The state of eternal war. There are medicines, but you will not get them.

International CF Week has started & mdash; the most common genetic disease in the world. Of which you can still painfully die before reaching adulthood, although effective therapy already exists. Not for everybody.

Average life expectancy of patients with cystic fibrosis in Russia & mdash; 25-27 years old. There are about a thousand adults with this diagnosis in our country, children & mdash; three more time. In Europe, the average patient life expectancy is 45 to 50 years, and adults among all patients diagnosed with cystic fibrosis (Cystic Fibrosis – this is how the diagnosis sounds in English) – & # 39; mdash; more than half. Why in Russia not all children with cystic fibrosis grow into adults and why only a few of them have a chance to lead full lives & mdash; read in the material of the charitable foundation “ AiF. Kind Heart '', providing support to critically ill children and adults with this disease. & Nbsp;

Cystic fibrosis & mdash; rare hereditary disease. In Russia, about four thousand people suffer from it, most & mdash; children and adolescents under the age of 18. All of them have been living with cystic fibrosis since birth. And the worst thing for anyone with this diagnosis & mdash; catch the infection. A family with cystic fibrosis quickly learns the words Pseudomonas aeruginosa. and “separation”. If you don't react quickly, then this is the second (Burkholderia cepacia – a complex of bacteria resistant to various antibiotics and especially dangerous for patients with cystic fibrosis) can invalidate the organism and lead to death within days or even hours. This is due to systemic organ damage & mdash; those that produce mucus are mainly affected: the lungs, pancreas, liver and intestines. Due to cystic fibrosis, the secretion secreted by them becomes too thick and forms clumps which easily become infected and prevent the patient from breathing and absorbing food. & Nbsp;

There is no cure for cystic fibrosis. And every day you need to control the symptoms: take antibiotics, mucolytics, enzymes to digest food, hormones, vitamins and other drugs. Every day & mdash; endless hours of therapy: coughing, inhalations, vibrating vests, physiotherapy, postural drainage, percussion, breathing exercises. Morning, afternoon, evening, no break, weekend and vacation. 4 to 8 hours a day & mdash; breathe, eat, live. & nbsp;

In addition, there is the so-called “ targeted '' therapy: along with drugs that relieve the symptoms of cystic fibrosis, “genetic correctors” are taken, which fight against the cause of the disease, that is, with a specific defect of the genes in the patient's body . & nbsp;

“ This is now the most promising and promising therapy of any in the world '', & mdash; said Stanislav Aleksandrovich Krasovsky, candidate in medical sciences, senior researcher at the Pulmonology Research Institute . Unfortunately, the prices of “target” drugs can reach twenty to thirty million rubles per year, and only a few manage to procure drugs in Russia.

Leila Morozova has succeeded. It took her a year and a half to get the initial purchase of a targeted therapy drug for her children. There are two & mdash; boy and girl, both with cystic fibrosis. Senior Miroslav was only diagnosed at the age of three (they didn't notice any gene degradation right away), after the younger Ulyana had cystic fibrosis immediately after birth. The sister actually saved the brother. And since then, not a single day of their life goes by without each other. And without symptomatic treatment: inhalations of antibacterials, vitamins, antibiotics and other drugs that prevent the appearance of mucus congestion, inflammation and infections. & Nbsp;

Leila calls herself the “Mother of Heroes” and says they've been lucky with children twice. First time & mdash; with the safety of the pancreas, which works so well that it saves Mira and Ulyu from taking tons of enzymes. The second & mdash; with the reception of the 'target'. & nbsp;

