New funding from Cure Parkinson's on Neurotrophic factor therapy for Parkinson's

26 Aug 2021

A new study funded by Cure Parkinson’s and led by Professors Gerard O’Keeffe and Aideen Sullivan at University College Cork, Ireland, will help lay the foundations to determine just how effective the neurotrphic factor GDF5 is at regenerating brain neurons and stopping Parkinson’s. 

GDF5 is a neurotrophic factor that has already shown promising pre-clinical results. A new study will build on early results and help to determine whether this drug could progress to clinical trials.

Neurons and your brain: Growth factors are proteins that are secreted by cells throughout the body. They stimulate cells to multiply. In the brain, the role of neurotrophic growth factors is to keep neurons alive. Growth factors signal to the neurons and they rely on this constant communication to stay alive. It is the death of dopamine producing neurons in the brain that leads to Parkinson’s.

Exploring the next generation of growth factors: In earlier studies a drug called GDNF showed encouraging signs. However, these signs did not consistently translate in clinical trials. GDNF is a big sticky protein and it cannot pass the bloodbrain-barrier. It requires direct delivery to the brain via surgery.
Researchers think they might have an answer for why the GDNF trials produced complex results. To function, GDNF requires a protein called ‘RET’. But in Parkinson’s, the levels of RET are disrupted in the body. It is thought that without sufficient RET, GDNF may not be able to provide the anticipated regenerative effects.
GDF5 acts through a different pathway to GDNF; it does not need the RET protein to work. Preclinical studies already completed have shown GDF5 to have protective effects in Parkinson’s.

"We are honoured to have the support of Cure Parkinson’s for our ongoing pre-clinical work on developing the neurotrophic factor GDF5 as a therapy for Parkinson’s. In our new study, which is funded by Cure Parkinson’s, we will examine GDF5’s ability to protect dopamine-producing neurons in the brain in a model of Parkinson’s which mimics aspects of the pathology and symptoms of the human condition. If successful, this will lay the foundation for the use of GDF5 in clinical trials aimed at preventing the worsening of Parkinson’s pathology." Professors Aideen Sullivan and Gerard O’Keeffe, Department of Anatomy and Neuroscience, University College Cork, Ireland.

 Photograph B Riedewald