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Why Doctors Are Trying A Skin Cancer Drug To Treat A Brain Tumor


3 Star


Why Doctors Are Trying A Skin Cancer Drug To Treat A Brain Tumor

Our Review Summary

The article briefly recounts the story of MaryAnn Anselmo and her experience with genetic targeting to treat her glioblastoma. NPR’s story is a nice primer on genetic targeting for cancer treatment, and how it can, in some patients, prove more effective and tolerable than standard cancer treatments. By zeroing in on one mutation in one person, the reader gains an understanding of genetic targeting’s promise. However, by not mentioning other types of targetable mutations, or the associated costs, or the larger base of evidence that is relevant to this approach, the piece could give readers an overly simplistic and incomplete understanding of genetic targeting. There are, in fact, more than 300 genetic variations in solid tumors (e.g. brain cancer) known to have some response to targeted treatments–BRAF is only one of them–and the costs can vary astronomically, depending on the drug used. Some acknowledgment of that context, and the studies that have already investigated genetic targeting, would have strengthened the story considerably.


Why This Matters

Glioblastoma and it’s highest-grade form, glioblastoma multiforme (GBM), is a very aggressive, deadly class of malignant tumor. Such cancers make up 12-15 percent of all brain cancers, and they arise from mutations in the connective, glue-like tissue of the brain, called the glia. Glial cells support and nourish the brain’s thinking cells (or develop into them)–they’re both well-nourished by blood vessels and can divide very rapidly. This is why only 40 percent of all people with glioblastoma today survive more than 2 years after they’re diagnosed, with the prognosis being worse for adults (30 percent survive 2 years) than for children. About 10 percent survive five years or more, but typically with significant deficiencies caused by the cancer, treatments, or both.

Surgery, radiation, and chemotherapy are the three modern standards of treatment for glioblastoma (as well as other solid tumors). Trouble is, they’re likely to harm healthy tissue as well as cancerous tissue–so researchers have sought new and different ways to target and destroy only cancer cells to keep the patient healthy, extend their survival, and/or improve their quality of life. With the lowering cost of sequencing a person’s entire genome–i.e. what used to cost $2.7 billion in the 1990s can now be done for less than $1,000–researchers and practitioners are increasingly turning to this information to identify and exploit genetic targets in cancerous tissue.


Does the story adequately discuss the costs of the intervention?

Not Satisfactory

The article generalizes some costs, e.g. “Zelboraf is expensive,” but no hard numbers are shared. (E.g. As of 2011, a common, six-month course of Zelboraf costs about $56,000.) With costs varying widely–and astronomically–among treatments, and name-brand vs. generic, this is critical and pertinent information to include. Another missing cost-related discussion regards the oral chemotherapy drug called temozolomide (which was alluded to in the article but never named). Temodar, the name-brand form of the drug, costs about $100,000 a year, while the generic alternative costs less than $10,000 a year–a 10-fold difference. The cost of genetic testing ($4,000 sequencing costs per tumor) was mentioned in the on-air piece but not in the online body copy.

Does the story adequately quantify the benefits of the treatment/test/product/procedure?


The benefits are described as extending a person’s survival while increasing her quality of life, and a number–1 year, so far–is attached to the treatment. The story also includes this quote from a researcher, which clarifies that the treatment is not expected to work indefinitely: “We all have patients that have been on these drugs for years. But I don’t know, I mean, I think if I was being honest eventually our expectation would be that it would stop working.”

We’ll call this good enough for a Satisfactory rating, but it would help to have more context here. For example, the article could have stated the median survival for the typical adult diagnosed today with glioblastoma, which is about 15 months. It would also be useful to know if there are studies of patients similar to the one profiled here, and what their experience has been. That’s a deficiency we’ll address below in the Evidence section.

Does the story adequately explain/quantify the harms of the intervention?


The article describes only one side effect of vemurafenib, the drug that targets the BRAF mutation in cancer. (“She’s lost some peripheral vision, though she’s been able to compensate.”) However, the piece does link to the NIH-run MedlinePlus description of the drug, including a full list of side effects and other important information–for a cursory online story, this is helpful and very responsible.

Does the story seem to grasp the quality of the evidence?

Not Satisfactory

This piece states “The pills have kept her cancer from growing,” quotes the patient’s neurooncologist on its efficacy (and limitations), and explains how the drug is part of a clinical trial. Together, this implies an appreciable quality of evidence for the drug in question. But the story never directly discusses or references any of that evidence. Nor is there any discussion of the evidence supporting genetic targeting as a potential new standard of care — the primary aim of the piece. The story notes that this patient is enrolled in a new kind of trial known as a basket trial — it would have helped to see a couple of sentences briefly describing the evidence from other such studies and its quality/efficacy, perhaps with other cancers or gene-drug combinations. As it stands, the story is a one-patient anecdote detached from any broader context of evidence.

Does the story commit disease-mongering?

Not Satisfactory

The opening quote from the patient profiled in this story is dramatic:”‘Oh my God, you’re history.’ It’s like a death sentence.”  Given the poor survival statistics associated with this type of cancer, that type of reaction is certainly understandable. But if a story is going to lead with an alarming statement like that, we’d also expect it to provide some backing in the form of dispassionate, evidence-based figures about survival. Otherwise it’s not clear whether patients typically have weeks or years to live following a diagnosis.

Does the story use independent sources and identify conflicts of interest?


Both sources quoted in this story–the patient and her doctor–have interests that are arguably aligned. It would have been helpful to hear from a second neurooncologist who has experience with the disease, drug, and/or genetic targeting technique. However, because the neurooncologist clearly describes the limitations of highly targeted cancer-treating drugs, i.e. how they can and probably will eventually resist the drug, we’ll rate this sourcing satisfactory.

Does the story compare the new approach with existing alternatives?


At least on one alternative is mentioned, e.g. “traditional chemotherapy” (Temodar/temozolomide), so we’ll rate the story satisfactory. However, it’s important to note that radiation therapy is not discussed at all, and it is part of the standard of care alongside chemotherapy and surgery. There are other potential alternatives, too, including drugs that limit blood flow to cancerous tissue and immunotherapy techniques (aka “cancer vaccines”).

Does the story establish the availability of the treatment/test/product/procedure?

Not Satisfactory

The skin cancer drug this patient received is stated to be available through a clinical trial, at least for GBM. The article also links to NIH-curated information about the drug. But this story is mainly about cancer treatment that is guided by genetic testing of the tumor, and the availability of that testing and related treatment is not satisfactorily established. The story states that the patient’s family sent samples of her tumor to several leading cancer hospitals, suggesting that this is not something that would’ve been done as a routine part of her care. But what about patients whose families may not be savvy enough to coordinate this kind of testing? And how much does it cost and would the testing be covered by insurance? This story is going to create interest and demand for such testing, so a few more details about the logistics of having it carried out would have been very useful.

Does the story establish the true novelty of the approach?


The fact that GBM patients respond to a drug commonly used in skin cancer patients (i.e. those with the BRAF mutation) is novel and clearly portrayed. It would have strengthened the article, however, to more clearly explain how genetic testing can generate new leads in treating cancer by briefly expanding on other types of mutations in cancer and the drugs that can target them.

Does the story appear to rely solely or largely on a news release?


The article went beyond any press release.

Total Score: 6 of 10 Satisfactory


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