Why are studies on Haemophilia A so important?
Haemophilia patients can experience secondary complications such as joint disease or muscle dystrophy. Haemophilia A patients, especially the severe patients, experience spontaneous bleedings without any (clear) cause. Recurrent bleeding in the joints, in particular elbow (25%) and knees (44% of all cases) can lead to irreversible joint damages and severely reduce mobility. Studies on haemophilia A are important to better understand the disease and this research helps to develop new treatment procedures to improve quality of life of haemophilia A patients.
What will be the contribution of HemAcure to haemophilia A research in Europe?
The main objective of the HemAcure project is to develop a novel cell based therapy to treat severe haemophilia A. The innovative idea behind HemAcure: The isolation of cells from blood of the haemophilia A patient and performing a genetic (FVIII) correction of those cells. The corrected cells will be expanded, meaning grown in a laboratory and if enough cells are available to produce sufficient FVIII, corrected cells will be transplanted back into the patient in a medical device designed for therapeutic cells (Cell Pouch™). The cells in this kind of cell bag or artificial organ are connected with the bloodstream of the patient and that allows a continuous release of FVIII into the patient’s blood. The overall aim of this new approach is to significantly improve the quality of life of haemophilia A patients and most importantly to reduce the side effects of the disease.
What will be the influence of the HemAcure project on the treatment of Haemophilia A?
Patients can finally hope for a curative treatment of their disease, haemophilia A. They will have constant systemic levels of the clotting factor with strong reduced or even without the need for infusions of FVIII anymore resulting in an improved safety and quality of life relative to the current standard of care.
How can patients benefit from HemAcure?
Currently, all process steps are brought to a high-quality level, called GMP. GMP stand for Good Manufacturing Practice this means we are standardizing the process to make it robust, efficient and as safe as possible for the patient. Good manufacturing practice guidelines provide guidance for manufacturing, testing, and quality assurance in order to ensure that a drug product is safe for human use.
Thus, next steps will be thorough preclinical investigations to demonstrate the safety of this new therapeutic cell product. Hereafter, first in man studies with severe haemophilia A patients are planned and we hope that many people are willing to participate in this study to make this new therapeutic approach available to all severe haemophilia A patients as soon as possible.
How can I contribute/ participate in HemAcure as a Haemophilia A patient?
Once the first in man studies or clinical trials start, haemophilia A patients are asked to participate to receive this new therapeutic approach. All patients are invited to check our HemAcure website regularly to get informed as soon as these studies will start.
Which patient groups will benefit from this therapy?
Initially the new cell based treatment will be offered to severe Haemophilia A patients but should be made available to other patients in the future as well.
When will the therapy be available for patients?
The new cell based therapy will be available for patients either during the clinical studies if patients are willing to participate, or after the clinical studies and approval by the regulatory bodies.
What is Gene Therapy?
Gene therapy is an approach to replace a mutated or non-functional gene that causes disease with a healthy copy of the gene. The genetic correction of the mutated gene, in case of Haemophilia A the gene for factor VIII, enables the corrected cells to produce functional factor VIII again.
What is a Cell Pouch?
Cell Pouch System™ is a novel implantable and scalable medical device which forms a natural environment in the body for the housing and long-term survival and function of therapeutic cells.
Who is funding this project?
This project received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 667421.
Horizon 2020 is the biggest EU Research and Innovation programme ever with nearly €80 billion of funding available over 7 years (2014 to 2020). It promises more breakthroughs, discoveries and world-firsts by taking great ideas from the lab to the market.