FDA must regulate stem cell therapies to mitigate risks to patients and the public

In December 2025, the US Food and Drug Administration's (FDA's) Center for Biologics Evaluation and Review (CBER) approved a gene-based stem cell therapy for Wiskott-Aldrich syndrome, a rare and life-threatening genetic disease.This decision was appropriately based on data from two clinical studies that demonstrated a clinical benefit.Unfortunately, this standard of evidence, and FDA approval itself, are not the norm for many stem cell therapies.Instead, hundreds, if not thousands, of clinics have made unproven and unapproved stem cell treatments widely available to treat everything from common sports injuries to neurological diseases.Officials from the FDA and other federal agencies have tried to stop this potentially dangerous trade.As part of those efforts, the FDA filed two lawsuits in 2018 against clinics involved in the online promotion of unapproved stem cell therapies.After years of court filings and appeals, the agency won an important court case in late 2024 that confirmed the FDA's power to regulate unproven stem cell therapies.In October 2025, the US Supreme Court declined to reconsider the decision, meaning that after seven years, the FDA had confirmed its regulatory authority over a burgeoning online industry.That's good news for proponents of evidence-based medicine.But for how long will this position hold?This may depend in significant part on the current leadership at the Department of Health and Human Services (HHS), which sits above the FDA.Thus far, the signs are not good.Since Congress' February 2025 confirmation of Robert F. Kennedy, Jr. (RFK Jr.) as Secretary of HHS, changes in the FDA's senior officials and policy priorities suggest that the FDA may back away from exercising the authority to regulate this important field, despite its seven-year battle to do so.Here, we highlight the changes that the current Administration might consider for FDA