Highlight:
- MEDIPOST based in Seoul, Korea: CARTISTEM®: successful commercialization: is a novel stem cell drug for the treatment of cartilage defects, such as in osteoarthritis, caused by degeneration or repeated traumatic injuries, leading to regeneration of cartilage. Through a single surgical procedure, human Umbilical Cord Blood-derived Mesenchymal Stem Cells (hUCB-MSCs) mixed with viscous biopolymer are transplanted.
- Gamida Cell: based in Jerusalem, Israel: StemEx® technology has been developed to expand the stem/progenitor cells present in UCB, in an ex-vivo environment using proprietary copper chelator technology, to improve clinical outcomes. A Phase III global study has recently been completed which encompassed 25 hospitals, 101 patients, a 180 day follow-up, and a goal of showing overall survival at 100 days. Dr. Margolin was pleased to announce that StemEx® improves survival at 100 days post transplantation and also decreases engraftment time, suggesting that StemEx® can serve as an alternative transplant treatment for patients who cannot find a matched bone marrow donor.
- Proteus Venture Partners, California, USA: Review: Future of the cord blood banking industry:
- 2010 the market for cord blood was valued at $3.4 billion, but is expected to rise to $14.9 billion by 2015.
- Currently there are over 150 cord blood banks that contain 575,000 public units and over 1 million private units, however only 1-3% of units are used each year.
- Nearly 50% of patients cannot find a match for a bone marrow transplant, making the cord blood industry the fastest growing segment of the cell transplant market.
- In 2010 cord blood transplants accounted for 22% of all transplants, and that number is expected to double by 2015.
- Over 60 therapeutic applications have been identified for cord blood, and with over 430 FDA clinical trials, 245 transplantation studies and 24 pivotal/Phase III trials – an exciting future awaits.
- Cord blood is the preferred cell source for pediatric use, due to the small unit size, however it is the over 50 age group that are the largest users, accounting for 41% of 2010 UCB transplants.
- Transplant outcomes have improved dramatically due to progress in HLA matching, advances in conditioning regimes, and post-transplant care.
- In the future, Mr. Bonfiglio sees consolidation of blood banks and eventually a vertically integrated business model. The bank will cater to both private and public clients; however the public side will invest in clinical research that will eventually emerge as a cell therapy treatment. This will then be available at a surgi-center allowing for treatment of private clients (??)
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original source:
http://www.oxbridgebiotech.com/review/research-and-policy/cord-blood-banking/
On May 20th 2013, the
LRMN held their third meeting focusing on cord blood banking, with three experts sharing their experiences in working with cell therapies derived from cord blood and their perspectives on the state of the industry. The event, held at University College London, was free to attend due to generous support from
Macopharma.
Dr. Emily Culme-Seymour, Director of LRMN, kicked off the evening by welcoming the attendees and speakers. She promptly introduced
Dr. Antonio Lee who is the Associate Director of Business Development for
MEDIPOST based in Seoul, Korea. MEDIPOST is a stem cell biotechnology company that provides cord blood banking services and innovative adult stem cell drugs. Currently they have over 170,000 units of cord blood stored privately and four adult stem cell therapeutics. In his talk, Dr. Lee described the World’s first allogeneic stem cell drug, CARTISTEM®, and the path to its successful commercialisation. CARTISTEM® is a
KFDA (Korean Food and Drug Administration) approved drug for the treatment of knee cartilage defects, which arise due to degenerative osteoarthritis (OA). A lyophilized biopolymer is combined with human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs) to form a viscous gel which is applied directly to the fracture. It has been successful in both open and arthroscopic surgery and thus far 432 patients have been treated with the compound. Clinical trials show a 97.67% success rate, compared to treatment with a competing therapy that was 71.74% successful. Dr. Lee also discussed the other adult stem cell drugs currently in the portfolio and mentioned that MEDIPOST is working on novel applications for diseases such as Cerebral Palsy, brain injury and Type I diabetes.
The second speaker,
Dr. Yael Margolin, President & CEO of
Gamida Cell, which is based in Jerusalem, Israel, described the first crude bone marrow transplantations over 40 years ago and highlighted how much progress has been made to date. Despite the advances, there are still approximately 35,000 patients per year who do not receive bone marrow transplantations due to donor incompatibility. Gamida Cell has been making progress on an alternative source for hematopoietic stem cell transplantation – the use of UCB for cord blood transplantation (CBT). UCB is easily collected and less donor matching is required, but cord blood provides a smaller quantity of viable cells, which results in delayed engraftment and can increase transplant related morbidity and mortality. StemEx® technology has been developed to expand the stem/progenitor cells present in UCB, in an ex-vivo environment using proprietary copper chelator technology, to improve clinical outcomes. A Phase III global study has recently been completed which encompassed 25 hospitals, 101 patients, a 180 day follow-up, and a goal of showing overall survival at 100 days. Dr. Margolin was pleased to announce that StemEx® improves survival at 100 days post transplantation and also decreases engraftment time, suggesting that StemEx® can serve as an alternative transplant treatment for patients who cannot find a matched bone marrow donor. As such, Gamida Cell is seeking a strategic partner for the global commercialization of StemEx®. Dr. Margolin also mentioned that Gamida Cell is pursuing several expanded cell graft products that have been developed using nicotinamide technology as they displayed improved functionality following in vivo infusion. These products could potentially be used to treat cancer, hematological diseases, autoimmune diseases, inflammatory conditions and regenerative medicine.
A discussion on the past, present and future of the cord blood banking industry and was then delivered by Mr.
Gregory A. Bonfiglio, who is the Founder & Managing Partner of
Proteus Venture Partners in California, USA. Mr. Bonfiglio explained that in 2010 the market for cord blood was valued at $3.4 billion, but is expected to rise to $14.9 billion by 2015. Currently there are over 150 cord blood banks that contain 575,000 public units and over 1 million private units, however only 1-3% of units are used each year. Nearly 50% of patients cannot find a match for a bone marrow transplant, making the cord blood industry the fastest growing segment of the cell transplant market. In 2010 cord blood transplants accounted for 22% of all transplants, and that number is expected to double by 2015. Over 60 therapeutic applications have been identified for cord blood, and with over 430 FDA clinical trials, 245 transplantation studies and 24 pivotal/Phase III trials – an exciting future awaits. Cord blood is the preferred cell source for pediatric use, due to the small unit size, however it is the over 50 age group that are the largest users, accounting for41% of 2010 UCB transplants. Transplant outcomes have improved dramatically due to progress in HLA matching, advances in conditioning regimes, and post-transplant care. In the future, Mr. Bonfiglio sees consolidation of blood banks and eventually a vertically integrated business model. The bank will cater to both private and public clients; however the public side will invest in clinical research that will eventually emerge as a cell therapy treatment. This will then be available at a surgi-center allowing for treatment of private clients
The evening concluded with closing remarks by
Professor Chris Mason from the Advanced Centre for Biochemical Engineering, UCL, and was followed by a reception.
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