Signals Blog

As with all of my posts that relate to anything that might end up being a medical treatment, it is important to be very clear that I am scientist and not a physician.  My comments are my own opinion and based on my experience as a stem cell biologist over the last 10+ years.

This blog carnival is about a series of laws in the United States described as Right to Try laws and they are rapidly emerging from U.S. lawmakers – 37 states now have a Right to Try law. These laws are meant to give patients the right to receive treatments that have been through Phase 1 clinical trials (the safety phase), but are not yet approved by the Food and Drug Administration (FDA). They are controversial amongst policymakers for numerous reasons and some have claimed that they are toothless or “feel good” legislation because they do not mandate drug companies to provide the therapy.

In this post, I will explore some of the main issues with the overarching theme of needing to give permission in some cases for non-approved experimental therapy whilst capturing the data in a public manner to inform future efforts.

The “feel good” bit can be bad

First thing first: people who are dying or rapidly declining deserve to have an option to try a legitimate therapy. I think the difficult part of “right to try” therapies is determining what might be a legitimate therapy and what is snake oil (especially in a private system where big dollars are involved). The good news on this front is that the majority of Right to Try legislation is related to therapies that have passed through Phase 1 clinical trials (e.g., they are safe to use), but this certainly doesn’t mean that they have a reasonable chance of working, meaning that there is ample room for people to peddle therapies that do absolutely nothing in a patient – and walk home with bags of cash.

Right idea, perhaps not the best approach…?

The spirit of these laws and the majority of arguments for them are to give dying people “one last chance” to treat their disease. This is an easy rallying cry for people to get behind; who wouldn’t want “one last chance” after all? The problem, however, is that some treatments and therapies are pretty much guaranteed not to work (and have solid evidence to support this lack of efficacy) and these laws do very little to establish a framework to give people enough information about these likelihoods. The laws do not discriminate and virtually give people the right to try anything, so long as they can find access to the therapy and a physician to administer it. This pushes the door wide open for those with less scruples to exploit hopeful patients or for physicians to be bought/persuaded to try things they wouldn’t normally try.

The need for accurate (and publicly available!) record keeping

In my opinion, the only way to learn from experiments is to keep accurate records and to be very open about the good, bad and ugly of a dataset. If people are treated (experimentally or otherwise), the results need to be made public so that others do not undertake the same therapy, assuming the outcome was negative. If you knew that the first 100 people who had the therapy did not get better, you might think twice about whether or not you signed up for it and paid for it.  Moreover, national rules would need to be established to determine when something has been “tried too often” without positive results.

Overall, I wholeheartedly agree that doctors (and patients) need to have room to experiment (even in people!) outside of the clinical trials structure. Some diseases are very rare (and therefore difficult to recruit enough patients); some patients are extremely unique (e.g., different medical/treatment history); and some diseases have no reasonable alternative cure or treatment strategy. That said, rules need to exist and recording successes and failures is as important as allowing treatments to take place in the first place. Toothless legislation won’t cut it and a nationalized framework of what has been tried and recommended for other patients would be far more effective.

 My blog is just one of many covering this topic as part of Signal’s second annual blog carnival on the theme ‘Right to Try.’ Please click here to read what other bloggers think about this.


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David Kent

David Kent

Principal Investigator at University of Cambridge
Dr. David Kent is a Principal Investigator at the University of Cambridge in the Cambridge Stem Cell Institute ( His laboratory's research focuses on fate choice in single blood stem cells and how changes in their regulation lead to cancers. David is currently the Stem Cell Institute’s Public Engagement Champion and has a long history of public engagement and outreach including the creation of The Black Hole in 2009. He has been writing for Signals since 2010.