October 1, 2013 (Vol. 33, No. 17)

Snapshot of the Evolving Market Landscape

Cellular therapy is a challenging discipline from a variety of perspectives: The cells themselves need to be prepared and proven to be the appropriate inoculum free of contaminating subpopulations; there need to be assays to demonstrate, safety, efficacy, and potency; there is a challenging regulatory environment; and eventual reimbursement of cellular therapeutics is uncertain.

In this environment, benchmarks are needed—i.e., scenarios utilizing cellular therapeutics wherein a trajectory has been established. For this sense of direction, we turn to hematopoietic stem cell transplantation (HSCT), which has a 40+ year operating track record and has evolved in terms of the types of cellular inoculum that are deployed. The Figure presents the evolution of this space and presents two time points: 1988 and 2012—note the evolution of the source of stem cells utilized.

Cord blood is gaining in popularity as a source of starting material and provides a roadmap for how the cellular therapeutics space may evolve. Indeed, one could argue that cord blood utilization provides a framework for how the evolution of the broader cellular therapeutics field may unfold.

In the companion report published in GEN online (http://bit.ly/1g0hLED), we present a snapshot of some of the quantitative metrics in the cord blood banking space and track the challenges in the field as it seeks to remake itself from purely a source of material for HSCTs to a source of stem cells for addressing a wide swath of therapeutics needs.

Indeed, the ongoing clinical trials in the cord blood space focusing on CNS and other disorders demonstrate how a focused cellular therapeutic for hematological malignancies and inborn errors of metabolism can find wide appeal as a general source of therapeutically relevant stem cells.

The fact that more than 580,000 units of cord blood are available in public banks worldwide (a fraction of which is probably actually usable as a cellular therapeutic) with rapid access to patient populations suggests the potential of cord blood and its associated cells as a generalized cellular therapeutic going forward.

Of course, there are challenges and limitations with cord blood, and some of these are addressed in the companion report. The takeaways therefore are that cord blood-based cellular therapeutics provide a roadmap for the evolution of the cellular therapeutics space. In addition, some of the challenges in getting cord blood approved and working within its QA/QC parameters, which allow it to be offered as a product, to an extent will also likely applicable to allogeneic cellular therapeutics in the future.

In summary, we continue to follow the cord blood market as a means to track the evolution of this space per se as well as to provide a path forward for the unfolding of the allogeneic therapies space using adult stem cells of various classes over the coming years.

Our industry analyses show the increased utilization of cord blood in the hematological malignancy space and this trend is “spilling over” into other disease classes. These data lead to the concept that we seek to promote that the cord blood adoption trajectory provides a benchmark for the allogeneic cell therapy space.

Evolution of the HSCT Space

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