Targeting abnormal Gαs signaling in myeloid blood cancers
Mechanisms and targeting of aberrant Gas activation in myeloid neoplasms
['FUNDING_R01'] · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · NIH-11313828
Researchers are developing ways to block harmful Gαs signaling in people with myelodysplastic syndromes and related acute myeloid leukemias linked to splicing-factor mutations.
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-11313828 on ClinicalTrials.gov |
What this research studies
This project uses human induced pluripotent stem cell models with common splicing-factor mutations (SRSF2 and U2AF1) created by CRISPR to mimic patient disease. Scientists analyze RNA and protein to find mis-splicing of the GNAS gene that produces a longer, overactive Gαs protein isoform (Gαs-L). They will test strategies to inhibit this abnormal Gαs-L signaling in lab models to see if normal blood cell development can be restored. The work aims to translate these findings into candidate drug approaches for patients with SF-mutant MDS/AML.
Who could benefit from this research
Good fit: Ideal candidates would be people diagnosed with myelodysplastic syndromes or acute myeloid leukemia whose cancer carries SRSF2 or U2AF1 splicing-factor mutations.
Not a fit: Patients without splicing-factor mutations (SRSF2/U2AF1) or with unrelated leukemia subtypes are unlikely to benefit from therapies specifically targeting Gαs-L.
Why it matters
Potential benefit: If successful, this work could lead to targeted treatments that block abnormal Gαs signaling and slow or prevent progression from MDS to AML for patients with these splicing-factor mutations.
How similar studies have performed: Broad splicing-modulator drugs have shown limited success in clinical testing, and targeting Gαs-L in SF-mutant myeloid neoplasms is a new, primarily preclinical approach.
Where this research is happening
NEW YORK, UNITED STATES
- ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: PAPAPETROU, EIRINI — ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
- Study coordinator: PAPAPETROU, EIRINI
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.