Biotechnology
November 3, 2014
Cell Therapy: What is Minimally Manipulated? New FDA-Proposed Guidelines Offer Insight
Summary:
The FDA released a draft of new guidance around regulations that govern the use of products based on “Human Cells, Tissues, and Cellular and Tissue-Based Products.” The regulations affect companies developing stem cell therapies that are hoping to be classified as minimally manipulated and, as such, developed through a device pathway that is generally believed to be less onerous than a traditional biologics license application (BLA). Companies this may impact include YYYY, XXXX and IntelliCell BioSciences (SVFC - $0.001 - NR), as well as "do it yourself" doctors.
Details:
The FDA has released new guidance for the use of products for cellular therapy. The new regulations are contained in Section 1271.15b of CFR21, titled "Human Cells, Tissues, and Cellular and Tissue-Based Products," known as HCT/Ps. The stated purpose is to establish practices and eligibility and prevent spread of diseases. The section requires providers to register with the FDA Center of Biologics Evaluation and Research and comply with regulations. Section 1271 gives several exceptions for products that are considered to have minimal changes to the cells or pose minimal risk. The exceptions to the regulations include: 1) autologous use, in which the HCT/P is removed and implanted back to the same individual; 2) implanting the HCT/P back to the “same individual within the same surgical procedure”; 3) the HCT/P cells have been “minimally manipulated” and remain in their “original form”; and 4) the risk of disease transmission is no different from surgical procedures.
Centrifugation is not specifically mentioned in the document. This point may be clarified in the final guidance, but until it is clarified, we are assuming it is permitted under the minimal manipulation rule. If centrifugation is not permitted, additional therapies would be required to register, and this would put pressure on "do it yourself" operators.
YYYY adipose-derived stem and regenerative cells (ADRC) are made from cells that are taken from adipose tissue and then extracted through a process of washing, enzymatic digestion, and filtration. According to Section 1271, the “combination with another article” and use of collagenase would mean that ADRC requires registration with the FDA. As such, we believe Cytori will need to purse a BLA pathway for product indications.
XXXX has bioprocess technologies that are used for both therapeutic development (SurgWerks) and biobanking (MarrowXpress System). These systems isolate and concentrate different types of bone marrow cells. This process does not use cell separation media or agents to fractionate the cells. This retains cells in their original form and would fit the definition of “minimally manipulated.” As such, we believe Cesca may pursue therapeutic approvals through a device pathway.
IntelliCell BioSciences: IntelliCell has developed an autologous product called stromal vascular fraction cells. This technology uses ultrasound, rather than enzymes or agents, to separate vascular cells from a sample of adipose tissue. It then transplants them back to the donor. This process seems to fit the definitions of “autologous” and “minimally manipulated.”
Conclusion. We see the new guidelines as a positive as regulators move to define what is manipulated and requires clinical trials through either device or biologic pathways.
Jason Kolbert
Head of Healthcare Research
Senior Managing Director and Biotechnology Analyst
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