The HemAcure Consortium, consisting of three European academic institutions, an enterprise for quality management and Ontario-based clinical stage regenerative medicine Company, Sernova Corp, reported significant scientific progress achieved in the development of a ‘first in world’ personalized regenerative medicine therapy for the treatment of Hemophilia A patients.
The therapy being developed by international scientific Consortium members is to treat severe Hemophilia A, a serious genetic bleeding disorder caused by missing or defective clotting factor VIII in the blood stream. This therapy consists of Sernova’s implanted Cell Pouch(TM) device transplanted with therapeutic cells, corrected to produce Factor VIII at a level sufficient to significantly reduce the side effects of the disease and improve patient quality of life. Sernova is developing medical technologies for the treatment of chronic debilitating metabolic diseases such as diabetes, blood disorders including hemophilia and other diseases treated through replacement of proteins or hormones missing or in short supply within the body. The company’s Cell Pouch, is an implantable medical device and therapeutic cells (donor, xenogeneic or stem cell derived therapeutic cells) which then release proteins and/or hormones as required.
The main objective of the HemAcure project is to develop and refine the tools and technologies for a novel ex vivo prepared cell based therapy within Sernova’s prevascularized Cell Pouch to treat this bleeding disorder that should ultimately lead to improved quality of life of the patients. “We are thrilled with the approval by the European Union of the next stage of funding for the HemAcure program based on our quality interim report. This is a strong validation of the Consortium’s dedication and teamwork and the importance of this regenerative medicine approach,” said Dr. Joris Braspenning, HemAcure Program Coordinator.
“The international HemAcure Consortium team members are pleased with the ground breaking scientific advances achieved at this point and are on track for this regenerative medicine solution to advance into human clinical evaluation,” remarked Dr. Philip Toleikis, Sernova President and CEO. Toleikis added, “Sernova’s Cell Pouch platform technologies are achieving important world first milestones in both diabetes and now hemophilia, two significant clinical indications which are being disrupted by its regenerative medicine approach aimed at significantly improving patient quality of life.”
In summary, the following ground-breaking developments have been achieved by the Consortium:
- A reliable procedure has been implemented to isolate and maintain required endothelial cells from a sample of the patient’s blood.
- Using a novel gene correction process, the cells have been corrected and tuned to reliably produce the required Factor VIII to treat Hemophilia A.
- The cells have been successfully scaled up to achieve the required therapeutic number, and cryopreserved for shipping and future transplant into the implanted Cell Pouch.
- A preliminary study confirmed survival of the Factor VIII corrected human cells injected into the hemophilia model, achieving sustained therapeutic Factor VIII levels. This preliminary work is being used to aid in dosing of these cells in the Cell Pouch.
- Safe Cell Pouch surgical implant and cell transplant procedures have been developed in the hemophilia A model in preparation for use in hemophilia patients.
- Development of Cell Pouch vascularized tissue chambers suitable for Factor VIII producing cell transplant has been demonstrated in the hemophilia A model, expected to mimic the predicted findings in human patients.
- In combination, this work is in preparation for safety and efficacy studies of the human hemophilia corrected Factor VIII producing cells in the Cell Pouch in a preclinical model of hemophilia.
This combination of advances by the HemAcure team represents a ‘first in world’ achievement towards developing a regenerative medicine therapy for the treatment of severe hemophilia A patients. “In this regard, these fundamental advancements have set the stage for further optimization and implementation of cell production processes under controlled GMP conditions,” stated Martin Zierau, IMS member consortium team leader responsible for coordination of GMP processes.
With Factor VIII corrected cells, studies are ongoing to optimize cell dosing within the Cell Pouch and for study of safety and efficacy of hemophilia corrected Factor VIII cells in the hemophilia model. These studies are in support of the current extensive regulatory package already assembled for the Cell Pouch in anticipation of human clinical evaluation of the Cell Pouch with hemophilia corrected Factor VIII producing cells.
The project has received funding from the European Union’s Horizon 2020 research and innovation program under grant agreement No 667421. The consortium members include the University Hospital Wuerzburg (Coordinating Institute), Germany, IMS – Integrierte Management, Heppenheim, Germany, Università del Piemonte Orientale “Amedeo Avogadro,” Novara, Italy, Loughborough University, Loughborough, United Kingdom, ARTTIC International Management Services, Munich, Germany and Sernova Corp., London, Ontario, Canada.
Sernova has developed its proprietary highly innovative Cell Pouch technologies for the placement and long-term survival and function of immune protected therapeutic cells. It has proven to be safe and efficacious in multiple small and large animal preclinical models and has demonstrated safety alone and with therapeutic cells in a clinical trial in humans for another therapeutic indication. The company believes the Cell Pouch platform is the first such patented technology proven to become incorporated with blood vessel enriched tissue-forming tissue chambers without fibrosis for the placement and long-term survival and function of immune protected therapeutic cells.
About Hemophilia A
People with Hemophilia have prolonged abnormal bleeding as a result of trauma. Hemophilia A, also called factor VIII (FVIII) deficiency is the most common form of Hemophilia and is a genetic disorder caused by missing or defective FVIII, a blood clotting protein. Severe hemophilia occurs in about 60% of cases where the deficiency of FVIII is less than 1% of normal blood concentration. While it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous change in the gene. According to the US Centers for Disease Control and Prevention hemophilia occurs in about 1 in 5,000 births. If the prolonged bleeding occurs in the brain of a person with hemophilia, it can be fatal. Prolonged bleeding in joints can cause inflammatory responses and permanent joint damage. Approximately 20,000 people in the United States and 10,000 in Europe have the moderate or severe form of hemophilia A, as well as approximately 2,500 in Canada. All races and ethnic groups are equally affected by hemophilia A. Though there is no cure for the disease, it can be controlled with regular infusions of recombinant clotting FVIII. Annual costs for the treatment of the disease for each patient may range from $60,000 to $260,000 US for a total cost of between $2-5B per year in North America and Europe.
Horizon 2020 is the biggest EU Research and Innovation program ever with nearly €80 billion of funding available over seven years (2014 to 2020). It promises more breakthroughs, discoveries and world-firsts by taking great ideas from the lab to the market. The project is funded as part of societal challenges “personalizing health and care” in a specific call about innovative treatments and technologies. New therapies, such as gene or cell therapies, often require technological innovation in the form of development of specific component tools and techniques such as isolation and multiplication of a cell or development of a scaffold, delivery of the therapy to the patient and for following-up the effect of the therapy in the patient. In particular, achieving therapeutic scale production and GMP standards at reasonable cost is often underestimated. The European Union aims to improve the development of advanced methods and devices for targeted and controlled delivery, and to bring these innovative treatments to the patient.