Genomics
References 1 Brown et al. Blood 2012:120 (18):3729-3740. 2 Strauss et al. PLoS Biology 2012:10(9):e10011389.
become a reality. Collaborative efforts are under- way to develop, optimise and validate these sys- tems and methods and to transition them from the laboratory to the bedside. Karolinska University Hospital (Stockholm, Sweden) – a pioneer in the advancement of cell therapies, with several clinical trials under way, including for the treatment of cancer and neurological and metabolic disorders – has partnered with GE Healthcare to research and identify solutions that will advance and accelerate the implementation of cell therapies as standard treatment options.
Another facet of cell therapy with tremendous potential for saving lives in the short term is cord blood transplantation to treat hematopoietic can- cers and diseases such as sickle cell anaemia. More than 14,000 patients each year in the United States alone are diagnosed with a life-threatening blood cancer such as leukaemia, lymphoma or myeloma. These cancers can often be successfully treated with long-term remissions and cures by replacing the patients’ blood-forming stem cells with healthy haematopoietic stem cells from the blood of a matching donor. Unfortunately, finding a donor that is a good match is often difficult. An alterna- tive solution is to transplant haematopoietic stem cells isolated from umbilical cord blood – an abun- dant source of stem cells that is typically discarded after a baby is born, but can be collected and banked. Cells from umbilical cord blood do not have to match the recipient as closely as would stem cells from a regular donor.
Cord blood banks and available stores of donor cord blood for transplantation are increasing in number worldwide. This approach to cell therapy would benefit from greater standardisation in cell processing and from advances in techniques to enable robust and reproducible expansion of stem cell populations from the banked cord blood sam- ples. The number of stem cells in any given sample of cord blood may be sufficient to treat a child in need of a transplant, but is typically not enough to treat an adult. A joint project undertaken by the University of California, San Francisco and GE Healthcare is applying HTS and automated high content imaging technology to screen 120,000 compounds to identify those best able to stimulate expansion of stem cells isolated from cord blood. The most promising compounds will then be eval- uated in studies designed to foster large-scale haematopoietic stem cell production in amounts needed for subsequent clinical trials. The future presents many challenges from a healthcare perspective, including an ageing popula- tion and economic pressures that will demand pre-
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ventive and regenerative strategies and less costly, safer therapeutic options that offer greater efficacy. Genomics is enabling stratification of patient popu- lations for drug testing and for treatment. Advances in cell imaging and stem cell technology is making it possible to develop more targeted, safer treat- ments tailored to specific patient populations. And progress in translational research is driving those treatments through clinical development and to the patient’s bedside. For cell therapies, continued improvements in technology and in workflows and automation to standardise and industrialise the supply chain for cell collection, processing, and delivery will make these new treatments more broadly accessible. By embracing the complexity of the cell and the ever-expanding database of ‘omic’ information, safer, more effective, more person- alised medicines are on the horizon.
DDW
Dr Amr Abid is Head of Global Strategic Growth at GE Healthcare Life Sciences. Before joining GE Healthcare in 2011, Abid held a number of posi- tions over 10 years at Invitrogen (now Life Technologies), finally as EMEA Leader, Cell Systems. Prior to entering the industry, he spent five years in pharmacology research at the University of Nancy, France.
Drug Discovery World Spring 2013
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