Expert Insight

The history of cord blood transplantation/biology & perspective for future efforts to enhance the field

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Expert Insight

Hal E Broxmeyer

Latest Advances in Cord Blood Applications & Commercialization

Cord blood hematopoietic stem and progenitor cells have been used successfully for hematopoietic cell transplantation to treat a variety of malignant and non-malignant disorders. Use of cord blood has advantages and disadvantages as a source of transplantable cells compared to that of bone marrow and mobilized peripheral blood. Most recently, haplo-identical transplants have been competing with cord blood as a source of transplanted cells. Efforts are ongoing to modulate cord blood and recipients of cord blood transplantation for more efficacious transplant outcomes, in part in order to overcome the slower time to neutrophil, platelet and immune cell recovery of these compared to the other sources of transplantable cells. This review briefly describes the history of cord blood biology and hematopoietic cell transplantation, efforts to collect more stem cells and to expand and better allow them to home to the bone marrow for more efficient and rapid engraftment. A personal perspective for future efforts in these areas is also provided.

Submitted for review: Feb 20 2017 Published: Sep 6 2017
DOI: 10.18609/cgti.2017.045
Citation: Cell Gene Therapy Insights 2017;3(7), 521-530.
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The Economics of Cord Blood Banking

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Interview

Michael Boo

Latest Advances in Cord Blood Applications & Commercialization

 

Michael Boo serves as a business consultant to the healthcare industry, particularly in the areas of blood and marrow transplantation. He is the former Chief Strategy Officer of the National Donor Program/Be The Match (NMDP), the national registry for the USA. He joined the NMDP in 2001. His contributions while at NMDP had included the redesign and implementation of a new strategic planning process that has helped guide substantial growth at NMDP, guide the development of the Center for Cord Blood in 2003, and expand NMDP growth in cord blood in the USA from 15% of the market to over 90%. Boo has identified and developed new products and services that have had significant bottom-line revenue impact and developed new relationships within the extensive NMDP network of national and international partners that have improved access to cell sources and markets worldwide. He has provided leadership and oversight for legislative activities, has helped establish a new federal funding program for cord blood inventory growth, which has provided more than $100 million to date, and pursued other strategies to continue and expand government funding sources. More recently, he has led a number of initiatives to address deficiencies in reimbursement for transplant related-costs by both the public and private payers.

DOI: 10.18609/cgti.2017.044
Citation: Cell Gene Therapy Insights 2017;3(7), 547-551.
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The critical role of shipping & logistics in the commercialization of cord blood therapies

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Innovator insight

David Murphy

David Murphy Quick Logistics

Latest Advances in Cord Blood Applications & Commercialization

 
 

David Murphy is a 30 year veteran of The Quick Group of Companies, holding various leadership roles in Quick’s Life Science division. Over the past 8 years, David has served as Executive Vice President of Quick Specialized Healthcare Logistics, and works closely with major healthcare organizations to develop specialized logistics solutions to safely transport human organs, tissue, blood and blood products for transplant or research. He also works with biotech and pharmaceutical companies to plan and implement transportation strategies for personalized medicine; including cell, gene and immunotherapy treatments. He develops scalable transportation solutions that preserve the product integrity of these life-saving shipments, and most importantly, the overall safety of patients. He helps to ensure adherence to the strict regulations of the life science industry and the chain of custody at every shipment milestone. David was instrumental in the logistics planning of the first FDA approved cancer vaccine, and subsequent commercialization roll out.

DOI: 10.18609/cgti.2017.048
Citation: Cell Gene Therapy Insights 2017; 3(6), 481-486.
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Emerging use of cord blood in regenerative medicine

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Expert Insight

Jessica Sun

Latest advances in cord blood applications and commercialization

Regenerative medicine is dedicated to the study of repairing, replacing or regenerating damaged human cells, tissues or organs to restore or establish normal function; and it has potential applications to treat a wide variety of conditions. Umbilical cord blood (CB) is a relatively safe, easily collected, readily available and non-controversial source of cells for regenerative medicine purposes. If it proves to be useful in this regard, there will be significant implications in both the treatment of diseases and the current models of CB collection.

Submitted for review: Aug 6 2017 Published: Sep 6 2017
DOI: 10.18609/cgti.2017.058
Citation: Cell Gene Therapy Insights 2017;3(7), 573-581.
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Optimal conditioning regimens for cord blood transplantation: advances and challenges

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Expert Insight

Kristin Page & Mitchell Horwitz

Latest advances in cord blood applications & commercialization

Conditioning regimens of all intensities have proven effective for patients in need of umbilical cord blood transplantation. While total body irradiation (TBI) continues to play a prominent role in most adult and pediatric regimens, there are now highly effective regimens without TBI. Children with malignant and non-malignant disorders receive predominantly myeloablative conditioning regimens. The clinical need for less toxic regimens for adult patients, particularly those with co-morbidities, has prompted clinical research activity in reduced intensity and non-myeloablative regimens capable or facilitating engraftment of single or double umbilical cord blood grafts. Here, we will describe the evolution of many of these regimens and how they are currently being utilized in stem cell transplant centers throughout the world.

