Release on potential therapy for rare bleeding disorder gets points for restraint
Rating
Gene therapy for rare bleeding disorder achieves proof-of-concept
Our Review Summary
Credit: UC Davis Comparative Oncology Program
This short news release describes a gene therapy experiment in four dogs that might turn out to help human patients with a rare blood disorder known as Factor VII deficiency. The release does a good job of making clear that this is just a proof-of-concept and needs human trials. Great specific language is used to make that clear. But it does not give any context for the problems of developing gene therapies for rare diseases. Companies may balk at developing a treatment than can’t be sold very widely, which contributes to high costs.
Why This Matters
Gene therapies are emotional minefields for patients and families, especially since so much has been promised and actual successes have been few. One gene therapy offered in Europe has a price tag of $1 million. This release would serve readers better if it gave some of that daunting context. A Washington Post story explains some of the challenges: “No [gene] therapy is approved yet in the United States, so discussions about price — as well as crucial questions about how much patients will pay directly — are hypothetical. But industry leaders are already talking about ways to get ahead of potentially massive one-time price tags that could make insurers and patients balk.”
Criteria
Not Applicable
The news release does not bring up costs, but we’ll rate this Not Applicable since the release is clear that this is early-stage work that is not yet ready for use in humans. Some mention of costs would have been valuable nonetheless. Gene therapy is known to be extremely costly, and when a disease is rare, as this is, that raises questions about what company would find it profitable to pursue the potential treatment. The University of Utah provides some excellent background on the “commercial viability” of gene therapy.
Satisfactory
The release squeaks by on this. There are no numbers used to quantify, but this excerpt at least touches on the method of measurement and the three-year follow up. “The treated dogs had expressed levels of clotting factor VII that would be therapeutic in humans, with long-term stability. In one dog, the effects persisted nearly three years.” The release could have explained what a “therapeutic” expression level of clotting factor actually means. A closer look at the paper reveals that a clinically therapeutic level is greater than or equal to 15% of normal.
Satisfactory
The release addresses harms in a couple of places, as in this excerpt:
“The FVII-deficient dogs tolerated the initial gene therapy infusions very well and have had no adverse side effects over several years of follow up.”
and in a quote by the lead researcher:
“Based on kidney function, liver function, and blood measurements in the dogs, the treatment was safe, and did not elicit unwanted immune responses.”
But as this study was done in canines, we would have liked a bit more detail on how harms might manifest in human clinical trials.
Satisfactory
The limited information given in the news release describes the quality of the evidence, particularly the fact that this was an animal study that is not directly applicable to humans. The release notes that the study “sets the stage for clinical trials in humans.” The release could have noted that a study with only four (canine) participants is very small.
Satisfactory
There is no disease mongering.
Satisfactory
There were no conflicts identified. All the funding sources were listed.
Satisfactory
The release tells us that this rare clotting disorder is currently treated by infusions of clotting factors. The release could have done a better job of explaining how difficult that therapy is for patients. Is the disorder regulated well by that method? We aren’t given any information or what those costs are vs. what a one-time gene therapy might cost.
Satisfactory
The release makes clear that this treatment will not be available until human clinical trials are conducted.
Satisfactory
This gene therapy is novel for this particular condition. The news release should also be commended for pointing out that a similar gene therapy approach has been tested by the same researchers for hemophilia. It’s admirable that they are not overselling the novelty of this approach by referencing their own previous work in related areas.
Satisfactory
We were pleased to see proof-of-concept in the headline, because this is a very useful way to immediately alert readers that an idea has been validated, but not a therapy for human patients.
Total Score: 9 of 9 Satisfactory
Comments
We Welcome Comments. But please note: We will delete comments left by anyone who doesn’t leave an actual first and last name and an actual email address.
We will delete comments that include personal attacks, unfounded allegations, unverified facts, product pitches, or profanity. We will also end any thread of repetitive comments. Comments should primarily discuss the quality (or lack thereof) in journalism or other media messages about health and medicine. This is not intended to be a forum for definitive discussions about medicine or science. Nor is it a forum to share your personal story about a disease or treatment -- your comment must relate to media messages about health care. If your comment doesn't adhere to these policies, we won't post it. Questions? Please see more on our comments policy.
Our Comments Policy
But before leaving a comment, please review these notes about our policy.
You are responsible for any comments you leave on this site.
This site is primarily a forum for discussion about the quality (or lack thereof) in journalism or other media messages (advertising, marketing, public relations, medical journals, etc.) It is not intended to be a forum for definitive discussions about medicine or science.
We will delete comments that include personal attacks, unfounded allegations, unverified claims, product pitches, profanity or any from anyone who does not list a full name and a functioning email address. We will also end any thread of repetitive comments. We don”t give medical advice so we won”t respond to questions asking for it.
We don”t have sufficient staffing to contact each commenter who left such a message. If you have a question about why your comment was edited or removed, you can email us at feedback@healthnewsreview.org.
There has been a recent burst of attention to troubles with many comments left on science and science news/communication websites. Read “Online science comments: trolls, trash and treasure.”
The authors of the Retraction Watch comments policy urge commenters:
We”re also concerned about anonymous comments. We ask that all commenters leave their full name and provide an actual email address in case we feel we need to contact them. We may delete any comment left by someone who does not leave their name and a legitimate email address.
And, as noted, product pitches of any sort – pushing treatments, tests, products, procedures, physicians, medical centers, books, websites – are likely to be deleted. We don”t accept advertising on this site and are not going to give it away free.
The ability to leave comments expires after a certain period of time. So you may find that you’re unable to leave a comment on an article that is more than a few months old.
You might also like