Is a cure for Parkinson's in sight? First ever human stem cell trials to replace those damaged by the disease

By KATE PICKLES FOR MAILONLINE

PUBLISHED: 12:05 EST, 30 March 2016 

The proposed trials have led to new hope for a cure for Parkinson's disease, which affects millions of people including boxer Mohammad Ali

A cure for Parkinson's disease could be on the horizon after a research team was given permission to start the first ever human stem-cell trials.

Parkinson’s is a progressive neurological condition that destroys the brain cells producing the chemical messenger dopamine, in the part of the brain that controls movement.

Scientists have so far struggled to make any significant advances in the pursuit of a cure, and treatments are limited to drugs which control symptoms.  

But with California-based International Stem Cell Corporation (ISCO) set to start clinical trials on 12 people with moderate to severe Parkinson's Disease, there are hopes of a breakthrough.

During the trial, doctors will implant replacement brain cells, called neural precursor cells, into the patients' brains.

It is hoped these cells will finish maturing into the kind of neurons which are destroyed by the movement disorder.

The trials, which are the first to be carried out on humans, will give participants varying doses of neural stem cells.

Patients will then have their neurological condition monitored for 12 months to see how their brains and bodies react.

But in a paper published in the Journal of Parkinson's Disease, Dr Roger Barker, from the John van Geest Centre for Brain Repair at the University of Cambridge warned it was still early days.

'As with many such exiting news items, one should react with caution,' he said.

Especially since the outcome of this trial can affect the development of other stem cell programs moving towards clinical trials.'

Experts urged discussion of five key points before the trials begin, specifically: 

• What is being transplanted, and what is the proposed mechanism of action?
• What are the pre-clinical safety and efficacy data supporting the use of the proposed stem cell product? 
• Can arguments concerning ethics, risk mitigation, or trial logistics outweigh concerns regarding the expected efficacy of the cell and constitute a primary justification for choosing one cell type over another in a clinical trial?
• What is being claimed regarding the potential therapeutic value of the stem cell-based therapy better control of symptoms or a cure?
• What is the regulatory oversight of the trial and is it guided by input from experts in the field
• 
  

Actor Michael J Fox, pictured here in Back to the Future, is a famous sufferer of Parkinson's, a progressive neurological condition that destroys the brain cells producing the chemical messenger dopamine

Patrik Brundin editor-in-chief of the Journal of Parkinson's Disease, and co-author of the article said the trials should only start once the questions had been addressed.'This is an exciting prospect but should only be undertaken when all the necessary pre-clinical data and regulatory approvals have been obtained and verified and the criteria for moving those cells to trials fully resolved and met,' he said.

'Acting prematurely has the potential not only to tarnish many years of scientific work, but can threaten to derail and damage this exciting field of regenerative medicine.

'Hopefully, in 2016, we are ready to take a more careful approach as we strive to repair the PD brain with stem cell-based therapies, avoiding many of the mistakes that have dogged this field over the last three decades.'

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