Lorlatinib found to be safe and effective for patients with ALK-driven relapsed/refractory high-risk neuroblastoma (63+% Response with or without chemo)

Medical Xpress / Children's Hospital of Philadelphia / Nature Medicine ^ | March 33, 2023 | Kelly C. Goldsmith et al

[ConservativeMind]

An international group of researchers has shown that the targeted therapy lorlatinib is safe and effective in treating high-risk neuroblastoma.

"The profound clinical responses seen in this trial, in a highly therapy-resistant, relapsed pediatric cancer population, allows us to now offer lorlatinib into frontline care for newly diagnosed patients with ALK mutated or amplified neuroblastoma, a population known to have inferior survival," said Kelly Goldsmith, MD. "This trial will truly change the paradigm of clinical care and improve outcomes for our neuroblastoma patients."

Neuroblastoma is an aggressive pediatric cancer that develops from early nerve cells, often appearing as a solid tumor in the chest or abdomen. The disease accounts for up to 10% of childhood cancer deaths, and survival rates are low—less than 50% of patients with the disease survive, and there is still no known curative therapy for patients.

In 2008, Dr. Mossé and colleagues discovered that the anaplastic lymphoma kinase (ALK) gene causes most cases of rare, inherited neuroblastoma.

…However, although crizotinib demonstrated impressive response rates in other ALK-driven cancers, children with neuroblastoma had a response rate of only about 15%.

After screening numerous anti-ALK agents, the researchers discovered in preclinical tests that lorlatinib, an ALK and ROS-1 inhibitor, surpassed results seen with crizotinib.

Researchers found that lorlatinib given alone or in combination with chemotherapy was safe and tolerable in pediatric, adolescent, and adult patients with relapsed/refractory ALK-driven neuroblastoma. Lorlatinib demonstrated clinical activity across patients of all ages harboring the three neuroblastoma-specific hotspot ALK mutations, including patients who had previously received other ALK inhibitors.

Approximately 30% of patients under the age of 18 responded to the drug, and approximately 67% of patients over 18 responded. Patients under the age of 18 had a better response in combination with chemotherapy, with 63% of patients responding to the combined treatment.

[ConservativeMind]

This is a cancer with few options, until now.

[ConservativeMind]

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