How Gene-Editing Made Two Incurable Diseases Curable

The U.K.’s approval of CRISPR-based gene-editing treatment, Casegvy, gives hope to patients with inherited blood disorders.

• Casgevy targets sickle cell disease and β-thalassemia, both characterized by genetic abnormalities affecting hemoglobin production in red blood cells.
• Results show sickle cell disease patients experiencing reduced pain, and β-thalassemia patients showing a decreased need for red-blood-cell transfusions.

The U.K. has approved Casgevy, the first-ever CRISPR-based gene-editing treatment for inherited blood disorders, specifically sickle cell disease and β-thalassemia.

Do you remember last week we talked about the technology that would permanently lower Low-Density Lipoprotein (LDL) cholesterol after a single therapy session?

Well, it’s the same application of the gene-editing technique called CRISPR. The scientists edit the DNA of the patients and eliminate the little bit of DNA responsible for the disease. However, Casgevy is the brainchild of Vertex Pharmaceuticals in Boston, Massachusetts, and CRISPR Therapeutics in Zug, Switzerland.

After a series of successful clinical trials, the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted its approval to treat two blood-related diseases through CRISPR. And now the FDA and the European Medicines Agency are looking into approving it.

The first, sickle-cell disease, is characterized by crescent-shaped red blood cells, which hinder their movement through the veins and can block blood flow. The second, β-thalassemia, reduces the production of hemoglobin, which is the protein in red blood cells responsible for the transport of oxygen to your various organs. Both of these diseases are the results of errors in the DNA bit responsible for hemoglobin.

The scientists managed to edit that bit of DNA in blood-producing stem cells from the patients’ bone marrows. The editing triggers the production of normal fetal hemoglobin, rather than the abnormal adult one.

In sickle-cell disease trials, 28 out of 29 participants experienced relief from debilitating pain for at least one year after the one-time intravenous infusion treatment. Similarly, for β-thalassemia, 93% of the 54 participants treated with Casgevy did not require red-blood-cell transfusions for at least a year post-treatment.

This is amazing. Every time I read about humans’ exploits in medicine at the DNA level, I’m amazed. To get a reprieve from the pain of chronic illness is… would…. does bring a person to their knees in gratitude.

Sometimes when you receive the diagnosis of a chronic illness or an incurable disease, it starts to feel like the walls are closing in on you and you’re being buried alive. It feels like a death sentence.

Cue the doctors’ appointments, the treatments, the needles, the side effects… and at times you feel so beaten down and angry. Then come those days when you feel like you’ve reached your finished line. You can’t take another transfusion. You can’t take another flare-up.

I’m well aware that the treatment is expensive. But with this technology, and the way they are slowly applying it to different genetic disorders, patients will get a new lease on life.

Perhaps worth every penny…

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