FDA advisers evaluate CRISPR-based gene therapy for Sickle Cell Disease

FDA advisers evaluate CRISPR-based gene therapy for Sickle Cell Disease | Credits: Google Images
FDA advisers evaluate CRISPR-based gene therapy for Sickle Cell Disease | Credits: Google Images

Washington, United States: Advisers to the Food and Drug Administration will be reviewing a gene therapy for the inherited blood disorder – sickle cell disease, on Tuesday. According to the reports, the disorder majorly affects the Black people in the US. After reviewing, they will consider whether more research is required for the suitable treatment.

Following the approval by the FDA, it will become the first gene therapy in the US market. It will be based on CRISPR – the gene editing tool that won its inventors the Nobel Prize in 2020. Before taking up a different sickle cell gene therapy later that month, the treatment will be decided by the agency.

Dr Allison King, a professor at Washington University School of Medicine in St Louis, is one of the caretakers of the children and young adults with sickle cell disease, outlined that she is excited to see the result the new treatments. She was quoted saying, “Anything that can help relieve somebody with this condition of the pain and the multiple health complications is amazing,” according to the news agency AP.

FDA advisers evaluate CRISPR-based gene therapy for Sickle Cell Disease | Credits: Google Images

She further outlined, “It’s horribly painful. Some people will say it’s like being stabbed all over.”

The disorder directly affects the protein in red blood cells that carriers oxygen – hemoglobin. A genetic problem makes cells change their shape and become like crescents, which can block blood from moving and lead to terrible pain, damage to organs, strokes, and other issues.

Many people around the world, including about 100,000 in the United States, have this problem. It’s more common among people from places where malaria was or still is a big issue, like Africa and India. It’s also more common in certain groups, like those with African, Middle Eastern, and Indian backgrounds. Some scientists think that having the sickle cell trait helps protect against very bad malaria.

Right now, doctors use medicines and blood transfusions to help with this issue. The only cure is a bone marrow transplant, but it’s tricky. It has to come from a closely matched donor without the problem, and there’s a risk that the body might reject it.

The new treatment involves changing of DNA

While elaborating the treatment, the health experts outlined that for one-time gene therapy no donor is required. The therapy is named “exa-cel,” made by Vertex Pharmaceuticals and CRISPR Therapeutics. Under this treatment DNA is permanently changed from the blood cells of the patient.

FDA advisers evaluate CRISPR-based gene therapy for Sickle Cell Disease | Credits: Google Image

The major goal behind the treatment is to produce a fetal form of hemoglobin, which is naturally present at birth. During the treatment, the stem cells are removed from the blood and CRISPR, to knock out the switching gene.

According to the health experts, flawed blood-producing cells are killed off through medicines and then given back their own altered stem cells. According to the data shared by Institute for Clinical and Economic Review – a nonprofit organisation, thus far, the treatment is tested in a relatively small number of patients.

What developing company, Vertex, has to say?

In a document released before a meeting, Vertex, the company that made the treatment, said that 46 people used it in the main study. Out of 30 people who were followed for at least 18 months, 29 didn’t have painful episodes for a year, and all 30 avoided going to the hospital for pain during that time.

The company said the treatment is a big positive change and is very safe.

Victoria Gray, the first person to try the treatment from Mississippi, talked about her experience at a science conference. She explained that she had been suffering from painful episodes since she was a child and had to take strong pain medicine and sometimes get blood from others. She said that she felt like she was given a fresh start on the day she got the gene therapy.

She highlighted that now, she has been taking care of her kids, along with doing a full-time job. She was quoted saying, “My children no longer have a fear of losing their mom to sickle cell disease,” according to AP.

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