Developing new therapies for genetic disorders affecting the RAS/MAPK pathway
Therapy Development for Genetic Disorders of the RAS/MAPK Pathway
['FUNDING_R01'] · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · NIH-11078785
This study is looking for better and safer treatments for RASopathies, which are genetic disorders, by testing a new promising drug called M1 in special models like fruit flies and human cells, to help people who have these conditions feel better without the harsh side effects of current options.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11078785 on ClinicalTrials.gov |
What this research studies
This research focuses on developing new treatments for RASopathies, which are genetic disorders that disrupt the RAS/MAPK signaling pathway. The team is using advanced models, including transgenic fruit flies and human stem cells, to identify effective drug candidates that can target these disorders without the severe side effects associated with current therapies. They have already discovered a promising compound, M1, and are working to enhance its effectiveness and understand its mechanisms. The goal is to create safer and more effective treatments for patients suffering from these complex genetic conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with RASopathies or related genetic disorders that affect the RAS/MAPK signaling pathway.
Not a fit: Patients with RASopathies who do not respond to therapies targeting the RAS/MAPK pathway may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective therapies for patients with RASopathies, improving their quality of life.
How similar studies have performed: Previous research has shown promise in targeting the RAS/MAPK pathway, but this approach aims to develop novel therapies that have not yet been tested.
Where this research is happening
NEW YORK, UNITED STATES
- ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: GELB, BRUCE D — ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
- Study coordinator: GELB, BRUCE D
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.
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