Doctors are not ready to treat a child with Spinraza after trying to use Zolgensma

In the Sverdlovsk region, doctors canceled treatment with Spinraza for three-year-old Misha Bakhtin, who suffers from SMA. Previously, his parents themselves refused therapy, trying to use Zolgensma. When the treatment didn’t work, the family decided to go back to Spinraza. But now doctors refuse to use this drug, since its effectiveness and safety after using Zolgensma have not been proven. The state fund “Circle of Goodness” explains that therapy using two drugs has experimental status, so the fund cannot pay for it.

A council of doctors and employees of the Ministry of Health of the Sverdlovsk Region decided to cancel treatment using the expensive drug Spinraza, which was prescribed to three-year-old Misha Bakhtin with spinal muscular atrophy (SMA). About it “Kommersant-Ural” said the boy’s father, Dmitry Bakhtin. According to him, the consultation took place on October 27, but the parents were not even notified about it.

Let us recall that in the fall of 2020, Misha Bakhtin, by decision of the medical commission, received from the regional Ministry of Health two ampoules of the drug Spinraza, which helps stop the development of the disease. Then philanthropists helped buy two more ampoules. Afterwards, Evgeny Roizman’s foundation (Evgeny Roizman is recognized as a foreign agent) opened a collection for the drug “Zolgensma” worth 160 million rubles.

Spinraza is significantly cheaper, but requires six injections in the first year of treatment and then every four months throughout your life. In turn, Zolgensma infusion is performed only once.

By the end of 2020, the foundation managed to collect the necessary amount, and the child received an infusion of Zolgensma. But sufficient positive dynamics did not happen, so the parents turned to the Regional Children’s Clinical Hospital (RCCH) with a request to continue treatment. However, the Sverdlovsk Ministry of Health and the hospital stated that after the Zolgensma injection it is not advisable to use Spinraza. Then the parents went to court. The Bakhtins also recorded a video message to the head of the Investigative Committee of Russia, Alexander Bastrykin, and the child’s father, Dmitry Bakhtin, went out with single pickets on Red Square. As a result, the court ordered doctors to “immediately begin treatment.” In addition, a case was initiated under Part 1 of Art. 293 of the Criminal Code of the Russian Federation (“Negligence”) in relation to CSTO employees.

Regional Minister of Health Andrey Karlov claimed, that the drug “Spinraza”, by court decision, was indeed purchased by the ministry, but they cannot yet use it. “In the structures of the Ministry of Health of the Sverdlovsk Region, in the Sverdlovsk state hospitals today there is not a single doctor ready to give the boy an injection. The reason is the same – in Russia and in the world there are no evidence-based cases of safe use of Spinraza after the drug Zolgensma. A decision by the federal council is necessary,” Mr. Karlov explained earlier.

The legal representative of the family, Yulia Lipinskaya, told Kommersant-Ural that the Bakhtins learned about the regional consultation only after the fact: “We received a paper stating that the Ministry of Health applied to the Kirovsky District Court of Yekaterinburg in order to stop enforcement proceedings regarding the provision of medicines, and there the decision of the council was discussed.”

She insists that the decision of the regional council has no legal force. According to her, the Bakhtins, the child’s legal representatives, indicated that without their participation no events affecting the interests of their son could be carried out. “This is an absolutely cannibalistic story that has no moral standards regarding a disabled child in need of life-saving medicine,” added Dmitry Bakhtin.

To prevent the child’s condition from worsening, it was decided by a neurologist to purchase the drug “Risdiplam” on a commercial basis – at the expense of funds and parents. “It is the same drug as Spinraza, but you can buy it and it is given as a syrup orally. Misha has been taking this drug for a year and three months,” Dmitry Bakhtin told Kommersant-Ural. He claims that the child is showing positive dynamics and there are no side effects.

Now parents are waiting for a new council of federal centers (Russian Children’s Clinical Hospital, NCCH, Veltishchev Research Institute of Pediatrics), which should decide whether it is possible to provide the child with the drug Risdiplam. The date of its holding should become known in the coming days, says Dmitry Bakhtin.

“If illegal actions from the healthcare system continue, then again there will be exits to Red Square and to the building of the Ministry of Health of the Russian Federation. I will not go anywhere, I will stand day and night until my child is given the medicine,” he said.

Kommersant-Ural sent a request to the head of the Ministry of Health of the Sverdlovsk region, but did not receive a response.

Kommersant also turned for comment to the Circle of Goodness state fund, which provides therapy to children with SMA. They reported that tandem therapy – taking the drug Evrysdi (Risdiplam) or administering the drug Spinraza after infusion of Zolgensma – has experimental status. “At the moment, there is no data on international or Russian clinical studies of the effectiveness of tandem therapy; the foundation has no right to pay for such therapy,” the organization’s press service reported, adding that the Bakhtins themselves did not contact them.

The fund noted that Zolgensma is a “very complex and relatively new” drug. Research into its long-term effects on the child’s body is still ongoing. The manufacturer’s instructions approved by the Ministry of Health of the Russian Federation indicate that the experience of using Zolgensma in combination with other drugs for the treatment of SMA is “limited.” Clinical guidelines for the treatment of SMA approved by the medical department do not provide for tandem therapy. “The logic is simple: the public budget should be used only for proven treatment methods, and experimental methods around the world are tested at the expense of pharmaceutical manufacturers. The foundation cannot pay for such experiments at the expense of Russian taxpayers,” the foundation concluded.

Maria Ignatova, Yekaterinburg; Natalia Kostarnova