scholarly journals Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)

Gene Therapy ◽  
2021 ◽  
Author(s):  
Jennifer E. Adair ◽  
Lindsay Androski ◽  
Lois Bayigga ◽  
Deus Bazira ◽  
Eugene Brandon ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Working Group ◽  
The United States ◽  
Phase I Clinical Trials ◽  
Middle Income ◽  
Gene Therapies ◽  
Middle Income Countries ◽  
Development Capacity ◽  
Therapy Development ◽  
Therapy Field

AbstractThe gene and cell therapy field saw its first approved treatments in Europe in 2012 and the United States in 2017 and is projected to be at least a $10B USD industry by 2025. Despite this success, a massive gap exists between the companies, clinics, and researchers developing these therapeutic approaches, and their availability to the patients who need them. The unacceptable reality is a geographic exclusion of low-and middle-income countries (LMIC) in gene therapy development and ultimately the provision of gene therapies to patients in LMIC. This is particularly relevant for gene therapies to treat human immunodeficiency virus infection and hemoglobinopathies, global health crises impacting tens of millions of people primarily located in LMIC. Bridging this divide will require research, clinical and regulatory infrastructural development, capacity-building, training, an approval pathway and community adoption for success and sustainable affordability. In 2020, the Global Gene Therapy Initiative was formed to tackle the barriers to LMIC inclusion in gene therapy development. This working group includes diverse stakeholders from all sectors and has set a goal of introducing two gene therapy Phase I clinical trials in two LMIC, Uganda and India, by 2024. Here we report on progress to date for this initiative.

scholarly journals The United States’ Regulatory Environment Is Evolving to Accommodate a Coming Boom in Gene Therapy Research

2019 ◽  
Vol 24 (3) ◽  
pp. 147-152 ◽  
Author(s):  
Daniel Eisenman
Keyword(s):  
United States ◽  
Gene Therapy ◽  
Clinical Trials ◽  
The United States ◽  
Regulatory Environment ◽  
Evidence Based ◽  
Regulatory Structure ◽  
Regulatory Changes ◽  
Current State ◽  
Therapy Field

Introduction: A dramatic increase in the number of clinical trials involving gene-modified cell therapy and gene therapy is taking place. The field is on the verge of a boom, and the regulatory environment is evolving to accommodate the growth. Discussion: This commentary summarizes the current state of the field, including an overview of the growth. The United States (US) regulatory structure for gene therapy will be summarized, and the evolution of the oversight structure will be explained. Conclusion: The gene therapy field has recently produced its first FDA-approved therapeutics and has a pipeline of other investigational products in the final stages of clinical trials before they can be evaluated by the FDA as safe and effective therapeutics. As research continues to evolve, so must the oversight structure. Biosafety professionals and IBCs have always played key roles in contributing to the safe, evidence-based advancement of gene therapy research. With the recent regulatory changes and current surge in gene therapy research, the importance of those roles has increased dramatically.

scholarly journals Global neurosurgery: a scoping review detailing the current state of international neurosurgical outreach

2020 ◽  
pp. 1-9
Author(s):  
Anthony T. Fuller ◽  
Ariana Barkley ◽  
Robin Du ◽  
Cyrus Elahi ◽  
Ali R. Tafreshi ◽  
...  
Keyword(s):  
Scoping Review ◽  
Data Extraction ◽  
Medical Personnel ◽  
The United States ◽  
Middle Income ◽  
Middle Income Countries ◽  
Gray Literature ◽  
Current State ◽  
Study Selection ◽  
Research And Education

OBJECTIVEGlobal neurosurgery is a rapidly emerging field that aims to address the worldwide shortages in neurosurgical care. Many published outreach efforts and initiatives exist to address the global disparity in neurosurgical care; however, there is no centralized report detailing these efforts. This scoping review aims to characterize the field of global neurosurgery by identifying partnerships between high-income countries (HICs) and low- and/or middle-income countries (LMICs) that seek to increase neurosurgical capacity.METHODSA scoping review was conducted using the Arksey and O’Malley framework. A search was conducted in five electronic databases and the gray literature, defined as literature not published through traditional commercial or academic means, to identify studies describing global neurosurgery partnerships. Study selection and data extraction were performed by four independent reviewers, and any disagreements were settled by the team and ultimately the team lead.RESULTSThe original database search produced 2221 articles, which was reduced to 183 final articles after applying inclusion and exclusion criteria. These final articles, along with 9 additional gray literature references, captured 169 unique global neurosurgery collaborations between HICs and LMICs. Of this total, 103 (61%) collaborations involved surgical intervention, while local training of medical personnel, research, and education were done in 48%, 38%, and 30% of efforts, respectively. Many of the collaborations (100 [59%]) are ongoing, and 93 (55%) of them resulted in an increase in capacity within the LMIC involved. The largest proportion of efforts began between 2005–2009 (28%) and 2010–2014 (17%). The most frequently involved HICs were the United States, Canada, and France, whereas the most frequently involved LMICs were Uganda, Tanzania, and Kenya.CONCLUSIONSThis review provides a detailed overview of current global neurosurgery efforts, elucidates gaps in the existing literature, and identifies the LMICs that may benefit from further efforts to improve accessibility to essential neurosurgical care worldwide.

