Completed Brain & Nervous System Cancer

Development of Mitochondrial Permeability Transition Pore Inhibitors for the Treatment of Neurodegenerative Diseases

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A small biotech company is developing drugs that protect the brain’s cellular batteries—mitochondria—to slow or halt Parkinson’s disease and motor neuron disease. This matters because current treatments for both conditions only manage symptoms, not the underlying disease. Parkinson’s affects roughly 145,000 people in the UK, while motor neuron disease typically kills within 3–5 years of diagnosis. A growing body of evidence links mitochondrial failure to the death of brain cells in these disorders, yet no approved drug targets this mechanism. If NRG Therapeutics’ drug candidates succeed, they could fundamentally change the trajectory of these diseases. Instead of merely masking symptoms, patients might gain years of preserved mobility, speech, and independence. For motor neuron disease, even a modest delay in progression would extend meaningful life beyond the current few months offered by the only available treatment. This project funds the final preclinical steps: proving the drugs reach the brain, protect mitochondria, prevent cell death in animal models, and remain safe with repeated dosing. If the data hold, the next step would be human trials. The research is applied, not fundamental—its entire purpose is to produce a candidate ready for clinical testing.

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Although life expectancy has increased steadily following major advances in public health and the development of modern medicines development, _healthy_ life expectancy has not increased to the same degree. Consequently, the UK's aging population is facing a crisis of extended periods of ill health in later life, with older adults often developing one or more age-related chronic disease such as arthritis, cancer, type 2 diabetes mellitus, cardiovascular and neurodegenerative disorders. Parkinson's disease (PD) is the second most prevalent neurodegenerative disorder in the UK, affecting ~145,000 individuals. People with Parkinson's experience debilitating motor (tremor, rigidity, muscle stiffness) and non-motor (dementia, depression) symptoms. Unfortunately, all current treatments for PD control symptoms only and do not halt or slow progression of disease. Motor neuron disease (MND), is a rare fatal neurodegenerative disorder that affects the nerve cells in the brain and the spinal cord resulting in a loss in control of the muscles needed to move, speak, eat, and breathe. This typically leads to death within 3-5 years of diagnosis. There is only one treatment for MND that has shown a modest (by a few months) benefit in slowing down progress of the disease. For both diseases, novel medicines that can cure or halt disease progression, are therefore desperately needed. NRG Therapeutics (NRG), an innovative UK-based biotech with expertise in neuroscience drug discovery and mitochondrial biology, is developing novel medicines for the treatment of chronic neurodegenerative diseases such as PD and MND. Mitochondria are the powerhouses or batteries of cells and therefore essential for maintaining cell health but there is now a substantial body of evidence showing that mitochondrial failure or dysfunction is common across many degenerative diseases. NRG's investigational new drugs have been shown to protect mitochondria and prevent the death of brain cells and therefore have the potential to halt or significantly slow the progression of disease in individuals with PD or MND. This project will fund progression of NRG's lead drug discovery project and generate a preclinical data package that demonstrates its drug candidates penetrate into the brain, protect mitochondria, prevent brain cell death in animal models and are safe & well tolerated following chronic dosing.

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