Last week in Part 1 of our series, we presented observations from industry leaders into how they plan for success by installing comprehensive quality management systems and laying down detailed groundwork for an optimized transition to GMP-compliant starting materials and technologies. This week in Part 2 of our series, cell therapy manufacturers present their own perceptions of what constitutes an ideal cell therapy starting material supplier, as well as what can be done on their end to ensure their cell therapy retains consistent quality and potency.
The emergence of cell and gene therapies onto the global stage is generating growing excitement, as unprecedented clinical success fuels the expectation of a promising new chapter in medicine. Alongside an extraordinary increase in the number of cell and gene therapies entering clinical trial, there are a lower yet still significant number of companies launching the commercialized manufacture of their cell therapy products.
A study out of Research Triangle Park, North Carolina [1] cites using HemaCare leukopaks as the starting material for new research on allogeneic CAR T cells.
As most people know, the cancer fighting technique known as CAR T cell therapy has been getting a lot of attention these days, due to some remarkably positive clinical trial results. Two different versions of CAR T therapy have now earned FDA approval, so it seems that this type of therapy will inevitably be expanded to meet the needs of a considerably wider patient population. There’s a fairly large obstacle standing in the way of this expectation, however. Most clinical trials to date have been based on CAR T cell treatment as an autologous therapy, with the starting materials coming straight from the patients themselves. The problem with this autologous model is that it’s difficult to implement on a commercial scale, due to the limited availability of starting material.
An independent publication cites the use of bone marrow and mobilized peripheral blood sourced from HemaCare in a study aimed at improving the efficiency of resource consumption in gene therapy.
The publication, published in the journal Molecular Therapy, focuses on the use of lentiviral vector (LV) to introduce corrected versions of defective genes into stem cells. The goal of gene therapy is to transplant these stem cells into people suffering from genetic disease, in hopes of restoring the function of the faulty gene. One of the main challenges in this clinical strategy is the cost; substantial amounts of LV are needed to modify a therapeutically effective dose of stem cells.
“As the industry and regulatory landscape continues to evolve, it’s vital that as an industry, we take a lifecycle approach to risk management –to ensure that we mitigate as much risk as possible all the way from discovery through to commercialization.” - Dr. Dominic Clarke, Global Head of Cell Therapy, HemaCare
Cell and Gene Therapy Insights has just published HemaCare’s white paper on how to manage starting material quality and stability to maximum effect during cell therapy manufacturing. [1] Quality cell therapies can only be created from quality starting materials, yet paradoxically, starting materials are the single greatest source of variability in the cell therapy manufacturing process. Mitigating risk requires minimizing that variability, and also managing shelf-life limitations and biopreservation logistics.