Leila Morozova with children. Photo: Extract from the family archives

“ The drugs targeted in Russia are now being received by a few, and they have hardly reached the regions, '' & mdash; says Stanislav Krasovski. The Morozovs & nbsp; live in Kronstadt, and in order to establish endless documents to obtain a 'target', they had to regularly travel to St. Petersburg. Almost a year of constant travel, official receptions and letters have produced no results & mdash; permission has been repeatedly refused citing a lack of necessary papers. Due to bureaucratic obstacles, little progress was made in the matter. “It looked like” bring this, I don't know what. “There was a feeling that an ordinary parent with no relationships and no money would not be able to break the wall of endless 'but' health systems ''. , & mdash; Leila recalls. The Morozov family are said to have received permission for the main purchase of targeted drugs as a 'gift' for the New Year 'after the next consultation in St. Petersburg, just before the New Year's holidays. & nbsp;

In May, the drugs arrived at the pharmacy. Since then, Miroslav and Ulyana take Kalideko & mdash; not registered in Russia, a targeted therapy drug against cystic fibrosis. It should be drunk at strictly defined times, but this will not surprise children & mdash; with Kalideko in their daily routine there are several antibacterial inhalations and doses of antibiotics, which should not be missed either. Their routine has remained largely unchanged with the new drug, but there is hope for “the future of ordinary people,” as Leila puts it. “ People who will not suffocate and die at the age of 20-25, if given the opportunity to receive targeted drugs. & # 39; & # 39;

Targeted cystic fibrosis drugs will suffice for Miroslav and Ulyana for another four months. Next & mdash; total uncertainty. “ In our country, it is often more difficult to make repeated purchases of drugs than primary purchases. There is no order in which to receive them & # 39 ;, & mdash; says Leïla. This means that you will have to wear the 'I don't know what' again, break down the doors of state institutions, and collect tons of papers. Because the abrupt cancellation of the targeted therapy, because it was decided to no longer deliver the drug, it is also impossible & nbsp; & mdash; there may be deterioration. It's almost the same as if diabetic patients were left without insulin, and asthmatics – and mdash; without inhalers. So the Morozovs don't know how many more of these roller coasters with permits, purchases, and waivers ahead of them. “ A family with a sick person, in principle, cannot afford a state of endless war. Of course, it's exhausting. We are fed up with fighting, constantly knocking something out and proving & raquo;, & mdash; said Leïla. Either way, she's willing to take that path as many times as she wants to get a treatment that works. And to regain the hope of living up to the moment when she tells her grandchildren all this amazing story. & Nbsp;

“ The state is struggling to buy drugs that are not registered. For example, Kalideko, Simdeko, and Trikaftu, despite their proven effectiveness, can still be found bypassing official purchases only on the black market & # 39; & # 39 ;, & mdash; says Stanislav Krasovski. In Russia, out of four existing targeted cystic fibrosis drugs, one is registered & mdash; Orcambi. It acts on a limited number of patients carrying strictly defined genetic mutations. & Nbsp;

The first purchase of Orcambi for patients with cystic fibrosis in the Volgograd region will take place in January 2022. Elena Ovchinnikova, ward of the AiF charitable foundation. Kind Heart ', providing support to CF patients and their families. With her help, Elena managed to raise the necessary amount for a year's supply of basic therapy drugs for her grandson with cystic fibrosis. Soon he will be two years old, and he will finally be on the list of those who need Orcambi. According to the Russian state program “14 high-cost nosologies”, free genetic correctors are offered to all children between the ages of two and eighteen with genetic mutations. But & mdash; only with transfers eligible for funding from this program. Ignatius, almost two years old, is like that. Now he has every chance to start targeted therapy thanks to the 'Circle of Kindness' state fund. and halt the progression of the disease. & nbsp;

Adult support is not included in the program.

