Novel Approaches to Targeting TSC1/TSC2 Across Malignancies

James J. Harding, MD, describes the GI tumors where TSC1 or TSC2 alterations can arise, how the mechanism of action of nab-sirolimus may help fulfill an unmet need in this population, and the importance of using genetic testing to identify patients with mutations in TSC1, TSC2, or other genes to drive targeted treatment.

James J. Harding, MD, discusses the prevalence of TSC1 and TSC2 mutations in gastrointestinal cancer, and the need to develop a standard-of-care treatment for patients with these genetic alterations.

Brian Schulte, MD, discusses the relevance of these mutations and an actively enrolling phase 2 PRECISION-1 basket trial investigating the efficacy of nab-sirolimus in solid tumors.

Brian Schulte, MD, discusses the ongoing phase 2 PRECISION 1 basket trial evaluating the use of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring TSC1/2 mutations.

Gopa Iyer, MD, explained the rarity of TSC1 and TSC2 mutations in GU cancers, discussed the rationale and design of PRECISION 1, and emphasized the important role next-generation sequencing plays when determining the most effective targeted therapies for patients with GU cancers and other tumor types.

Gopa Iyer, MD, discusses the unmet needs for patients with solid tumors harboring TSC1/2 mutations.

With no approved agents for patients with malignant tumors harboring TSC1 or TSC2 inactivating alterations, investigators seek to establish a new pathway forward for patients with solid tumors in the PRECISION 1 trial.

Brian Schulte, MD, discusses the prevalence of TSC1/TSC2 mutations in cancer.

Kathleen N. Moore, MD, MS, discusses the investigation of nab-sirolimus in TSC1/2-mutated gynecologic cancers.

Kathleen N. Moore, MD, MS, discusses the incidence of TSC1 and TSC2 mutations in different gynecologic tumor types, the trial design and eligibility criteria for the PRECISION 1 study, and the importance of genetic testing to identify these mutations and treat them early.