CRISPR gene therapy ‘Exa-cel’ shows promise for Sickle Cell, FDA nears approval

CRISPR gene therapy 'Exa-cel' shows promise for Sickle Cell, FDA nears approval | Credits: Google
CRISPR gene therapy 'Exa-cel' shows promise for Sickle Cell, FDA nears approval | Credits: Google

United States: The US Food and Drug Administration (FDA) announced that the therapy, named exa-cel, has been proven to be effective for sickle cell on Tuesday. The experts have also suggested that the Administration will pave the way for full approval by early December.

The gene therapy has been developed by Vertex Pharmaceuticals of Boston and CRISPR Therapeutics of Switzerland. The medical experts suggested that the therapy frees the patients from the excruciating symptoms of sickle cell disease.

It is to be noted that exa-cel will become the first medicine to treat a genetic disease with a CRISPR gene-editing technique if it is approved by December 8 (as expected). The statement was provided by CRISPR Therapeutics through a news release.

CRISPR gene therapy ‘Exa-cel’ shows promise for Sickle Cell, FDA nears approval | Credits: iStock

A second potential cure for the disease, developed by Bluebird Bio of Somerville, Mass, will be decided by the FDA by December 20. This potential cure will specifically strike Black people.

Director of the cellular and molecular therapeutics branch at the National Heart, Lung and Blood Institute and a member of the advisory committee – Dr John Tisdale, as per the New York Times, reported, “We are finally at a spot where we can envision broadly available cures for sickle cell disease.”

Exa-cel treatment

The experts have outlined that this gene therapy is a one-time treatment that changes the DNA of blood cells in a patient.

How does the treatment work?

Stem cells from your blood are taken out, superhero-like cells with incredible healing potential. Next, the medical experts call in CRISPR, the gene-editing superhero, to target and eliminate the gene behind those troublesome crescent-shaped blood cells.

Simultaneously, medication is used to rid the body of the problematic blood-producing cells. Then, the upgraded stem cells are reintroduced, creating a supercharged blood cell production system, potentially transforming the health condition of a patient. It’s like a fresh start with a team of cells inside the body of the patient.

Dr Allison King, a professional at Washington University School of Medicine in St Louis, recently interacted with the Associated Press and mentioned, “Anything that can help relieve somebody with this condition of the pain and the multiple health complications is amazing.”

Dr King added, “It’s horribly painful. Some people will say it’s like being stabbed all over.”

Vertex Pharmaceutical’s remark

CRISPR gene therapy ‘Exa-cel’ shows promise for Sickle Cell, FDA nears approval | Credits: Google Images

Vertex Pharmaceutical, the developer of the therapy, outlined that for this research, around 46 people received treatment. “Among the 30 who had 18 months of follow-up, 29 were free of pain crises for at least a year and all 30 avoided being hospitalized for pain crises,” the report noted.

Standard treatments

It is to be noted that temporary treatments for the disease include medications and blood transfusions. However, a bone marrow transplant from a closely matched donor with the disease is only a cure.

According to the reports by AP, the second gene therapy for sickle cell disease will expectedly intend to work by making functional copies of a modified gene. It is to be noted that the therapy will assist the red blood cells to produce hemoglobin – which is not misshapen.

The authorities have not yet released the prices of both gene therapies. However, recent research has outlined that the expected cost-efficient price tag will be around US$ 2 million, as the existing treatment is worth US$ 1.6 million for women and US$ 1.7 million for men between the ages of 0-65.

In this regard, Dr King was quoted saying, “But if you think about it. How much is it worth for someone to feel better and not be in pain and not be in the hospital all the time.”

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