“They're the ugliest scissors around. Because the children will be able to start this therapy, achieve a significant effect, have confidence in the future, and at exactly 18 years of age they will be deprived of the opportunity to continue the treatment ',' ' 39; & # 39; mdash; says Stanislav Krasovsky. & nbsp;

In the Volgograd region, only one adult patient with cystic fibrosis was able to obtain the right to targeted therapy in court. Others still have to fight even after taking basic therapy drugs. This is especially difficult with expensive inhaled antibiotics and mdash; they are often not given even to children. And if they do, they try to replace them with more affordable analogues. So another neighborhood in the AiF. Good heart & # 39; & # 39 ;, Vika Nagornyachenko , nine years from Volgograd, contrary to medical recommendations, they bought Indian antibacterial inhalations instead of European Colistin & mdash; to save the regional budget. But the replacement only made the girl's condition worse: the side effects and toxic properties of the generic were quickly felt. “ The Department of Health always has a reason to refuse needed drugs, so they cut costs and save the budget '' & mdash; says Tatiana Nagornyachenko , whose daughter of such a “ treatment '' it just gets worse and worse.

In May, Vika Nagornyachenko finally received a medical prescription for targeted therapy with another drug not registered in Russia, but effective & mdash; Simdeco. Clearance was given in May, now & mdash; November. And the family is still trying to get at least one initial purchase of the drug from the local health committee. In response to official letters & mdash; stamped refusals without explanation. & nbsp;

& laquo; Endless unsubscriptions & mdash; it is a huge stress for the parents. To withstand this, you need a strong core inside. '' & mdash; said Vicki's mother. The worst thing for their family & mdash; it's unknown. Will they give permission to buy the drug (at least a year and a half after the medical appointment) & mdash; as was the case with Miroslav and Ulyana Morozov & mdash; not clear. The “ Circle of Kindness '' state fund Will he commit to supporting the family, who are obligated to provide a 'target'? children with cystic fibrosis & mdash; still not clear too. “ We still only live on empty promises & # 39; & # 39 ;, & mdash; says Tatiana Nagornyachenko. In the near future, they don't have to look forward to receiving targeted drugs. & Nbsp;

The daily battles for the life and health of their children take almost all of their time and energy from parents. And it seems obvious that every family with a critically ill child is in dire need of a & nbsp; Support. But where to get it & mdash; free and one-time if you live in a small town, in a remote area, far from big clinics, foundations and hotlines? And without such support, as the mother of the parish of our foundation once said: “It's like we fall into a black hole of despair every day, and the bottom doesn't. ; is not visible. This is why AiF. Good heart & # 39; & # 39; offers her wards with cystic fibrosis help on all fronts of their never-ending struggle. The Route of Help program, created in collaboration with the Presidential Grants Fund, includes lawyers who walk families through to get the drugs they need, and psychologists who lift parents out of this black hole. Legal support, patient communities, peer counseling & nbsp; & mdash; this is what helps parents feel confident that they can handle it, that their child will receive treatment. That they will have time. Thus, supporting the work of the specialists of “ Help Route '' through your donations, each of you provides entire weeks and months of peaceful living not only to children with cystic fibrosis, but also to their parents.

A magic pill for cystic fibrosis does not exist yet. Even patients on advanced targeted therapy have to take their entire life and, alas, this is not for everyone. Different types of mutations, different organisms, different responses to treatment. But right now, new types of drugs are being developed, new ingredients are being tested, genetic mutations are being studied, and new breakthroughs are being made every day. And it is already clear that only a complex treatment of cystic fibrosis (target + symptomatic treatment) has a chance of lastingly stopping the development of the disease, of no longer being afraid of any infection and, in the end, of living to see your children. . and grandchildren. & Nbsp;

In some countries, all cystic fibrosis treatments are paid for by the state by default, and families don't need to 'knock out' drugs, save for inhalers, collect money for anti-vibration vests. It is not necessary to consult a lawyer every time life-saving therapy is refused. These are the countries where there is virtually no infant mortality from cystic fibrosis, and the adult mortality rate is steadily declining. Russia also has its own achievements: we have learned very well about saving regional budgets and bureaucratic resources. It remains to learn to value the lives of children and the time of their parents. And this & nbsp; and the other in patients with cystic fibrosis is not so much. & Nbsp;


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