A few months ago, the whole country joined forces to raise HUF 1.4 billion for two toddlers suffering from SMA (spinal muscular atrophy), as only the most expensive medicine in the world could save them. With the help of generous donations from the country and beyond, Zente and Levente were able to start the treatment in time and they finally stood on their feet. Following the successful treatment of the toddlers, three Slovakian children suffering from the same condition received the expensive treatment in Hungary, VálaszOnline reports.
Fortunately, the families did not have to travel to Boston because the medical staff at the Bethesda Children’s Hospital in Budapest learned how to use the gene therapy medicine. Since then, the small reformed church hospital has become the largest center for gene therapy in Europe. In the middle of the coronavirus epidemic when the European borders closed, following the successful treatment of the Hungarian boys, three Slovak children were also treated there.
The children suffer from SMA-1, an extremely rare genetic disorder which affects only one in eight to ten thousand people. Because of a defective gene, the body does not produce the protein that protects muscle cells, so muscles slowly deteriorate. SMA is currently classified as an incurable disease, but in recent decades, thanks to developments in the pharmaceutical industry, patients have been given some hope. However, the development and testing of the new drugs, especially for rare diseases are unimaginably expensive. However, a drug called Zolgensma may cure the patients, but its price is set at 2.1 million USD (HUF 641 million) by its manufacturer, Novartis. Additionally, parents are usually not only competing with the disease, but with time as well, as children are only allowed to receive the drug before reaching the age of two.
A few years ago, 90% of these children were lost before the age of two, because muscles are needed not only for movement, but also for breathing and swallowing. However, the most expensive medicine in the world, Zolgesma, gave them some hope. Last year, both Hungarian boys’ parents started fundraising for the HUF 700 million treatment and Zente and Levente received the gene therapy in Budapest’s Bethesda Hospital.
Three Months After 700 Million HUF Treatment, SMA-Baby Zente Stands on His Feet
One of the Slovak boys, Riško, was four months old when his parents noticed that he could no longer lift his leg, and later he was diagnosed with SMA. His parents researched the disease on the Internet, and they came across the story of a young Hungarian child, Zente.
They immediately contacted Zente’s mom, who by the time had been in contact with 50 SMA parents worldwide. She helped them too by giving contacts at the Bethesda Hospital and tips for collecting the money for the treatment. In Slovakia, however, the parents of four SMA-babies needed help at the same time, so they asked for help together – and just as the Hungarians were touched by Zente’s story and raised the money for the treatment in a matter of days, so did Slovakians.
However, raising money for the children did not mean an immediate start to treatment either. The borders closing due to the coronavirus and the distance to Boston for the children were all a problem. That’s when they turned to the Bethesda Hospital.
György Velkey, director general of Bethesda Children’s Hospital, told Hungarian news portal valaszonline.hu that they had several dilemmas when the Slovak parents contacted them. He said that “when Zente’s mother approached us last year, we came to the conclusion that it was best for us to do the treatment here. But they were Hungarian children. How can we help a foreign child with a product that is not yet authorized in the European Union? How does a drug manufactured in Chicago get to Budapest safely at temperatures below minus 60 degrees through borders closed due to the coronavirus?”
In addition, Bethesda Hospital has a total annual budget of HUF 3 billion. It is less than the price of a drug for five children, while the risk factors are greater if something goes wrong during the treatment. However, in the end, they decided to help the Slovakian children as well. Bethesda has thus become the largest center for gene therapy in Europe, with no other hospitals giving the therapy for five children anywhere else.
Since then, in May, the medicine was also registered with the European Medicines Agency (EMA), making it easier to apply for health insurance support. At the end of November, Gulyás Gergely, head of the Prime Minister’s Office announced at this week’s government briefing that the government will provide HUF 34 billion (EUR 101 million) in additional funds for the health fund for 2019. Partly for pharmaceutical and medical aids, and for the care of patients with rare diseases, including SMA.
Gov’t to Support People Suffering from Rare Diseases after Rejecting Opposition Proposals
From the HUF 34 million fund, the medical care and expensive treatment of those suffering from rare diseases can be supported. However, at the moment, there is only one Hungarian child with SMA who could receive the treatment – in Romania. His fundraising currently stands a little over HUF 500 million.
featured photo: Riško’s official facebook page