Cell Therapies Come of Age: Overcoming Challenges Within the Regulatory Framework

Cell Therapies Come of Age

By Tracy TreDenick, Head of Regulatory and Quality Assurance, BioTechLogic

While the field of stem cell therapy has been in development for decades, most notably with the first successful bone marrow transplant in 1968, regenerative medicine is now experiencing rapid progress fueled by scientific and technology advancements. Although cellular therapeutics benefits greatly from embryonic stem cells, debates over the ethics of this type of research has led to the discovery of a more sustainable alternative, somatic stem cell research.

This said, many of the emerging cell therapies are scientifically and medically complex with many learnings and understandings still to come. As if core scientific and medical complexities were not enough, the industry, medical community and regulators are grappling with the challenge of conforming the regulatory framework to support the commercialization of cellular therapies, for instance cellular therapy classification, quicker approval pathways, and CMC challenges.

Cell Therapies:  A Drug or not a Drug?

Within the cell therapy arena, regulators, the medical community and industry alike are often confronted with an unsettling question. What is this, really? Should a given cell therapy be regulated as human cells, tissues, and cellular and tissue-based products (HCT/Ps), or should the cell therapy be regulated as a drug with the FDA oversight and regulatory approval process that accompanies that designation?

As cell therapies have become more common and indications have expanded, it’s becoming increasingly common to be trapped in “no man’s land” trying to answer this question, creating tension and confusion for the range of stakeholders involved in the decision-making process. Today, the dispute primarily centers around somatic stem cells that are harvested and prepared for transplantation through minor surgical procedures at stem cell clinics and other medical service providers.

Mesenchymal stem cells (MSCs) are collected from a patient’s own bone marrow or fat tissue, or from donor tissue not altered or manipulated and can form fat, bone or cartilage, making them useful for repairing bones and joints, minimizing inflammation caused by conditions such as rheumatoid arthritis, and promote the repair of a range of tissues. Hundreds of stem cell clinics now perform procedures with MSCs that are regulated as HCT/Ps under Public Health Service Act (PHSA) section 361. Procedures falling under section 361 classification are subject to regulations similar to that of other surgical procedures that are primarily aimed at avoiding contamination, infection and the spread of infectious disease.

HCT/Ps that require “manipulation” or alteration are governed by PHSA section 351. These products/procedures are considered to be indistinguishable from drugs and must undergo a rigorous regulatory approval process before being administered to patients. Some of the lines that separate section 351 products from those of section 361 are clearly drawn. For example, cells and tissues used homologously, meaning they perform the same function in the recipient as they do in the donor — such as the transplantation of bone marrow to restore healthy blood-cell production, are regulated under section 361. And therapies that employ a patient’s own stem cells (autologous) are more likely to fall under section 361 than those that use allogeneic cells (from a donor).

So back to the question at hand: A drug or not a drug? Broadly, the FDA considers a product to be a drug if “more than minimal manipulation” is required for its effectiveness. Ambiguities can arise, however, because merely separating stem cells from their neighboring cells always entails some degree of manipulation.

The question of minimal manipulation was… click to continue reading article.


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