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Foundation Supports ALS Inflammation Treatments

Brain activity graphic

(Gordon Johnson, Pixabay)

17 Feb. 2020. A foundation is funding a biotechnology company’s research on therapies to block proteins encouraging neuron inflammation in people with amyotrophic lateral sclerosis, or ALS. The ALS Association in Arlington, Virginia is providing a $500,000 grant to support the work of Neuropore Therapies Inc. in San Diego.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure.

Neuropore Therapies discovers and develops treatments for neurodegenerative disorders. The company focuses particularly on diseases from accumulations of toxic misfolded proteins in neurons in the brain, and the chronic and damaging inflammation of neurons that instigates or drives progressive neurodegeneration. Neuropore’s pipeline includes therapies in clinical trials and preclinical development for Parkinson’s disease and ALS.

One of Neuropore’s targets is a set of proteins called toll-like receptors that detect foreign microbes in the body. Toll-like receptors stimulate innate, or general, defense mechanisms in the immune system, as well as responses to specific pathogens. In the brain, however, toll-like receptors can drive chronic inflammation that damages neurons in people with ALS and Parkinson’s disease.

Neuropore developed a treatment code-named NPT1220-312 that blocks a subset of toll-like receptors in the brain, known as toll-like receptor 2 or TLR2 proteins associated with ALS and Parkinson’s disease inflammation. “Targeting TLR2 is an exciting new approach to the treatment of ALS,” says Doug Bonhaus, Neuropore’s CEO and chief scientist in a company statement released through BusinessWire. “NPT1220-312 is a potent, selective, orally-bioavailable TLR2 antagonist. It has shown robust beneficial actions in cell-based assays and in an animal models of ALS.”

ALS Association is an advocacy and research funding organization seeking to find effective treatments for ALS, and provide support for patients with ALS and their families. The group is funding the Neuropore work from its drug development program that supports preclinical studies and clinical trials by academic labs and industry teams.

Bonhaus adds, “Pending positive outcomes in ongoing studies, our goal is to submit an IND application for NPT1220-312 to FDA by first half of 2021.” IND is short for investigational new drug application, in effect clearance from FDA to begin clinical trials of an experimental drug or biologic therapy.

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