Autologous Interventions Research Articles

Autologous stem cell-based interventions in Australia: exploring patient experience in light of regulatory exceptionalism

Background & Aim Over the last decade there has been a growing prevalence of privately owned clinics in Australia providing autologous interventions to patients based on ‘stem cells’ isolated from their own fat, blood or bone marrow. These claimed stem cell-based interventions have primarily been marketed for osteoarthritis treatment, but have also been available for facial rejuvenation, hair restoration, and the treatment of motor neurone disease, Parkinson's disease and dementia. Such interventions are rarely part of a clinical research program, lack high level evidence of efficacy, and are not reimbursed by public or private insurers, resulting in patients paying significant out of pocket expenses (AUD$6,000 or more per intervention). Due to a regulatory exemption that broadly excluded the manufacturing and administration of autologous cells and tissues from the oversight of the Australian Therapeutic Goods Administration, clinics have been able to market directly and aggressively to patients in a manner that both exaggerates benefits and underplays risks. This study explores how Australian patients and their carers understood and experienced these interventions, and in particular how their presumptions about autologous stem cells, the ethics of medical practice, and the regulatory environment in Australia have conditioned their preparedness to undergo unproven treatments. Methods, Results & Conclusion Findings were drawn from two workshops conducted in 2016 with patients, their carers and family members (22 participants) and 15 semi-structured interviews (conducted in 2017). Patients’ consideration of, and decision to undergo, an autologous stem cell-based intervention was shaped by five factors: illness experience, disillusion with current medical practice, unrealistic expectations about the intervention, lack of information from providers, and trust in the health care system and clinicians more broadly. While recently announced regulatory amendments may in time provide greater patient protection, the previous ‘light touch’ regulatory approach clearly created the preconditions in Australia for clinical practices with questionable evidentiary basis, and for a market where patient vulnerability and risk could be monetized. In future, we argue, efforts to enhance consumer awareness of the unregulated and unproven nature of autologous stem cell-based interventions need to be coupled with sound regulation and periodic policy review to avoid exceptionalism and patient exploitation.

Challenges in the translation and commercialization of cell therapies.

BackgroundCell therapies are an emerging form of healthcare that offer significant potential to improve the practice of medicine and provide benefits to patients who currently have limited or no treatment options. Ideally, these innovative therapies can complement existing small molecule, biologic and device approaches, forming a so-called fourth pillar of medicine and allowing clinicians to identify the best treatment approach for each patient. Despite this potential, cell therapies are substantially more complex than small molecule or biologic interventions. This complexity poses challenges for scientists and firms developing cell therapies and regulators seeking to oversee this growing area of medicine.ResultsIn this project, we retrospectively examined the development of seven cell therapies – including three autologous interventions and four allogeneic interventions – with the aim of identifying common challenges hindering attempts to bring new cell therapies to market. We complemented this analysis with a series of qualitative interviews with experts in various aspects of cell therapy. Through our analysis, which included review of extant literature collected from company documents, newspapers, journals, analyst reports and similar sources, and analysis of the qualitative interviews, we identified several common challenges that cell therapy firms must address in both the pre- and post-market stages. Key pre-market challenges included identifying and maintaining stable funding to see firms through lengthy developmental timelines and uncertain regulatory processes. These challenges are not unique to cell therapies, of course, but the novelty of cell-based interventions complicates these efforts compared to small molecule or biologic approaches. The atypical nature of cell therapies also led to post-market difficulties, including challenges navigating the reimbursement process and convincing providers to change their treatment approaches. In addition, scaling up production, distributing cell therapies and managing the costs of production were challenges that started pre-market and continued into the post-market phase.ConclusionsOur analysis highlights several interrelated challenges hindering the development of cell therapies. Identifying strategies to address these challenges may accelerate the development and increase the impact of novel cell therapies.

Challenges in the translation and commercialization of cellular therapies

Background: Cell therapies are an emerging form of healthcare that offer significant potential to improve the practice of medicine and provide benefits to patients who currently have limited or no treatment options. Ideally, these innovative therapies can complement existing small molecule, biologic and device approaches, forming a so-called fourth pillar of medicine and allowing clinicians to identify the best treatment approach for each patient. Despite this potential, cell therapies are substantially more complex than small molecule or biologic interventions. This complexity poses challenges for scientists and firms developing cell therapies and regulators seeking to oversee this growing area of medicine. Results: In this project, we retrospectively examined the development of seven cell therapies – including three autologous interventions and four allogeneic interventions – with the aim of identifying common challenges hindering attempts to bring new cell therapies to market. We complemented this analysis with a series of qualitative interviews with experts in various aspects of cell therapy. Through our analysis, which included review of extant literature collected from company documents, newspapers, journals, analyst reports and similar sources, and analysis of the qualitative interviews, we identified several common challenges that cell therapy firms must address in both the pre- and post-market stages. Key pre-market challenges included identifying and maintaining stable funding to see firms through lengthy developmental timelines and uncertain regulatory processes. These challenges are not unique to cell therapies, of course, but the novelty of cell-based interventions complicates these efforts compared to small molecule or biologic approaches. The atypical nature of cell therapies also led to post-market difficulties, including challenges navigating the reimbursement process and convincing providers to change their treatment approaches. In addition, scaling up production, distributing cell therapies and managing the costs of production were challenges that started pre-market and continued into the post-market phase. Conclusions: Our analysis highlights several interrelated challenges hindering the development of cell therapies. Identifying strategies to address these challenges may accelerate the development and increase the impact of novel cell therapies.

Hair Follicle Plasticity with Complemented Immune-modulation Following Follicular Unit Extraction.

Background:During hair transplantation as an effective therapy for androgenetic alopecia, hair follicles were typically trans-located from the nonaffected occipital to the balding frontal or vertex region of the scalp. Although this is an autologous intervention, the donor and recipient hair follicle tissue differ in composition and local environment.Settings and Design:In two case studies, we investigated the changes in hair follicle morphology and the immune status of scalp and body hair follicles from different origins transplanted to the eyebrows and the frontal scalp using follicular unit extraction.Results:Quantitative histomorphometry and immunohistochemistry revealed a transformation in hair follicle length and dermal papilla size of the scalp, chest and beard hair follicles, which had been re-extracted after a 6-month period posttransplantation. Furthermore, a significant infiltration of B and T lymphocytes as well as macrophages could be observed most prominently in the infundibulum of transplanted hair follicles.Conclusion:The presented results demonstrate that hair follicle units from different body sites are capable to replace miniaturized or degraded hair follicles in different recipient areas like scalp or eyebrows as they keep their intrinsic capability or acquire the potential to readjust plastically within the beneficiary skin region. The essential secretory crosstalk underlying the observed tissue remodeling is possibly mediated by the infiltrating immune cells.