Today, Ain Shams University Specialized Hospital performed the injection of the second Egyptian child suffering from spinal muscular atrophy, a one-year and ten-month-old boy named Asser Karim el-Din. Doctors at Ain Shams Hospital, under the supervision of Dr. Nagia Ali Fahmy, Professor of Neurology and Director of the Neuromuscular Diseases Unit at Ain Shams University Faculty of Medicine, administered the drug Zolgensma, which is the most expensive drug in the world, costing $2.125 million or approximately 34 million Egyptian pounds.
Dr. Mahmoud Al-Metini, President of Ain Shams University, explained that this drug is the first of its kind in the world, administered to the patient via intravenous injection just once, and has been approved by the U.S. Food and Drug Administration since May 2019.
Dr. Fahmy noted that the drug manufacturer had offered 100 opportunities to obtain the drug for free in countries where the drug has not yet been registered, along with criteria for selecting eligible children for injection, including that the child must be under two years old and that the mutation should be in the first gene.
In March, the Neuromuscular Diseases Unit at Ain Shams submitted files for eight children to the drug manufacturer, and the case of a child named Ryan from Alexandria was chosen to be the first in Egypt to receive the drug for muscular atrophy. Following the success of the medical teams at Ain Shams Hospital with the first case, Asser was also selected for the injection.
In this context, Dr. Al-Metini mentioned that the hospital provided full support to complete the procedures for obtaining the drug, coordinating with the Minister of Finance and the Head of the Medical Professions Union to facilitate customs clearance and exempt from taxes and duties amounting to 1.750 million pounds. The global and local Novartis companies also contributed training support for doctors on how to administer the treatment to the patient and facilitated the transport of the treatment to Egypt at a temperature of minus 70 degrees Celsius.
Regarding the cure rates with this drug, Dr. Fahmy confirmed that spinal muscular atrophy types one and two lead to inevitable death within the first two years of a child's life due to respiratory failure. This drug has been clinically tested for years, and the first child injected with it is now five and a half years old. She explained that the drug improves respiratory functions and motor weakness, placing the child on a path of normal growth, though improvements occur gradually through stages, where the child undergoes physical therapy and respiratory rehabilitation.
On the condition of the first child, Ryan, Dr. Fahmy confirmed that since he was injected with the gene therapy, he has been monitored over the past months to ensure all his bodily functions are intact, through a comprehensive team of specialists in physical medicine, cardiology, genetics, pulmonology, and orthopedics at Ain Shams Specialized Hospital, in addition to the Neuromuscular Diseases Unit team. This will also apply to the child Asser.
Regarding the potential availability of this drug in Egypt, Dr. Fahmy emphasized that there are hundreds of children with spinal muscular atrophy who would benefit from this drug, but its costs exceed individuals' capabilities. She pointed out that the Neuromuscular Diseases Unit, established 25 years ago at Ain Shams, has been able to offer services that were previously unavailable in diagnostics and treatment, and the unit aims for Egypt to become a center for research and clinical trials on genetic and hereditary diseases, which would provide treatment in Egypt at an acceptable price, especially with pharmaceutical companies, including local ones, shifting their research efforts in this field.
