The Cure Parkinson’s Trust, by definition, focuses on cure. It is a bold aim, and made even bolder by the urgency and priority by which we seek it. Yet we are greatly encouraged by recent progress in several different scientific fields that relate directly to Parkinson’s Disease and our growing understanding of why it starts, and how to limit or even arrest its progression.
Whilst the cause of Parkinson’s Disease in individual patients may be genetic, environmental or even trauma, the precise biochemical or cellular mechanism by which the disease is triggered is not known. Parkinson’s Disease has long been known to relate to a loss of specific brain cells responsible for helping to control movement. For the past 30-40 years treatment has centred on limiting this compromise so as to improve symptoms. In the first few years of treatment this often works fairly well but thereafter control of symptoms can become progressively more difficult. For many years no definitive treatment emerged that might slow, stop or reverse disease progression.
Two very important aspects have provided a vital backdrop towards current and imminent progress. These are the sequencing of the human genome, and some startling achievements in cancer research that have a direct bearing on Parkinson’s Disease (including a fundamental understanding of cell signalling and communication, what triggers cell death, what makes immature cells grow into working maturity, as well as an understanding of a large number of relevant intracellular biochemical processes).
In the past 2-3 years, scientific progress in Parkinson’s Disease has been astonishing:-
We have seen the first clinical trial using gene therapy, and are working with the group that did this groundbreaking work on developing further follow-up studies.
The genetic basic for 5%-10% of all Parkinson’s cases has been clarified, leading to recognition of what are some of the important biochemical processes involved with Parkinson’s Disease. What is more, just about to start is a definitive ‘genome association’ study on 3000 patients, already funded by the Wellcome Trust, which promises to identify ALL genes significantly involved in Parkinson’s Disease.
The Cure Parkinson’s Trust will be assisting with the complex analysis of these genes. The aim is to work out which may be responsible for the disease, by what biochemical process they act, and how to modify these processes towards therapy or prevention. There will also be a massive amount of information to sort out on genes that affect drug treatment. This is a one-time definitive study; it will form the ‘central knowledge base’ that all future workers on Parkinson’s will interrogate for answers.
It has been realised that Parkinson’s Disease is, biochemically, a far more diverse disease than previously thought. For example, we now know the adrenaline system plays an important part where, for many years, it was disregarded.
In another example, many suspect that inflammation may also be part of the disease process – much as it would be in many injuries in other organs, some scientists even think it is central to disease progression in Parkinsons, rather than just a response to cellular damage. Scientists have consequently been trying standard anti-inflammatory drugs to see if this slows disease progression.
Whilst ‘neuro-protection’ is a powerful goal, ‘neuro-regeneration’ represents a more fundamental approach. Neuro-regeneration aims to grow new functioning brain cells to replace those that have been damaged. This is an exciting time for Parkinson’s. We are determined, focused and, we believe, close to our goal.
Dr. Richard Wyse, Head of Research & Development, The Cure Parkinson's Trust, March 2008