Developing Induced Pluripotent Stem Cell-Based Therapy for the Masses

Abstract

The discovery of induced pluripotent stem cells and the ability to manufacture them using clinically compliant protocols has the potential to revolutionize the field of regenerative medicine. However, realizing this potential requires the development of processes that are reliable, reproducible, and cost-effective and that at the same time do not compromise the safety of the individuals receiving this therapy. In the present report, we discuss how cost reductions can be obtained using our experience with obtaining approval of biologic agents, autologous therapy, and the recent approval of cord blood banks. Significance: For therapy to be widely available, the cost of manufacturing stem cells must be reduced. The steps proposed in the present report, when implemented, have the potential to reduce these costs significantly.

Distributed automated manufacturing of pluripotent stem cell products

“…Core enabler for work in [16] and current work. [17] Haplobanked cell therapy, see [3][4][5]18] IPSC Automated process (based on [16]; and also see [19] addressing cost). US [6] and EU [7] regulatory environment.…”

“…Automation is essential in any mature production pipeline. 4 In-process quality control is automated, and the batch records are maintained by the automated platform.…”

“…The aim was to run batches for multiple automated passages at each site from a standardised starting material that had been banked at one of the sites. This is significant for a number of reasons: (i) manufacturing of cell therapies for global markets will require aseptic manufacturing in multiple locations in different countries (sometimes within different regulatory regimes); (ii) manufacturing may be close to the point-of-care or use rare (autologous) starting materials [1,2]; and (iii) the emerging model for international human-induced pluripotent stem cell (hiPSC) haplobanking requires the sharing of starting materials and their subsequent expansion in different countries and within different regulatory regimes [3][4][5]. The requirement to manufacture products at multiple sites is recognised by both EU and US regulators as challenging for biological products of this complexity [6,7].…”

Survival and Functionality of Human Induced Pluripotent Stem Cell-Derived Oligodendrocytes in a Nonhuman Primate Model for Multiple Sclerosis

“…Moreover, recent improvements in the protocol for the differentiation of human iPSCs into OPCs have led to a significant reduction in the duration of this procedure [36, 37]. Finally, in a recent report, Rao and Atala have proposed important steps to reduce the costs for iPSC production significantly [38].…”

Ips Progression a Decade Devoted

“…An international collaboration in the form of GAIT (global alliance for iPS based therapy) aims to formulate a GMP iPS-haplobank in harmony with mutually recognized iPS banks for maximizing utilization and offering hope in the future to facilitate iPS therapy globally [111][112][113].…”