Overcoming the challenges of conducting translational research in cell therapy

Abstract

Cell therapy holds promise in addressing a number of unmet medical needs but despite its considerable impetus, faces numerous challenges including support for translational research in this area. Here, we examine the challenges confronting cell therapy research including the limited funding available for translational research partly due to the uncompetitive nature of required validation studies in cell therapy that of necessity are not hypothesis-driven. Other issues include lack of standardization of definitions of cell products, uncertainty regarding optimum preclinical animal models, duplication of efforts to secure regulatory approval and a lack of uniformity of nomenclature for some cell products. Nonetheless, several initiatives to address all of these issues are underway.

Keywords

stem cells / clinical trials / funding / animal models / standardization / common definitions

References

[1] DiMasi JA, Hansen RW, Grabowski HG. The price of innovation: new estimates of drug development costs. J Health Econ 2003; 22(2): 151-185

[2] DiMasi JA, Hansen RW, Grabowski HG, Lasagna L. Cost of innovation in the pharmaceutical industry. J Health Econ 1991; 10(2): 107-142

[3] Williams DA, Keating A. Enhancing research in regenerative medicine. Blood 2010; 116(6): 866-867

[4] Group NW. Enhancing Translational Research and Early Phase Trials for Cellular Therapy. Keating AWGC, Editor. 2008. Bethesday, Maryland

[5] ICH. Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals (S6), in Safety Guidelines. 1997

[6] ICH. Addendum to ICH S6: Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals (S6R1), in Safety Guidelines. 2009

[7] FDA.Guidance for Industry: Considerations for Allogeneic Pancreatic Islet Cell Products. 2009

[8] FDA. Guidance for Industry: Cellular Therapy for Cardiac Disease. 2010

[9] FDA. Draft Guidance for Industry: Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage. 2007

[10] EMEA. Reflection paper on stem cell-based medicinal products. Therapies CfA, Editor. 2010

[11] Dominici M, Le Blanc K, Mueller I, Slaper-Cortenbach I, Marini F, Krause D, Deans R, Keating A, Prockop Dj, Horwitz E. Minimal criteria for defining multipotent mesenchymal stromal cells. The International Society for Cellular Therapy position statement. Cytotherapy 2006; 8(4): 315-317

[12] Horwitz EM, Le Blanc K, Dominici M, Mueller I, Slaper-Cortenbach I, Marini FC, Deans RJ, Krause DS, Keating A. Clarification of the nomenclature for MSC: the International Society for Cellular Therapy position statement. Cytotherapy 2005; 7(5): 393-395

RIGHTS & PERMISSIONS

Higher Education Press and Springer-Verlag Berlin Heidelberg