Recipient: Dr Richard Gordon

Evaluating a new therapeutic target to block inflammation and slow motor neuron loss in MND using repurposed drugs.

Funding from MND and Me is enabling our team to establish a new MND research program which aims to understand and treat the underlying mechanisms which drive disease progression. Persistent activation of the immune system has been shown to drive ongoing inflammation which is believed to accelerate the loss of motor neurons in MND. Importantly, this persistent inflammation is evident across all genetic and sporadic forms of the disease, suggesting that it is a common underlying feature of MND.

Our project is evaluating the role of a promising new pathway that we recently discovered to be important in regulating the persistent inflammation which occurs during MND. This pathway is expressed both in immune cells, which drive inflammation in MND, as well as the neurons which are lost as the disease progresses. Our research has uncovered that this pathway is highly activated in models of MND and drives both inflammation and neuronal death. Crucially, this pathway can be blocked by a drug that is already approved, and which is currently in use for treating certain types of blood cancers arising from immune cells. Additionally, emerging studies with this drug in human trials show that it can efficiently cross the blood brain barrier.  This is critical for the drug to reach the brain and spinal cord at levels that may be effective to block inflammation and neuron loss in MND. Therefore, this repurposed drug has already overcome one of the biggest challenges associated with developing new treatments for brain diseases such as MND. In collaboration with highly experienced MND researchers and clinicians, our team is currently evaluating the effectiveness of this repurposed cancer drug as a potential new treatment for MND using animal models. We are also confirming if this pathway is activated in MND patients similar to what we have already shown in animal models.  The results from these studies will guide the next steps in progressing this drug to the clinic as a potential new treatment for MND.

This is the first grant for MND research for our lab and we are grateful for the funding support from MND and Me during these difficult times. This funding will enable our team to establish and grow our new MND research program with the goal of developing new treatments for this devastating disease.