HemaCare Corporation’s latest publication in BioPharm International  sheds light on a topic that has been preoccupying some of the best minds in cell therapy research; the need to ensure a reliable supply of top-notch starting materials for up-and-coming cell and gene therapies.
Unlike traditional medical treatments, the raw materials for these therapies cannot simply be manufactured. Instead, the supply of starting materials relies on a steady influx of voluntary donors willing to take the time to undertake a fairly complex screening process and donate peripheral blood or bone marrow for altruistic reasons. Nor is a reliable donor pool the only criteria necessary to supplying materials for a successful cellular therapeutic. Starting material quality and consistency ultimately determine the reliable efficacy of the final product.
The impressive clinical results of the first few cell and gene therapy products to go through the regulatory approval process are prompting a fundamental transformation of the cell and gene therapy industry. Federal regulatory agencies, including the Food and Drug Administration (FDA) in the U.S., and the European Medicines Agency (EMA) in Europe responded to the exciting potential of these new therapies by committing to an overhaul of their approval process. New guidance was introduced specifically aimed at accelerating the approval process for these life-saving medicines, and new staff are being hired to manage the robust pipeline and introduce the expertise necessary to understanding these “living” drug products.
The FDA has developed four distinct pathways to speed the progress of promising new therapies through clinical trials, based on their meeting safety and efficacy milestones, as well as establishing the critical need for new life-saving treatments. These new approval pathways have encouraged and facilitated a marked increase in the number of cell and gene therapies entering clinical trial.  Simultaneously, the advance of new therapies into late stage clinical trials has significantly expanded the number of patients enrolled for treatment.
Cell therapy related Phase I to III clinical trials over a four-year period. (Data courtesy of the Alliance for Regenerative Medicine Annual Data Reports)
Accelerated approval pathways, strong pipelines, and increased patient enrollment are all putting pressure on cell therapy starting material providers to increase their production capabilities, while at the same time, safeguarding the quality and efficacy of their products.
As starting material suppliers rise to the challenge of the emerging cell therapy industry, it is becoming clear that multiple solutions need to be implemented to ensure success. Improved access to healthy donors, optimized and standardized cell collection methods, and early transition to GMP-compliant materials can all make a big difference in ensuring a high-quality product.
For greater insight into the challenges and solutions involved, access the full-length article here.
- Taylor B., and Clarke D. The Evolving Role of Starting Materials in Cell and Gene Therapy. BioPharm International. 32(12); 30-35. Dec 2019.
- Scull L., “Cell & Gene Therapy Sector Overview,” Presentation for the Alliance for Regenerative Medicine, Feb. 27, 2019.