The Transcarpathian Annácska can be the next Zolgensma gene therapy patient in Hungary

Szerző: | 2021.03.22., 12:39 | News

This week, the 10th Zolgensma gene therapy SMA treatment was performed in the to a young Serbian boy, Olivér Pál Gajódi. Over the past year and a half, the hospital has applied Zolgensma gene therapy to two Hungarian, four Slovak, one Dutch, a Hungarian child from Romania, a Turkish and now a Serbian child. With this, Bethesda Children’s Hospital has become one of the most experienced institutions in Europe in the use of this medicine.

SMA (spinal muscular atrophy) is a rare genetic disease, the essence of which is the loss of function of the neurons that move the skeletal muscles of the spinal cord, which leads to the weakening and loss of muscle movements. As a result of muscle atrophy, the development of normal movement of children fails. In severe cases, the respiratory muscles also atrophy, respiratory failure develops which makes mechanical respiratory support necessary. Zolgensma gene therapy medicine, considered one of the most expensive treatments in the world, provides effective help to these patients.

The 21th century brought a new perspective in the treatment of patients. For the first time, so-called gene modification therapy became available in 2016 and since 2019, gene replacement therapy has also been introduced. The essence of this therapy is that the gene responsible for the development of the disease, but missing from the cells, is introduced into the patient’s body. Gene therapy differs from, among other things, gene modification treatment in that the child receives the missing gene itself.

Bethesda Children’s Hospital has begun gene therapy treatment in October 29, 2019. In the nearly one and a half years since then, 10 children have been treated with this treatment.

The 10th treatment took place this week, and they are also preparing the 11th treatment in the near future, in the case of the Hungarian Burinda Zsombor.

Anna Szalcevics, also coming from abroad, from Ukraine can be the 12th child who can get the chance to a new life if the total cost of the treatment can be gathered.

The experiences so far are encouraging: „Two children no longer need breathing support at all, and one only needs breathing support when sleeping during the day.

In two children, swallowing function was also partially restored Children who have not had impaired swallowing function at the time of gene therapy have all retained it and are feeding independently and developing well. This may have significantly increased their daytime freedom and activity, which in turn greatly aids their intellectual development. The development in the movement of the treated children varies depending on the condition from which they started. There are those who sit and play permanently, independently, there are those who have become able to turn, crawl, all of them purposefully using and moving their upper limbs and fingers of the hand. There are also children who have taken the first steps since then. ” reports dr. Borbála Mikos, medical director of Bethesda Children’s Hospital, leader of the gene therapy working group.

The chances of development of children also depend on how early they receive the gene therapy treatment, the age limit is 2-2.5 years old according to the current recommendations.

So, the Zolgensma gene therapy treatment can not be delayed and the COVID-19 pandemic is a particularly difficult challenge for the hospital and those waiting to be treated. Crossing the border, performing medical tests while the borders are closed, the high level of isolation – in order to maintain to highest recovery with the medicine are very hard tasks to the children with weakened immune system, which have to be treated with utmost care and speed by Bethesda Children’s Hospital.

The hospital places special emphasis on segregating the child and their parents to avoid infections.

In the days before treatment, the weakening of the child’s immune system begins that the medicine to be administered achieves its purpose. Administering and control meetings with the child take place under strict isolation rules.

As a result of the therapy, the muscles of children affected by SMA begin to develop, resulting in significant movement development. Changes in the state of movement are constantly monitored by gymnastic therapist before the start of gene therapy and after the administration, and they develop it by every child individually. It should be emphasized that the improvement of the state of movement after gene therapy can be enhanced by appropriate complex development. Bethesda Children’s Hospital undertakes long-term follow-up of children.

Oliver, who comes from Serbia, is still treated in Bethesda, as his gene therapy treatment took place this Tuesday. Dr. Nenad Barisic, professor of cardiology, Oliver’s Serbian doctor, said that

„Oliver was considered as uncurable by everyone in Serbia, as he could not be removed from the ventillator, so it was not possible to treat him with gene therapy. He arrived in Bethesda to assess his condition and after weeks of struggle, it was possible to detach him from the ventillator and it was possible to administer the medicine. I was allowed to be present at the administration of the medicine, I could be part of a fantastic professional teamwork where everything happens for the benefit of the patient. This is a great hope not only for Hungarian children, but also for Serbian children waiting to be treated”

The mother of a Dutch child cried during their farewell from the hospital and highlighted:„Seen from afar, we seem to have come from a more advanced health care system, where, however, Jayme was renounced. Our little boy could get a chance to a new life here in Bethesda, we will never forget what we got here..”

Turkish Ali’s mother said so two weeks after treatment that “they’ve performed a miracle here with my child, I see a change in Ali’s condition day by day.”

At the time of the current epidemic, Bethesda Children’s Hospital is particularly proud of the cross-border cooperation that has been established with Slovakia, Romania, Turkey and Serbia.

In connection with the last two children treated, the Turkish and Serbian ambassadors also spoke at the press conference.

Ahmet Akif Oktay, Ambassador of Turkey, and Barbara Avdalovic, Ambassador Counselor to Serbia, also attended, thanking for the high-level treatment of their compatriots and providing further cooperation and support for Hospital.

Dr. György Velkey, the director general of the hospital, emphasized that „Gene therapy treatment is the success of collaborations, as different cultures and countries have spoken out for the benefit of sick children. This success could only be achieved through the cooperation of diplomats, doctors-nurses, authorities, pharmaceutical wholesalers, economic and legal professionals, and charities.

Bethesda Children’s Hospital

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