Mutant calreticulin in certain blood cancers (MPN)
Functional and Molecular Dissection of Mutant Calreticulin in Myeloproliferative Neoplasms
Looking for new treatments that target a mutant protein called calreticulin to help people with CALR‑mutant myeloproliferative neoplasms.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Palo Alto Veterans Instit for Research NIH-funded |
| Lab location | 1 site (Palo Alto, United States) |
| Project ID | NIH-11189783 on NIH RePORTER |
What this research studies
This project focuses on blood cancers called myeloproliferative neoplasms (MPN) driven by mutations in the calreticulin (CALR) protein. The research team will study how CALR mutations change blood stem cell behavior, including protein handling pathways such as N‑glycosylation and secretion, and how co‑mutations like ASXL1 alter chromatin to drive disease. They will use laboratory models, molecular analyses, and patient‑derived cells or samples to find biochemical vulnerabilities in CALR‑mutant cells. The ultimate aim is to turn those vulnerabilities into new therapies that could modify or possibly cure CALR‑mutant MPN.
Who could benefit from this research
Good fit: People diagnosed with CALR‑mutant myeloproliferative neoplasms (for example essential thrombocythemia or primary myelofibrosis), including those with concurrent ASXL1 mutations, are the ideal candidates.
Not a fit: Patients whose MPN is driven by other mutations (for example JAK2 or MPL without CALR) or unrelated blood disorders may not directly benefit from therapies developed here.
Why it matters
Potential benefit: If successful, this work could produce more precise, disease‑modifying treatments for people with CALR‑mutant MPN.
How similar studies have performed: Existing drugs target related pathways (such as JAK inhibitors) but directly targeting mutant CALR is a newer approach that remains largely preclinical.
Where this research is happening
Palo Alto, United States
- Palo Alto Veterans Instit for Research — Palo Alto, United States (Active)
Researchers
- Principal investigator: Mullally, Ann — Palo Alto Veterans Instit for Research
- Study coordinator: Mullally, Ann
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.