A drug shrank one child's spreading neuroblastoma and delayed radiation
Original title: Lorlatinib-Containing Therapy for MYCN-Amplified, ALK-Mutated Neuroblastoma With Bulky Leptomeningeal Relapse: A Case Report.
How far along is this research?
- Lab cells
- Animals
- Review
- Tested in people
This was tested in people. That is the most reliable kind of research we share.
This is a report on just one child, not a study in many people.
The short version
In one toddler, a drug called lorlatinib shrank cancer spread and put off radiation.
What was studied. Doctors report on one toddler with neuroblastoma. The cancer had spread to the lining of the brain and spinal cord, and the child could not move their legs. The tumor had changes in two genes, MYCN and ALK.
What they found. The child got chemotherapy that included lorlatinib. Almost all of the spread in the brain and spine lining went away, and the child fully recovered and could move again. The cancer later came back in the brain, and radiation was given. But the first response let doctors wait 11 months before that radiation.
What this means, and what it doesn't
What it could mean: For some children with these gene changes, this drug may shrink cancer that has spread to the brain and spine. It may also let doctors wait before giving radiation to the whole brain and spine. That matters, because such radiation can cause lasting harm in very young children. Gene testing is what shows whether a tumor has these changes.
What it doesn't mean: This is a report on one child, not a study. One case cannot show that a drug works for others. It is not proof, and it is not a cure. The cancer did come back, and radiation was still needed in the end. Many more children would need to be studied before this becomes normal care. Talk with your own care team before changing any plan.
Source: PubMed, October 29, 2026 · Read the original
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