US gives green light to first gene editing treatment

US gives green light to first gene editing treatment

The revolution has begun. The dream of curing diseases by rewriting the genetic code is no longer just an illusion. Finally, the United States gave the green light to the first treatment based on genetic editing in humans. In particular, a drug from the company Vertex Pharmaceuticals is approved for the treatment of sickle cell anemia, a rare, very painful blood disorder for which there is no effective treatment. The new treatment, Casgevy, is approved for use in patients aged twelve and over.

At the beginning of the month, a panel of experts concluded that the drug is effective and safe after evaluating clinical trials. The final approval from the FDA, the US drug agency, is missing, it was announced this Friday. With this, the United States became the second country to bring this revolutionary therapy to the clinic. The United Kingdom continued the approval process, approving the same drug on November 17. In addition, the FDA also approved this Friday another type of more traditional gene therapy to correct this rare anemia. It’s called Lyfgenia and it restores the correct copy of the affected gene to patients’ cells.

Both products are made from the patient’s own blood stem cells, which are modified, and returned to the patient in an infusion with a blood stem cell transplant. Before the treatment, stem cells are taken from the patient, who must undergo chemotherapy treatment. In this way, bone marrow cells are removed to replace them with modified Casgevy and Lyfgenia cells. Both are treatments that have been approved in an accelerated manner due to the absence of therapies. Therefore, patients receiving Casgevy or Lyfgenia will be followed in a long-term study to assess the safety and effectiveness of each product.

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In the case of Lyfgenia from the pharmaceutical company BlueBird, a black label was also included, the highest warning sign in the United States for a drug. The decision is due to the appearance of cases of hematological neoplasia (cancer of the blood) in treated patients. Treated patients should undergo lifelong follow-up for these malignancies.

Another obstacle is its high price: Vertex assures that the price of gene editing per patient will reach 2.2 million dollars and that of Bluebird will exceed three million.

Jump to the clinic

Approvals from the United Kingdom and the United States are the starting shot for a technology that seeks to change the way many diseases are treated. The leap into the clinic is a strategy that has so far only benefited a few hundred patients in clinical trials.

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The protagonist of this revolution is a device that won the Nobel Prize in Chemistry just three years ago. : CRISPR/Cas9. These ‘molecular scissors’ make it possible to edit the human genome, deleting and adding genes at will. In fact, it is a tool that allows you to change any form of life and its use seems to have no limits. It has long been used in agriculture to improve crops, for example gluten-free wheat, or varieties that are resistant to drought and adverse weather conditions. Or in livestock farming to improve the production of sheep, cattle and pigs. It is even believed to have the power to revive extinct species.

But in medicine, genetic editing may be the key to curing diseases that still have no cure and cause a lot of pain and suffering due to genetic errors. One of them is sickle cell anemia, where the red blood cells, which should be round, adopt a crescent shape and become sticky and hard, causing them to clump together. These ‘lumps’ block blood vessels and deprive tissues of oxygen, leading to severe organ damage and episodes of severe pain. Its victims are forced to undergo between 15 and 30 transfusions per year and suffer sudden and severe pain crises. The new treatment almost completely eliminated the symptoms.

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Eliminate “bad” cholesterol

Like this type of anemia, other rare diseases with no treatment options await a gene editing solution. Although these many rare patients are not the only ones to benefit. A clinical trial also wants to show if this is the answer to a problem as common as excess cholesterol and the prevention of strokes and heart attacks. The goal of the Heart1 study is to modify the PCSK9 gene that holds the key to destroying ‘bad’ cholesterol or LDL. The journal ‘Nature Medicine’ chose this trial as one of the most promising clinical studies that will affect our future health.

Oncologists also hope that cancer treatments can benefit from Crispr or fight infections or aging. The list is endless. Currently, it is an important tool in research laboratories where it allows you to study the function of genes, playing with them. Rewrite, life in short.