Stem cells offers hope in battle against motor neurone disease

Published on Monday 24 August 2009 20:03

NEURODEGENERATIVE diseases represent one of the major public health burdens of our time.

These untreatable and progressive disorders that include Alzheimer's disease, Parkinson's disease, multiple sclerosis and motor neurone disease (MND) have a devastating impact on the lives of patients, and families as well as enormous costs to society.

Although there have been advances in our understanding of these diseases, there remains a great and unmet need for more rapid diagnosis and, crucially, treatments that will slow and ultimately stop them.

Improving treatments for neurodegenerative diseases is an area where stem cell research holds enormous potential. In the past decade, not only have we been able to grow human embryonic stem cells in the lab, but we can now generate stem cells unique to the patient from a skin biopsy.

In effect, we can create bespoke cells that allow us to both model the disease in a dish and look to generate replacement cells, for some conditions, that will not be rejected after transplantation.

Perhaps the major immediate dividend from the stem cell revolution is that this technology provides new ways to understand more about the underlying causes of these diseases as well as to test and discover new drugs.

It is paramount that clinicians and researchers are cautious and realistic in managing the rising patient expectation of stem cell medicine. This is a long journey, and progress will be inevitably slower that one would wish.

Nonetheless, and accepting that cures are not imminent, there are grounds for optimism that insights from work on stem cells will begin to make a significant impact in improving quality of life. If we can, for instance, put off the day when a patient with MND is put on a ventilator by even a few months, this apparently modest gain will make a huge difference.

The Euan MacDonald Centre at Edinburgh University builds on the world-leading stem cell expertise in Edinburgh at the MRC Centre for Regenerative Medicine with a mission to improve the quality of MND patients lives through research.

Its research will be interdisciplinary and collaborative. Collaborating with NHS colleagues and MND Scotland, it will make possible the establishment of a national register of MND patients. Because Scotland has a relatively small and stable population, this will help us share expertise in diagnosing and managing patients and in translating developments made in the lab to the clinic.

The national register will also help us understand the differences between various types of MND. For instance, we know that some cases of MND are inherited, while some forms target different muscle groups over others.

Repair of the damaged brain remains arguably the greatest challenge of medicine. Rapid advances in human stem cell biology allied to the genetic revolution promise, through improved knowledge of these disorders, new treatments.

Time to clinic remains difficult to predict, and will vary among diseases, with a balance to be struck between justifiable risk of innovative experimental therapies and the potential benefit. MND highlights both the challenges of neurodegenerative disease and the opportunities of experimental medicine.

&#149 Siddharthan Chandran is professor of neurology at Edinburgh University.