Finding small molecules to help repair myelin in multiple sclerosis
OPC-X screen: Discovery of small molecule inhibitors to overcome extrinsic inhibition of remyelination.
Searching for drugs that help brain precursor cells rebuild the myelin coating lost in people with multiple sclerosis.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | J. David Gladstone Institutes NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11134777 on NIH RePORTER |
What this research studies
Researchers are using a lab-based screening approach to find small molecules that let oligodendrocyte precursor cells (OPCs) turn into myelin-producing cells even when blocked by fibrinogen. They developed an OPC-X assay and will scale it to screen many compounds in cell-based tests, then follow up promising hits in animal models of MS. Prior work showed that blocking BMP receptors with certain inhibitors increased myelin-producing cells in the assay and improved symptoms in two mouse models, and the team will use similar tests to validate new compounds. The overall aim is to identify candidate drugs that could move toward safety testing and, eventually, trials in people with MS.
Who could benefit from this research
Good fit: People with multiple sclerosis, especially those with active demyelination or recent loss of myelin, would be the eventual candidates for treatments developed from this work.
Not a fit: People without demyelinating conditions or those with long-standing, irreversible nerve damage may not benefit from remyelination-focused therapies.
Why it matters
Potential benefit: If successful, these compounds could promote remyelination and help slow disability or improve function in people with multiple sclerosis.
How similar studies have performed: Related preclinical studies, including use of BMP receptor inhibitors like LDN-212854, improved remyelination and clinical scores in animal models, but these approaches have not yet been tested in humans.
Where this research is happening
San Francisco, United States
- J. David Gladstone Institutes — San Francisco, United States (Active)
Researchers
- Principal investigator: Akassoglou, Katerina — J. David Gladstone Institutes
- Study coordinator: Akassoglou, Katerina
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.