Positive results announced from early stage stem cell therapy trial

The results from this Phase 1 clinical trial were announced by pharmaceutical company Bayer AG and BlueRock Therapeutics LP at the International Congress of Parkinson's Disease and Movement Disorders in Copenhagen, Denmark.

In the trial, 12 people with Parkinson’s underwent surgery to receive either a high or low dose of dopamine-producing cells developed from stem cells. The experimental therapy is called bemdaneprocel or BRT-DA01.

The results show that the treatment was well tolerated with no major safety issues. And after 12 months, participants experienced improvement in their Parkinson’s symptoms, with those who received the higher dose showing greater improvement.

Planning is now underway for a Phase 2 study to investigate this promising therapy further, with recruitment expected to begin in 2024.

Why stem cells hold hope for Parkinson’s

Stem cells have the ability to develop into different types of specialised cells, such as skin, muscle or brain cells.

Researchers have been working for many years to harness the potential of stem cells for Parkinson’s and find ways to convert them into dopamine-producing brain cells that can then be used to replace those that are lost in people with the condition.

There are many challenges to getting stem cell therapies right for Parkinson’s, including: 

• developing cells for transplant that are able to survive and integrate into the brain
   
• ensuring transplanted cells do what they are supposed to and don’t cause unwanted side effects
   
• ensuring transplanted cells are not ‘rejected’
   
• the overall safety and acceptability of the treatment.

Many years of painstaking research to address these challenges mean that there is now a range of promising stem cell based therapies being tested in early-stage clinical trials for Parkinson’s around the world.  

Early signs of promise need further investigation

Claire Bale, Associate Director of Research at Parkinson’s UK, comments:

“These results indicate that this pioneering therapy is safe and produces improvements in symptoms for people with Parkinson’s which is very encouraging.

“It’s important to note that this was an 'open label' trial with no comparison group. This means that all the participants and investigators knew that they were receiving the active treatment - in this case a stem cell derived transplant.

“We know the placebo effect - the belief that a treatment will work - is very strong in Parkinson's, so we need to bear that in mind when looking at these results.

“We’re glad that the team are planning further studies very soon to investigate the potential benefits of this therapy in more depth and we’ll share any further news as soon as it’s available.”