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Fundraising for Toddler’s Expensive Treatment Moves Hungary

Fanni Kaszás 2019.09.24.

Only the most expensive medicine in the world could save a Hungarian toddler, who is suffering from SMA (spinal muscular atrophy). The parents of Zente have started fundraising for the boy’s 700 million HUF treatment. The fundraising started last week and as of today, nearly 60% of the amount, almost 450 million HUF has been collected. The expensive medicine called Zolgensma, that may cure the boy, has already generated huge controversy in the United States, and because of its astronomical price, many insurance companies are only willing to finance it on strict terms, as its long-term effect has not yet been proven. In Hungary, another treatment may have given hope to SMA patients since last year, but for the time being, it is only funded on a case-by-case basis, exclusively for children.

Zente was born on February 28th of last year with perfect medical scores, and in the first days nothing suggested that he had any medical problems. But then, after several worrying signs and countless tests, Zente was diagnosed with the most severe type of muscular atrophy, SMA-1.

SMA-1 is an extremely rare genetic disorder which affects only one in eight to ten thousand people. Because of a defective gene, their body does not produce the protein that protects muscle cells, so their muscles slowly deteriorate. Symptoms of SMA-1 – the type that Zente has – usually occur during the first months of the patient’s life. In most cases, due to respiratory paralysis, children do not reach age two. There are approximately 120 SMA patients officially registered in Hungary (this applies to all types of SMA, not just SMA-1) but due to the outdated registration system, professionals say that the actual number is around 300.

Why does it cost so much?

SMA is currently classified as an incurable disease, but in recent decades, thanks to developments in the pharmaceutical industry, patients were given some hope. However, the development and testing of the new drugs, especially for rare diseases are unimaginably expensive. Zente’s condition could probably be improved with gene therapy with the medicine Zolgensma, but its price was set at 2.1 million USD (HUF 641 million) by its manufacturer, Novartis. Another difficulty is that although the treatment was authorized by the US pharmaceutical authority in May, it is not yet patented in Europe. In the meantime, Zente’s parents are also competing with time, as children are only allowed to receive the drug before reaching the age of two.

Zolgensma has already generated huge controversy in the United States, and because of its astronomical price, many insurance companies are only willing to finance it on strict terms, as its long-term effect has not yet been proven. Novartis argues with the criticism of the medicine’s price, saying that decades of research precede such therapies, and if they are developed for rare diseases such as SMA, the price will inevitably be higher, which both patients and insurers need to understand.

However, according to affected parents, their child’s life is priceless, and the 2.1 million dollar price is nothing compared to having their children go untreated – as without medicine, they may die or need to be treated for the rest of their lives, in many cases, with a respirator.

How does it work?

Zolgensma is currently the most expensive medicine in the world and it has only been on the market since May. The essence of the treatment is that the patients are given a virus by gene therapy that “infects” and replaces the gene pool of defective or missing motor neurons, thus preventing muscle atrophy. 36 infants aged between two weeks and eight months have participated in the clinical trials. According to statistics from September, all children receiving treatment showed improvements in their condition.

However, it is important to note that the drug does not work in the same way with everyone: it cannot rebuild muscles that have already atrophied, so patients can only be treated completely if they have not yet developed the symptoms of the disease. The long-term effects of Zolgensma cannot be seen as well, but according to researchers, it can bring about a 90% improvement.

Is there another option?

Zente has already received a medicine called Spinraza, financed completely by the National Health Insurance Fund (NEAK). The vaccine was patented just last year, when Zente was just a couple of months old and he has received it ever since. This treatment is not cheap either, as an injection costs 23 million HUF, but the cost is entirely borne by NEAK. However, this medicine is needed by patients for the rest of their lives. The boy has shown results from the treatment, and thanks to it and physiotherapy, he can sit steadily and hold his head for up to 25 minutes.

Last week, Zente’s parents finally decided to start collecting the money for his expensive treatment on the Internet. The target amount is 700 million HUF, which has to be raised within six months as the boy is now 1.5 years old, and the whole country has been moved for the toddler’s recovery. Supporters include children who have offered their own Christmas presents, other patients with severe illnesses, while others are auctioning their services to help. Among the donors were quite big names such as: violinist Zoltán Mága, for example, gave one million HUf towards the treatment and called on the richest people in the country to donate as well.

According to the latest information, nearly 450 million HUF was raised by Tuesday morning.

featured photo:  Mira Budafoki/GoFundMe