Trump's Declaration of the Global Gag Rule

2021 ◽  
pp. 277-294
Author(s):  
Sudeep Uprety ◽  
Obindra B. Chand
Keyword(s):  
United States ◽  
Health Research ◽  
The United States ◽  
Middle Income ◽  
Government Organization ◽  
Middle Income Countries ◽  
Us Government ◽  
Securitization Theory ◽  
The Media ◽  
The Impact

The current expanded policy on the Global Gag Rule by the United States (US) government and President Donald Trump has led to wider debate and discussions among the non-government organization (NGO) sector, especially in low and middle income countries (LMICs) such as Nepal that are heavily reliant on US funding for health research and intervention projects. Debates and discussions are also shaped by how the media shapes the narrative. Using the securitization theory, this chapter attempts to unfold the trend and the nature of stories reported in Nepali media on the Global Gag Rule declaration, meticulously unfolding the impact it has had in Nepal.

scholarly journals Tracheostomy During the COVID-19 Pandemic: Comparison of International Perioperative Care Protocols and Practices in 26 Countries

2020 ◽  
pp. 019459982096198
Author(s):  
Carol Bier-Laning ◽  
John D. Cramer ◽  
Soham Roy ◽  
Patrick A. Palmieri ◽  
Ayman Amin ◽  
...  
Keyword(s):  
Resource Constraints ◽  
Invasive Mechanical Ventilation ◽  
The United States ◽  
Postoperative Management ◽  
Reference Points ◽  
Data Sources ◽  
Cross Sectional ◽  
Middle Income ◽  
Middle Income Countries ◽  
Evidence Based Care

Objective The coronavirus disease 2019 (COVID-19) pandemic has led to a global surge in critically ill patients requiring invasive mechanical ventilation, some of whom may benefit from tracheostomy. Decisions on if, when, and how to perform tracheostomy in patients with COVID-19 have major implications for patients, clinicians, and hospitals. We investigated the tracheostomy protocols and practices that institutions around the world have put into place in response to the COVID-19 pandemic. Data Sources Protocols for tracheostomy in patients with severe acute respiratory syndrome coronavirus 2 infection from individual institutions (n = 59) were obtained from the United States and 25 other countries, including data from several low- and middle-income countries, 23 published or society-endorsed protocols, and 36 institutional protocols. Review Methods The comparative document analysis involved cross-sectional review of institutional protocols and practices. Data sources were analyzed for timing of tracheostomy, contraindications, preoperative testing, personal protective equipment (PPE), surgical technique, and postoperative management. Conclusions Timing of tracheostomy varied from 3 to >21 days, with over 90% of protocols recommending 14 days of intubation prior to tracheostomy. Most protocols advocate delaying tracheostomy until COVID-19 testing was negative. All protocols involved use of N95 or higher PPE. Both open and percutaneous techniques were reported. Timing of tracheostomy changes ranged from 5 to >30 days postoperatively, sometimes contingent on negative COVID-19 test results. Implications for Practice Wide variation exists in tracheostomy protocols, reflecting geographical variation, different resource constraints, and limited data to drive evidence-based care standards. Findings presented herein may provide reference points and a framework for evolving care standards.

scholarly journals Implementation of Multicolor Melt Curve Analysis for High-Risk Human Papilloma Virus Detection in Low- and Middle-Income Countries: A Pilot Study for Expanded Cervical Cancer Screening in Honduras

2018 ◽  
pp. 1-8 ◽  
Author(s):  
Scott A. Turner ◽  
Sophie J. Deharvengt ◽  
Kathleen Doyle Lyons ◽  
Jorge Arturo Plata Espinal ◽  
Ethan P.M. LaRochelle ◽  
...  
Keyword(s):  
Cervical Cancer ◽  
High Risk ◽  
Medical Center ◽  
Virus Detection ◽  
The United States ◽  
Middle Income ◽  
Rural Village ◽  
Middle Income Countries ◽  
Dartmouth Hitchcock Medical ◽  
Low And Middle Income

