CAR T Cell Therapy Targets CALR Mutation Driving Rare Blood Cancers
核心洞察
Researchers at UCL developed CAR T cells that precisely kill cancer cells carrying the CALR (搜索) mutation without harming other blood cells.
The therapy was validated using patient samples, a lab model recreating human bone marrow, and studies in mice.
A Phase I clinical trial is planned at UCLH, aiming to begin within one to two years pending funding and regulatory approval.
Researchers at UCL have developed a CAR T cell therapy (搜索) capable of precisely killing cancer cells carrying the CALR (搜索) mutation, a key driver of rare blood cancers, without harming other blood cells. The findings represent a significant step toward a targeted treatment for myeloproliferative neoplasms (搜索) (MPNs), chronic blood cancers that begin when a mutation arises in the DNA of a blood stem cell.
The therapy was validated across multiple experimental systems, including patient samples, a laboratory model that recreates human bone marrow, and studies in mice. In these models, the CAR T cells demonstrated the ability to selectively eliminate cancer cells bearing the CALR (搜索) mutation while sparing healthy blood cells.
MPNs are chronic blood cancers that originate from a mutation in a blood stem cell. Over time, some patients progress to myelofibrosis (搜索), a serious condition marked by scarring of the bone marrow and anaemia. The CALR (搜索) mutation represents a key target in this disease pathway, and the new CAR T cell approach is designed to home in on this specific genetic alteration.
A Phase I clinical trial is being planned at UCLH, with the aim to begin within the next one to two years, subject to funding and regulatory approval. The development underscores the potential of cell-based immunotherapies to address genetically defined subsets of hematologic malignancies with greater precision.