Scientists are now seeing incredible early results from the first round of studies that are testing gene editing for blood disorders that are known to be painful, inherited and plague millions of people worldwide.
Sickle cell disease treatment
Medical experts hope that the one-time treatment, which involves altering the DNA in blood cells permanently by using a tool called CRISPR, can help treat and cure sickle cell disease and beta thalassemia.
On December 5, the partial results of the studies were presented at an American Society of Hematology conference and some were even published by the New England Journal of Medicine.
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In the study, the doctors described 10 patients who were removed from their respective treatments for several months. All of the patients no longer need regular blood transfusions and all are free from pain crises that they have suffered from for years.
Sickle cell affects millions of people worldwide, but it is more common in Black people. Beta thalassemia affects about one in 100,000 people. The only cure now for beta thalassemia is a bone marrow transplant from a closely matched donor without the disease like a sibling. However, not everyone has a close match donor.
Both of the diseases involve mutations in a gene for hemoglobin, which is the substance in red blood cells that carries oxygen in the body.
In sickle cell, the defective hemoglobin leads to deformed and crescent-shaped blood cells that do not carry oxygen well in the body. They usually stick together and they clog small vessels. This can cause pain, organ damage and strokes to the patient.
Meanwhile, those with beta thalassemia do not have enough normal hemoglobin. They suffer from fatigue, anemia and shortness of breath. Severe cases of beta thalassemia require transfusions every two to five weeks.
Gene problem
The study about the new gene-editing treatment attacks the source of the problem, which is the gene. Fetuses make a special type of hemoglobin in the womb.
After birth, when babies breathe on their own, a gene is automatically activated and it instructs cells to change and make an adult form of hemoglobin instead. The adult hemoglobin is what is defective in those with one of these disease. The CRISPR editing in the study aims to remove the switching gene.
Dr. Haydar Frangoul from Sarah Cannon Research Institute in Nashville, Tennessee, said that what they are doing is turning the switch off and making the cells think that they are back in the utero so they make fetal hemoglobin again instead of the adult ones.
The new treatment would involve removing stem cells from the patient's blood and using CRISPR in a lab to turn off the switching gene. Patients will be given strong medicines in order to kill off the other damaged cells, afterwards they are given back their altered stem cells.
The results of the study on December 5 showed 10 patients, seven have beta thalassemia and the three have sickle cell.
According to NBC News, the studies are still on going simultaneously in the United States and in Europe and they aim to enroll at least 45 patients each.
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Written by Sieeka Khan