Purpose Cervical cancer is a leading cause of cancer-related mortality in low- and middle-income countries (LMICs) and screening in LMICs is extremely limited. We aimed to implement on-site high-risk human papillomavirus (hrHPV) DNA testing in cohorts of women from an urban factory and from a rural village. Methods A total of 802 women were recruited for this study in partnership with La Liga Contra el Cancer through the establishment of women’s health resource fairs at two locations in Honduras: a textile factory (n = 401) in the city of San Pedro Sula and the rural village of El Rosario (n = 401) in Yoro. Participants received a routine cervical examination during which three sterile cytobrushes were used to collect cervical samples for testing. hrHPV genotyping was performed using a hrHPV genotyping assay and a real-time polymerase chain reaction instrument. Results hrHPV status across all participants at both sites was 13% hrHPV positive and 67% hrHPV negative. When hrHPV status was compared across all three testing sites, hrHPV-positive rates were approximately equal among the factory (13%), village (12%), and confirmatory testing at Dartmouth-Hitchcock Medical Center (Lebanon, NH; 14%). hrHPV genotype was compared across sites, with HPV16 showing the highest infection rate (15%), followed by HPV59 (12%), and HPV68 (11%). There was a low prevalence of HPV18 observed in both populations compared with the hrHPV-positive population in the United States. Conclusion In collaboration with oncologists and pathologists from La Liga Contra el Cancer, we were able to provide a continuum of care once health-fair testing was performed. We established a method and implementation plan for hrHPV testing that is sustainable in LMICs.

scholarly journals Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective

2020 ◽  
Vol 19 ◽  
pp. 89-98
Author(s):  
Michael W. Bolt ◽  
Laurence O. Whiteley ◽  
Jessica L. Lynch ◽  
Brian Lauritzen ◽  
Antonio R. Fernández de Henestrosa ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Working Group ◽  
Viral Vector ◽  
Gene Therapies

Gene therapy comes of age

Science ◽  
2018 ◽  
Vol 359 (6372) ◽  
pp. eaan4672 ◽  
Author(s):  
Cynthia E. Dunbar ◽  
Katherine A. High ◽  
J. Keith Joung ◽  
Donald B. Kohn ◽  
Keiya Ozawa ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
The United States ◽  
Genetically Engineered ◽  
Drug Status ◽  
Hematopoietic Stem ◽  
Gene Therapies ◽  
Engineered T Cells ◽  
Near Future

After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers recently progressed to approved drug status in the United States and Europe, or are anticipated to receive approval in the near future. In this Review, we discuss milestones in the development of gene therapies, focusing on direct in vivo administration of viral vectors and adoptive transfer of genetically engineered T cells or hematopoietic stem cells. We also discuss emerging genome editing technologies that should further advance the scope and efficacy of gene therapy approaches.

scholarly journals COVID-19 Therapeutics for Low- and Middle-Income Countries: A Review of Candidate Agents with Potential for Near-Term Use and Impact

2021 ◽  
Author(s):  
Daniel Maxwell ◽  
Kelly C. Sanders ◽  
Oliver Sabot ◽  
Ahmad Hachem ◽  
Alejandro Llanos-Cuentas ◽  
...  
Keyword(s):  
United States ◽  
European Union ◽  
The United States ◽  
Middle Income ◽  
Middle Income Countries ◽  
Negative Results ◽  
Clinical Trial Registries ◽  
Oral Agents ◽  
Near Term ◽  
Low And Middle Income

Low- and middle-income countries (LMICs) face significant challenges in the control of COVID-19, given limited resources, especially for inpatient care. In a parallel effort to that for vaccines, the identification of therapeutics that have near-term potential to be available and easily administered is critical. Using the United States, European Union, and WHO clinical trial registries, we reviewed COVID-19 therapeutic agents currently under investigation. The search was limited to oral or potentially oral agents, with at least a putative anti-SARS-CoV-2 virus mechanism and with at least five registered trials. The search yielded 1,001, 203, and 1,128 trials, in the United States, European Union, and WHO trial registers, respectively. These trials covered 13 oral or potentially oral repurposed agents that are currently used as antimicrobials and immunomodulatory therapeutics with established safety profiles. The available evidence regarding proposed mechanism of actions, potential limitations, and trial status is summarized. The results of the search demonstrate few published studies of high quality, a low proportion of trials completed, and the vast majority with negative results. These findings reflect limited investment in COVID-19 therapeutics development compared with vaccines. We also identified the need for better coordination of trials of accessible agents and their combinations in LMICs. To prevent COVID-19 from becoming a neglected tropical disease, there is critical need for rapid and coordinated effort in the evaluation and deployment of those agents found to be efficacious.

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