Inclusivity and diversity: Integrating international perspectives on stem cell challenges and potential.

Regenerative medicine has great potential. The pace of scientific advance is exciting and the medical opportunities for regeneration and repair may be transformative. However, concerns continue to grow, relating to problems caused both by unscrupulous private clinics offering unregulated therapies based on little or no evidence and by premature regulatory approval on the basis of insufficient scientific rationale and clinical evidence. An initiative by the InterAcademy Partnership convened experts worldwide to identify opportunities and challenges, with a focus on stem cells. This was designed to be inclusive and consensus outputs reflected the diversity of the global research population. Among issues addressed for supporting research and innovation while protecting patients were ethical assessment; pre-clinical and clinical research; regulatory authorization and medicines access; and engagement with patients, policy makers, and the public. The InterAcademy Partnership (IAP) identified options for action for sharing good practice and building collaboration within the scientific community and with other stakeholders worldwide.

A design-thinking approach to therapeutic translation: tympanic regeneration.

PURPOSE OF REVIEW: Clinician researchers face the pressures of meeting academic benchmarks combined with advancing new therapies to patients. The vast majority of drug discoveries fail in translation. A new method of meeting the challenges of preclinical therapeutic translation is presented using the example of tympanic regeneration. RECENT FINDINGS: The key to a design-thinking approach to therapeutic translation is to 'begin with the end in mind' by widening the scope of the problem, with multiple points of view, to not only understand the disease but the context for the patient and the health system in which it occurs. Idea for therapeutics should be tested in relevant models early and once proof of efficacy is established, translational milestones that represent the greatest risk, such as safety and toxicity should be addressed first. It is important to seek the feedback of industry early to understand what milestones should be best addressed next with limited academic resources. Whenever proceeding, guidelines for maintaining scientific reproducibility should be followed to minimize risk of failure during transfer into industry. SUMMARY: A Design-thinking approach addresses the potential failures in drug discovery and preclinical translation.

A SY-Stematic approach towards understanding stem cell biology.

The 2nd SY-Stem Symposium - a symposium for 'the next generation of stem cell researchers' - was held on the 21-23 March 2019 at the Vienna BioCenter in Austria. After the great success of the initial SY-Stem meeting in 2018, this year's event again focused on the work of young scientists. Here, we summarize the impressive amount of new research covering stem cell-related fields that was discussed at the meeting.

Regenerative Medicine Venturing at the University-Industry Boundary: Implications for Institutions, Entrepreneurs, and Industry.

Regenerative medicine research at university laboratories has outpaced commercial activity. Legal, regulatory, funding, technological, and operational uncertainty have slowed market entry of regenerative medicine treatments. As a result, commercial development has often been led by entrepreneurial ventures rather than large biopharma firms. Translating regenerative medicine across the university-industry boundary links academic scientists, technology transfer organizations, funders, and entrepreneurs. Conflicting motivations among the participants may significantly hinder these efforts. Unproven downstream business models for regenerative medicine delivery further complicate the entrepreneurial process. This chapter explores the challenges associated with entrepreneurial activity commercializing regenerative medicine science developed at research institutions.

Evolution of Business Models in Regenerative Medicine: Effects of a Disruptive Innovation on the Innovation Ecosystem.

PURPOSE: This article focuses on 10 case studies of companies/organizations that are part of the current innovation ecosystem of regenerative medicine (RM) in the United Kingdom. It analyzes the actors, linkages, and influences that will determine the future shape of the RM industry sector and its capacity to live up to its initial expectations. METHODS: Using the case study approach, purposive sampling was used to get 18 interview respondents from 10 RM companies/organizations in the United Kingdom. We used semistructured interviews for data gathering and thematic analysis for identifying gaps in the RM value chain (ie, the range of activities required for bringing a product from conception to market and end-use) and the influences of the innovation ecosystem on the evolving RM business models. FINDINGS: RM promises to address currently unmet health care needs by restoring the normal form and function of cells, tissues, and organs. The innovations emerging to support the progress of RM to satisfy these important health care markets will disrupt the business models of incumbent industry sectors, particularly pharmaceuticals. Companies involved in this area must develop innovative business models and value chains and negotiate the complex influences of the innovation ecosystem, including regulatory systems and standards, financial support systems, and new market dynamics. IMPLICATIONS: This article highlights the needs for more systemic analyses of the needs of potentially disruptive innovations, in RM and more widely, and for policymakers to give greater attention to these insights in planning regulatory and other supporting initiatives, with the promotion of innovation in mind.

