Pulmonary Fibrosis Foundation Warns Against Stem Cell Infusions

Janis C. Kelly

March 18, 2019

In response to an uptick in direct-to-consumer advertising for stem cell therapies by for-profit centers, the Pulmonary Fibrosis Foundation (PFF) has issued an updated warning against any type of stem cell treatment of pulmonary fibrosis (PF) outside of an approved clinical trial.

"The decision to update the PFF stem cell statement was multifactorial," Gregory P. Cosgrove, MD, chief medical officer of the PFF and associate professor of medicine, National Jewish Health and the University of Colorado Denver, told Medscape Medical News.

"The PFF continues to receive questions regarding the role of stem cells/cell-based therapy in the treatment of PF from patients, caregivers, and providers on a regular basis through our support line and information hub, the Patient Communication Center.... Additionally, there appears to be an increase in direct-to-consumer marketing of stem cell therapies to specific groups of patients, notably those with pulmonary fibrosis and emphysema. For these reasons, our medical advisory board decided to update the PFF stem cell statement to clarify the issue for our community."

The warning reiterates the foundation's 2015 statement on stem cell treatments, noting that experimental treatments provided by unregulated, commercial stem cell centers can cause great harm to individuals with life-threatening diseases such as PF.

As previously reported, unapproved treatments using umbilical cord blood–derived stem cells have led to serious bloodstream infections and other infections in patients in multiple US states, according to an ongoing Centers for Disease Prevention and Control investigation.

"Modern-Day Equivalent of Snake Oil"

The updated warning from the PFF medical advisory board explains, "The evolving field of regenerative medicine holds tremendous promise, but at present stem cell/cell-based therapies remain unproven, experimental, and may be detrimental for patients with pulmonary fibrosis. International guidelines have been developed to direct the efficient and appropriate assessment of innovative treatments that can be evaluated in clinical trials and approved only after appropriate regulatory review. This contrasts sharply with the unproven interventions offered by practitioners operating with minimal oversight at for-profit stem cell centers that represent 'the modern-day equivalent of snake oil.' "

The PFF noted that several reported cases of severe respiratory illness were temporally associated with stem cell infusions from for-profit centers. The organization warns, "Without a proven mechanism of action, no set standards for cell preparation, cultivation, storage and treatment regimen, the direct to consumer marketing of stem cell therapies have exaggerated claims of safety and efficacy, often with weak or absent scientific rationale."

Andrew Limper, MD, chair of the PFF medical advisory board, said in a press statement, "Desperate patients and their physicians continue to succumb to an onslaught of marketing and branding of as yet unproven stem cell treatments."

Cosgrove said that enthusiasm for a potential use of stem cell/cell-based therapies in the treatment of lung diseases based on results from animal studies is understandable but that the safety and efficacy of these therapies in PF patients have not been established.

"Given the limited number of studies investigating the particular type of stem cell/cell-based therapy, the route of delivery of the agents, and the side effects and benefits, further investigation is necessary and required to understand the dangers of these therapies. The path forward to rapidly evaluating these therapies and truly understanding their value in the treatment of patients with pulmonary fibrosis is through well-designed clinical trials, which can be identified via resources such as the PFF Clinical Trial Finder. By utilizing stem cell/cell-based therapies outside of this arena, patients expose themselves to potentially serious side effects without a clear benefit," Cosgrove said.

The foundation said that several small phase 1 studies of mesenchymal stem cells or multipotent mesenchymal stromal cells showed acceptable safety profiles in patients with PF, COPD, acute respiratory distress syndrome, and lung dysfunction after lung transplant. More advanced clinical trials are being designed.

Cosgrove has disclosed no relevant financial relationships.

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