You can be born with Parkinson’s

Stem cell study offers drug hope

PARKINSON’S disease may start in the womb, leading scientists suggested yesterday.

Their study found that abnormal brain cells may put people at risk right from the very start of their life.

The insight opens up the possibility of protecting those vulnerable to Parkinson’s before the age of 50 by giving them a drug that can prevent the cell damage.

‘Young- onset Parkinson’s is especially heartbreaking because it strikes people at the prime of life,’ said Dr Michele Tagliati, a co-author of the study from CedarsSinai Medical Centre in California.

‘This exciting new research provides hope that one day we may be able to detect and take early action to prevent this disease in at-risk individuals.’

Blood was taken from three patients in their thirties with early-onset Parkinson’s disease but no family history of the illness. In the laboratory the blood cells were first transformed into stem cells then brain cells, or neurons, to allow the researchers to see where the abnormalities had arisen.

They found that the cells built up a protein – alpha-synuclein – that is believed to play a part in causing the damage that leads to Parkinson’s.

The cells also had a malfunction in the cell structures that are supposed to dispose of alpha-synuclein.

The result is thought to be a reduction in levels of dopamine, a chemical which controls movement in the brain.

This would explain the tremors, slowness and loss of balance that Parkinson’s victims are forced to live with.

Clive Svendsen, a professor of biomedical sciences and medicine at Cedars-Sinai, said: ‘What we are seeing using this new model are the very first signs of young-onset Parkinson’s.

‘It appears that dopamine neurons in these individuals may continue to mishandle alpha-synuclein over a period of 20 or 30 years, causing Parkinson’s symptoms to emerge.’

Published in the journal Nature Medicine, the study tested a number of drugs to see whether they could reverse the abnormalities in the brain cells.

They found that one drug, PEP005, which is already used in the US to treat pre-cancers of the skin, reduced levels of alphasy nuclein in the cells.

It also reduced levels of the same protein in mice when they were injected with the drug.

Next the team plan to investigate how PEP005, which is available in gel form, might be delivered to the brain to potentially treat Parkinson’s.

The disease is the fastest growing neurological condition in the world and there is no cure.

The main symptoms are slow movement, stiff muscles which make victims struggle with walking or fall over, and involuntary tremors in parts of the body such as the hands.

There are more than 140,000 sufferers in the UK.