A groundbreaking new delivery method for Brineura (cerliponase alfa) is transforming treatment for patients with CLN2 disease—commonly known as late infantile Batten disease. This innovative chest port system is not only proving more stable than traditional scalp-based infusions, but it's also enhancing the overall quality of life for these individuals, according to a recent case series.

In a noteworthy report published in the journal *Child’s Nervous System*, researchers detailed their success with this new device, highlighting that none of the seven patients who received this chest port experienced any complications such as access issues or infections since implantation.

The Shift from Scalp to Chest: A Seamless Transition

The researchers proudly noted, “Our conversion of the traditional scalp port to a chest port was easily performed and has resulted in better quality of life for these patients, with no reported adverse reactions or complications since implantation.” This shift represents a significant advancement in the delivery of Brineura, which is essential for managing the debilitating effects of CLN2.

What is CLN2 Disease?

CLN2 disease is a genetic disorder characterized by the deficiency of the TPP1 enzyme, responsible for clearing cellular waste. Without this enzyme, toxic accumulations lead to various neurological symptoms including seizures, vision loss, and substantial motor difficulties.

Brineura, developed by Biomarin Pharmaceutical, is administered directly into the cerebrospinal fluid surrounding the brain. However, the traditional methods of delivery have burdensome implications, such as lengthy infusions that can last up to five hours every two weeks.

The Complications of Traditional Infusion Methods

Scalp-based delivery systems have been fraught with challenges, including infections, device replacements every four years, and various complications like blockage and leakage.

A Promising Alternative: Modified Chest Port System

The novel chest port system circumvents these issues by connecting a catheter directly to a port implanted under the skin on the chest. This allows for a smoother infusion process from within the body to the brain, significantly reducing the need for frequent, invasive scalp interventions.

Real-Life Success Stories

The study analyzed seven patients aged between 3 and 24, showcasing the effectiveness of the new system. The first patient, an 11-year-old boy who had undergone numerous scalp infusions, experienced relief post-implantation, marking over 160 subsequent treatments without complications.

Another success story includes an 8-year-old girl who, after her diagnosis at age 4, received the chest port and has since had over 100 successful accesses without any issues.

Positive outcomes continued with a 24-year-old man and his younger sister, both also diagnosed with atypical CLN2. They had faced serious complications with previous methods but are thriving with the new port system.

The other patients, a 3-year-old boy along with two further siblings aged 5 and 7, similarly exhibited no access problems after switching to the chest port system, showcasing its effectiveness across various ages.

A Safer Future for CLN2 Patients

The researchers concluded that this new method could significantly reduce device failure rates and infection risks, paving the way for a safer, more manageable treatment process for families battling CLN2 disease. This revolutionary approach offers hope for improving the experience and outcomes for many patients and their families moving forward.