Brain-targeted kinase blockers that spare the rest of the body
Brain-Restricted Kinase Inhibition with Binary Pharmacology
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · NIH-11284092
A two-part drug strategy to block harmful enzyme activity inside the brain while reducing side effects elsewhere, aimed at people with brain tumors and other brain disorders.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA, SAN FRANCISCO (nih funded) |
| Locations | 1 site (SAN FRANCISCO, UNITED STATES) |
| Trial ID | NIH-11284092 on ClinicalTrials.gov |
What this research studies
This work develops brain-permeable kinase inhibitors that tightly bind a helper protein (FKBP12) to shut down disease-driving signaling in brain cells. The team pairs those brain-active drugs with a second, brain-impermeable blocker to protect the rest of the body from drug effects, keeping treatment focused in the brain. They've tested this approach in lab models including mouse brain tumor grafts and are designing similar FKBP12-dependent kinase inhibitors from known drug scaffolds. The goal is to deliver stronger, more precise brain treatments with fewer systemic side effects.
Who could benefit from this research
Good fit: Most directly relevant to people with glioblastoma or other brain tumors today, and potentially to patients with brain disorders tied to the same kinase pathways if clinical testing expands.
Not a fit: Patients whose illnesses are unrelated to brain kinase signaling or who need only approved standard treatments are unlikely to benefit in the near term.
Why it matters
Potential benefit: If successful, this could allow more effective, higher-potency drugs to treat brain diseases while greatly reducing harmful side effects outside the brain.
How similar studies have performed: Preclinical studies of RapaLink-1 and the complementary RapaBlock approach showed strong results in animal brain-tumor models, but human clinical data remain limited.
Where this research is happening
SAN FRANCISCO, UNITED STATES
- UNIVERSITY OF CALIFORNIA, SAN FRANCISCO — SAN FRANCISCO, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: WEISS, WILLIAM A — UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
- Study coordinator: WEISS, WILLIAM A
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.
Conditions: Alzheimer disease dementia, Alzheimer syndrome, Alzheimer's Disease