This AP story covers a recent study evaluating an experimental gene therapy, “chimeric antigen receptor T-cell therapy” or CAR-T, on 101 patients with a very specific type of lymphoma who had exhausted conventional therapies.
The story does well to point out the results of the study had some caveats–they were released by a company developing the treatment, the findings have not been published, nor have they been reviewed by independent experts. The story also does a good job discussing the risks of the therapy, and it included quotes from an independent expert.
The story would have been stronger if it had contrasted the company results with data from therapies that are currently in widespread use for this very small subset of aggressive lymphomas.
The fact that CAR-T therapy is being heavily invested in by major drug companies pushing for FDA approval, that preliminary results have been encouraging, and that many of the cancers that may respond to it are considered untreatable, makes any study on the subject enticing to readers, doctors and journalists. That’s reason enough to approach these studies – as this author did – with a balanced perspective that places an emphasis on independent sources, potential harms, study limitations, and –perhaps, most importantly — careful documentation of potential financial conflicts of interest.
The companies competing to bring this therapy to market have not said how much they will charge. The story acknowledges cost this way:
“Company officials would not say what the treatment might cost, but other types of immune system therapies have been very expensive.”
This is a barely passing satisfactory. Other news sources have brought up that, since CAR-T cell therapy is an individualized gene therapy, the cost may run into the hundreds of thousands of dollars per patient.
The story makes note of quantitative results related to tumor shrinkage, as well as remission data six months after treatment. It also pointed out that median survival in the study is not known.
There is one anecdotal quote by a patient who was facing potential stem cell transplant, but opted for CAR-T therapy, and went into complete remission and saw his tumors “shrink like ice cubes.”
This is a major strength of this article. It makes it clear CAR-T treatment is not without major risks which include: treatable anemia, brain toxicity, and a “dangerous condition where the immune system overreacts in fighting the cancer” (likely, “cytokine release syndrome”). Deaths directly caused by CAR-T therapy in this and other trials are mentioned.
The headline labels it a “major study.” That’s a subjective term. We need to know was the study randomized, blinded and controlled? What did the control group receive? If there was no control group, the story should caution readers as to the limitations of the study design (something that didn’t occur here).
That being said, the story deserves praise for mentioning two important limitations. First, it notes that the study is company-funded, unpublished, and the results have not been peer-reviewed. Second, an independent source provides the important caveat that follow-up beyond 6 months is a critical piece of missing information in judging how beneficial the treatment is.
The story did not disease monger. These lymphomas are serious and aggressive forms of blood cancer.
The story notes that the study was funded by Kite and it quotes two outside sources. First, Dr. Roy Herbst of the Yale Cancer Center, who has not disclosed any relationship with Kite Pharma. Also quoted is Dr. Steven A. Rosenberg of the National Cancer Institute “who had no role in Kite’s study” but who has had T-Cell immunotherapy research funded by Kite Pharma within the past year (a fact not disclosed in the piece).
The story makes a good faith effort to provide independent, objective perspectives on the research. However, to make a point of saying Dr. Rosenberg had no role in “this” study but not mention he has been funded by Kite Pharma in the past for similar research is enough of a red flag to warrant an unsatisfactory mark for potential conflict of interest.
There is passing mention of chemotherapy, as well as stem cell transplant, and the story does note that these patients have exhausted all other available options. However, there is no mention of what the current accepted treatments for these specific lymphomas are. Furthermore, a clear understanding of treatment alternatives is compromised because the story does not clearly state that CAR-T therapy is currently only being tested for Acute Lymphocytic Leukemia (ALL) in children and young adults, as well as some B-cell lymphomas and leukemias. Without knowing this the reader can not answer the obvious: “what exactly does CAR-T treat and what are the alternatives?”
The story mentions this is an “experimental gene therapy” and that, if approved, it could become the first such treatment to be available in the United States. The story notes that the company is seeking approval for the drug by the end of March. While this is sufficient for a satisfactory grade, we think the story could have elaborated on this a bit, and noted that the approval would be based on preliminary phase 2 data — not the typical phase 3 study data that the FDA normally requires for approval. The drug appears to be eligible for expedited approval based on the FDA’s “breakthrough” therapies program.
The story immediately states that this is an experimental therapy and that there are no approved gene therapies currently available in the United States. It would have helped readers to know that over a dozen medical centers across the country are entering their second year of Phase I or II trials with CAR-T.
The story included the perspective of one independent source and did not appear to rely heavily on the news release.