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HOPE for FATIGUE

Part 1 - Mitochondrial Failure: The Root Cause of Alzheimer's Disease?

Mitochondria, the energy generators of our cells, are essential for life. When they are under attack - from a combination of stress, environmental toxins, poor nutrition and genetic mutations – our cells can no longer function at an optimal level and they begin to shut down.

There is now growing recognition that effective Alzheimer’s disease treatments will need to improve mitochondrial function as a way to slow, and potentially reverse, the negative effects of this disease on cognitive function.

Significant mitochondrial damage has been identified early in the course of Alzheimer’s disease (AD) and it often predates the accumulation of amyloid-beta, the cellular waste product historically believed to be responsible for the premature death of brain cells. A growing body of evidence now points to progressive mitochondrial failure as the trigger and driving force of amyloid-beta accumulation, eventually leading to the premature loss of brain cells, progressive defects in memory and poor cognitive functioning. 

Treatment Approaches

The landscape for treating Alzheimer's disease has been bleak for many years. Following the failure of multiple efforts at targeting amyloid-beta, the pharmaceutical industry has now realized the need to focus on alternative AD targets. 

The mitochondrion is a highly complex organelle and addressing mitochondrial dysfunction in AD will require innovative strategies. These strategies could focus on supporting the thousands of biochemical reactions occurring within the mitochondria with key micronutrient cofactors that can significantly reduce oxidative stress. Also, addressing MT dysfunction by utilizing a compound that broadly supports mitochondrial functionmay prove more effective than relying on a single small molecule that only addresses a single molecular target.

K-PAX Pharmaceuticals is currently planning to perform Phase 2 proof-of-concept trial of a repurposed drug (methylphenidate) augmented with high potency mitochondrial support [KPAX002] to demonstrate improvement in cognitive function compared to placebo (24 weeks). 

We have already observed KPAX002’s ability to improve cognitive functioning in patients with mitochondrial diseases in several settings. In three previous clinical trials, we have observed asubstantial improvement in cognitive function, averaging a mean improvement of over 20% after 12 weeks of treatment. Furthermore, we have recently administered KPAX002 to several AD patients in the clinic where it has been both well tolerated and generated substantial improvement in cognitive function (approximately 30% within 90 days) as assessed with validated measurement tools.

Conclusion

There are compelling reasons driving us to understand how altered mitochondrial function can be addressed to inhibit the progressive loss of brain cells, and the memory loss that follows. By delivering a well-studied mitochondrial support compound designed to decrease oxidative stress and promote mitochondrial repair, and by gently stimulating CNS neurons, improvement in cognitive functioning can occur and the rescue of previously failing CNS neurons can be achieved despite conditions of significant metabolic stress.

In Part Two of this series, we will describe in detail the component parts of KPAX002 and how its unique mechanism of action can rescue failing brain cells, thereby improving cognitive function in Alzheimer’s patients.

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