Serious health threats possible with underregulated commercial stem cell clinics

Stem cells hold potential benefits for the treatment of age-related macular degeneration, but commercial stem cell clinics that skirt under FDA regulations and oversights can pose serious health threats to patients, according to an editorial in the New England Journal of Medicine.

Two articles, one by Mandai and colleagues and the other by Kuriyan and colleagues, which Ocular Surgery News previously reported about, describe different stem cell approaches for the treatment of AMD, George Q. Daley, MD, PhD, wrote. Mandai and colleagues describe the treatment of a woman with AMD with the transplantation of a sheet of retinal pigment epithelial (RPE) cells derived from autologous induced pluripotent stem cells (iPSCs).

“The iPSCs were generated from one of the patient’s skin fibroblasts; their autologous origin renders them — and the RPE cells derived from them — theoretically safe from attack by the immune system. In fact, Mandai et al. generated iPSCs from two patients and subjected both cell lines to extensive functional and molecular characterization, which included whole-genome sequencing, to determine their genetic integrity,” Daley wrote in the editorial.

The woman received the sheet of stem cells in one eye, and a year after surgery, her vision stabilized, with no evidence of any permanent adverse events, he wrote.

However, Kuriyan and colleagues describe three patients “who received intravitreal injections of a poorly defined slurry of autologous adipose tissue-derived cells in both eyes,” harvested by liposuction.

These treatments resulted in the blindness of one woman and “marked visual loss in the other two women,” Daley wrote.

“The provision of autologous cellular ‘therapies’ outside the experimental clinical trial setting — and on a for-profit basis — is a gross violation of professional and possibly legal standards; it carries the risk of worsening human health and violates the long-standing medical tradition of primum non nocere,” Daley wrote. – by Robert Linnehan

Reference s :

Daley GQ. N Engl J Med. 2017;doi:10.1056/NEJMe1701379.

Kuriyan AE, et al. N Engl J Med. 2017;doi:10.1056/NEJMoa1609583.

Mandai M, et al. N Engl J Med. 2017;doi:10.1056/NEJMoa1608368.

Disclosure: Daley reports grant support from Megakaryon, personal fees from MPM Capital and 28/7 Therapeutics, personal fees and other support from iPierian and Ocata Therapeutics, and other support from True North, Epizyme and Solasia. He reports pending and issued patents related to the derivation of blood stem cells and blood products from iPS cells, on reprogramming methodologies and characterization of stem cells. He reports he anchored the development of international guidelines that govern the evaluation and quality control around stem cells and transplants.