The decision will have widespread implications for the cell and gene therapy (CGT) sector as well as for patient safety, says the International Society for Cell & Gene Therapy (ISCT).
The judgement is flawed, according to the industry organization. It said the decision has inserted regulatory uncertainty into the CGT market, creating opportunities for clinics offering purported treatments that are scientifically unproven and potentially dangerous to patients:
“The ruling reinforces the imperative market need for informative resources that establish scientific consensus, standards, and best practices,” commented ISCT president, Jacques Galipeau.
Late August saw a US federal judge, Jesus G Bernal of the Central District of California, issue a landmark decision siding with the California Stem Cell Treatment Center (CSCTC) and Cell Surgical Network Corporation, in a lawsuit brought by the US Food and Drug Administration (FDA).
The FDA took the legal action against CSCTC in 2018 to assert regulatory authority over stem cell therapies. The agency’s position is that taking a patient’s fat and digesting it with an enzyme to isolate the stem cell fraction creates a biologic drug that requires full FDA approval.
The court rejected this argument. Instead, the judge accepted CSCTC’s position that its treatments qualified for an exception from FDA regulations, in part because they were tantamount to surgical procedures.
The court wrote: "The adipose tissue the defendants remove from patients clearly consists of human cells. And whatever is injected back into patients as part of [the] defendants' SVF surgical procedure and expanded MSC surgical procedure certainly contains such cells."
Reacting to the decision, Dr Elliott Lander, co-founder of CSCTC, said the centre appreciated the court's “clear and unequivocal ruling, which affirms what we have been saying for 12 years: that our innovative surgical approach to personal cell therapy is safe and legal.”
However, Professor Paul Knoepfler, who is based at the UC Davis Department of Cell Biology and Human Anatomy, disagrees. He claims the judge wrongly concluded that stromal vascular fraction (SVF) cells are not changed by the procedure to isolate them.
And the ISCT also believes that scientific inaccuracies in the ruling may have impacted the judge’s decision.
Its committee on the ethics of cell and gene therapy identified several examples of statements that are problematic and unfounded:
Firstly, they found that the ruling made several statements concerning SVF that are both inaccurate and unsupported by current scientific knowledge. "The ruling mistakenly claims the production of SVF is essentially equivalent to surgery and mischaracterizes SVF as a naturally occurring, circulating, unaltered biological entity that is simply relocated from adipose tissue to other diseased parts of the body by surgical means."
Secondly, the committee argues that the court's assertion that the clinical networks use FDA-authorized devices to produce autologous stem cell-based interventions does not consider that the devices in question may not have been authorized by the FDA, or authorized for other purposes, and have not been designed to produce stem cell therapies.
They also found that the statement that culture-expanded mesenchymal stem cells (MSCs) should not be regulated as drugs conflicts with scientific evidence. "This statement opens the door to unchecked administration of poorly characterized and non-standardized cell preparations with unknown safety and efficacy and may pose significant risks to patients."
Laertis Ikonomou, chair of the ISCT committee on the CGT ethics, said: "ISCT has worked for many years now, alongside the FDA and other regulators across the globe, to ensure all those offering cell and gene, and advanced therapies, operate within established clinical regulatory frameworks to uphold scientific standards and ensure treatments are safe and effective before they reach patients. CGTs currently hold unparalleled potential to treat a vast range of conditions that are underserved needs. However, as one of the most advanced and novel fields of medicine, enhancing patient's own cells, these therapies must operate entirely through a global regulatory framework subject to the most stringent scientific standards."