Mohamed Abou-el-Enein

Mohamed Abou-el-Enein
Keck School of Medicine USC · Department of Medicine

MD PhD MSPH
Executive Director, USC/CHLA Cell Therapy Program

About

43
Publications
23,162
Reads
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839
Citations
Additional affiliations
February 2010 - December 2020
Charité Universitätsmedizin Berlin
Position
  • Head of Department

Publications

Publications (43)
Article
Full-text available
Chimeric antigen receptor (CAR) T cells have emerged as a potent therapeutic approach for patients with certain haematological cancers, with multiple CAR T cell products currently approved by the FDA for those with relapsed and/or refractory B cell malignancies. However, in order to derive the desired level of effectiveness, patients need to succes...
Article
Full-text available
Graft-versus-Host-Disease (GvHD) is still the major non-relapse, life-limiting complication following hematopoietic stem cell transplantation. Modern pharmacologic immunosuppression is often insufficient and associated with significant side effects. Novel treatment strategies now include adoptive transfer of ex vivo expanded regulatory T-cells (Tre...
Chapter
Full-text available
The development of genetically modified chimeric antigen receptor (CAR) T-cells to target cancer by conferring tumour antigen recognition has tremendously improved the fight against the disease and broadened treatment options for haematological malignancies. However, in contrast to conventional drugs that patients can easily access, the implementat...
Chapter
Full-text available
Advanced therapy medicinal products (ATMPs), comprising cell, gene therapy, and tissue-engineered products, represent a new line of defense against diseases with currently limited or no effective treatment options. For instance, chimeric antigen receptor (CAR) T cell approaches have been showing substantial clinical benefits in some hematologic mal...
Article
Full-text available
The emergence of new cell and gene-based therapies (CGTs) utilizing innovative technologies has recently intensified. Long-standing efforts in publicly funded biomedical research have resulted in breakthrough therapeutic approaches for patients with devastating and life-threatening diseases. Transformative gene-based therapeutic tools include human...
Article
Full-text available
As of April 2021, there are five commercially available chimeric antigen receptor (CAR) T cell therapies for hematologic malignancies. With the current transition of CAR T cell manufacturing from academia to industry, there is a shift toward Good Manufacturing Practice (GMP)–compliant closed and automated systems to ensure reproducibility and to me...
Article
Full-text available
The unproven stem cell intervention (SCI) industry is a global health problem. Despite efforts of some nations, the industry continues to flourish. In this paper, we call for a global approach and the establishment of a World Health Organization (WHO) Expert Advisory Committee on Regenerative Medicine to tackle this issue and provide guidance. The...
Article
Full-text available
Patient-derived T cells genetically reprogrammed to express CD19-specific chimeric antigen receptors (CARs) have shown remarkable clinical responses and are commercially available for the treatment of patients with certain advanced-stage B cell malignancies. Nonetheless, several trials have revealed pre-existing and/or treatment-induced immune resp...
Preprint
Full-text available
Background: The ongoing COVID-19 pandemic, caused by the novel coronavirus SARS-CoV-2, represents a serious worldwide health concern. A deeper understanding of the immune response to SARS-CoV-2 will be required to refine vaccine development and efficacy as well as to evaluate long-term immunity in convalescent patients. With this in mind, we invest...
Article
Full-text available
Objective To assess whether reshaping of the immune balance by infusion of autologous natural regulatory T cells (nTregs) in patients after kidney transplantation is safe, feasible, and enables the tapering of lifelong high dose immunosuppression, with its limited efficacy, adverse effects, and high direct and indirect costs, along with addressing...
Article
Full-text available
Preventing the progression to acute respiratory distress syndrome (ARDS) in COVID-19 is an unsolved challenge. The involvement of T cell immunity in this exacerbation remains unclear. To identify predictive markers of COVID-19 progress and outcome, we analyzed peripheral blood of 10 COVID-19-associated ARDS patients and 35 mild/moderate COVID-19 pa...
Article
Full-text available
Chimeric antigen receptor (CAR) technology and its application to regulatory T cells (Tregs) has garnered interest among researchers in the field of cell and gene therapy. Merging the benefits of CAR technology with Tregs offers a novel and promising therapeutic option for durable reshaping of undesired immune responses following solid organ or hem...
Article
Full-text available
We aim to improve the clinical/translational methodologies that developers (including us) use to generate evidence on the safety and efficacy of cell and gene therapies, de-risk the process and provide guidance as to ‘best practices’ in this field.
Article
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Cellular senescence is implicated as a contributing factor in the pathology of several age-related disorders and cancer development. Therefore, targeted removal of senescent cells has emerged as a promising treatment strategy for these conditions. However, current senolytic strategies that selectively eliminate senescent cells are limited by their...
Article
Full-text available
Advanced therapy medicinal products (ATMPs) comprising cell therapy, gene therapy, and tissue-engineered products, offer a multitude of novel therapeutic approaches to a wide range of severe and debilitating diseases. To date, several advanced therapies have received marketing authorization for a variety of indications. However, some products showe...
Article
Full-text available
Chimeric antigen receptor (CAR) T cells represent a potent new approach to treat haematological malignancies. Two CAR T-cell therapies, tisagenlecleucel and axicabtagene ciloleucel, have been approved in Europe and the USA, as well as several other countries, for the treatment of leukaemia and lymphoma. These approvals marked a major milestone in t...
Article
Full-text available
The implementation of new regulatory tools, such as the PRIority MEdicine (PRIME) scheme, by regulatory authorities in Europe enabled faster patient access to innovative therapies. This early access tool goes along with a clear need for a thorough assessment of safety and efficacy upon marketing authorization. Due to the higher degree of uncertaint...
Article
Medicinal products based on genome editing must undergo rigorous preclinical testing and are subject to regulatory oversight for proper risk assessment prior to first evaluation in humans. We give a European perspective on the regulatory expectations to translate genome editing to the clinic to ensure their timely progress to market.
Article
Full-text available
