FDA ought to promote stem cell therapy by easing up on regulation and its aggressive enforcement.
On International Rare Disease Day 2017, one month after being sworn in as President, Donald Trump gave his 2017 Joint Address to Congress. During his speech, he took particular note of the “slow and burdensome approval process at the Food and Drug Administration” (FDA) that “keeps too many advances … from reaching those in need.” With a specific emphasis on the health of sick children, President Trump argued that if we “slash the restraints” at FDA, then “we will be blessed with far more miracles.”
In attendance that night was Sarah Hughes, a young woman who was forced to travel to Mexico for stem cell therapy (SCT) to treat her systemic idiopathic juvenile diabetes. In 2014, Hughes had her own cells extracted, processed and then infused back into her in a process known as adult autologous stem cell therapy. The results were life-changing.
Before the SCT, Hughes was taking 23 medications a day. After nearly two dozen stem cell infusions over a two-year period, Hughes was down to eight medications a day, and at lower doses. SCT alleviated Hughes’ chronic pain, allowed her to eat normally and absorb nutrients from food, and gave her choices in life she never had before. Despite her progress, she lamented the fact that that other Americans in her position could not avail themselves of SCT.
Since delivering his address to Congress, President Trump has in fact made progress in modernizing FDA, most notably by signing into law the Right to Try Act of 2017, which allows terminally ill patients increased access to experimental drugs that have completed Phase I of the clinical trial process but have not been approved by FDA.
President Trump’s actions continue a broader trend in easing patients’ access to emerging medical treatments. In December 2016, for example, President Obama signed the 21st Century Cures Act into law, which contains special provisions for the “accelerated approval for advanced regenerative therapies” like SCT.
Despite the clear trend toward FDA modernization and the easing of restrictions by Presidents Obama and Trump, the U.S. House of Representatives’ Energy and Commerce Committee has recently signaled that it wants to see enhanced FDA regulatory enforcement over SCT. Leaders of the committee sent a letter to Acting FDA Commissioner Ned Sharpless voicing its concern about FDA’s seemingly permissive use of its discretionary regulatory enforcement authority against potentially violative clinics.
The Committee is seeking more information about FDA’s long-term enforcement strategy, including: financial resources dedicated to approving “legitimate” SCT products; human resources dedicated to the reporting of adverse events; and the possibility of state-federal partnerships to revoke the medical licenses of SCT clinicians.
To be sure, concerns over the safety of patients receiving SCT are reasonable and necessary. But any call for increased regulatory enforcement against clinics offering SCT is premature and will likely disadvantage far more Americans than it helps. At a time when an increasing number of Americans suffer from debilitating chronic medical conditions, we need more medical choice, not less.
The unspoken truth is that despite the constant invocation of the threat of harm from SCT, the actual number of reported cases of adverse harm is remarkably few. Conversely, success stories are numerous.
These SCT successes are built upon robust scientific literature and clinical practice that demonstrate the safety and efficacy of SCT for certain medical indications. The scientific and anecdotal evidence on SCT’s efficacy in treating orthopedic conditions is substantial. Research has shown that it can facilitate the healing of bone fractures, stimulate cartilage regeneration, treat meniscus repair, and decrease lower back pain—the greatest contributor to global disability according to 2010 Global Burden of Disease data.
Stem cell therapy has also been shown to treat both the chronic pain caused by opioid abuse and the effects of opioid tolerance. Likewise, SCT’s ability to treat the symptoms of certain autoimmune conditions is well established. Perhaps most noteworthy is the virtual absence of adverse events that can be directly ascribed to adult SCT.
Although officials such as former FDA Commissioner Scott Gottlieb and current Commissioner Sharpless acknowledge the power and uniqueness of SCT, federal policy on stem cell research and rulemaking has a Janusian quality. On the one hand, it has expanded significantly in the past decade as a result of the relaxing of rules restricting embryonic and other types of stem cell research and the passage of federal laws aimed at expediting regenerative medicine therapies to market. On the other hand, FDA regulations that define the standards for determining which therapies can be offered without FDA approval and those that require approval—legally deemed “drugs”—tend to operate to slow down medical innovation.
In fulfillment of its obligations under the Cures Act, FDA released a guidance document in November 2017 with a new framework that is intended to “balance the agency’s commitment to safety with mechanisms to drive further advances in regenerative medicine so innovators can bring new, effective therapies to patients as quickly and safely as possible.” Importantly, the guidance purports to clarify the terms “minimally manipulated” and “homologous use,” key standards that determine the availability of stem cell therapies to patients. Stem cells that are “minimally manipulated” and used for “homologous purposes” do not need to undergo clinical trials. However, by all accounts, the guidance document interprets these terms quite narrowly, effectively proscribing the therapies altogether.
In an effort to facilitate compliance for clinics that offer unapproved SCT services, FDA has stated that for the first 36 months following issuance of the guidance, it will adopt a “risk-based approach” to enforcement of the new rules. So far, FDA has indeed exercised its enforcement authority judiciously, targeting clinics that it deems flagrant in their marketing or medical practices. Enforcement has generally taken the form of either warning letters or federal lawsuits. The combination of FDA’s narrow interpretations with increasing public demand for alternative medical therapies, however, has meant that the number of clinics offering unapproved SCT products has grown.
The Energy and Commerce Committee should consider the following four factors in determining how to proceed. First, it must recognize that SCT is a unique and unprecedented medical modality that requires a unique regulatory enforcement approach that balances the interest of regulators, scientists, clinician and patients.
Second, FDA’s stated three-year grace period has not yet run. Any evaluation of enhanced enforcement should be deferred until that time comes.
Third, the reporting of adverse events resulting from the use of SCT in clinics is astonishingly small. Of course, any adverse events should be meticulously documented, investigated, and taken seriously. That said, to indict an entire practice because of the negligence or recklessness of a few is a step too far, especially given how many Americans have benefitted from SCT.
Fourth, it is precisely because so many people with no viable medical alternatives have benefitted from the therapy that the Committee should re-think its aggressive posture toward enforcement. When taken together, these four factors weigh against enhanced federal enforcement at this time.
Furthermore, states have played a critical role in SCT regulation. In 2017, Texas Governor Greg Abbott signed a law that makes Texas the first state to authorize the use of SCT for patients with certain severe chronic condition or terminal illnesses. Arkansas is on course to be the first state to require medical insurance companies to cover stem cell therapy.
States are also actively participating in enforcement against clinics that they believe fraudulently market SCT. New York State Attorney General Letitia James recently filed a lawsuit against a clinic offering SCT, maintaining that it misled patients with deceptive marketing practices. The Illinois Department of Financial and Professional Regulation is also investigating patient complaints. These two cases show that states are more than capable of weeding out alleged SCT bad actors without enhanced efforts by FDA.
Like Sarah Hughes, I was forced to travel abroad for SCT. In 2010, I traveled to Nanjing, China for SCT to treat a progressive neuromuscular condition. Although my time in China was wonderful, no Americans should have to travel for SCT—a safe and inexpensive therapy—to save or improve their lives. Stem cell therapy is a paradigm-shifting medical modality that allows persons to use cells from their own bodies to heal themselves. As such, it embodies the democratization of medicine. To unduly stifle, impair, or otherwise restrict the availability and affordability of SCT would not only injure the constituents that members of Congress fight for, but also wound our democratic ideals.
Despite disagreements over policy, all interested parties agree that SCT represents a revolution in medicine. Americans have recognized this shift, and the desire for SCT has reached a tipping point. Thoughtful, judicious, and balanced regulatory enforcement that targets the most flagrant bad actors and allows states to take the lead is the proper way forward at this point.