Gene drug ‘only hope’ for Turkish child, but health costs millions

Gebze, Turkey Yusuf Polat is only five years old, but he has accumulated enough medical conditions to last a lifetime.

He has scoliosis and a hole in his stomach, his bones and muscles have deteriorated so much that he can’t walk or swallow or breathe properly, and he has to brace himself with seven devices when he sleeps.

Yusuf’s parents have lived through this nightmare before. His older sister Melek died when he was one and a half years old of the same condition.

But this time his parents have a plan.

Joseph’s condition, spinal muscular atrophy (SMA) type 1, is a rare genetic disease, but there is now a cutting-edge drug that can halt disease progression in eligible infants, and in some cases improve motor functions in a single dose. Can ,

Yet the genetic therapy, onasemnogene abeparvovec, marketed as Zolgensma, is also known as the world’s most expensive drug at $2.1m – a price that becomes less accessible every day with inflation in Turkey Is.

“The people who discovered this remedy did not prioritize helping people, or healing children, or eradicating disease from the face of the earth; They prioritize making money,” said Zeynep Ceylan, one of Yusuf’s “volunteer sisters”, who met the family a few months ago through their fundraising campaign.

The company that owns the drug, Basel-based Novartis, has come under worldwide criticism over its cost. Novartis said it initially planned to price it between $4m and $5m, although a drug price watchdog said a fair price would have been between $310,000 and $900,000.

Novartis justifies the price by arguing that it uses advanced technology and saves families a lifetime of medical visits.

A Novartis spokesperson said, “With a one-time therapy, Novartis gene therapy is upending the current treatment paradigm in spinal muscular atrophy (SMA), which is built around chronic therapies that can kill millions of patients over a patient’s lifetime.” Euros can spend.” told Al Jazeera.

It also points out that its potential buyers are few because SMA is a rare condition, affecting around one in 10,000 babies globally.

In Turkey, the rate is estimated to be approximately one in 6,000. As a genetic disease, it is more prevalent in children of related couples; Like Yusuf’s parents, these couples are often the least able to afford treatment.

Yusuf Polat’s family is using every possible means to raise funds for his treatment – such as a hoarding displaying his bank account details [Naomi Cohen/Al Jazeera]

‘a difficult situation’

Kayhan and Mensure Polat live in Gebze, one of Turkey’s manufacturing centers, where Kayhan worked in construction. He quit his job to be able to keep up with the fundraising campaign and doctors’ visits, which caused the family to lose their health insurance.

Through it all, Youssef puts on a happy face for his guests and the camera: his smile is pasted on billboards and cropped up on online images that share his story and his bank account number, so that To make it easy for people to donate for Yusuf’s treatment. ,

“We have to ask people for their money,” Mensure said. “Some people donate small amounts, some just walk away. It’s a very difficult situation.”

When Yusuf’s parents started the campaign last September, the negotiated price was roughly equivalent to 16 million Turkish lire. Since then, the lira has fallen against the dollar, dropping its value to more than 34 million lira ($1.83m). As the lira declines, their targets climb and pockets get tighter.

“We can’t reach people who are in better shape,” Ceylan said. Had Yusuf been the son of the MP, the campaign would have been over in a week.

Some parents get celebrities or politicians – like Istanbul Mayor acrem imamoglu — to support his drive, and a group of them is working on meeting with the president. Without such connections, Youssef’s campaign has focused on holding concerts, holding stands at transit hubs, protesting in front of Novartis headquarters and organizing drives on his Instagram account, which now has 22,000 followers.

The worldwide distribution of COVID-19 vaccines made it clear that it is difficult for poorer countries with less bargaining power to negotiate prices and access. Patients in these countries must travel to authorized countries to dispense the drug, and therefore must pay extra for a stay of several months.

For example, the total price tag for those arriving from abroad in the United States can go up to $3m. Residents of the US and a few dozen countries that have an agreement with Novartis can get insurance to cover the treatment.

no government approval

The Turkish Ministry of Health held talks with Novartis in 2020, but the talks failed.

Health Minister Fahrettin Koca told Turkey’s parliament’s budget committee last year that the ministry’s SMA scientific committee was still evaluating the drug’s effectiveness and had not received the requested information from Novartis.

“The sensitivity of the subject obliges us to act with the utmost care,” he said. “We cannot play with the hopes of these fragile lives.”

Turkey’s health ministry did not respond to multiple requests for comment.

Fikri and Serpil Tezkan, whose son Yigit was diagnosed with SMA type 1 at 60 days old, won a court case last year to import the gene therapy to Turkey. While the agency approving drugs in Turkey has added Zolgensma to its list, the Tejcan family says the ministry continues to block its importation.

Rather than wait, the couple went to Germany, where Yigit could be treated for free because Serpil is a German citizen. Now three years old, Yigit can grasp His neck is straight and is learning to walk. Her parents are struggling to access drug use in Turkey; He applied to the European Court of Human Rights this year to have it covered by the Turkish Social Security.

While Turkey already includes another SMA type 1 treatment, Spinraza, its effects only last for months before more doses are needed. Turkey now also covers genetic testing for couples getting married, and it covers their in vitro fertilization if they carry the gene.

Yusuf’s parents and their 11-year-old daughter have now been tested. He said that because of Spinraza, Yusuf was able to live so long, which is why he waited before deciding to try gene therapy. Yet Joseph is already three years past the recommended age for treatment. He was rejected by hospitals in the US and Germany, but a Dubai hospital has said it will treat him as long as he stays below the 13.5 kg (30 lb) limit.

Until they reach their goal, Mensure rations the milk she feeds Joseph so that he does not gain a pound.

Had he gone over the limit, the provincial government would not have extended his fundraising campaign for another year.

Other families are having a hard time just starting campaigns: Since the Ministry of Health doesn’t recognize Zolgensma’s effectiveness, they can only launch them through a court case.

“It was found unfavorable to organize campaigns in our country to carry out gene therapy, for which there is not yet sufficient evidence in terms of efficacy and safety,” Coca said in a statement last year.

When asked about the Turkish minister’s claims, Novartis pointed to several studies showing Zolgensma’s effectiveness. While Novartis said it could not comment on Turkey’s regulatory processes, it said that “no matter the country”, it aims to find “sustainable access solutions for transformative gene therapies like Zolgensma”.

Mehmet Rıfat Bacanlı, the lawyer of the Tezcan family, has opened 15 cases against the Ministry of Health to shut down the campaigns.

“The money these families collect is not sustainable, as the number of children diagnosed increases every day,” Backnally said. Last year, he said he knew of about 12 fundraising campaigns; This year, because of more genetic testing and awareness, he said there should be more than 100.

More than a year into their campaign, Polats is finally close to reaching the end. Yet once they raise the full amount, the balance is uncertain. The Dubai hospital has not yet shared details on the procedure; For now, he says, they’ve only talked about money.

“We don’t know what the drug will do,” Kahan said. “But whatever it is, we want to get it. We have to. We have no choice.”

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