Hope on nerve diseases

The research focused on one particular protein

Scientists have discovered a new approach for treating diseases caused by abnormal build up of misshapen proteins in the body.

The breakthrough could eventually lead to new ways to treat conditions such as Parkinson's and Alzheimer's disease which are thought to be caused in this way.

However, for the moment the finding only applies to similar "amyloid" diseases.

Discovering how to interfere with these protein folding events at a cellular level offers enormous potential

Dr Richard Harvey

These diseases result when proteins fold up in a way they are not supposed to and cluster together in sticky lumps.

These microscopic plaques build up in the body's organs, and disrupt their function, sometimes lethally.

Scientists from the Scripps Research Institute in California have found a way to stop one particular protein called transthyretin from folding up in an abnormal fashion.

Transthyretin is secreted by the liver into the bloodstream. It helps to circulate hormones and vitamins around the body.

Many diseases

Abnormal folding of the protein causes the build up of plaques around the nerves and muscle tissue.

This causes a collection of over 80 rare "amyloid" diseases known collectively as familial amyloid polyneuropathy.

Symptoms include numbness, muscle weakness, and - in advanced cases - failure of part of the nervous system.

Transthyretin is made up for four sub-units that bind to each other.

Sometimes mutant forms of the sub-units are produced which undermine the binding process, and cause the protein to fold up in an abnormal way.

The new approach counters this by exposing transthyretin to tiny designer molecules which bind to its sub-units, and make it less likely that they will not bind together.

Lead researcher Professor Jeffery Kelly said: "I'm very excited about pursuing these potential therapeutic opportunities.

"The same approach may also work with other amyloid diseases.

"Any protein that misfolds and causes pathology could, in principle, be targeted."

Significant finding

Dr Richard Harvey, director of research at the Alzheimer's Society, said it was becoming increasingly clear that a range of degenerative neurological diseases all have problems with the folding of proteins at the molecular level at their core.

"Even though these are all different diseases, and are caused by the accumulation of different neurotoxic proteins, it is the way these proteins fold up that determines whether or not they cause disease.

"Discovering how to interfere with these protein folding events at a cellular level offers enormous potential for preventing, halting or even reversing some of the most disabling degenerative neurological diseases that affect hundreds of thousands of people in the UK alone.

"Unfortunately, almost all of this research is still in the test tube. What is now needed is greatly increased funding from governments, industry and from the public through charities to translate these exciting findings into the treatments of tomorrow."

Harriet Millward, deputy chief executive, Alzheimer's Research Trust, described the research as "novel and interesting".

But she said: "It may be difficult to produce similar results for Alzheimer's due to the nature of the particular amyloid protein associated with the disease.

"There is a continued need to fund major research into the possible causes of Alzheimer's so we can find an answer to this terrible disease."

The research is published in the journal Science.