Can Regenerative Medicine Help Close the Gap Between the Medicine Pipeline and Public Health Burden of Cardiovascular and Musculoskeletal Diseases?

PURPOSE: This commentary discusses the therapeutic and economic potentials of regenerative medicine (RM) by addressing how the reprioritization of resources in drug development may alleviate unmet medical need across many diseases, but especially cardiovascular diseases (CVDs) and musculoskeletal diseases (MSDs), the leading causes of mortality and morbidity, respectively, in the United States. METHODS: Data and perspectives represented in this commentary were obtained through an online literature search, public press releases from federal agencies and companies, online opinion pieces, published journal articles, and consulting agency reports; however, there were limitations to the available data because of the breadth and novelty of the therapeutic modalities involved. FINDINGS: Currently, the misallocation of resources within the therapeutic areas of CVDs and MSDs are possibly contributing to low approval rates, high cost of drug treatments, and consequently, disease burden. With a 2025 global market estimate of US $50.5 billion, RM is expected to become a major player in the pharmaceutical industry, with a potential to change the treatment paradigm and lessen disease burden across multiple disease areas, most notably in CVDs and MSDs. IMPLICATIONS: While the public sector appears to be doing its fair share by funding basic research and revamping regulatory regimes to address the vagaries of RM as a rapidly emerging novel technology, the support framework necessary for transforming the field from a promising concept to available therapy requires levels of resource allocation and marketing support that only the private sector can provide.

FDA and NIST collaboration on standards development activities supporting innovation and translation of regenerative medicine products.

The development of standards for the field of regenerative medicine has been noted as a high priority by several road-mapping activities. Additionally, the U.S. Congress recognizes the importance of standards in the 21st Century Cure Act. Standards will help to accelerate and streamline cell and gene therapy product development, ensure the quality and consistency of processes and products, and facilitate their regulatory approval. Although there is general agreement for the need of additional standards for regenerative medicine products, a shared understanding of standards is required for real progress toward the development of standards to advance regenerative medicine. Here, we describe the roles of standards in regenerative medicine as well as the process for standards development and the interactions of different entities in the standards development process. Highlighted are recent coordinated efforts between the U.S. Food and Drug Administration and the National Institute of Standards and Technology to facilitate standards development and foster science that underpins standards development.

How to build an allogeneic hematopoietic cell transplant unit in 2016: Proposal for a practical framework.

Allogeneic hematopoietic cell transplantation is part of the standard of care for many hematological diseases. Over the last decades, significant advances in patient and donor selection, conditioning regimens as well as supportive care of patients undergoing allogeneic hematopoietic cell transplantation leading to improved overall survival have been made. In view of many new treatment options in cellular and molecular targeted therapies, the place of allogeneic transplantation in therapy concepts must be reviewed. Most aspects of hematopoietic cell transplantation are well standardized by national guidelines or laws as well as by certification labels such as FACT-JACIE. However, the requirements for the construction and layout of a unit treating patients during the acute phase of the transplantation procedure or at readmission for different complications are not well defined. In addition, the infrastructure of such a unit may be decisive for optimized care of these fragile patients. Here we describe the process of planning a transplant unit in order to open a discussion that could lead to more precise guidelines in the field of infrastructural requirements for hospitals caring for people with severe immunosuppression.

Rebuilding a broken heart: lessons from developmental and regenerative biology.

In May 2016, the annual Weinstein Cardiovascular Development and Regeneration Conference was held in Durham, North Carolina, USA. The meeting assembled leading investigators, junior scientists and trainees from around the world to discuss developmental and regenerative biological approaches to understanding the etiology of congenital heart defects and the repair of diseased cardiac tissue. In this Meeting Review, we present several of the major themes that were discussed throughout the meeting and highlight the depth and range of research currently being performed to uncover the causes of human cardiac diseases and develop potential therapies.

The social management of biomedical novelty: Facilitating translation in regenerative medicine.

