Correspondence: Warren Sherman, Center for Interventional Vascular Therapy, Columbia University Medical Center, 173 Ft. Washington Avenue, New York, NY 10032, USA. E-mail: ws2157@mail.cumc.columbia.edu CORPORATE VISIONS Commercialization of trials for peripheral artery disease WARREN SHERMAN 1 , CHAYA MAZOUZ 2 , ROBERT DEANS 3 & AMIT N. PATEL 4 1 Center for Interventional Vascular Therapy, Columbia University Medical Center, New York, New York, USA, 2 Pluristem Therapeutics, 3 Regenerative Medicine, Athersys Inc., Cleveland, Ohio, USA, and 4 University Of Utah Commercialization of trials for peripheral artery disease Cell-based clinical studies for peripheral arterial dis- ease (PAD) are moving forward at a brisk pace, to early pivotal trials, a fact that offers considerable hope and encouragement for the cardiovascular regenera- tive field. Doors are opening to novel agents, further insights and important questions, while some aspects of clinical development, such as multiple dosing, opti- mal administration techniques and bio-equivalence testing, remain to be addressed. The regenerative approach to advanced PAD is dynamic, and open to creative input from industry and clinical scientists, but mandatory for the success of the clinical trial process and commercialization of its products, is that a level of collaboration be undertaken among those invested in this field, particularly regarding optimiza- tion of trial design and recruitment. The International Society for Cellular Therapy (ISCT) focuses on driving the translation of scien- tific research and developing technology platforms essential to cell therapy. In keeping with its goal of expediting the development of cell therapies and advancing global patient access, ISCT has given priority to creating forums directed toward emerg- ing cell therapeutics and commercialization perspec- tives. This goal takes on particular importance as pivotal cell therapy studies have commenced regard- ing acute myocardial infarction, refractory angina, congestive heart failure and PAD. Results will have significant bearing on subsequent trials. Therefore, ensuring that clinical trial design and endpoints are well selected and that investigators have the tools to evaluate comparative cell types and select the most appropriate among them, is crucial to the field at this time. These imperatives align well with key missions of ISCT: to foster open-code development of specific cell products targeting specific diseases, and to pro- vide regulatory agencies with a background and data integral to their decision-making processes. With the Cardiovascular Research Foundation (CRF), ISCT convened a strategic Clinical Development Focus Group in January 2011 at the Sixth International Conference on Cell Therapy for Cardiovascular Dis- eases (IC3D), involving experts from academia and industry for a focused discussion on clinical studies in advanced PAD. Cell therapy in PAD PAD is defined in a consensus document (1). It pres- ents as intermittent claudication (IC) and critical limb ischemia (CLI). Revascularization by surgical or transcatheter methods is effective therapy for PAD of proximal vessels, but not of small-caliber distal vessels. In patients with CLI, collateral-dependent flow is insufficient to prevent tissue necrosis and infection, leaving amputation as the alternative. The prevalence of PAD in a primary care population is approximately one in three (2). CLI carries a 5-year mortality rate of 30–50%. Investigative approaches center on inducing neovascularization by modifying the various mechanisms regulating vascular growth. The goals of the strategic Clinical Development Focus Group are detailed in Table I. Patient populations and accrual for clinical study Most PAD patients enrolled into cell-based clinical trials have CLI. Fewer studies are open to IC. Failure to respond to approved therapies is a requirement for participation in these studies. The panel voiced concerns that trial enrollment is low, in part because of competition from clinical Cytotherapy, 2011; 13: 1157–1161 ISSN 1465-3249 print/ISSN 1477-2566 online © 2011 Informa Healthcare DOI: 10.3109/14653249.2011.620795 Cytotherapy Downloaded from informahealthcare.com by 187.72.175.250 on 05/20/14 For personal use only.