Fanconi Hope is pleased to announce that in collaboration with the US Fanconi Anemia Research Fund we have provided a grant of £30,000 for the first UK research programme relating to the use of stem cell technology in conjunction with gene therapy which may in future provide a cure for Fanconi Anaemia; ‘Using iPSC technology to understand early haematopoietic development in Fanconi Anaemia patients’. Fanconi Hope’s research grant of £30,000 is now funding the first phase of the work, and we have set ourselves a target of raising a further £100,000 for the remaining phases.
Background:
Recent joint research by the Salk Institute for Biological Studies in Calfornia, the Center of Regenerative Medicine in Barcelona (CMRB) and the CIEMAT Centre in Barcelona have shown for the first time that in principle human genetic diseases such as Fanconi Anaemia can be cured using a combination of gene therapy and induced pluripotent stem (iPS) cell technology.
The potential significance of this is that corrected cells from the patient’s own tissue would be used in a bone marrow transplant thereby avoiding the issue of tissue rejection which often causes cancers subsequently in transplanted patients.
This groundbreaking research has been shown to cure an FA-affected cell and in theory this could then be transplanted into a FA-affected patient to cure the blood-related element of the disease. However many hurdles remain before the theory can become practice not least in preventing the reprogrammed cells from inducing tumours, and the international team are now funded to pursue research aimed at translating basic science into clinical cures.
Prof Majlinda Lako
Prof Chris Mathew
Now for the first time in the UK a collaborative research programme into this exciting new technology has been initiated between a group in Newcastle led by Prof Majlinda Lako working with induced pluripotent stem (iPS) cell technology and a group in London led by Prof Chris Mathew researching in Fanconi Anaemia.
In brief, the programme involves generating and characterising iPS cell lines (the first phase of the programme). Next, the team will study whether these iPS cell lines behave the same way as normal FA cells with respect to DNA repair, genomic instability and the ability to make blood cells. If they do, they will then have a good FA model (which can be used in a laboratory environment rather than testing on humans) to test different corrective measures/drugs for the faulty DNA repair mechanism. The team will then investigate to see whether the process of creation of blood producing cells is different for FA and control patients. This should give a better understanding of what goes on so that hopefully they can help improve the process of curing patients through new therapeutic regimes using gene therapy and iPS cell technology.
This research project is being funded by Fanconi Hope in collaboration with the Fanconi Anemia Research Fund (FARF) in the US. Fanconi Hope has provided a grant of £30,000 for the first phase of the work and has set a target of raising a further £100,000 for the remaining phases. This research topic was selected by the Trustees of Fanconi Hope from a shortlist of candidate UK research programmes which have been approved for funding by the FARF Scientific Advisory Board, whose 14 members comprise FA specialists from the US, Canada, Holland and the UK.
Further details of the research can be found on the Fanconi Hope Funded Research page.
