The premium medical provider has offered gene therapy as part of a global programme (Managed Access Programme) that has given100 Spinal Muscular Atrophy (SMA)patients around the world, access to the world’s most ground-breaking treatment, in 2020. Medcare Hospitals has become the first healthcare provider in the Middle East to offer the complimentary gene therapy to two infant patients suffering from Spinal Muscular Atrophy (SMA) type 1 as part of the Managed Access Programme (MAP) by Novartis.
Novartis, a pharmaceutical company that develops this gene therapy for SMA, started this programme in 2020 to offer gene replacement therapy for free to 100random patients in the world diagnosed with SMA. An infant is selected every 15 days as the part of this programme, and the first two in the Middle East have been chosen from Medcare Women & Children Hospital (MWCH).
Dr Shamsa Abdulla bin Hammad, Chief Operating Officer, Medcare Women and Children Hospital said, “We, at Medcare Women and Children Hospital, are proud to be the first hospital in UAE to start the treatment of Gene therapy for Spinal Muscular Atrophy! Under the #AveXisMAP program in the Middle East. This treatment is like giving the child the gift of life which is a very fulfilling feeling and this is the reason we do what we do. Spreading smiles and health to all our patients has always been our priority”
Spinal Muscular Atrophy (SMA) is a genetic condition in which an essential protein called SMN is not produced, as a gene responsible for its production called SMN1 is deleted or mutated, making the muscles weaker. The condition worsens over time, and can be fatal. A severe case (type 1) can mean that the child may not survive beyond a couple of years. However, with the gene therapy, the patient can lead a normal, or close to normal life.
Dr Vivek Mundada, Paediatric Neurologist at Medcare Women & Children Hospital, said: “Gene replacement therapy means replacing a fully functional copy of the missing SMN1 gene, which is the root cause of the disease. The gene then enters targeted body cells and starts producing the missing SMN protein.
“Children with SMA Type 1, who usually never achieve the developmental milestone of sitting, having received this gene replacement therapy, have learnt to sit and have survived with a good quality of life. Some have even achieved the milestone of walking. This therapy changes the natural course of the disease as their muscle power increases significantly and hence is a life-saving drug for this condition,” Dr Mundada added.
Both the children receiving gene therapy suffer from SMA type 1, and will be the first in the Middle East to receive gene therapy under the MAP program. For one of them, the rapid genetic testing was performed using a clinically validated assay at Al Jalila Children’s Genomic Center, Dubai. The test result was ready within 48 hours, a rapid turnaround that would have taken 2-4 weeks if the test was sent abroad. This insured timely confirmation of the clinical diagnosis and suitability of the patient for the gene replacement therapy. MWCH is one of the few hospitals in the UAE that can accommodate this treatment programme. Dr Mundada also adds over ten years of expertise in this field leading a highly specialized clinical and research unit in London, aimed at helping children with neuromuscular problems.
“The infants had pre-therapy clinical assessments and necessary blood tests required for the therapy. The drug is specifically made for these children according to their weight. The children will be admitted to the intensive care unit, and all the staff, including nurses, pharmacists, and medical crew involved in the care of these children have received specialized training for storing, handling, and preparing the drug for infusion within the hospital setting,” Dr Mundada explained.
The children will be monitored for at least 24 hours after the infusion. After discharge, periodic assessments will be done by Dr Mundada and the pediatric physiotherapist at Medcare Women & Children Hospital to monitor their progress and other parameters like side effects.
Parents have been educated on the condition of SMA, including various monitoring requirements of the children. Apart from Dr Mundada, they have been seen by pediatric respiratory and gastrointestinal specialists. The children are receiving regular physiotherapy, with regular updates on the progress of this therapy being shared with the parents.