• Yusuf Polat, 5, has enough medical problems for a lifetime.
• His bones and muscles have deteriorated so terribly.
• Gene may arrest the disease’s progression in eligible babies and enhance motor abilities in one dose.

Yusuf Polat, 5, has enough medical problems for a lifetime.

His bones and muscles have deteriorated so terribly that he can’t walk, drink, or breathe properly, and he must be strapped to seven gadgets when he sleeps.

Yusuf’s parents had lived this nightmare. Melek, his older sister, died a year and a half after him.

His parents have a plan.

Yusuf has spinal muscular atrophy (SMA) type 1, a rare genetic disorder. A new medicine may arrest the disease’s progression in eligible babies and enhance motor abilities in one dose.

Onasemnogene abeparvovec, marketed as Zolgensma, is the world’s most costly medication at $2.1m. With rampant inflation in Turkey, this price becomes less attainable every day.

“Those who developed this medicine don’t prioritize helping people, treating children, or wiping the disease off the face of the world; they prioritize making money,” stated one of Yusuf’s “volunteer sisters” who met the family through their fundraising effort.

Basel-based Novartis has been criticized for its price. Novartis had planned to price it between $4m and $5m, but a medication price watchdog said $310,000 to $900,000 was fair.

Novartis says the drug’s innovative technology saves families a lifetime of medical visits.

Novartis Gene Therapies is expanding the treatment paradigm for spinal muscular atrophy (SMA) with a one-time therapy, which can save healthcare systems tens of millions of euros over a patient’s lifetime.

SMA affects one in 10,000 babies globally, thus there are fewer prospective customers.

In Turkey, it’s 1 in 6,000. As a genetic condition, it affects more offspring of related couples; these couples, like Yusuf’s parents, can typically least afford therapy.

Kayhan and Mensure Polat live in Gebze, one of Turkey’s manufacturing centers. He resigned his job to fundraise and see doctors, causing the family to lose health insurance.

Yusuf keeps a smiling face for his guests and the camera: his grin is on billboards and online photographs that offer his narrative and bank account number to help people give to his treatment.

Mensure: “We need money.” “Some donate little, others pass by. It’s a tough spot.

Yusuf’s parents launched the campaign in September for 16 million Turkish lira. Since then, the lira has fallen against the dollar, bringing the price to $1.83m. The decreasing lira raises their objective and tightens their finances.

Ceylan: “We can’t reach the wealthy.” “Had Yusuf been a politician’s kid, the campaign would have concluded in a week.”

Some parents persuade celebrities or politicians to support their campaigns, like Istanbul Mayor Ekrem Imamolu. A group of them is seeking to meet with the president. Without such links, Yusuf’s campaign has concentrated on concerts, protests at transit hubs, and drives on his Instagram account, which has 22,000 followers.

Poorer countries with less bargaining power have a tougher difficulty negotiating rates and access. These patients must go to nations authorised to administer the medicine and pay for several months of lodging.

In the U.S., foreigners can pay up to $3m. US and other countries with Novartis agreements cover the treatment.

In 2020, Turkey’s health ministry negotiated with Novartis.

Health Minister Fahrettin Koca told the Turkish parliament’s budget committee last year that the SMA Scientific Committee is currently examining the drug’s effectiveness and has not received information from Novartis.

The topic’s sensitivity requires extreme caution, he said. “We can’t gamble with their hopes.”

The Turkish health ministry did not comment.

Fikri and Serpil Tezcan won a court case last year to import gene therapy for their SMA type 1 son Yiit. Zolgensma was added to Turkey’s list of approved pharmaceuticals, but the Tezcan family alleges the ministry still blocks its importation.

Instead of waiting, the pair flew to Germany, where Serpil may cure Yiit for free. Yiit is 3 years old and starting to walk. His parents continue to struggle for access to the drug in Turkey; they asked the European Court of Human Rights to cover it this year.

Turkey already covers Spinraza for SMA type 1, but its effect lasts just months. Turkey provides genetic testing for engaged couples and IVF if they carry the gene.

Yusuf’s parents and 11-year-old daughter were tested. They said Yusuf’s long life was due to Spinraza, so they waited to try gene therapy. Yusuf is three years past the treatment age. A hospital in Dubai will treat him as long as he keeps below 13.5kg (30 pounds).

Mensure rations Yusuf’s milk until they accomplish their objective, so he doesn’t gain 500 grams.

If he’d gone over, the province wouldn’t have prolonged his fundraising campaign.

As the health ministry doesn’t recognise Zolgensma’s success, other families can only initiate campaigns in court.

“Organizing gene therapy campaigns in our country was deemed unfavourable due to insufficient efficacy and safety evidence,” Koca stated last year.

Novartis pointed to studies showing Zolgensma’s effectiveness when challenged about the Turkish minister’s assertions. Novartis said it couldn’t comment on Turkey’s regulatory processes but hoped to establish “sustainable access options for breakthrough gene treatments like Zolgensma”

Mehmet Rfat Bacanl has initiated 15 charges against the health ministry for shutting down campaigns.

Bacanl: “The money these families earn is unsustainable because the number of diagnosed babies climbs daily.” Last year, he knew of 12 fundraising efforts; this year, possibly due to greater genetic testing and awareness, there must be over 100.

The Polats’ campaign has lasted more than a year. Even after they raise enough, the future is uncertain. The Dubai hospital hasn’t shared procedure details; they’ve simply discussed money.

Kayhan remarked, “We don’t know the drug’s effects.” We want it everything. Must. We’re stuck.”

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