Submitted for review: Jun 29 2017 Published: Sep 6 2017
DOI: 10.18609/cgti.2017.057
Citation: Cell Gene Therapy Insights 2017;3(7), 559-571.
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Cord blood transplantation in pediatric inherited metabolic diseases: an update on clinical developments

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Expert Insight

Jaap Jan Boelens

Latest Advances in Cord Blood Applications & Optimization

Inherited metabolic diseases (IMDs), arising from genetic defects, including deficiencies in the production of the lysosomal enzymes (lysosomal storage disease [LSDs]) and abnormalities of peroxisomal function, are associated with devastating systemic processes and premature death. Allogeneic hematopoietic cell transplantation (HCT) has been shown to be a treatment option for a selected group of patients with IMD. Cord blood has proven to be the preferred cell source for HCT for a variety of reasons; most importantly due to the superior long-term outcomes associated with cord blood. In this article, the evolution of the use of cord blood as a cell source for HCT and the potential of this source as an adjuvant cellular therapy (e.g., targeting specific organs or cell types) to potentially reduce or completely prevent residual disease burden after HCT is reviewed.

Submitted for review: Feb 8 2017 Published:Sep 5 2017
DOI: 10.18609/cgti.2017.046
Citation: Cell Gene Therapy Insights 2017;3(7), 531-539.
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Advances in the use of cord blood as a potential source of MSCs

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Interview

Giuseppe Astori

Latest advances in cord blood applications & commercialization

 

Giuseppe Astori is the head of the Advanced Cellular Therapy Laboratory (LTCA) at the Hematology of the Vicenza Hospital (Italy). His team focuses on the development of cellular therapies for the treatment of steroid-resistant graft-versus-host disease by using cord blood-derived mesenchymal stromal cells and for the immunotherapy of tumor relapse and viral reactivations after allogeneic hematopoietic stem cell transplantation. LTCA has an accredited laboratory at controlled contamination for the manipulation of transplants as for Directive 2004/23/EU and a GMP facility. Another area of interest of his group is the production and characterization of platelet derivatives. He has been nominated International Society of Cellular Therapy (ISCT) representative in the JACIE board and has been member of the European Leadership Committee of the ISCT (2014-2017). Before joining the Vicenza Hospital he worked at Cardiocentro Ticino, Switzerland where he was appointed Qualified Person of the GMP facility.

DOI: 10.18609/cgti.2017.054
Citation: Cell Gene Therapy Insights 2017; 3(7), 553-557.
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Advances and challenges in umbilical cord blood and tissue bioprocessing: procurement and storage

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Expert Insight

Lindsey Parker, Shaun Mansfield, Kate Sneddon, Ben Charles and Qasim A. Rafiq

Overcoming downstream bottlenecks in cell and gene therapy manufacturing

Umbilical cord tissue and blood is banked to complement the rapidly advancing field of tissue engineering and regenerative medicine for both autologous and allogeneic therapeutic applications. Whilst many problems concerning the use of the hematopoietic and multipotential mesenchymal stromal cells contained therein may be addressed through the future development of GMP-compliant manufacturing strategies, collection and bioprocessing of these tissues can be optimised in the present to maximise clinical outcomes. In this review, we describe current procurement, processing and storage approaches for umbilical cord blood and tissue; current challenges and how these may be met to augment translation and use of therapeutics harnessing their derivatives.

Submitted for review: May 18 2017 Published: Aug 4 2017
DOI: 10.18609/cgti.2017.042
Citation: Cell Gene Therapy Insights 2017;3(6), 489-508.
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Advances in the use of cord blood-derived iPSCs

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Interview

Mahendra Rao

Latest Advances in Cord Blood Applications & Commercialization

 

Mahendra Rao received his MD from Bombay University in India and his PhD in Developmental Neurobiology from the California Institute of Technology. He is widely known for his research involving hESCs, iPSCs, and other somatic stem cells, having worked in the stem cell field for more than 20 years with stints in academia, government, regulatory affairs and industry. Dr Rao has published more than 300 papers on stem cell research and is the co-founder of a neural stem cell company, Q therapeutics, based in Salt lake City (Utah). He continues to work with the NIH, FDA and other regulatory authorities on ESC related issues, most recently as the CIRM and ISSCR liaison to the ISCT. Dr Rao is currently the Vice president of Research in Regenerative Medicine at Q therapeutics and its subsidiary Neuro Q. He currently serves on the Board of CESCA, XCell and Stempeutics and on the SABs and as a consultant of various stem cell companies including the New York Stem Cell foundation. He continues to maintain an active research program in neural development and in evaluating cell-based screening and therapy to treat disorders of the nervous system.

DOI: 10.18609/cgti.2017.047
Citation: Cell Gene Therapy Insights 2017;3(7), 541-545.
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