Consistently, pharmacological inhibition of Hedgehog signaling using vismodegib suppressed cell migration, invasion, and lung metastases in murine osteosarcoma cells. In human osteosarcoma cells, LDHA silencing downregulated Hedgehog signaling, and inhibition of Hedgehog signaling suppressed cell migration and invasion. These findings highlight the involvement of the Ldha-Hedgehog signaling axis in osteosarcoma lung metastases, providing insights into its molecular mechanisms and potential therapeutic targets.

P2, N=12, Recruiting, University of Florida | Trial completion date: Apr 2026 --> Apr 2027 | Trial primary completion date: Apr 2026 --> Apr 2027

P2, N=21, Active, not recruiting, Gruppo Oncologico del Nord-Ovest | Recruiting --> Active, not recruiting | Trial completion date: Jan 2026 --> Dec 2026

P1, N=57, Recruiting, Endeavor Biomedicines, Inc. | Not yet recruiting --> Recruiting

P2, N=40, Not yet recruiting, Nelum Corp

In this study, we present a strategy that merges structural features of the previously identified inhibitors AZ1 and vismodegib to develop a new class of potent dual inhibitors...Moreover, a suitable negative control has also been identified, supporting the validity of the observed effects in cellular assays. These results highlight the potential of these compounds to serve as advanced chemical probes for dual USP25/USP28 inhibition and as candidates for further therapeutic development.

The patient was managed with complete excision of the lesion, along with a neck dissection for level V lymph nodes, and adjuvant chemotherapy with Vismodegib...However, considering the possibility of metastasis, the patient is being followed up by a multi-disciplinary team (MDT) for evaluation of level IIB cervical lymph nodes. This case highlights the diagnostic and therapeutic challenges that may arise in rare cases of BCC with metastasis.

An inhibition of SHH signaling by cyclopamine or GANT61 entirely normalized the differentiation deficit of Rxrg-/- OPCs, but also myelination of newly generated Rxrg-/- OLs...Importantly, hyperactivation of the SHH pathway by purmorphamine or SAG inhibited the oligodendrogenesis and myelination potential of wild-type OPCs, indicating that SHH hyperactivity can also be a sufficient factor to block OPC differentiation. These results point to RXRg as an important regulator of SHH pathway signaling and underline the need of an optimal, fine-tuning of SHH signaling to assure successful oligodendrogenesis.

P1, N=55, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Mar 2026 --> Mar 2027

P1, N=36, Active, not recruiting, Mayo Clinic | Trial completion date: Nov 2026 --> Jul 2027 | Trial primary completion date: Jan 2026 --> May 2025

Dysgeusia and alopecia occurred with daily dosing, whereas every-other-day dosing was well tolerated. This case highlights the value of transcript-level functional assays for interpreting PTCH1 splice-site variants and supports individualized, toxicity-guided sonidegib scheduling in selected patients with Gorlin-Goltz syndrome.

P1, N=57, Not yet recruiting, Endeavor Biomedicines, Inc.