A gene change may point to who else could try a skin cancer drug
Original title: PTCH1 mutations and tumor-agnostic clinical outcomes with hedgehog pathway inhibitors across cancer types.
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 was a look back at a small number of patients, not a real trial, so it only shows something worth testing next.
The short version
A small look at patients found that a drug used for one skin cancer helped a few people with other cancers, including one brain cancer.
What was studied. Researchers looked at 121,490 tumor samples to see how often a gene called PTCH1 was broken. They then checked 13 patients who did not have basal cell carcinoma and who were given a Hedgehog inhibitor, a drug already approved for that skin cancer.
What they found. A broken PTCH1 gene showed up in 2064 of the samples. That is 1.7% of them. Among the 13 patients treated with the drug, the response rate was 31%. Tumors shrank in some people with sebaceous adenocarcinoma, squamous cell lung cancer, skin squamous cell cancer, and glioblastoma: The fastest-growing type of glioma (grade 4). Treatment usually starts soon after diagnosis: surgery first, then radiation and chemotherapy. See the glossary.
What this means, and what it doesn't
What it could mean: If you have glioblastoma: The fastest-growing type of glioma (grade 4). Treatment usually starts soon after diagnosis: surgery first, then radiation and chemotherapy. See the glossary, this raises a question worth asking your doctor: whether your tumor has been tested for gene changes like PTCH1. It hints that a drug already used for a different cancer might help a small group of people whose tumors share this gene change.
What it doesn't mean: This does not mean there is a new treatment for brain tumors. Only 13 patients in total were looked at, and most did not respond. This was a look back at records, not a planned trial, so it cannot prove the drug works for glioblastoma: The fastest-growing type of glioma (grade 4). Treatment usually starts soon after diagnosis: surgery first, then radiation and chemotherapy. See the glossary. The drug is not approved for brain tumors. The researchers themselves say this needs to be tested properly first. This is not a cure, and it is a long way from everyday care.
Source: PubMed, July 17, 2026 · Read the original
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