Evaluating the FDA’s approval of two gene therapies for sickle cell disease, Genetics Specialist Prof. Dr. Korkut Ulucan emphasized that if the treatment’s success targets are achieved, the ‘Genome Age’ will officially begin. Prof. Dr. Korkut Ulucan said, "Considering other gene therapies, I believe the cost of this treatment will approach a million dollars."

Prof. Dr. Korkut Ulucan, a faculty member at Üsküdar University's Department of Molecular Biology and Genetics, evaluated the U.S. Food and Drug Administration's (FDA) approval of two gene therapies for the treatment of sickle cell disease.

If the treatment's success targets are met, the ‘Genome Age’ will officially begin

"We are all very, very excited. Although it will take some time for wider application, the initial results have given us clues about where we need to be," said Prof. Dr. Korkut Ulucan, stating that if the treatment's success targets are achieved, the ‘Genome Age’ will officially begin. 

Prof. Dr. Ulucan made the following remarks on the matter:

"Actually, this is a beginning. In 2019, preliminary studies related to gene-editing based therapies were approved for some small patient groups. Based on the outcomes of the data obtained, the approval was officially announced on December 8, 2023. However, the trials have not yet been fully or sufficiently followed up. The absence of disease symptoms is extremely gratifying. 

They genetically modified bone marrow stem cells and transplanted them back to the patient

Scientists from two research companies genetically modified, or briefly edited, bone marrow stem cells obtained from patients using a molecular editing method called CRISPR for the genetic treatment of a disease called sickle cell anemia, and transplanted them back to the patient. And after successful applications, they reported that the patients are currently quite healthy."

Patient follow-ups continue

Prof. Dr. Korkut Ulucan also stated that patient follow-ups are still ongoing, adding, "It has been reported that for 29 out of 30 patients with sickle cell anemia, everything went as planned at the end of approximately 18 months of follow-up. Additionally, for thalassemia patients, it was reported that everything went as planned for 39 out of a 42-person patient group," providing information about the initial results of gene-editing based therapies.

Patient follow-ups planned to last 15 years

Pointing out that the process will be long, Prof. Dr. Korkut Ulucan said, "It was already planned that patient follow-ups for this treatment would last approximately 15 years. However, the initial data yielded results beyond expectations, which is very pleasing. This success will likely lead to applications from new biotechnology companies and the formation of alternative treatment protocols related to this work. This, in turn, will cause time and treatment costs to decrease significantly over time." 

Treatment costs will reach millions of dollars

Prof. Dr. Ulucan also stated that there are different approaches regarding treatment costs, saying, "Treatment costs are another topic that needs consideration. Initially, of course, the success, applicability, and sustainability of the treatment are important. However, treatment accessibility comes next, which will take some time. My estimate is that the cost of this treatment, considering other gene therapies, will again approach a million dollars. I wouldn't be surprised if it exceeds that. This will not only be the application cost but will include all surgical and application costs. However, I believe that over time, if other companies also receive approval in this area, costs will be driven down."