Opinion: iPS cell pre-clinical data should be published before starting first human clinical trials

It is always exciting when a biomedical technology as revolutionary as iPS cells is on the cusp of being used in patients for the first time. In the last few days stories have reported about how a proposed clinical trial based on iPS cells has completed several regulatory steps and is closer to starting.

This is exciting, but the trial also has the potential for many risks. Just one risk stems from the fact that how cells behave in mice or other rodents or even in large animal models may not be how they turn out to behave in humans.

iPS cells, as do other pluripotent stem cells, have some characteristics in common with cancer cells as well and indeed actual cancer cells are frequent byproducts of the iPS cell production process.

Dr. Masayo Takahashi is a wonderful scientist who is leading a team (see more at the Laboratory for Retinal Regeneration and here on her Riken website) that is nearing the point of having all the needed regulatory approvals to start human trials of an iPS cell product for treatment of blindness. Reportedly, this trial could begin as early as later this year or early 2014.

This is extremely exciting research. I find it remarkable to see this technology advance, but I do have some concerns about whether the field is going too fast.

Reportedly, Dr. Takahashi presented some of her lab’s preclinical data at the recent ISSCR meeting that supports the notion that the iPS cell product has a strong safety profile.

In my opinion, given the momentous nature of the step of taking iPS cells into clinical trials, prior to initiating the human studies, the full breadth of pre-clinical data should be published.

There is no requirement that pre-clinical data be published (just made available to regulatory officials) before initiating a clinical trial, but I think publishing it would be the wisest and best course of action in this case.


I believe getting the pre-clinical data out there is crucial because of the historical importance of this step and the reasonable concerns about iPS cell product safety that are out there. More broadly there are very few studies that are clinically relevant that shine any light on iPS cell safety.

The pre-clinical data in this case should be published in an open-access format (e.g. even if it goes into a fire-walled journal like Nature, the journal and/or authors should make it freely available) as well so that there are no barriers to any scientists in the world reading the paper and giving thought to it.

What do you think?


4 thoughts on “Opinion: iPS cell pre-clinical data should be published before starting first human clinical trials”

  1. I tend to agree with Paul K. on this one. Paul F. makes a good point that forcing publication of preclinical studies might give away secrets to the competition, stifling the incentive to develop new therapies. But isn’t this what the patent system is for? As far as I can tell in oncology, the structures of drugs and preclinical data are usually released far in advance of FDA approval. The Institute of Medicine released a report last year in the wake of the Duke fraud scandal that speaks to the importance of a climate of openness when it comes to data seeding clinical trials.

  2. While publication of all of the preclinical data from Dr. Takahashi’s lab would be most welcome, it would also be most unusual. Publication of preclinical studies of therapeutics in development (small molecules, biologics and cells) normally lags behind the entry into clinical trials since disclosure of the design and results from these preclinical studies gives a tremendous boost to the competition, particularly if protection of intellectual property (IP) is at stake or where IP may difficult to acquire. Therefore, I feel that as long as Dr. Takahashi has submitted the required preclinical safety and efficacy data through the appropriate channels for regulatory and ethics review (IRB or ethics committee), I have no problem with the delay in publication. Regarding the historical significance, that will really come once human subjects have been treated and evaluated for response!

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