As the U.S. Food and Drug Administration (FDA) readies a public hearing on stem cell therapy regulation, an ongoing debate over the clinical application of stem cell therapy has been heating up.

The hearing, “Draft Guidances Relating to the Regulation of Human Cells, Tissues, and Cellular and Tissue-Based Products,” will be held Sept. 12–13 at the campus of the National Institutes of Health (NIH) in Bethesda, Maryland. The public is invited to follow the hearing live via an FDA webcast.

The hearing’s purpose, according to the FDA, is to obtain comment from a broad group of stakeholders, including tissue establishments, biological and device product manufacturers, healthcare professionals, clinicians, biomedical researchers, and the public on four draft guidance documents relating to the regulation of human cells, tissues, and cellular and tissue-based products (HCT/Ps). The FDA would like comments on the scope of the guidances, including the particular topics covered, particular questions posed, whether there are additional issues for which guidance would be helpful, and whether FDA recommendations for each topic are sufficiently clear and consistent to provide meaningful guidance.

Information obtained from the public hearing will be considered when finalizing the four documents.

The Medical Advisory Board of the Pulmonary Fibrosis Foundation (PFF) has issued a cautionary statement, noting that we know very little about the impact of cell-based therapies, including stem cells, for the treatment of idiopathic pulmonary fibrosis (IPF) in humans, since rigorous clinical trials have not been yet completed to test the role (or adverse effects) of stem cells in IPF management. It also notes that few such studies have been conducted in animals or humans for the treatment of any lung condition.

PFF acknowledges that patients are understandably eager to receive treatment, and are approaching physicians and care providers for access to interventions they hope can help. But, it says, patients need to be aware that the  safety and efficacy of stem cell therapies need to be determined in approved clinical trials now underway in the United States, Europe, and other countries. More information about these trials can be found on the PFF website.

Until trial results are available, most support for stem cell therapies has been anecdotal, based on patient claims of success.  For example, the Lung Institute recently reported that a pulmonary fibrosis patient who had been given a year to live upon diagnosis now has a new lease on life, after receiving adult stem cell therapy at the Institute’s facility in Tampa, Florida, in March 2015.

The patient, identified only as Joseph O., said in a Lung Institute press release that pulmonary fibrosis had made his life a daily struggle. He was on oxygen 24/7, walking up stairs was a challenge, and that conducting conversations or even brushing his teeth while standing were difficult. He affirms that the stem cell therapy helped restore quality to his life.

“My oxygen level is steady at 98-99, where it was in the 80s before,” he said in the release. “I haven’t seen oxygen in over six months. Haven’t touched it. I was on oxygen 24 hours a day, and now, zero.” He also said that a post-treatment CT scan (identified only as being done three weeks prior to his Lung Institute follow-up call) showed the inflammation in his lungs was gone. “My pulmonologist is blown away,” he said, adding: “I feel like a million dollars.”

The Lung Institute said that this doctor’s astonishment at the treatment’s success is not unusual, and maintains that while many are skeptical of clinical application of stem cells for lung disease, they need only to witness the success achieved and outcomes such as Joseph’s in order to be persuaded to rethink their position.

However, even apparently dramatic anecdotal outcomes are unlikely to convince those advising a cautious approach. The PFF Medical Advisory Board, for example, resolutely maintains that until trial data becomes available, and the safety and effectiveness of stem cell therapies have been rigorously documented, patients should be skeptical about the usefulness of stem cell therapies. It also recommends that patients who engage in such treatments do so through approved clinical trials, where they are closely monitored and the treatment’s potential benefits and harm objectively assessed.

For its part, the Lung Institute cautions that not all stem cell clinics are equal, suggesting that important boxes for prospective patients to check in selecting a stem cell clinic include: focus on treatment of specific diseases as opposed to a broad range of disorders, outcomes data collection and publication, and supervisory oversight by a third-party, such as an institutional review board.

Meanwhile, patients like Joseph O. insist that stem cells have essentially saved their lives, and are working in concert with several members of the U.S. Senate to advance legislation that will make stem cell therapy more affordable and accessible to the general public.

According to a recently published Lung Institute in-house pilot study, “Autologous Stem Cell Therapy and its Effects on COPD,” authored by Jack Coleman Jr., MD, medical director of the Lung Institute facility in Nashville, Tennessee, a large majority of patients treated by Institute clinics — in Tampa; Nashville; Scottsdale, Arizona; Pittsburgh, Pennsylvania; and Dallas, Texas — experienced improvement. The Institute acknowledges that stem cell therapy is not a cure for pulmonary fibrosis, but insists that for many patients with chronic lung diseases, the treatment has potential to improve quality of life.

“After testing approximately 100 patients, the Lung Institute has discovered that within three months of treatment, 84 percent of patients found their quality of life had improved,” Coleman writes. “Among other promising statistics, this discovery could change lives significantly.”

Pulmonary fibrosis (literally “scarring of the lungs”) is an often fatal lung disease belonging to a larger group of disorders called interstitial lung diseases, and is characterized by permanent lung tissue scarring. The Lung Institute estimates that about 140,000 Americans have been diagnosed with pulmonary fibrosis, which typically affects people ages 50 to 75.

Potential causes of pulmonary fibrosis include exposure to airborne toxins, receiving radiation treatment, genetic vulnerabilities, and as a side effect of certain medications. When doctors are unable to pinpoint a specific cause or the disease appears to have occurred spontaneously, it is known as idiopathic pulmonary fibrosis. It is estimated that 50,000 new idiopathic pulmonary fibrosis cases are diagnosed in the U.S. each year, taking an annual toll of nearly 40,000 lives.

For more background on the FDA hearing and stem cell therapy controversy, particularly in the context of multiple sclerosis, see Ian Frank’s recent Multiple Sclerosis News Today column, “Welcome or Not, FDA Focuses on Stem Cell Treatments.”

Sources:
The Pulmonary Fibrosis Foundation
The Lung Institute
The American Lung Association