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University of Pennsylvania gene therapy shows promise in eradicating some blood cancers

University of Pennsylvania gene therapy shows promise in eradicating some blood cancers

Our Review Summary

Suggestions for improvement:

  • The story should have explained why we need to be cautious when evaluating results from just 10 people being presented at a medical conference.
  • It should have provided perspective from someone not directly involved with the research.
  • It could have mentioned costs, as the competing NY Times story did.
  • It should have made entirely clear that this is not ‘first line’ therapy for leukemia but rather highly experimental therapy for people who have not responded to conventional (proven) leukemia treatment and are expected to die of their disease, thus making the extreme risk acceptable.


Why This Matters

The experimental treatment being reported on here, which recruits the patient’s own immune system to selectively target cancer cells, represents an interesting new area of research with exciting preliminary results in a few patients with untreatable cancers. Even scientists can get caught up in the excitement surrounding this kind of research, which makes it incredibly important for journalists to dispassionately present a balanced take on the findings, including strengths and weaknesses, benefits and harms. Stories that don’t take this responsibility seriously risk inspiring false hope in people desperate for positive news.


Does the story adequately discuss the costs of the intervention?

Not Satisfactory

The story did not mention costs. While it’s too early for an exact figure, we know that this sort of treatment with tailoring the patient’s own cells is very expensive. The competing NY Times story went into detail on costs, reporting:

“But such drugs tend to be extremely expensive. A prime example is the Novartis drug Gleevec, which won rapid approval in 2001 for use against certain types of leukemia and gastrointestinal tumors. It can cost more than $5,000 a month, depending on the dosage.

Dr. June said that producing engineered T-cells costs about $20,000 per patient — far less than the cost of a bone-marrow transplant. Scaling up the procedure should make it even less expensive, he said, but he added, “Our costs do not include any profit margin, facility depreciation costs or other clinical care costs, and other research costs.”

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


The story focuses primarily on one girl’s miraculous recovery from leukemia. But unlike the competing New York Times piece, it did provide the balancing perspective of other patients receiving this experimental treatment, at least one of whom has not recovered completely. That gives a more realistic take on what the story calls the “roller-coaster learning curve” associated with the treatment. The story also largely refrained from using the hyperbolic language that was sprinkled throughout the Times’ account.

The reader comes away with the idea that this is highly experimental and that only a handful of sustained remissions have occurred from a handful of attempts.

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

Not Satisfactory

Although it discusses the potentially catastrophic side effects of acute treatment, the story did not explain that these patients have compromised immune systems following treatment and need ongoing therapy with immunoglobulins for an unknown amount of time. That’s a considerable omission.

The New York Times story, which we also reviewed, did a better job on this.


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

Not Satisfactory

The story is based on a case series of 10 patients that was scheduled to be presented at a medical conference. The story acknowledges that this is a “tiny” number of patients, but it never goes into any detail regarding the limitations and caveats that we should attach to this data — including the fact that medical conference reports may not be subject to rigorous peer review by other scientists prior to presentation.

Does the story commit disease-mongering?


The story mentions that treatment for childhood leukemia has an 85% success rate, so it’s clear that the case being discussed here is not a typical outcome.  That brief mention gives this story the edge over the competing NY Times story on this criterion.

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

Not Satisfactory

The only expert sources were physicians/researchers involved in the development of the experimental treatment or the care of patients receiving it. A perspective from someone not affiliated with this research would have helped identify the caveats and limitations that we felt were missing from this report.

Does the story compare the new approach with existing alternatives?


The story mentions chemotherapy and bone marrow transplants as treatment options for leukemia.

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


It’s clear from the story that this treatment is not yet available commercially.

But it would have been useful to include some information about whether and how someone might get enrolled in a clinical trial. Although it is unlikely that this program is ready to handle an influx of new cases, patients and parents might be desperate for any information on this question.

Does the story establish the true novelty of the approach?


The story mentions that 20 years of studies involving genetically engineering T cells preceded this research.

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


There is enough original reporting that we can be sure this story wasn’t based on a press release.

Total Score: 6 of 10 Satisfactory


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