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HMC achieves rare distinction with gene therapy breakthrough

  • 19 November 2019
  • Author: QT01
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HMC achieves rare distinction with  gene therapy breakthrough
Tribune News Network
Doha
Hamad Medical Corporation (HMC) has become the second healthcare organization outside the United States and Canada to treat children with congenital spinal muscular atrophy using a new gene therapy. Yesterday a team of doctors at Hamad General Hospital administered the innovative gene therapy to two pediatric patients diagnosed with the congenital disease. A single dose of the newly introduced medication was injected into each patient and will be followed by a prescribed therapeutic plan, as per the internationally approved protocol.
Qatar has become the second country outside North America to provide the new treatment, which is said to cost USD 2.1 million a dose and is being provided to Qatari pediatric patients free of charge.
Commenting on the new gene therapy being available to patients in Qatar, Dr. Abdulla Al Ansari, Acting Chief Medical Officer at HMC said: “The application of this new gene therapy only six months after its approval by the Food and Drug Administration (FDA) in the United States reiterates HMC’s resolve and commitment to making the latest and most effective medications available to patients.”
Dr. Al Ansari extended thanks and gratitude to His Highness the Amir, the wise Cabinet, and the Minister of Public Health, for their ongoing support of the healthcare sector and enabling HMC to provide its patients with the medications they need, regardless of cost.
“We wish a speedy recovery to these children. I would like to highlight the efforts of HMC’s clinical teams who provide care for, and treatment of, all patients with this condition. In the past, we have provided spinal muscular atrophy patients with the latest treatment available. The cost of that medication was QAR 500,000 per dose, and each patient required several doses. With the emergence of this new drug, which was approved by the FDA only 6 months ago, we immediately sought to obtain it for the benefit of our patients, despite its high price. HMC will always put its patients first and will always remain committed to providing them with the best and latest treatments,” added Dr. Al Ansari.
Dr. Al Ansari concluded by thanking HMC’s management and all clinical, nursing, and administrative teams for their efforts and continued commitment to delivering the safest, most effective, and compassionate healthcare to all patients.
Dr. Tawfeg Ben-Omran, Senior Consultant, Pediatrics, and Head of Clinical and Metabolic Genetics said HMC becoming the second center outside North America to offer the innovative therapy is a significant achievement. 
“Spinal muscular atrophy (SMA) is a genetic disorder and a leading cause of death in children under two years of age. The disease is characterized by weakness and atrophy in the muscles used for movement, breathing, and swallowing. Spinal muscular atrophy can be prevented through premarital screening and genetic testing before commencing IVF treatment for couples with a family history of the condition,” said Dr. Ben-Omran. 
“This highly advanced treatment involves the one-time intravenous administration of a healthy and functional gene which is delivered by a vector (a vehicle for delivering genetic material) to target the cause of SMA. The gene therapy improves the response of the child’s motor neurons. Dosage of treatment is determined based on the child’s weight,” added Dr. Ben-Omran.
Dr. Abdullah Al Houthi, Deputy Chief, Pediatrics Department, Hamad General Hospital, said Qatar is one of the few countries in the world to use the innovative gene therapy. 
“This morning, in the Pediatric Intensive Care Unit (PICU) at Hamad General Hospital, we administered the first dose of the innovative gene therapy to children with spinal muscular atrophy. The first dose was injected by a team that included Dr. Khaled Al Laithy, Head of the Pediatric Intensive Care Unit at Hamad General Hospital and Dr. Husam Ali, Clinical Pharmacist. Both patients are currently undergoing observation in the PICU. This time of careful observation will be followed by a prescribed treatment plan,” said Dr. Al Houthi.     
Dr. Al Laithy said that the proper dose of gene therapy has been successfully administered to two patients, noting the patients are 17 and 18 months old. Prior to the introduction of the new gene therapy, the two patients had been treated with repetitive spinal injections, a cumbersome procedure, but the new therapy is thought to be safer and more effective in the management of the disease.
“The new gene therapy, which is prescribed to pediatric patients under the age of two years old, will be administered to three more patients at HMC in the near future. A full clinical assessment of these patients will be conducted before the gene therapy procedure to ensure the treatment is safe and appropriate for the patients. Healthcare professionals at HMC’s PICU are capable of administering the new medication and undertaking the necessary follow-up care,” added Dr. Al Laithy.  
Dr. Ali noted that the proper dose of the gene therapy medication for each pediatric patient is determined in close collaboration with the pharmaceutical company manufacturing the product. 
“Based on medical and laboratory analysis reports provided by HMC clinical departments, the manufacturer supplies the medication where it is prepared by highly qualified HMC pharmacists in accordance with internationally approved protocols. The pediatric patient is injected with the gene therapy in an Intensive Care Unit environment under the supervision of ICU consultant and trained nursing teams. This medication is, by all standards, deemed revolutionary and is considered a major pharmaceutical breakthrough, being the first gene therapy of its kind directly administered to patients,” added Dr. Ali.     
Mr. Mohamed Ahmad Al Meer, the father of one of the two pediatric patients who received the new treatment, expressed his sincere gratitude to His Highness the Amir and thanked the government of Qatar, Her Excellency Dr. Hanan Mohamed Al Kuwari, Minister of Public Health, and all clinical and nursing teams who provided continued support and care to his child since his diagnosis, which was made a few months after birth. 
Mr. Al Meer commended the efforts of the State of Qatar to enhance healthcare services and provide the latest international treatments to all citizens, regardless of cost. Mr. Al Meer concluded by wishing health and wellness to everyone in Qatar. 
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