January 21, 2021

The Niche

Trusted stem cell blog & resources

Push for National Right-To-Try Law Raises Concerns in Stem Cell Community

Right To TryRight-To-Try laws have been passed in many states across America, paving the way in theory for gravely ill patients to have the right to try unproven treatments of various kinds, and now there is a serious push underway for a national Right-To-Try law.

What is a Right-To-Try law?

Typically, these Right-To-Try laws allow for a patient facing serious illness, with consent of their physician and the business that makes the still investigational therapy, to not have to wait for full vetting of the new treatment. They can take the risk, for example, on a new chemical drug or stem cell-based regenerative medicine therapy before it is proven to work or definitely to be safe. For a patient with a fatal illness such as ALS, it seems reasonable that they should have a right to try different options, but it should be noted that the FDA already has a program for this kind of situation called compassionate use or as the agency calls it, “expanded access”.

On the stem cell front, one of the biggest concerns over Right-To-Try is that it could be exploited by some of the hundreds of stem cell clinics out there. The new stem cell law in Texas, which in an earlier incarnation as a bill I called “Right-To-Profit” for stem cell clinics, has some Right-To-Try elements. Another concern is that some proponents of Right-To-Try don’t want it limited to fatal illnesses.

Overall, the campaign for a federal Right-To-Try law is gaining momentum via a legislative push called the Trickett Wendler Right to Try Act. Yesterday, for instance, Sen. Ron Johnson (R-Wis.) released a statement saying Right-To-Try should be included as part of the FDA reauthorization bill. Johnson is a sponsor of Trickett Wendler and has threatened to hold up FDA funding over this cause.

I asked stem cell scientist Sean Morrison, who is also the Chair of the ISSCR public policy committee, for his take on the push for a federal RTT law, particularly as it relates to stem cells, and here is what he said:

“1. These right to try bills are bad for patients because most of the new therapies patients will be able to access as a result of these bills would be expected to prove unsafe or ineffective once additional clinical testing is done. This is because we know that most therapeutics that seem successful in phase 1 clinical trials prove to be unsafe or ineffective upon additional testing in phase 2 and 3 clinical trials.

2. These bills are often introduced by well-meaning legislators who have very little understanding of medical research or clinical testing. They don’t realize these bills will victimize patients by opening the door to snake oil salesmen to sell unproven therapies to patients. Patients will be forced to make life and death decisions without the benefit of clinical data on whether the therapies are safe or effective.

3. These bills have been introduced nationwide as part of a general assault on FDA regulation. Most people who support these bills don’t realize that the FDA already has a very effective, and efficient, accelerated access program under which patients can get therapeutics prior to FDA approval. Most don’t realize the extent to which the FDA protects Americans from snake oil salesmen trying to make a buck by exploiting the hopes of desperate patients, by selling unproven and often scientifically implausible therapies that are promised to cure all manner of incurable diseases.

4. There are scores of companies in the United States who want to sell unproven and scientifically implausible stem cell therapies to patients, some of whom are among the main supporters of right-to-try legislation.”

It’s better than 50-50 that Right-To-Try will become federal law, but it’s not a done deal and if it does become law it isn’t entirely clear what the practical impact would be since state laws of this kind haven’t to my knowledge led to dramatic changes in terminal patient access to investigational drugs plus many drug manufacturers do not want their experimental treatments utilized in this manner for various reasons. For stem cells specifically, I expect that if there is a federal Right-To-Try law that we’ll start seeing “Right-To-Try” popping up in clinic advertisements, which wouldn’t help matters.

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