larotrektinib

Larotrektinib is an investigational targeted cancer therapy. It is a small molecule inhibitor of tropomyosin receptor kinases (TRKs), which are proteins that play a role in cell growth and development. In certain types of cancer, the genes that encode TRKs can become rearranged or mutated, leading to their abnormal activation. This abnormal activation can promote the growth and survival of cancer cells.

Larotrektinib works by blocking the activity of these abnormally activated TRKs. By inhibiting TRK signaling, larotrektinib aims to slow down or stop the growth of cancer cells that rely on this pathway. It is designed to target cancers with specific genetic alterations in the TRK genes, regardless of the cancer's origin in the body. This approach is known as tumor-agnostic therapy.

Clinical trials have investigated larotrektinib in various solid tumors that harbor NTRK gene fusions. These NTRK gene fusions are the genetic alterations that lead to the overactivation of TRKs. Studies have shown promising responses in a range of cancer types, including lung cancer, salivary gland tumors, and soft tissue sarcomas. The development of larotrektinib represents an advancement in personalized medicine, focusing on the underlying genetic drivers of cancer rather than the tissue of origin. Regulatory reviews are ongoing for its potential use in treating NTRK fusion-positive solid tumors.