DEPARTMENT OF DEFENSE - CONGRESSIONALLY DIRECTED MEDICAL RESEARCH PROGRAMS

Targeting Neurotoxic Gila to Promote Myelin Repair and Neuroprotection

Principal Investigator: CALABRESI, PETER
Institution Receiving Award: JOHNS HOPKINS UNIVERSITY
Program: MSRP
Proposal Number: MS180181
Award Number: W81XWH-19-1-0622
Funding Mechanism: Investigator-Initiated Research Award
Partnering Awards:
Award Amount: $970,635.00


PUBLIC ABSTRACT

Multiple sclerosis (MS) is a major cause of non-traumatic progressive disability in young people. While we have several drugs to treat relapsing forms of the disease, there is an unmet need to prevent disability progression. This proposal directly addresses the focus area of obstacles to remyelination and neuroprotection. In this application we propose to examine how certain brain cells called glia become activated in a way that causes damage to the myelin and nerves as occurs in people suffering from multiple sclerosis.

Our team has discovered that a new drug may be especially effective at getting into the brain and suppressing the type of brain inflammation that we think causes damage to the nerve cells in people with MS. We have preliminary data that this drug suppresses the onset of brain inflammation in animals that is similar to what we see in people with multiple sclerosis. In this study we plan to treat the animals, after they are already diseased, to see if the new drug can suppress the ongoing brain inflammation and prevent nerve damage or facilitate repair of the myelin coating around the nerve projections. These research lab studies will provide critical new information to scientists about how inflammation in the brain causes damage and will speed up the translation of this drug into clinical trials in people suffering with multiple sclerosis.

This area of research addresses an important need in that presently available drugs predominantly target immune cells in the blood stream and help in relapsing forms of multiple sclerosis. It is believed, however, that inflammation in the brain is likely to be the major problem in progressive disease, which is a major cause of disability in young people including some active military personnel and many veterans.