CAR T-cell therapy — Why it’s too soon to speculate about a ‘cure’ for multiple myeloma

An experimental immunotherapy treatment for multiple myeloma from a biotech company in China captured wide attention at the just-concluded American Society of Clinical Oncology (ASCO) meeting.

We’ve been following news reports from the meeting closely since it kicked off last Friday:

• High drug costs (and stock values) take center stage at final day of ASCO
• Small studies get big headlines at ASCO 2017
• 6 things to keep in mind if you read cancer-related news in the next few days

ASCO’s news release on the new treatment, a form of CAR T-cell therapy, noted that the phase 1 results are preliminary and that the small trial was short-term.  But then it blew past those cautions by speculating that the treatment could send multiple myeloma into long-term remission and might represent “a cure” for this incurable disease. The treatment costs weren’t included, either.

Phase I studies are mainly to assess safety, not benefits

Andrae Vandross, MD, a hematology/oncology fellow at UCLA and a contributor to HealthNewsReview.org, said the news release had some important details, such as a discussion of adverse events, particularly cytokine release syndrome (CRS), which affected many patients.

But “overall, I wish more attention was paid to the fact that this small trial was to examine safety and efficacy and that much more needs to be done to make statements regarding clinically meaningful impact on disease,” Vandross said.

The shortcomings of the news release can be found repeated in some of the news coverage.

Reuters’s story, “Chinese cell therapy effective in small multiple myeloma trial” didn’t explain harms well nor mention costs.

FierceBiotech’s article played up the “horse race” aspect of the pharmaceutical industry — where competing companies race to get media and investor attention for their preliminary research, “ASCO dark horse Nanjing Legend Biotech shines with promising CAR-T data.” But costs didn’t make it into the discussion.

Nor was the price tag mentioned in The Scientist’s piece, “CAR T-Cell trials boast promising results.”

Deborah Korenstein, MD, an internist at Memorial Sloan-Kettering Cancer Center, who did attend the meeting, found some of the reports on the CAR-T study lacking.

“None of these articles mentions cost, though CAR-T therapy is very expensive. They do note that the follow-up is short-term so far, though they also talk about long-term remission (in the case of the Reuters piece) or “cure” (in the ASCO press release), which at the very least sends a mixed message.”

NBC News’ piece, “CAR-T Cancer Approach Has Surprising Success in Multiple Myeloma,” reflects some of this mixed messaging. After reporting that the results were “impressive” and “remarkable” and that the study “brings hope” of a possible cure, the NBC story eventually broaches the issues of adverse effects and costs: “The treatment is expected to cost $200,000 to $300,000, and “who’s going to pay for that is a big issue,” according to a study author.

Help, not hype: 3 tips for reporting on early-stage research

What does Korenstein think news organizations and public relations people could do better?

Stick to the evidence at hand, avoid speculating about outcomes that haven’t been determined, and make sure that potential harms are described as thoroughly as benefits.

“Certainly there is no evidence so far to support those claims about longer term outcomes. They say it in a ‘hedgy’ way but to me they shouldn’t be mentioning it at all. They also play down the fact that the huge majority of patients had CRS, even if it was often not ‘severe’ – which is generally defined as requiring hospitalization. It’s not clear here what ‘severe’ means.”

Dr. Korenstein disclosed that her spouse consults for Vedanta Biosciences.