FDA’s Forward Focus: Will Hypesters Finally Be Sent Packing?

Since my somewhat back-ended entry into the regenerative medicine spotlight almost three years ago, I’ve felt a bit like a 55-year-old poster child for what I soon realized was an inconvenient truth: there are patients in the U.S. receiving significant benefit from not-yet-proven, not-yet-approved cell therapy interventions.

My own outcome of sight restoration is an unusual one, especially to the degree I’ve experienced it. After my story hit the press, early on I shared my experience and thoughts with Dr. Bob Califf, then Commissioner of the FDA, as well as the hundreds of patients I interacted with. It’s poor form to turn away a patient, especially one who is knowledgeable, and Dr. Califf was gracious toward my enthusiasm.

Even so, as we talked it soon became clear we were in agreement. The outcome I enjoyed was a “hit or miss” proposition at best, and I was “hit.” I’m sure he detected by my demeanor and enthusiasm that I had resolved to not shy away from what I considered to be a privileged position in the stem cell movement: serving as a potential lens through which light from a new balance between social and regulatory policy could be observed, debated, and rigorously tested. Not only that, I knew this this could happen with more curiosity and less contempt for the fact that patients like me don’t just “get lucky” (as some of even the most rational scientists have characterized it) but rather with the admittance that these treatments actually can work. Careful exploration was warranted to better understand what was “not-yet-proven.”

For the greater good, I’ll strategically assume whatever position others choose to project upon me as a patient, because I am both an advocate for science done the right way, and a witness of the changed lives of people across the country, indeed the world, who are being helped by stem cell therapies still in their first “iteration.” This has led me to fight for balance over hyperbole, curiosity over hubris, and shared goals over stakeholder turfism.

A story of “I was blind, but now I see” is incredibly inspiring, especially if the story gets told. It touches the place in us that is perfectly suited to our natural perplexities and journeys of faith. Nothing is more powerful in getting the conversation started than an unanticipated success.

Encouraging hope and faith is essential when dealing with miracles. I had to be very careful in talking with prospective patients, from young single mothers to U.S. Senators and - believe it or not - CEOs of major pharmaceutical companies, who all wanted to know if the treatment I had might work for them or a loved one.

The Miracle Factor isn’t the only predisposition we share. There is also a “baser” part of each of us that is fascinated by a train wreck. We lament and are angered by the pain and injustice, but we can’t take our eyes off the tragedy. We rubberneck driving by flashing lights on the side of the highway - we’re drawn to alarmism, and we’re usually not waiting too long before we are given a front row seat.

This could not have been more true than when my “blind to sight after stem cell treatment” miracle was countered by a “blinded after a stem cell treatment” tragedy.

Despite the reality that both outcomes are clearly uncommon, happening in that same “hit or miss” fashion Dr. Califf and I discussed, these distinct events had the unfortunate effect of helping to fuel a controversy ripe for hype, inviting opportunistic interests to exploit the ambiguity and fill vacuum.

Truly, the FDA has had its work cut out for it. In one corner of the ring, they had pressure from what I will call the “Doctor Good Collective,” a small group of what the FDA has come to call “bad actors” in the stem cell clinic world, that were demanding freedom from accountability and the right to peddle a promise of miraculous outcomes, as well as administer biologic substances that in some cases were most assuredly dangerous to patients. In the other corner, they faced a group I think of as the “Chicken Little Brigade,” a small group of zealous and loud town criers with a propensity to sound an alarm notifying us that “the sky is falling.”

In its mandated obligation to find balance between these extremes, maintain the integrity of science, educate the public, formulate enforcement strategy, and speed patient access, the FDA has been facing a daunting task.

They’ve been executing it flawlessly. During a recent trip to D.C., I sat down with Dr. Peter Marks, Director of CBER. (Our conversation occurred in the context of the Regenerative Outcomes Foundation’s Pioneers Of Hope Interview Series and will be published in April.) We talked about specific ways in which CBER is preparing to fulfill its mandate under the 21st Century Cures Act. Among other strategies, they have developed what Dr. Marks describes as a more “nimble” approach to trial design and development, as well as acceptance for a market of initial (what I call “1.0”) therapies, allowing relative efficacy for patient access.

You might imagine where our dialog went on from there, but you’ll have to visit our blog when the interview is published!

Until now, moderate thinkers within the stem cell movement, who I consider as having the voice of reason on their side, have been longing in vain for balance in regulatory policy. Their concern that the Chicken Little Brigade would succeed in its effort to convince regulators that a categorical and indiscriminate “crack down” on stem cell clinics was the only solution to the risk that an unchecked Doctor Good Collective would ravage havoc and harm patients, has been justified. The chilling effect on stem cell clinic operators moving forward sooner toward responsible validation of their work has been palpable, and has actually slowed the movement toward productive accountability in my view. Trepidation caused by hype is real. In the case of stem cell policy, the delay, likely about two years’ worth, has meant more people suffering than we are likely to discover have suffered otherwise by the drama of miracle vs. train wreck we’ve been distracted by.

Fortunately, we have recognized from various examples that both of these drumbeats of expansive messaging, what you might call stem cell “spinsanity”, have at times been overexploited with artificial intensity, and that counterintuitive consequence of this kind of bipolar hype we has actually been a new gravity at the middle of policy influence that is pulling stem cell practitioners away from the extreme twin fringes of alarmism carelessness and drawn their much-needed loyalties toward the middle ground where it belongs, and where patients need it.

After my talk with Dr. Marks, as well as others who interact with him regularly, I’m convinced that they are moving forward with new creativity in crafting and supporting opportunities for successful testing of stem cell therapies. I’m finally seeing signs that the Chicken Little Brigade is losing its voice, and the Doctor Good Collective is losing its foothold.

If successful, the FDA’s more balanced, nimble approach to trialing and approving cellular therapeutics should propel the science forward while simultaneously validating the sensibility of shared decision-making, in the context of a patient’s functioning and their experience of an impactful, serious condition. Equally important, the timing of their initiative could now help to validate the patients (and providers) that are tired of being told the sky is falling, and keep patients from feeling as vulnerable that they may get sold a bill of goods, or put themselves or loved ones in harm’s way.

Now it’s more important to focus on asking, “Is the FDA actually willing to genuinely begin engaging physician practitioners and physician groups operating stem cell clinics, and help them enter a pathway to sponsoring clinical trials?”

Stem cell therapy providers are beginning to believe the answer is “yes.” They have been calling me, several per week, asking how they can get help conducting clinical trials. In addition, educational workshops that train would-be stem cell therapy providers are now urging these practitioners to pursue clinical trial sponsorship, and have begun providing training in regulatory compliance and ethical patient engagement.

I believe we should now take the FDA at its word that they are pursuing operational policy aiming to include “the good,” co-opt innovation, improve upon it, submit it to rigorous science within a reasonable time frame, and provide early access to patients with fewer options than most people will ever experience. The decision-makers and influencers at the FDA should be applauded, from the leadership of its Commissioner and Director of Center for Biologics Evaluation and Research, to its RMAT team working long hours with the constant challenge of needing more staff.

Now that the FDA is in effect clarifying its policy and filling the vacuum hypesters were squatting in, is the fuel needed in order to keep peddling their controversy running out? If so, that can mean only one thing for the Doctor Good Collective and the Chicken Little Brigade: it’s time to pack up and leave town. As far as I’m concerned, they’re no longer welcome to dominate our conversation.

Perhaps now we’ll all breathe fresher air, seize the opportunity before us, and collaborate more effectively with each other. Perhaps we will move forward, now that the FDA is paving the way.