At the Russian Children’s Clinical Hospital, the first patient from Astrakhan has received an innovative therapy for Hunter syndrome (mucopolysaccharidosis type II). Thanks to the unique opportunity provided by the Krug Dobra Foundation for children in need – and in accordance with the decisions of the foundation’s expert council, which includes leading medical specialists – Russia has become only the second country in the world to commercially offer this technology, following Japan.

Enzyme replacement therapy (ERT) with recombinant human idursulfase has been used to treat mucopolysaccharidosis type II (MPS II) since 2006. Patients receive the drug intravenously once a week; however, this route of administration does not allow the enzyme to cross the blood–brain barrier and affect the brain. As a result, patients with the neuropathic form of the disease continue to experience progressive damage to the central nervous system.

Hunterase® Neuro* (idursulfase beta) is the first and only registered ERT in the world that delivers the enzyme directly into the brain’s ventricles. Intracerebroventricular administration via an Ommaya reservoir allows the therapy to bypass the blood–brain barrier and address the neurological manifestations of MPS II.

The port implantation surgery and the first infusion were performed by Dmitry Aleksandrovich Reshchikov, a neurosurgeon at the Russian Children’s Clinical Hospital (RCCH).

“Today marks the launch of a new type of treatment for patients with the neuropathic form of mucopolysaccharidosis type II. Until now, children received the medication intravenously. Now the enzyme is delivered exactly where it needs to go and begins working immediately,” the doctor noted. “We hope this will be a major step forward in the quality of care for these patients.”

Clinical trial data** show that the use of Hunterase Neuro in combination with standard intravenous ERT can stabilize or improve neurological outcomes in patients with the neuropathic form of the disease.

Russian patients have gained access to this therapy thanks to the long-standing partnership between Nanolek and South Korea’s GC Biopharma.

An important aspect of introducing this new therapy is that patients will be able to receive treatment in their home regions. To make this possible, doctors from the Astrakhan region underwent specialized training at the RCCH. The first administration of the drug was carried out together with them, after they had completed both practical and theoretical training.

“We witnessed a crucial moment and are very glad that our patients can now receive such innovative medicines,” said Yulia Aleksandrovna Romakhova, a pediatrician from Znamensk. “We were able to attend both the port implantation procedure and the first drug administration. We were very impressed by how well the entire process was organized and explained. We gained both theoretical and practical knowledge, which is extremely important in our profession for continuous improvement. Now we are able to better support our patients going forward.”

The training of regional doctors was organized with the support of the Expert Assistance Center ‘House of Rare’. This work is an essential part of implementing innovative treatment methods in Russia, contributing not only to improving the qualifications of medical specialists but also to the successful application of new therapies in practice.

*https://grls.minzdrav.gov.ru/Grls_View_v2.aspx?routingGuid=a92a7222-c0b4-4c22-bcdf-0049154c3105

**Seo JH, Kosuga M, Hamazaki T, Shintaku H, Okuyama T. Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II. Mol Ther Methods Clin Dev. 2021.

**Joo-Hyun Seo, et al Intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysacchar idosis type II: Final report of 5-year results from a Japanese open-label phase 1/2 study, 2023.