Although there is great potential for cell and gene therapies, most of those currently approved for clinical use, particularly CAR-T cell products, were evaluated in pivotal trials of less than 100 patients. Having such limited data means that there may be a larger-than-usual gap between what is known about the safety and efficacy of these therapie...
Article
The biomedical field has witnessed remarkable advances in analytical tools and technologies that have expanded our understanding of healthy and diseased human tissue and, at the same time, enable extensive molecular characterization of living cells. The volume of scientific data generated is expanding in an unprecedented manner; however, these data...
Data
Figure S1. Telbivudine pretreatment of parvovirus B19‐infected endothelial cells reduces the pro‐apoptotic potential of parvovirus B19‐infected endothelial cells upon co‐culture in HL‐1 cardiomyocytes. A. Experimental design: HMEC‐1 were 24 h after plating, treated with/out Telbivudine for 2 h, next infected with 10 000 viral particles/cell and col...
Article
Full-text available
Aims Myocarditis is often associated with parvovirus B19 (B19V) persistence, which can induce vascular damage. Based on the antiviral and anti‐inflammatory properties of telbivudine, we aimed to evaluate its efficacy to protect B19V‐infected endothelial cells in vitro and to treat chronic lymphocytic myocarditis patients with B19V transcriptional a...
Article
Full-text available
The promise of stem cell (SC) therapies to restore functions of damaged tissues and organs brings enormous hope to patients, their families, loved ones and caregivers. However, limits may exist for which indications SC therapies might be useful, efficacious, and safe. Applications of innovative therapies within regulatory boundaries and within the...
Article
Full-text available
Clinical registries collecting patient data from cell and gene therapies represent an essential activity that support therapeutic translational evidence necessary for confident product approval.
Article
Clinical trials are crucial to determining the human safety and efficacy of new therapeutic innovations. Extraordinary amounts of human experiential data are generated over the course of any clinical trial, however, much of these data is never made publicly accessible. Improved, reliable data sharing is essential to inform clinical decisions and in...
Article
Full-text available
ATMPs are expected to provide a major contribution to the field of next generation medicines. Acknowledging the innovative nature of these products, EU lawmakers and regulatory bodies have not insisted on conventional rigid regulatory expectations, but have installed a number of flexible tools to facilitate marketing and patient access. Here, we di...
Article
As genome editing rapidly progresses toward the realization of its clinical promise, assessing the suitability of current tools and processes used for its benefit-risk assessment is critical. Although current regulations may initially provide an adequate regulatory framework, improvements are recommended to overcome several existing technology-base...
Article
Full-text available
Gene therapy has, after years of setbacks, returned as a highly advantageous novel therapy for the treatment of severely debilitating diseases for which there were no treatment options. As marketed therapeutic products, gene therapies will add an arsenal of new options to provide life-saving clinical benefits to many patients worldwide.
Article
Full-text available
Often, novel gene and cell therapies provide hope for many people living with incurable diseases. To facilitate and accelerate a successful regulatory approval and commercialization path for effective, safe and affordable cell and gene therapies, the involvement of patient advocacy groups (PAGs) should be considered early in the development process...
Article
Full-text available
Cellular therapies have potential to treat a wide range of diseases with autologous immunotherapies showing unprecedented therapeutic promise in clinical trials. Such therapies are mainly developed by academic researchers applying small-scale production, targeting rare and unmet medical needs. Here we highlight the clinical translation of immunothe...
Article
While advanced therapy medicinal products offer great clinical promise, most EU-approved products have not achieved satisfactory commercial performance. Here we highlight a number of issues that prevent current products from obtaining commercial success and pitfalls that developers must overcome in future product development.
Article
Inefficient translational processes for technology-oriented biomedical research have led to some prominent and frequent failures in the development of many lead drug candidates, several designated investigational drugs, and some medical devices, as well as documented patient harm and post-market product withdrawals. De-risking this process, particu...
Article
Full-text available
Cell therapies, especially autologous therapies, pose significant challenges to researchers who wish to move from small, probably academic, methods of manufacture to full commercial scale. There is a dearth of reliable information about the costs of operation, and this makes it difficult to predict with confidence the investment needed to translate...
Article
Full-text available
The new EU Clinical Trials Regulation will replace the current Directive, providing a directly applicable legal act throughout the EU. It also offers new principles and provisions that aim to counteract the limitations of working with the Directive, promote better harmonization and increase transparency in the reporting of clinical trial results. W...
Article
Full-text available
Despite undeniable accomplishments in developing cell and gene therapeutic strategies to combat HIV infection, key social, economic, and policy-related challenges still need to be overcome for any future commercialization efforts of these novel therapies to be successful. Here, we address these challenges and structure a framework for eradicating H...
Article
Full-text available
We believe that gene therapies should be made affordable, that development of gene therapy applications already funded by public funds should be licensed to manufacturers at a reasonable price and that less cost-intensive manufacturing technologies should be developed. Gene therapies should also be accessible to patients in developing nations.
Article
Full-text available
During the past decade, successful gene therapies for immunodeficiencies were finally brought to the clinic. This was accomplished through new gene therapy vectors and improved procedures for genetic modification of autologous hematopoietic stem cells. For HIV, autologous hematopoietic stem cell (HSC) gene therapy with ‘anti-HIV genes’ promises a f...

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Projects (3)
Project
Notably, several limiting factors exist that block advanced therapies from commercial viability and universal adoption by providers. Tailored strategies need to be in place in order to enhance the process of translating these novel therapies efficiently into clinical applications that are sustainable on the market while involving all stakeholders at the most appropriate points early in the process.