There are at least two manufacturing strategies to bring stem cell therapies to market. In first strategy, you can build the GMP facilities in small sections in each hospital to use in patients, whereas in second strategy you can produce your product in a larger cGMP facility. My question is, in your opinion which of these strategies is best suitable to scale up over the next 10 years?

Answer

The answer to this question is unequivocally the latter strategy – it is imperative that you start as early as possible using cGMP (current good manufacturing practice) that may be scaled-up and scaled-out as your process moves through clinical development. While having dispersant manufacturing areas at various hospitals may be suitable for phase 1 studies involving small numbers of patients, this process will not easily scale for phase 2 and particularly phase 3 trials involving larger number of patients (and it certainly will not work for commercial scale production).

On June 25, 2013, CIRM hosted a Roundtable with experts from around the country entitled “Key Tools and Technology Hurdles in Advancing Stem Cell Therapies” and one of the three topics discussed were the challenges in scaling up manufacturing for cell therapies products (the other two were imaging and assay & biomarker development). It was the recommendation of the Roundtable that cGMP processes be implemented as early in the process as possible in order to ensure a smooth transition to phase 3 and commercial-scale production. The Roundtable participants agreed that the most expensive and time consuming steps in manufacturing often involve removing processes that worked for phase 1 (or even phase 2 trials in some cases depending on patient numbers), but will need to be removed or changed for larger scale production. Thus, developing a commercially-viable process early on is crucial to saving time and money down the road as your product advances through clinical development and commercialization.

CIRM is currently in the process of writing a White Paper about the key themes and outcomes of the June 25th Roundtable – please check back on our website in early September for the full report which will discuss cGMP and manufacturing scale-up in greater detail.