It's an exciting time for cell therapy, but quality doesn't come easy, as HemaCare reports
HemaCare recently shared with readers of Drug Discovery World the tremendous prospective benefit of cell therapy, as well as considerations that should be noted in order to deliver optimal patient care. Cell therapy, using products made up of functional living cells to mediate a therapeutic effect, may potentially be applied for disorders such as cardiovascular disease, cancer, autoimmune diseases, and diabetes, afflictions that affect 130 million people in the United States alone. Already, over 2700 cell therapy clinical trials have been initiated, and 51 cell therapy products have received marketing authorization worldwide.
But cell therapy products are no simple, tidy drug. Such material is heterogeneous and defies easy definition. Certain components may be considered vital for one type of therapy but “impurities” in another application. For example, lymphocytes in peripheral blood mononuclear cells (PBMCs) are used to generate regulatory T-cells (among other therapies); however, in the production of dendritic cell-based therapeutic vaccines, lymphocytes are contaminating cells.
Clinical and commercial success depends on a thorough understanding of the product’s Critical Quality Attributes (CQAs). CQAs, encompassing safety, identity, purity, and potency characterization testing, are characteristics of a product that should be within certain limits in order for safety and efficacy to be obtained. In turn, CQAs depend on manufacturing process parameters (Critical Process Parameters, CPPs) and raw material characteristics (Critical Raw Material Attributes, CRMs). The manufacturing process, a chief determinant of the CQAs, must undergo rigorous oversight. In the article, HemaCare provided examples of how various parameters affect the quality and consistency of the end product.
As the cell therapy field matures, it will be all the more important that care is taken to provide high quality biospecimens, so that the treatments that come out of the pipeline are safe and effective. The phrase “Garbage in, garbage out” has been used as a warning that a therapy is only as good as the materials and the preparation that went into it. “Quality in, quality out” sounds much better. As HemaCare states, “Controlled consistent processes yield controlled, consistent products.”
1. Burger, SR et al. Cellular raw material collection in cell therapy: critical determinant of product quality. Drug Discovery World. (Summer, 2014) 15(3):29-34.
2. Compton, C. Getting to personalized medicine. Cancer (2007) 110:1641-1643.