"You feel more helpless than your child": RTD releases a film about children diagnosed with SMA

The film begins with the story of Alyosha Gulyants.

He lived for only three months.

His mother, Yulia, says that the pregnancy and childbirth went well, all indicators were excellent: “At some point I noticed that my son was dry.

When they donated blood, the doctors said: "Mom, are you really afraid that such a beautiful baby has some kind of genetic disease?"

I was ashamed: why am I so worried.

It is hard to believe that he has such a serious illness ... "

Alyosha's family was not the only one who faced the tragic diagnosis of spinal muscular atrophy.

There are 1,045 people, including 817 children, in the non-state register of the “SMA Families” foundation.

They suffer from a deadly genetic disease that prevents them from making the protein needed for motor neurons, the nerve cells that set muscles in motion.

The neurons die, and the muscles, including those responsible for breathing, atrophy.

68% of children diagnosed with type I SMA do not live to see their second birthday, 82% die before they are four years old.

Until recently, SMA was thought to be untreated.

However, at the end of 2016, the first drug appeared that could stop the progress of the disease - Spinraza.

It must be taken for life; injections are made into the spinal cord.

The medicine is very expensive: the first year of injections alone costs about 48 million rubles.

Spinraza was registered in Russia in the summer of 2019, and in fact, until now, it was the only medicine that could be officially prescribed by domestic doctors.

There are two other drugs in the world that help with SMA.

One of them - "Evrisdi" (registered in Russia on November 26) - is taken orally and also for life.

How much it will cost and how Russian patients will be able to get it is not yet known.

The second, Zolgensma, is a gene therapy drug.

Unlike the other two, Zolgensmu only needs to be taken once.

The drug "reprograms" the broken gene to produce the protein needed by motor neurons.

This is one of the most expensive drugs in the world, even because of this it got into the Guinness Book of Records: the "injection of life" costs $ 2.125 million, or about 150 million rubles.

As David Lennon, head of gene therapy at Novartis (Zolgensma's manufacturers) explains in the film, gene therapy is an extremely complex process that requires a lot of investment.

“The lifelong therapy that SMA patients need is costing the healthcare system tens of millions of dollars,” he says.

“We believe that in the long term, Zolgensma will help reduce the cost of treating these patients and take the financial burden off both individual families and the entire healthcare system.

This is where we see the value of the drug.

Therefore, we consider our pricing policy absolutely justified. "

As Lennon noted, manufacturers do not expect families to pay for the drug themselves, implying that it is "the responsibility and responsibility of the health ministries and government."

However, the heroes of the film "You Can't Kill Cure" tell that in Russia parents of children with SMA often have to rely only on themselves and the help of caring fellow citizens.

“You feel more helpless than your child.

If there was no medicine at all, you already know that this is a sentence.

You will make sure that your child lives the rest of his life normally, - says Varya Rostova's dad Stas.

- The person who created the medicine (Zolgensmu.

- RT

), I would say thank you.

And to the one who sells it, except for mats, I can't say anything.

I understand that this is a business, not a desire to help. "

Varya was lucky: her family closed the camp and the baby was given a long-awaited injection.

At the time of the beginning of work on the film, only three Russian children received Zolgensma, and during the filming period another 25 families closed multimillion-dollar fees.

Only 10% of SMA patients in need of Spinraza treatment receive the drug: since it was the only one registered in the country, the regions are obliged to purchase it.

Obliged - but sometimes help comes too late.

Perhaps the most poignant moment in the film is when Svetlana Rukosueva lists what she will dress her son Zakhar in after his exhumation: “I bought clothes.

With ninjas, with minecraft, cowards with dinosaurs - now he has steam locomotives.

The vest will be.

Fashionable guy.

Why are you shaking? "

Zakhar was two years and eight months old.

He died on January 30 without waiting for the medicine he needed.

For several months, the family was sent from geneticists to neurologists, from neurologists to orthopedists - and vice versa.

Regional doctors said that in order to receive an appointment for Spinraza, the boy had to go to the Moscow Research Institute of Pediatrics.

Veltischev (having appointed hospitalization only for June 2020), although federal experts said that the conclusion of regional doctors was enough.

The doctors also referred to the fact that the procedure for the treatment of children with SMA in Russia by Spinraza and the sources of funding have not yet been determined.

On February 13, the Main Investigation Department of the Investigative Committee of the Russian Federation for the Krasnoyarsk Territory and the Republic of Khakassia initiated two criminal cases on the death of Zakhar Rukosuev under Art.

109 of the Criminal Code of the Russian Federation (causing death by negligence) and 293 of the Criminal Code of the Russian Federation (negligence).

For the investigative actions, the exhumation of the boy's body was required.

The elder brother Zakhara Dobrynya, who also has SMA, received Spinraza, which his family had been seeking for several months.

“Dobrynya received medicine at the cost of not 48 million, but at the cost of his brother’s life,” his mother says.

The media and volunteers helped raise awareness of the diagnosis and treatment of patients with spinal muscular atrophy.

In June, Russian President Vladimir Putin proposed raising the personal income tax rate from 13 to 15% for citizens earning more than 5 million rubles a year.

This measure will only apply to income over this amount.

It is assumed that this way the budget will receive about 60 billion rubles.

“I propose, as experts say, to“ dye ”these funds, protect them from any other use and target them for the treatment of children with severe, rare diseases, for the purchase of expensive drugs, equipment and rehabilitation equipment, for high-tech operations,” Putin said. ...

In October, Deputy Prime Minister Tatyana Golikova announced that the state would additionally fund the treatment of children with spinal muscular atrophy and 18 more orphan and 29 life-threatening diseases.

According to her, this will provide treatment for about 25 thousand children.

A special fund will manage funds, select patients and purchase drugs for them.

The funds of the fund should become an addition to the already available resources of the federal and regional budgets allocated for these purposes, the Deputy Prime Minister emphasized.

In November, Prime Minister Mikhail Mishustin signed a decree on the inclusion of Spinraza in the list of vital and essential medicines (VED).

This will allow the state to regulate the price of an expensive drug, as well as simplify the procedure for providing drugs to patients in need.

The film by Maria Finoshina can be viewed on the RTD website.