Administration of Zolgensma to little Oliver

Oliver was examined for the first time at FN Motol on May 4, long after the collection, which his parents initiated. Until then, the small patient was not in the care of our hospital, ie his exact internal condition was not known by the doctors of the Motol University Hospital and the parents did not have any binding opinion from FN Motol that it would be suitable for treatment with Zolgensma. The parents contacted the hospital only when they had already announced a collection for treatment, without consulting our doctors in person about the possibility of administering Zolgensma. Since the first visit (4.5.2020) MUDr. Jana Haberlová, chief physician of the Neuromuscular Center of the Department of Pediatric Neurology of the 2nd Medical Faculty of Charles University and the Motol University Hospital, informed little Oliver's parents that she would do her best to be given Zolgensma. Inclusion in preparation for gene therapy does not automatically mean its administration, but the preparation for administration and the final decision is always necessary to assess the overall health of the patient at the time.

The hospital's efforts to ensure the administration of Zolgensma at the Motol University Hospital near Oliver were always conducted with the aim of relieving Oliver of the need for repeated treatment, ie resolving the cause once. But because Oliver's state of health is very severe and treatment with Zolgensma has very serious health risks, the administration of this drug by Oliver was contraindicated due to life-threatening risks, because they far outweigh the potential benefit of treatment.. Abroad, for example, a similarly severe patient treated with Zolgensma recently died on the third day after administration. The doctors at FN Motol did their best to be treated with Zolgensma while Oliver was in their care. However, they cannot recommend its administration if the risk to it is far higher than the effect of the treatment and the consequences can be fatal.

The decision is in accordance with the Indicative Criteria of the Society of Pediatric Neurology of the Czech Medical Association JEP valid as of 15 June 6 and in accordance with the recommendation of the international expert team in the field of neuromuscular diseases. The reasons were repeatedly explained to the parents during the last visit last week. Nevertheless, according to their statements on social networks, despite our best efforts, they do not understand this explanation according to their words.

It should be clarified that:

1. It is not true that Oliver dies without gene therapy for Zolgensma.
Oliver is already on similar and also highly expensive treatment with Spinraza (more than CZK 5 million per year). This medicine has a similar effect to Zolgens

2. It is not true that Zolgensm's medicine will cure Oliver.
Unfortunately, no cure, not even Zolgensma, can improve the severity of Oliver's health. There is also no theoretical possibility that Oliver would be able to sit, stand or walk on his own after treatment with Zolgensma.

3. It is not true that Zolgensma can only be given to Oliver until his second birthday.
By registering Zolgensma in the EU in May this year, the limits for the administration of the drug were extended. Currently, the age limit for submission is not strictly set, it depends on the regional rules of the country in the EU.

The neuroluscular center of the Motol University Hospital was established about 5 years ago and provides patients with even the most serious neuromuscular diseases with highly specialized super-specialized care. This year, for the first time in the Czech Republic, the revolutionary drug Zolgensma was successfully administered to two patients. The Motol University Hospital Neuromuscular Center provides care at the international level, as evidenced by its membership in the European network of rare neuromuscular diseases - ERN NMD and accreditation of care by the World Duchenne Organization, which became the first neuromuscular center in Europe.

Press Release: Reaction - administration of Zolgensma to Oliver