A team of UCL and MRC researchers has developed a rating scale for prion diseases that has major implications for future treatment trials.
Prion diseases are rare neurodegenerative diseases and the team has developed a precise way to track how patients are progressing.
According to results published in the journal Brain, use of the rating scale (called the MRC Scale) in the BRC-funded National Monitoring Prion Cohort showed three distinct patterns of change:
- slow progress associated with genetic forms of disease
- rapid associated with all forms, which quickly leads to death
- an important subset of patients who stay in a comatose state, but do not die for some time.
Furthermore, there is substantial variation in the fast progressors, for which a genetic subtype has a strong determining effect.
The paper is based on work following on from an MRC funded trial PRION-1 (2004-2007) of the anti-malarial drug quinacrine as a treatment option which revealed the need for a definitive rating scale.
Commenting on the importance of the BRC funded Prion Cohort, Dr Simon Mead, senior clinician and National Prion Clinic lead, said “The PRION-1 study raised lots of questions, in particular about choice of outcome measures. Some scales were poor at measuring patient progression, though the functionally orientated measures seemed to match clinical reality. It is the BRC’s support that has allowed us to develop the Cohort to refine the MRC Scale and isolate distinct patterns of disease progression”.
Dr Mead added “The new rating scale is based on interviews with 71 families. We’ve taken into account the experience of the disease from the perspective of patients, their relatives and carers to find out what are the important symptoms of the disease.”
The UCL research group is now poised to do more experimental medicine studies with patients that aim to develop new therapies after finding a way to manufacture a human form of a monoclonal antibody which effectively treats prion infection in animals.
