This story presents the early findings of a new drug shown to reduce tumor size in two rare cancers: advanced basel cell carcinoma and medulloblastoma. While the drug showed good response rates, the story reminds the reader that this is a new drug in the early stages of testing and its true efficacy is currently unknown. More information regarding what “response” really meant in this case, and more about the potential harms would have strengthened the piece.
The drug has only been tested in a phase I trial and a discussion of cost is not applicable at this time.
The story indicates that 55% of patients (or 18 out of 33) with advanced basel cell carcinoma responded to the treatment. Only one person with medulloblastoma was enrolled in the study and the story adequately describes this case. One point we wish the story had addressed: what is meant by "response?" Typically it’s a standard percentage shrinkage on an imaging study. What did it really mean in this case?
The story states that the drug is associated with “few significant side effects,” however, the specifics of these side effects are not provided. Six patients in the advanced basel cell carcinoma study experienced 8 grade 3 adverse events, including fatigue, hyponatremia, muscle spasms, and atrial fibrillation. One patient withdrew from the study citing adverse effects.
The writer makes it clear that this is a small, experimental study that enrolled only 33 patients with advanced basel cell carcinoma and 1 patient with medullablastoma.
This story does not engage in disease-mongering. It clearly states that medullablastoma and advanced basel cell carcinoma are rare forms of cancer, a percentage of which involve the pathway targeted by the new drug.
This story provided comments from co-authors of the two studies, as well as the author of the accompanying editorial.
This story briefly mentions chemotherapy, surgery and radiation as treatment options for cancer.
The story makes it clear that this drug is still in the experimental phase and the FDA has not yet approved it.
This is story makes it clear this is a novel drug in the early phase of testing.
There does not appear to be a press release associated with this story.