Parkinson’s and nerve growth factors

Nerve growth factors, or neurotrophic factors, are proteins that support nerve cells (neurons) by encouraging their growth, development, and survival. There has been considerable research exploring their potential use in Parkinson’s. 

How could nerve growth factors help to treat Parkinson’s? 

Researchers believe that increasing levels of specific nerve growth factors associated with dopamine neurons could slow Parkinson’s progression. While the translation of nerve growth factor research from the laboratory into a potential treatment for people with Parkinson’s has posed its challenges, there is progress being made by researchers to find solutions. Cure Parkinson’s is actively working to overcome obstacles, and the journey towards a promising treatment is testament to the effort of all those involved. Learn more about the nerve growth factors being explored for Parkinson’s and the research being conducted in these areas below.

GDNF 

Glial cell-derived neurotrophic factor (GDNF) is thought to play a role in dopamine neuron differentiation – the process by which cells are instructed what type of neuron to develop into – and survival. There is much laboratory evidence to suggest that increasing levels of GDNF can slow or prevent neuron loss; however, this has yet to be replicated in a clinical trial. Cure Parkinson’s has supported further research into GDNF therapies, including exploring another closely related nerve growth factor, GDF5.  

How is gene therapy being explored for GDNF?

One of the greatest challenges associated with testing these therapies is their administration. Our brains are surrounded by a protective layer of cells called the blood-brain-barrier, which controls what comes in to and out of the brain. Most nerve growth factors are too large to pass over the blood-brain-barrier, meaning these therapies cannot be taken orally or via injection. Instead, an invasive surgical approach is necessary to allow these proteins to be administered directly to the brain, which needs to be done several times within one trial.  

Researchers are exploring different ways to overcome this challenge, one being gene therapy. Gene therapy uses modified, inactive forms of viruses to direct cell activity. In this case, researchers are hoping to use this to tell neurons to increase production of neurotrophic factors, like GDNF. This method would only require a one-time surgery and the effects of this treatment could potentially be longer as cells would be able to produce their own GDNF.  

CDNF 

Cure Parkinson’s has also supported work looking at another nerve growth factor for the treatment of Parkinson’s: cerebral dopamine neurotrophic factor (CDNF). More specifically, researchers in Finland have been developing a small fragment version of CDNF, called C-CDNF, that would be able to pass through the blood-brain-barrier. If effective in the laboratory, C-CDNF could eliminate the need for surgery, making it a more accessible treatment for people with Parkinson’s.