More than 1050 clinical trials are registered at FDA.gov that explore multipotent mesenchymal stromal cells (MSCs) for nearly every clinical application imaginable, including neurodegenerative and cardiac disorders, perianal fistulas, graft-versus-host disease, COVID-19, and cancer. In this review, we discuss the major clinical challenges with MSC therapies, the details of these challenges, and the potential bioengineering approaches that leverage the unique biology of MSCs to overcome the challenges and achieve more potent and versatile therapies.
The European Commission’s forthcoming Pharmaceutical Strategy could help stop advanced therapy medicinal product (ATMP) development moving away from the EU if it removes obstacles that impede access to and development of such products, says the Alliance for Regenerative Medicine.
The COVID-19 crisis could be a proving ground to legitimate the still early-stage technology of mesenchymal stem cell therapy, but only if clinical trials are of high quality – blinded and randomized – in a context of regulatory clarity.
Advanced therapy medicinal products include cell therapies, gene therapies, and tissue-engineered products. These highly complex treatments differ from traditional medicines, both in how they are made and administered and in the type of benefits they may provide. These are products for which regulations were established relatively recently and that present challenges for regulatory agencies on how to best they should be regulated within the existing regulatory framework.
The Alliance for Regenerative Medicine (ARM) in Brussels published its response to the EU Pharmaceutical Strategy Roadmap, which is designed to improve and accelerate patients’ access to safe and affordable medicines and to support innovation in the EU pharmaceutical industry. While ARM welcomes the Roadmap’s recognition of the wider industry’s contribution to the EU economy and its focus on ensuring patients’ access to safe, state-of-the-art therapies, the Alliance wants to ensure a continued pathway for the EU to benefit from innovative and life-changing advanced therapy medicinal products.
More focus on value-based pricing, less restrictive GMO designations, and more attention to hospital exemptions are all priorities for the Alliance for Regenerative Medicine, an industry group representing companies working in the gene and cell therapy space. Carlo spoke to Paige Bischoff, senior vice president of public affairs, about what ARM wanted ahead of their upcoming response to the EU’s Pharma Strategy roadmap.
In the midst of a global public health emergency, some businesses are taking advantage of widespread fears by marketing purported stem cell treatments for COVID-19. Such businesses target prospective clients with misleading claims, expose patients to potentially risky stem cell-based products, and undermine efforts to develop evidence-based treatments for COVID-19.
It’s been four years since the first CAR T cell therapies were approved and inadequate reimbursement is still a barrier to access in the U.S. A new payment rule proposed by CMS aims to change that, and its adoption could provide a road map to faster implementation of payment models for the next new technologies.
