stem cells in their environment
stem cell research on the road to clinical therapies
29 May 2014
By Andreas Reimer
Recently, I went to the 2nd BIRAX Regenerative Medicine Conference in Haifa, Israel. Scientists from all over the UK joined more than 200 Israeli scientists and assembled at the Technion – Israel Institute of Technology. Situated near the historic harbour, the Ruth and Bruce Rappaport Faculty of Medicine provided a suitable environment for a conference on regenerative medicine. The faculty is renowned for its research on high-tech medical products and lies opposite a large hospital, pointing the focus of its translational research in the right direction: patients.
One thing that sprang to mind during the opening conference dinner was the frequent use of the word "clinical". It's common jargon for medics but not usually heard of in a basic research environment – but maybe it should be? All key scientific articles conclude with a remark on how important findings show relevance to disease or therapeutic applications. It is right to start a project which may, one day, benefit people. So to finish the publication with a translational statement means to round off the idea by giving it a meaning. But should the idea be forgotten on the way? Clearly, there are other topics on the agenda of a regular research day but it helps to be reminded of the target you are working towards. And walking past a hospital on the way to the meeting did provide that extra reminder.
Amongst the opening speakers of the conference was David Willets, the UK Minister for Universities and Science, who gave a talk about the importance of regenerative medicine for the UK. Embryonic-like stem cells, which can be generated from adult tissue, possess great potential to provide a source for tissue replacement after injury or disease. After the opening remarks, the speakers in the first session highlighted novel manufacturing concepts that will facilitate the generation of medicinal products for stem cell-based therapies. Stephen Ward from the Cell Therapy Catapult, a partially government-funded organisation to advance cell therapies, gave an informative insight into what is required to bring cell therapies from the laboratory into the clinic. Based on his presentation, the large scale production of cells to make cell therapies commercially viable is still in its infancy but will be addressed in future projects. Sarah Moyle from the Jenner Institute in Oxford held a talk about the fundamentals of translational stem cell research including points which are not normally considered. For example, the transport of cells from the lab until they reach patients will need to be considered and standardised to ensure the safe delivery of cells.
As I've outlined above, the morning talks were not based around basic research problems. Instead, manufacturing and logistics - common topics in commercial industries - were addressed to highlight their importance. The many factors above and beyond basic research required to make translational research successful became apparent.
In the subsequent session, Sian Harding from Imperial College London held an interesting talk covering cardiovascular cell and gene therapy. She outlined a recently launched clinical trial which involves targeted gene correction to repair heart tissue after injury such as heart attack. Two decades of research, including stem cell research, has led to the identification of the SERCA2a gene as the ideal target for gene therapy against heart disease. The strategy involves the delivery of a specifically bioengineered gene snippet via a vector that mimics the infection machinery of a virus and only targets the affected tissue without damaging the host genome. It will be interesting to see the results of this trial in the following years.
The next sessions covered major aspects of basic research in the regenerative medicine field including the widespread usage of pluripotent stem cells to develop disease models in a dish. Jacob Hanna presented his views on the ground state of pluripotency and Ludovic Vallier showed how regulating the cell cycle can affect the fate of stem cells. Further talks included the epigenetic regulation of cell fate decisions by Yehudit Bergman and Shukry Habib's work on asymmetrical cell division using an innovative bioengineering approach. The challenges of immunogenicity, meaning the risk of rejecting the received cells by the body's own immune system, were also addressed. The most promising solutions include the targeted suppression of the host's immune system or generating a centralised cell bank to cover the majority of the population, in a similar way to blood banks but using small frozen plastic tubes instead of red bags.
Before the conference came to an end, a panel of speakers from small to medium-sized biotech companies gave an insight into their view on regenerative medicine. To me, these talks seemed a little lengthy, but this may have been caused by the thought of the delicious Israeli cuisine which was the next stop on the agenda. Nevertheless this final session provided an important message to the audience. Without company efforts patients might never see the results of our work. This is a different mindset but one that free-thinking basic scientists need to adapt in order to propel cell therapies into the clinic.