly bitter that this product has been developed exclusively in Europe in EU projects. The vector (the virus that delivers the gene to the correct ‘address’, ed.) was developed in Italy and France, but is now unavailable in Europe.” This is due, among other things, to the very complicated European network of regulations, which is much more complex and more expensive than in the USA, explains Staal.

“In Europe, the substance should therefore become even more expensive. This is something I am trying to change with my colleagues in our NWA project CURE4LIFE, which I coordinate.” This consortium wants to bring gene therapy to patients faster. “By the way, it is not the only gene therapy that is not available to patients for non-medical reasons. A lot.”

Sources: FDA, New Atlas, LOOK summer edition 2020

Image: VCHAL/iStock/Getty Images

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