Today at World Stem Cells & Regenerative Medicine Congress 2012, Gil Van Bokkelen, CEO, at Athersys, delivered a presentation about clinical trials in stem cell.
Gil Van Bokkelen congratulated Osiris on approval of Prochymal for treating pediatric GvHD in Canada. This is an important event, because it demonstrates the growing regulatory acceptance of cell therapy and provides an example of how the field is progressing and maturing for the investors. He also highlighted that over 325, 000 patients have been treated with FDA approved cell therapy products.
Gil mentioned that not all of the programmes will be successful, so we need to be prepared that some clinical experiences will fail. However it is important to learn from the mistakes that have been made along the way.
Gil highlighted some major areas of medical need & opportunity:
- Cardiovascular disease
- Neurological injury & disease
- Inflammatory & immune disease
- Pulmonary disease
- Hematopoietic conditions
- Renal disease
- Orthopedic conditions
Some of the key clinical development goals that Gil identified are:
- What type of data do we need?
- What are the potential problems to overcome?
- What is in place to ensure failure at the late-stage clinical trial is avoided?
- How are the clinical end-points established to depict significant clinical added value?
- How to achieve a better regulatory transparency for pre-clinical development process?
- How to enhance the efficiency of clinical development?
Gil Van Bokkelen covered some of the ways to achieve these goals in his presentation. In conclusion Gil stressed the importance of establishing a clearly defined list of priorities: i.e. explicitly recognised unmet medical needs, recognised by appropriate bodies such as Health & Human Services and FDA, National Institute for Health & Clinical Excellence, European Medicines Agency. Therapies designed to specifically address these needs should be priority status & ideally list acceptable clinical end-points.
Gil enjoyed the Congress, he commented: "World Stem Cells & Regenerative Medicine Congress is Big enough – to represent all the leaders from the industry, but small enough to see and meet all the old friends."
Check back here in a couple of days for the presentation. Excellent presentation Gil Van!