Neuralstem flirting with noncompliance via Right To Try Law?

Is Neuralstem flirting with noncompliance via a Right To Try Law in Colorado?

Colorado is an independent-minded state. For example, it is one of only a few states including Washington State with legalized marijuana use.

More recently, Colorado passed a new law supported by the ultra-conservative Goldwater Institute, the so-called Right To Try Law. This law allows patients to try experimental, unapproved medical interventions that have completed a Phase 1 clinical trial. Most Phase 1 drugs ultimately fail to be proven safe and effective and Phase 1 only demonstrates a minimum baseline level of safety.

Interestingly, Colorado is also a hot bed for experimental stem cell-related interventions. Putting these two realities together, when the Right-To-Try Law passed it seemed likely that we’d be seeing Colorado ending up as a magnet for patients wanting to try experimental, non-FDA approved stem cell therapies and that stem cell companies, including some that previously had records of compliance, would eventually begin federally non-compliant or at least unapproved operations in Colorado.

It didn’t take long for these predictions to be realized as stem cell biotech Neuralstem announced on June 5 that it would be offering its experimental, non-FDA approved human neural stem cell therapy for ALS in Colorado. And it might do it in a for-profit manner. Further, Neuralstem CEO Richard Garr has indicated that the company will not work with the FDA for the human experiments in Colorado. It is worth pointing out that CEO Garr is also Chair of the Goldwater Institute’s Right To Try National Advisory Council.

One of the big concerns about Right To Try-enabled operations of this kind is they could well end up being conducted in a manner that is noncompliant with federal law such as by exposing relatively large numbers of patients to risks.

Apparently the Colorado Right-To-Try Law was inspired by the movie, The Dallas Buyer’s Club, at least in a quote attributed to Colorado State Sen. Irene Aguilar. In his column on the law, David Kroll in turn quoted a column by bioethicist Art Caplan criticizing the law:

“Passing bills full of empty promises based on Hollywood rather than the real world of today is not compassionate but, actually, flat-out cruel.”

As much as I support expanded compassionate use for patients with a fatal illness such as ALS or terminal cancer, Right To Try is not likely to be the solution. It is not the same thing as carefully administered compassionate use, it could put patients at risk, and it could falsely raise hopes of patients. I can understand how some patients and their families would support Right To Try though.

There is a not so easily resolved tension between the completely understandable desire of terminally ill patients to be allowed to try experimental drugs and the concerns of those who advocate for appropriate regulatory oversight as well as evidence-based medicine. This is made all the more complicated by the current clinical trial system that is slow, expensive, and clunky, but absolutely something that should not be thrown out entirely by any means.

Until you are facing a potential terminal illness you don’t know what it is like to be in those shoes. When I was diagnosed with a very serious form of prostate cancer in late 2009, I thought for a while it might ultimately be a terminal cancer for me given what my doctors were telling me. Now four and a half years after surgery I’m doing great (knock on wood), but the cancer could come back. I know that and in part because of my own experience I respect patients greatly.

Still there are definitely some complexities and potential problems that are going to pop up in Colorado due to this new law. Patients are likely going to be put at risk and potentially end up spending tens of thousands of dollars for interventions that don’t work, but as a company Neuralstem is also potentially putting itself in jeopardy. Given that federal law trumps state law, it is unclear to me that Neuralstem can simply ignore the FDA.

It is also likely that the Right To Try Law will embolden other for-profit stem cell entities in Colorado to push the limits of what is permissible in terms of experiments on patients and may well lead companies from other states to do some stem cell interventions in Colorado thinking they are shielded by the state law. They might not be. To protect patients at least financially, I propose that Right To Try laws require that companies that engage in these activities be required to do so only in a not-for-profit manner. It would also be ideal if such companies consult with bioethicists in advance of beginning such operations.

Given that other states have already passed Right To Try laws including Louisiana and Missouri that are awaiting their respective governor’s signatures and that Arizona voters will see a similar bill on the ballot in November, will the FDA feel it needs to take swift action to avoid things spiraling out of control?

Importantly, PBS reports that a federal law has been proposed that is similar to Right To Try. The so-called the Compassionate Freedom Choice Act of 2014, if passed, would in my view introduce chaos into the federal system of careful medical oversight. A key part of this bill reads that it would:

“Amends the Federal Food, Drug, and Cosmetic Act to declare that nothing in it or in the Public Health Service Act shall prevent or restrict, and the Food and Drug Administration shall not implement or enforce any law to prevent or restrict, the manufacture, importation, distribution, or sale of investigational drugs or devices for terminally ill patient.”

In a sense, the Colorado Right-To-Try Law as it pertains to stem cells is itself a big human experiment and it will be probably end up being fascinating and perhaps disturbing to see how this all plays out even as there are concerns over risks it brings with it. I worry it might do more harm than good. What do you think?

For other takes on Right To Try laws, see this very thorough piece by Orac and a piece supporting these kinds of laws from the Goldwater Institute.