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Research Groups Fund Phase III Trials of Pfizer's Lorbrena in Pediatric Neuroblastoma

NEW YORK – Children's Hospital of Philadelphia (CHOP) and Gustave Roussy in France have received $1.3 million to conduct parallel Phase III trials of Pfizer's ALK inhibitor lorlatinib (Lorbrena) in ALK-mutated, pediatric neuroblastoma patients.

The grant was awarded by a group of research charities, led by Solving Kids' Cancer UK and Solving Kids' Cancer US. The funds will be split between CHOP and Gustave Roussy and spread over four years.

The lorlatinib trials were developed by the Children's Oncology Group (COG) and the International Society of Pediatric Oncology Europe Neuroblastoma Group (SIOPEN). The trans-Atlantic collaboration will evaluate lorlatinib in combination with the standard treatments developed by COG and SIOPEN in ALK-mutated pediatric neuroblastoma patients.

Approximately 14 percent of newly diagnosed, high-risk neuroblastoma patients have mutations in ALK, previous research has found. Neuroblastoma is also the most common cancer among infants, with approximately 1,500 children in North America and Europe diagnosed annually with a high-risk form of the disease that necessitates aggressive treatment.

"Only one new drug has been approved by the [US Food and Drug Administration] for neuroblastoma in the past 30 years," Yael Mossé, a neuroblastoma specialist at CHOP, said in a statement. "We hope this collaboration between North American and European researchers will bring more targeted therapies to children newly diagnosed with this disease.”

Lorlatinib is already approved in the US as a second- and third-line treatment for advanced non-small cell lung cancer patients with ALK mutations, and Pfizer is seeking approval for the drug as a first-line lung cancer treatment based on results from the Phase III CROWN trial. In that study, metastatic lung cancer patients whose cancer had spread to the brain experienced an 82 percent response rate with the third-generation ALK inhibitor compared to 23 percent for those on the first-generation ALK inhibitor crizotinib (Pfizer's Xalkori).