Piqray (alpelisib)

This consensus document provides operational and interpretative guidelines aimed at standardizing PIK3CAtesting and assessing related genes in clinical practice. These recommendations promote a harmonized approach to patient care, in line with the latest scientific evidence.

These findings identify actionable molecular subtypes of cervical cancer and support targeting PI3Kα in PIK3CA-mutant tumors. Moreover, combining PI3K inhibition with antigen-specific immunotherapy represents a promising strategy to improve outcomes in advanced HPV16-associated cervical cancer.

Background: Alpelisib plus fulvestrant improves outcomes in PIK3CA-mutated, hormone receptor-positive, HER2-negative metastatic breast cancer. Evening alpelisib preceded by fasting and low-carbohydrate guidance may improve metabolic tolerability without compromising efficacy or QoL. These findings support evaluation in a larger trial incorporating prospective metabolic adherence and pharmacokinetic assessments.

This study reports ANXA3 as a biomarker o predict poor prognosis and lenvatinib resistance in HCC. The combined use of Alpelisib and lenvatinib may serve as a potential therapeutic strategy for lenvatinib-resistant HCC.

Alpelisib plus fulvestrant provides clinical benefit for patients with PIK3CA-altered, HR+, HER2- ABC across a range of concomitant alterations, including those previously implicated in endocrine therapy or CDK4/6i resistance. Machine learning models identified factors including gene mutations that influenced PFS.

Interestingly, treatment with the TRPM8 antagonist led to a notable increase in PD-1 expression on CD8+ T cells. These findings suggest that the decreased expression of inhibitory receptors on CD8+ T cells after treatment with the CB1 antagonist whether alone or with alpelisib and TRPM8 highlights the potential of ECS as a promising therapeutic target in cancer treatment.

P1, N=32, Recruiting, Columbia University | Trial primary completion date: May 2026 --> Dec 2026

Comprehensive molecular analysis and MTB discussion revealed several potential treatment targets, leading to subsequent treatment including dinutuximab beta, nivolumab, cabozantinib, I-131-mIBG radionuclide therapy and alpelisib, unfortunately, all followed by disease progression. This case demonstrates the potential of comprehensive molecular analysis including methylation profiling for diagnosis and treatment guidance in rare tumors. Additional research is urgently required to improve outcomes in elderly patients with neuroblastoma.

A PDC with a PIK3CA activating mutation was sensitive to the PI3K inhibitor Alpelisib. We also demonstrated that PDCs harboring chromosome segregation errors were vulnerable to KIF18A deletion and pharmacological inhibition. These results support the value of HNSCC-derived PDCs as a platform for advances in precision medicine in oncology research.

The discovery that the non-selective beta-blocker propranolol induces rapid regression of proliferating IHs established the first widely adopted systemic pharmacologic therapy in vascular anomaly care and provided a clinical proof-of-concept that targeting lesion-specific endothelial biology can alter disease course. In parallel, recurrent somatic variants affecting PI3K/AKT/mTOR (e.g., PIK3CA, TEK/TIE2, AKT1) and RAS/MAPK (e.g., KRAS, NRAS) signalling have reframed many malformations as mosaic disorders amenable to targeted inhibition with agents such as sirolimus, alpelisib, AKT inhibitors and MEK inhibitors. This review synthesizes translational mechanisms, clinical evidence and safety considerations for beta-blockers and emerging targeted therapies, emphasizing lesion phenotype, timing of intervention and molecular stratification as determinants of response. We highlight current limitations, including toxicity, durability and pathway escape, and outline future directions for precision therapy and genotype-guided trial design in vascular anomalies.

P2, N=69, Active, not recruiting, Novartis Pharmaceuticals | Trial completion date: Mar 2026 --> Jan 2027 | Trial primary completion date: Mar 2026 --> Jan 2027

P2, N=39, Recruiting, Peking Union Medical College | Trial primary completion date: Jul 2026 --> Jul 2028