ISCT 2021 – VJRegenMed

Evolution of cell therapy for cardiovascular disease

admin_vjr_user — Fri, 07 Jan 2022 12:26:06 +0000

Stefan Janssens, MD, PhD, University Hospital Leuven, Leuven, Belgium, describes the evolving landscape of regenerative cell-based therapies for cardiovascular disease. Lessons learned from early clinical studies led to the investigation of whether cardiac repair mechanisms could be modulated by leveraging the paracrine activity of bone marrow-derived MSCs. Given the reduced cardiomyogenic capacity of these cells, it was later deemed that cardiac progenitor cells have a greater capacity for cardiac muscle regeneration, however, at an insufficient level to mediate a clinical effect. There is therefore ongoing research into the potential application of induced pluripotent stem cell (iPSC)-derived cardiac myocytes for the repair of advanced stage of heart failure. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.

Advances in organ bioengineering

admin_vjr_user — Tue, 04 Jan 2022 17:52:19 +0000

Paolo De Coppi, MD, PhD, FMedSci, UCL Great Ormond Street Institute of Child Health & NIHR Biomedical Research Centre, London, UK, provides an overview of the current landscape of bioengineering for organ replacement and some of the key considerations during the development of tissue-engineered organs, including the types of cell and polymers used. He notes that for the regeneration of simple tissues, autologous somatic cells may be sufficient, however, for more complex organs, pluripotent stem cells would likely be required, which would require immunosuppression upon implantation in order to avoid tissue rejection. Dr De Coppi also describes ongoing research into the use of biliary organoids for the repair of the damaged liver. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.

Driving the translation of intestinal tissue engineering

admin_vjr_user — Tue, 04 Jan 2022 17:52:17 +0000

Paolo De Coppi, MD, PhD, FMedSci, UCL Great Ormond Street Institute of Child Health & NIHR Biomedical Research Centre, London, UK, describes ongoing research aiming to address challenges facing the development of tissue engineering strategies for the treatment of intestinal failure and their translation into patients. While it has been demonstrated that organoid technology can used to replace the intestinal mucosa, the development of Good Manufacturing Practice (GMP)-compliant methods for organoid expansion are required before human transplantation. Given the organ complexity of the intestine, instead of engineering the entire intestine, ongoing research is focused on engineering a functional small intestine mucosa which could be transplanted into the large intestine. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.

The potential of vesicular secretome fraction in hearing loss

admin_vjr_user — Thu, 26 Aug 2021 14:05:30 +0000

Eva Rohde, MD, Medical University Salzburg, Salzburg, Austria, describes ongoing research evaluating the therapeutic potential of the vesicular secretome fraction (VSF) in hearing loss. A single patient with Meniere’s disease, a condition of the inner ear, received a co-injection of VSF in one ear during surgical cochlear implantation, which resulted in a slight improvement in cochlear electrode function and no short- or long-term adverse events. The initiation of a Phase I safety clinical trial is pending, depending on the outcomes of ongoing pre-clinical safety testing, including biodistribution and toxicology assays. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.

Promising avenues in extracellular vesicle-based therapeutics

admin_vjr_user — Tue, 24 Aug 2021 13:06:54 +0000

Eva Rohde, MD, Medical University Salzburg, Salzburg, Austria, provides an overview of the current landscape of research into extracellular vesicle (EV)-based therapeutics. She notes that in the last decade, the field has moved from basic, discovery research towards translation into the clinic, with a number of approaches currently being evaluated in clinical trials. Dr Rohde describes a number of EV-based therapeutic strategies under investigation, including the use of mesenchymal stromal cell (MSC)-derived EVs in neurodegenerative disorders, as well as the use of EVs in bone and tendon regeneration. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.

Remaining barriers to the translation of EV-based therapies

admin_vjr_user — Tue, 24 Aug 2021 13:06:53 +0000

Eva Rohde, MD, Medical University Salzburg, Salzburg, Austria, considers some of the major bottlenecks that need to be addressed in order to drive the translation of extracellular vesicle-derived therapeutics to the clinic. She highlights the importance of collecting comparable preclinical data, the thorough preclinical characterization of EVs and secreted factors, as well an early focus on Chemistry, Manufacturing, and Control (CMC) processes. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.

MSCs for type I diabetes

admin_vjr_user — Mon, 23 Aug 2021 17:29:15 +0000

Lindsay Davies, PhD, Karolinska Institutet & NextCell Pharma AB, Huddinge, Sweden, describes the rationale behind mesenchymal stromal cell (MSC)-based therapy for Type I diabetes (T1D). Dr Davies describes a strategy that involves the administration of a single dose of MSCs, with the aim of re-educating of the patient’s immune system and restoring beta cell function, thus reducing the co-morbidities associated with late-stage T1D, such as chronic wounds. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.

The benefits of Wharton’s jelly-derived MSCs

admin_vjr_user — Mon, 23 Aug 2021 17:29:14 +0000

Lindsay Davies, PhD, Karolinska Institutet & NextCell Pharma AB, Huddinge, Sweden, describes the benefits associated with the use of Wharton’s jelly-derived mesenchymal stromal cells (WJ-MSCs) compared to other MSC sources. WJ-MSCs are easily accessible from umbilical cord tissue, are highly immunomodulatory, can be used to generate large doses of cell product and are associated with fewer ethical issues compared to bone marrow-derived MSCs, given their ease of access and non-invasive methods of procurement. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.

ProTrans: Wharton’s jelly-derived MSC therapy for type I diabetes

admin_vjr_user — Mon, 23 Aug 2021 17:29:12 +0000

Lindsay Davies, PhD, Karolinska Institutet & NextCell Pharma AB, Huddinge, Sweden, describes a single center Phase II trial (NCT03406585) evaluating ProTrans, an allogeneic Wharton’s jelly-derived mesenchymal stromal cell (WJ-MSC)-based product, for the treatment of type I diabetes (T1D). This study demonstrated that a single infusion of ProTrans in adult patients with T1D resulted in maintenance of beta cell function, measured through the delta-change of C-peptide Area Under the Curve (AUC). There are currently plans to evaluate this therapy in a multicenter Phase III trial, as well as in a pediatric patient population. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.

Developing the bioartificial pancreas

admin_vjr_user — Thu, 19 Aug 2021 11:47:29 +0000

Ekaterine Berishvili, MD, PhD, University of Geneva, Geneva, Switzerland, describes ongoing research towards the development of the bioartificial pancreas for the treatment of type I diabetes, through the use of a range of tissue engineering, immune modulation and genome editing strategies. Given their immunomodulatory characteristics, he discusses strategies under investigation that utilize placental derivatives, as well as the development of placenta extracellular matrix-based biomaterials. The generation and transplantation of insulin-secreting organoids into a diabetic mouse model demonstrated successful engraftment, vascularization and therapeutic benefit. This interview took place during the International Society for Cell & Gene Therapy (ISCT) Annual Meeting 2021.