Genetics Specialist Prof. Dr. Erol Baysal, stating that the FDA approved the first cell-based gene therapy for thalassemia disease, said, “I have dreamed of today in my nearly 40-year professional life.”
TRNC Presidential Science and Health Committee Member, Genetics Specialist Prof. Dr. Erol Baysal made a written statement and announced that ‘gene therapy’ for thalassemia disease (Mediterranean Anemia) was approved.
The eyes of the thalassemia world are enlightened. In his statement with the title ‘Today is a groundbreaking day in treatment’, Baysal stated that he dreamed of today in his 40 years of specialization and professional life.
Baysal announced that as of August 17, 2022, the US Food and Drug Administration (FDA) approved the first cell-based gene therapy that treats adult and pediatric patients with beta-thalassemia who require regular blood transfusions.
Prof. Dr. Erol Baysal’s explanation is as follows:
“I have dreamed of today in my 40 years of specialization and professional life. As a TRNC citizen, only 22 years old, my hematology teacher at UCL, Prof. Modell told me at the end of a lecture, “you are from Cyprus, I want you to do your doctoral thesis on Thalassemia with me after you graduate, because it is very needed in your country Cyprus” still ringing in my ears. Yes, great progress has been made in this field in the past 40 years.
However, today’s development will go down in history as the last point we can reach in the treatment of thalassemia. That’s because the FDA has approved the first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular blood transfusions (August 17, 2022).
With this new development, I am very happy to give the good news of offering a remedy to our brothers and sisters with thalassemia instead of difficult and troublesome treatment.
Of course, I hope that the states will give all kinds of support to our sick brothers and sisters for such a brand new, highly effective and arduous treatment.
Since the TRNC has the highest incidence of thalassemia in the world, this is an extremely important development for our country.
I would like to thank BLUEBIRD BIO INC. in New York, who achieved this success, for choosing me as an ‘expert consultant’ especially in patient and mutation selections in the aforementioned Gene Therapy studies.
I am very proud and happy to share this good news with all my brothers and sisters with thalassemia in TRNC and TR. For those who are curious about the details and content of gene therapy studies, I explain below:
The US Food and Drug Administration FDA has approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.
Director of the FDA Center for Biological Evaluation and Research, Dr. “Today’s confirmation is a significant advance in the treatment of beta-thalassemia, especially in individuals who require continuous red blood cell transfusions,” said Peter Marks MD, PhD. “Given the potential health complications associated with this serious disease, this new development highlights the FDA’s commitment to supporting the development of innovative treatments for patients with limited treatment options.”
Beta-thalassemia is a type of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells in the blood through mutations in the beta-globin subunit, leading to insufficient oxygen delivery to the body. Decreased red blood cell levels can lead to a number of health problems, including dizziness, weakness, fatigue, bone abnormalities and more serious complications.
Transfusion-induced beta-thalassemia, the most severe form of the condition, often requires lifelong red blood cell transfusions as the standard of care. These regular transfusions can in themselves be associated with many health complications, including problems with the heart, liver, spleen, and other organs due to excess iron buildup in the body.
Zynteglo is a one-time gene therapy product administered as a single dose.
Each dose of Zynteglo is a customized treatment created using the patient’s own cells (bone marrow stem cells) that have been genetically modified to produce functional beta-globin (a component of hemoglobin).
The safety and efficacy of Zynteglo have been established in two multicenter clinical studies involving adult and pediatric patients with beta-thalassemia requiring regular transfusions. Efficacy is based on achieving transfusion independence achieved when the patient maintains a predetermined hemoglobin level without the need for any red blood cell transfusion for at least 12 months. Of the 41 patients who received Zynteglo, 89% achieved transfusion independence.
The most common side effects associated with Zynteglo include decreased platelet and other blood cell levels, as well as mucositis, febrile neutropenia, vomiting, fever, hair loss, epistaxis, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation. , nausea, decreased appetite, pigmentation disorder and pruritus.
There is a potential risk of blood cancer (leukemia) associated with this treatment; however, no cases were seen in studies on Zynteglo. Patients taking Zynteglo should have their blood monitored for at least 15 years for any evidence of cancer. Patients should also be monitored for hypersensitivity reactions, thrombocytopenia and bleeding during Zynteglo administration.”