Regenerative medicine (RM) is championed as a potential source of curative treatments for a variety of illnesses, and as a generator of economic wealth and prosperity. Alongside this optimism, however, is a sense of concern that the translation of basic science into useful RM therapies will be laboriously slow due to a range of challenges relating to live tissue handling and manufacturing, regulation, reimbursement and commissioning, and clinical adoption. This paper explores the attempts of stakeholders to overcome these innovation challenges and thus facilitate the emergence of useful RM therapies. The paper uses the notion of innovation niches as an analytical frame. Innovation niches are collectively constructed socio-technical spaces in which a novel technology can be tested and further developed, with the intention of enabling wider adoption. Drawing on primary and secondary data, we explore the motivation for, and the attempted construction of, niches in three domains which are central to the adoption of innovative technologies: the regulatory, the health economic, and the clinical. We illustrate that these niches are collectively constructed via both formal and informal initiatives, and we argue that they reflect wider socio-political trends in the social management of biomedical novelty.

[Clinical and organizational way of innovative development of regenerative medicine in security agencies].

The article covers organizational aspects of development of innovative technologies in the field of regenerative medicine. It is shown that for the effective design and implementation into medical practice of regenerative medicine requires a united complex of military health care, military medical research and education. The main goal is to formate a biological insurance of personnel to treat different consequences of radiological incidents, burn disease, identification of the remains of the victims; the maximum returning to action after disturbed as a result of health services. Proposes the creation of "Interdepartmental Clinical Research and Education Center for Regenerative Medicine", combining research, clinical, industrial and educational potential of the leading institutions of various departments that will enhance the national security of the Russian Federation.

Organizational profile: UK regenerative medicine platform immunomodulation hub.

The UK Regenerative Medicine Platform was launched in 2013 as a jointly funded venture by the Biotechnology and Biological Sciences Research Council (BBSRC), Biotechnology and Biological Sciences Research Council, Engineering and Physical Sciences Research Council (EPSRC) and Medical Research Council (MRC) to address the technical and scientific challenges associated with translating promising scientific discoveries into the clinical setting. The first stage of the Platform involved the establishment of five interdisciplinary and cross-institutional research Hubs and the final Hub, the Immunomodulation Hub, was formed in 2014. The Immunomodulation Hub comprises scientists from diverse clinical and nonclinical research backgrounds. Collectively, they provide expertise in tissues for which there is an unmet clinical need for regenerative treatments, in innate and adaptive immunity and in whole organ transplantation. Their vision is that by working together to determine how regenerative medicine cell therapies in a laboratory setting are affected by the immune system, they will make a substantial contribution to long-term clinical deliverables that include improved efficacy of photoreceptor cell therapy to treat blindness; improved repair of damaged heart tissue; and improved survival and functionality of transplanted hepatocytes as an alternative to liver transplantation.

Manufacturing road map for tissue engineering and regenerative medicine technologies.

The Regenerative Medicine Foundation Annual Conference held on May 6 and 7, 2014, had a vision of assisting with translating tissue engineering and regenerative medicine (TERM)-based technologies closer to the clinic. This vision was achieved by assembling leaders in the field to cover critical areas. Some of these critical areas included regulatory pathways for regenerative medicine therapies, strategic partnerships, coordination of resources, developing standards for the field, government support, priorities for industry, biobanking, and new technologies. The final day of this conference featured focused sessions on manufacturing, during which expert speakers were invited from industry, government, and academia. The speakers identified and accessed roadblocks plaguing the field where improvements in advanced manufacturing offered many solutions. The manufacturing sessions included (a) product development toward commercialization in regenerative medicine, (b) process challenges to scale up manufacturing in regenerative medicine, and (c) infrastructure needs for manufacturing in regenerative medicine. Subsequent to this, industry was invited to participate in a survey to further elucidate the challenges to translation and scale-up. This perspective article will cover the lessons learned from these manufacturing sessions and early results from the survey. We also outline a road map for developing the manufacturing infrastructure, resources, standards, capabilities, education, training, and workforce development to realize the promise of TERM.

Can we assure quality without stifling innovation?

The emergence of regenerative medicine raises new questions about the best ways to ensure the safety and efficacy of stem cell therapies for patients. The Foundation for the Accreditation of Cellular Therapy (FACT) believes minimum requirements for cellular therapy for regenerative medicine will promote patient safety, protect the research environment, and aid in the swift advancement of regenerative therapies from bench to bedside. The standards development process has demonstrated that consensus on minimum requirements advances the field. FACT's accreditation process and newly established "Common Standards for Cellular Therapies" can help meet unmet needs in regenerative medicine that will drive commercialization of new cell therapies.