Remaking life to conquer degenerative brain diseases
How is it possible to rewind life and take cells back to their embryonic state? Enter the world of stem cell generation and regenerative medicine! This field of science for medical treatment is at the forefront of technology and therapeutics, and is controversial. The potential to replace damaged cells in the brain, and even to enable recovery of lost functions, in devastating conditions such as Parkinson’s disease and Huntington's disease, holds amazing hope for sufferers. The scientists involved in the NeuroStemCell project are faced with the daily challenge of big unanswered questions, together with pressing ethical issues.
Parkinson’s and Huntington’s diseases are both responsible for the death of specific neurons in the brain and for a progressive loss of cognitive and motor functions. A tantalising new future can, however, be opened up if brand new cells can be grown with the potential to become the type of cell that is needed to repair damaged organs. These are so-called ‘pluripotent cells’ and they lie at the heart of stem cell research. Embryonic stem cells have so far proved to be the purest form of natural stem cell material.
The NeuroStemCell project is dealing with some of the biggest and most fascinating issues and questions. First and foremost, what is the best possible way to produce pure stem cells? Is it even possible to take adult cells back in time, as it were, to an embryonic state and start development afresh? Could treatment make use of a ‘cell bank’ at an intermediate stage of development? How can we generate a sufficient quantity of high quality material that remains stable over time and does not lose any of its purity?
Recently, the Nobel Prize in Physiology or Medicine 2012 was awarded jointly to Sir John B. Gurdon and Shinya Yamanaka “for the discovery that mature cells can be reprogrammed to become pluripotent”. These are Induced Pluripotent Cells (iPS), with the potential to regenerate diseased or damaged tissues for an individual patient, using their own adult cells as the source material. It’s all mind-boggling stuff; even though, at this stage many vital pieces of information are still unknown and the investigation of the therapeutic potential of iPS in animals is still at an early stage.
As NeuroStemCell faces these fascinating issues, the project is rooted in sound, thorough procedures aimed at taking a completely fresh look at the best possible source of stem cell material for the future. There are numerous steps involved in creating neurons that can reliably replace those damaged by Parkinson’s or Huntington’s. Professor Cattaneo is bringing together a whole range of studies to achieve this massive goal, including work to mimic the development of the nervous system and ways of instructing stem cells to become neurons, which in turn requires new expertise in transplanting cells. Here are just some of the programmes within the project.
In Lund, Sweden, a team led by Professor Anders Bjorklund, deputy coordinator of NeuroStemCell, is specialising in generating pure populations of cells to be used in cell replacement therapy for Parkinson’s and establishing safe methods of grafting cells that survive and function in the long term.
In Cambridge, UK, Professor Austin Smith is identifying what determines pluripotency, while Professor Roger Barker and his Transneuro Consortium are focusing on taking transplant procedures for injecting cells into the brains of Parkinson’s patients to a new level of reliability.
In New York, USA, Dr Lorenz Studer is investing years of scientific expertise and financial resources into producing dopamine cells required to treat Parkinson’s that stay stable after transplantation and therefore enable sustained recovery of lost functions. In Bonn, Germany, Professor Oliver Brüstle is researching embryonic and iPS cells to produce stem cell material from an intermediate stage of cell development or ‘cell bank’. On yet another front, Professor Elena Cattaneo, the NeuroStemCell project coordinator, and her team in Milan, Italy, are using the same techniques to develop sources of striatal neurons, specific cells required to treat Huntington’s disease.
Given the broad range of cutting edge science that is involved in the project, it is easy to see why the partners are so motivated by the NeuroStemCell project. There is no room here for those who prefer to go it alone; the partners know they need to build on each element of the project as they create safe and transplantable cells that are increasingly authentic and offer stable, long-term qualities beyond transplantation.
The project has recently applied for a 6 month extension because there are so many exciting strands to this research that can yield more results with just some extra time. Work towards generating pure populations of cells and advancing transplant techniques, for example, are now set to progress towards clinical trials.