NIH Grant to Advance Multiple Sclerosis Therapy Targeting Remyelination

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Novoron Bioscience, Inc., a biotech company focused on developing therapies for disorders of the central nervous system, recently announced that it has received a $680,000 National Institutes of Health (NIH) grant to assess the potential of a new therapeutic approach to induce remyelination in multiple sclerosis (MS).

MS is an autoimmune disease where the body’s own immune system attacks the protective coat around nerves in the brain and spinal cord, known as myelin. The destruction of myelin (demyelination) is irreversible in MS, and it disrupts signal transmission between nerves, leading to impairments in movement, vision, and speech. Demyelination is the primary cause of disability and death in MS patients.

Novoron received six NIH grants (totaling more than $1.2 million) to investigate novel therapeutic strategies for the treatment of several neurological disorders, namely MS, spinal cord injury, and stroke. The latest grant was awarded under the NIH’s Small Business Innovation Research (SBIR) Program.

For MS, the company will assess a therapeutic approach based on remyelination.

“These NIH grants are invaluable in helping us advance our novel therapeutic approaches, addressing fundamental limitations in the treatment of disorders of the central nervous system such as multiple sclerosis, spinal cord injury and optic neuropathies,” said Dr. Travis Stiles, president and CEO of Novoron Bioscience, in a press release. “We look forward to the opportunity to continue to extend our unique technological approach to CNS [central nervous system] disease and assess our capacity to promote remyelination and preserve neuronal viability in multiple sclerosis.”

Oligodendrocytes are the cells responsible for producing myelin, and when these cells die, due to trauma or environmental factors such as toxicity, the myelin sheath may become degraded. Normally, oligodendrocyte precursor cells (OPCs) infiltrate the damaged area and take over the function of oligodendrocytes, synthesizing new myelin and repairing the damage. However, in MS, a persistent myelin loss together with inflammation (due to immune infiltrates) inhibit the OPCs’ ability to produce new myelin. Such inhibition is the result of an unwanted activation of a molecule called RhoA. Novoron’s therapeutic lead compound, NOVO-117, targets RhoA by acting on a previously unknown mediator of RhoA activation — LRP1 (low density lipoprotein-related protein 1).

“Today’s approved therapies are aimed solely at dampening the autoimmune attack on myelin, but there are currently no approved treatments to repair damage after an attack has occurred,” explained Dr. Stiles. “Our approach shows substantial promise towards repairing this damage, a process known as remyelination, which not only protects neurons from further damage, but can lead to recovery of cell function and prevention of death.”

“Rho and Rho-kinase are well considered targets for multiple sclerosis, but are difficult to effectively inhibit therapeutically in the brain. Novoron’s technological foundation relies on our unique approach to targeting RhoA in a fashion that is amenable to disorders of the brain and spinal cord,” concluded Dr. Stiles.

The NIH grant will allow the development of a proof-of-concept study to validate further preclinical studies on NOVO-117 as a therapy for MS.

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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
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