Gebze, Turkey Yusuf Polat is only five years old, but he has had enough illnesses to last him a lifetime.
He has scoliosis and a hole in his stomach, his bones and muscles are so damaged that he can’t walk or swallow or breathe properly, and he has to tie himself to seven devices while he sleeps.
Yusuf’s parents faced the same problem. His sister Melek died when she was one and a half years old with the same problem.
But this time, his parents have a plan.
Yusuf’s condition, spinal muscular atrophy (SMA) type 1, is a rare genetic disease, but now there are drugs that can prevent the spread of the disease in the right babies, and sometimes change the motor functions at one level. .
However, the gene therapy, onasemnogene abeparvovec, marketed as Zolgensma, is also known as the most expensive drug in the world, at $2.1m – a price that, with inflation in Turkey, is becoming easier to find every day.
“Those who found this medicine do not prioritize helping people, or curing children, or removing the disease from the world; they prioritize making money,” said Zeynep Ceylan, one of Yusuf’s “volunteer sisters” who met the family a few months ago because of their fundraising work.
The company that owns the drug, Basel-based Novartis, has come under fire around the world for its prices. Novartis said it initially planned to buy between $4m and $5m, although a pharmaceuticals analyst said the price would be between $310,000 and $900,000.
Novartis justified the price by arguing that it uses the latest technology and saves families a lifetime of medical trips.
“With this one-time treatment, Novartis Gene Therapies is improving the existing treatment system in spinal muscular atrophy (SMA), which is built around a treatment that can cost health systems millions of euros over a patient’s life,” a Novartis spokesman. he told Al Jazeera.
It also shows that it has few buyers because SMA is so serious, it affects about 10,000 babies worldwide.
In Turkey, this number is estimated to be around 1 in 6,000 people. As a hereditary disease, it is more common in children of consanguineous families; These couples, like Yusuf’s parents, often cannot afford to get help.
Kayhan and Mensure Polat live in Gebze, one of Turkey’s industrial centers, where Kayhan works in construction. He quit his job so he could continue fundraising and doctor’s appointments, which left the family broke.
Despite this, Yusuf manages a happy face for his visitors and the camera: his smile is plastered on posters and posted on the Internet that share his story and his bank account, to make it easier for people to support Yusuf. .
“We have to ask people for their money,” Mensure said. “Some give a small amount of money, others just pass by. It’s a big problem.”
When Yusuf’s parents launched the campaign last September, the equivalent of what they negotiated was about 16 million Turkish lira. Since then, the lira has depreciated significantly against the dollar, raising its value to more than 34 million lira ($1.83m). As the lira continues to fall, their target continues to rise and the purses only increase.
Ceylan said: “We cannot reach people who are well. “If Yusuf had been the son of an MP, the campaign would have ended in a week.”
Some parents find celebrities or politicians – like the Mayor of Istanbul Ekrem Imamoglu – to support their campaigns, and their team is working to meet the president. In the absence of such connections, Yusuf’s drive is focused on throwing concerts, having a shopping center, protesting in front of the Novartis headquarters and organizing drives on his Instagram account, which now has 22,000 followers.
The global distribution of the COVID-19 vaccine showed that poor countries, with limited negotiating power, have a difficult time negotiating prices and opportunities. Patients in these countries have to travel to authorized countries to provide the drug, so they pay extra for a few months of accommodation.
In the United States, for example, the total cost can go up to $3m for foreign investors. Residents of the US and twelve countries that have an agreement with Novartis can have their own insurance for the drug.
There is no government approval
The Turkish Ministry of Health held talks with Novartis in 2020, but the talks ended.
Health Minister Fahrettin Koca told a Turkish parliamentary committee last year that the ministry’s SMA scientific committee was still evaluating the drug’s effectiveness and had not received a request from Novartis.
“The sensitivity of the issue forces us to do things very carefully,” he said. “We cannot play with the hopes of this fragile life.”
Turkey’s Ministry of Health did not respond to several requests for comment.
Fikri and Serpil Tezcan, whose son Yiğit was diagnosed with SMA type 1 at 60 days old, won a court case last year for Turkey to import the gene therapy. Although Turkey’s drug approval agency added Zolgensma to its list, Tezcan’s family says the ministry continues to block its importation.
Instead of waiting, the couple went to Germany, where Yiğit could receive treatment for free since Serpil is a German citizen. Now he is three years old, Yiğit can hold on His neck is straight and he is learning to walk. His parents continue to fight for access to the drug in Turkey; They have asked this year to the European Court of Human Rights to be granted human protection in Turkey.
Although Turkey already covers another treatment for SMA 1, Spinraza, the results take several months before they are needed. Turkey also has genetic testing for couples about to get married, and if they have the gene, it covers their in vitro fertilization.
Yusuf’s parents have now been tested, as has their 11-year-old daughter. They said Yusuf lived longer because of Spinraza, which is why they waited before considering trying gene therapy. However, Yusuf is already three years past the legal age to receive treatment. He was rejected by hospitals in the US and Germany, but a hospital in Dubai said he would be treated as long as he remained under 13.5kg (30 pounds).
Until they reach their goal, Mensure gives Yusuf milk through a syringe to keep him from gaining half a kilo.
Had they exceeded that limit, the county government would not have extended its fundraising campaign for another year.
Some families are facing difficulties just starting the campaign: since the Ministry of Health does not recognize the power of Zolgensma, it can only start court cases.
“It was found that it is not useful to create a campaign in our country to carry out gene therapy, for which there is no sufficient evidence in terms of efficacy and safety,” Koca said in a statement last year.
When asked about the Turkish minister’s statement, Novartis presented several studies that showed the effectiveness of Zolgensma. Although Novartis said that it could not comment on the Turkish administration, it said that “it does not matter the country”, its goal was to find “sustainable solutions for gene therapy such as Zolgensma”.
The lawyer of the family of Tezcan Mehmet Rıfat Bacanlı has opened 15 cases against the Ministry of Health for closing the campaign.
“The money these families collect is not sustainable, because the number of children diagnosed with diseases increases every day,” said Bacanlı. Last year, he said he knew about 12 fundraising events; this year, perhaps because of more genetic testing and diagnosis, he said there should be more than 100.
More than a year into their campaign, the Polats are nearing the end. However, even if they raise all the money, the rest is unknown. The hospital in Dubai did not disclose details about the procedure; right now they say they only talk about money.
“We don’t know what the medicine will do,” said Kayhan. “But whatever it is, we want to have it.” We have to. We have no other choice.”