Geneticist Prof. evaluated the FDA's approval of two gene therapies in the treatment of sickle cell disease. Dr. Korkut Ulucan, He emphasized that if the success goals of the treatment are achieved, the 'Genome Age' will officially begin. Prof. Dr. Korkut Ulucan: “I think the cost of this treatment will approach a million dollars when I consider other gene therapies.” said.
Üsküdar University Molecular Biology and Genetics Department faculty member Prof. Dr. Korkut Ulucan evaluated the US Food and Drug Administration's (FDA) approval of two gene therapies for the treatment of sickle cell disease.
If the success goals of the treatment are achieved, the 'Genome Age' will officially begin.
“We are all very, very excited. "Although there is still time to implement it, the first results gave clues about where we need to be." said Prof. Dr. Korkut Ulucan stated that if the success goals of the treatment are achieved, the 'Genome Age' will officially begin.
Prof. Dr. Ulucan made the following evaluations regarding the issue:
“Actually, this is a start. In 2019, preliminary studies on treatments based on gene editing were approved in some small patient groups. Based on the outputs of the data obtained, its approval was officially announced on December 8, 2023. But the trials have not yet been followed fully or sufficiently. It is very pleasing that the symptoms of the disease do not appear.
They genetically edited marrow stem cells and transplanted them back into the patient
For the genetic treatment of a disease called sickle cell anemia, scientists from two research companies genetically modified the marrow stem cells obtained from patients with a molecular editing method called CRISPR, briefly edited them and transplanted them back to the patient. And after successful applications, they reported that the patients are very healthy for now.”
Patient follow-up continues
Prof. also stated that the follow-up of the patients is still continuing. Dr. Korkut Ulucan said, “It was reported that in 30 of 29 patients with sickle cell anemia, everything went as planned after a follow-up period of approximately 18 months. In addition, they reported that in thalassemia patients, everything went as planned in 42 of the 39 patient group.” He gave information about the first results of treatments based on genetic editing.
Patient follow-up was planned to last 15 years
Pointing out that the process will be long, Prof. Dr. Korkut Ulucan said, “In the planning of this treatment, patient follow-up was planned to last approximately 15 years. However, the first data turned out to be beyond expectations, which is very pleasing. This success will probably lead to applications from new biotechnology companies and the creation of alternative treatment protocols. “This will cause time and treatment costs to decrease much more over time.” he said.
Treatment costs will reach millions of dollars
Prof. also stated that there are different approaches about treatment costs. Dr. Ulucan said, “Treatment costs are another issue that should be considered. Of course, the success, applicability and sustainability of the treatment are important in the first place. But then comes treatment accessibility, which will take some time. My assumption is that this treatment, when I consider other gene therapies, will approach the million dollar again. I wouldn't be surprised if it passes. This will not only be the application cost, but all surgery and application costs. However, I think that over time, if other companies receive approval on this issue, the costs will be reduced.” He completed his words by saying.
