PellePharm announced its launch today, with financing from BridgeBio Pharma. The Menlo Park, Calif.-based company is developing a topical gel to treat basal cell carcinoma, including the tumors that result from Gorlin syndrome.
The company’s topical inhibitor compound, patidegib, acts on the hedgehog pathway – a signaling pathway that transmits cellular information for adult tissue maintenance. Abnormal hedgehog signaling has been correlated with a number of cancers including basal cell carcinoma. PellePharm is studying the topical formulation of patidegib in 2 phase II trials. A study evaluating the gel’s use in patients with Gorlin syndrome just completed enrollment, according to the company.
Get the full story at our sister site, Drug Delivery Business News.
The post PellePharm announces launch to develop topical gel for basal cell carcinoma appeared first on MassDevice.

Source: Mass DeviceCategory: Medical Equipment Authors: Tags: Clinical Trials Drug-Device Combinations Oncology Pharmaceuticals BridgeBio Pharma PellePharm Source Type: news

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We report a case of a 6-year-old female presenting with ventricular tachycardia secondary to cardiac fibroma. After diagnosis of recurrent jaw keratocysts, she was clinically and molecularly diagnosed with NBCCS. Identification of a cardiac fibroma should prompt careful assessment of past medical and family history with consideration of a diagnosis of NBCCS.Mol Syndromol

Authors: Skoda AM, Simovic D, Karin V, Kardum V, Vranic S, Serman L
The Hedgehog (Hh) signaling pathway was first identified in the common fruit fly. It is a highly conserved evolutionary pathway of signal transmission from the cell membrane to the nucleus. The Hh signaling pathway plays an important role in the embryonic development. It exerts its biological effects through a signaling cascade that culminates in a change of balance between activator and repressor forms of glioma-associated oncogene (Gli) transcription factors. The components of the Hh signaling pathway involved in the signaling transfer t…

Basal cell carcinoma (BCC) is the most common type of skin cancer worldwide [1 –4]. In the United States, BCC is diagnosed in> 2 million patients annually, and global incidence rates continue to rise [1,5,6]. Risk factors for the development of BCC include fair skin type, exposure to ultraviolet radiation, age, history of BCC, genetic disorders (eg, Gorlin syndrome, xeroderma pigmentosum), and immunosuppression [1,2,4,7]. BCC tumors are generally slow growing and rarely metastasize, and the prognosis for patients who receive appropriate therapy is typically very good [1 –3].

Source: Cancer Treatment ReviewsCategory: Cancer & Oncology Authors: Tags: Anti-Tumour Treatment Source Type: research

ConclusionOn the basis of our results, OKCs do not present recurrent hotspot mutations in these 50 genes commonly mutated in cancer. In addition, BRAFV600E does not play a central role in OKC pathogenesis.This article is protected by copyright. All rights reserved.

Source: Journal of Oral Pathology and MedicineCategory: Pathology Authors: Tags: Original Article Source Type: research

CONCLUSIONS: Our study reported the identification of 17 novel candidate genes in a Han Chinese family through WES. ZFHX4 may be a susceptibility gene for NBCCS in Chinese population.
PMID: 29081410 [PubMed – as supplied by publisher]

Source: Cancer BiomarkersCategory: Cancer & Oncology Tags: Cancer Biomark Source Type: research

Hedgehog signaling is highly conserved across species and governs proper embryonic development. Germline gene mutations that reduce this signaling activity cause a variety of developmental abnormalities such as holoprosencephaly, while those that enhance Hedgehog signaling activity induce a tumor‐predisposition condition Nevoid basal cell carcinoma syndrome. Furthermore, dysregulated activation of Hedgehog signaling has been recognized in various sporadic malignancies, including pancreatic adenocarcinoma. Pancreatic adenocarcinoma develops through a multistep carcinogenesis starting with oncogenic mutation of th…

Source: Pathology InternationalCategory: Pathology Authors: Tags: Review Article Source Type: research

The etiology of most childhood cancer remains largely unknown, but is likely attributable to random or induced genetic aberrations in somatic tissue. However, a subset of children develops cancer in the setting of an underlying inheritable condition involving a germline genetic mutation or chromosomal aberration. The term “neurocutaneous syndrome” encompasses a group of multisystem, hereditary disorders that are associated with skin manifestations as well as central and/or peripheral nervous system lesions of variable severity. This review outlines the central nervous system tumors associated with underlying neur…

Source: Journal of Child NeurologyCategory: Neurology Authors: Tags: Original Articles Source Type: research

AbstractNevoid basal cell carcinoma syndrome (NBCCS) is an autosomal dominant disorder characterized by developmental defects and tumorigenesis such as medulloblastomas and basal cell carcinomas, caused by mutations of thepatched-1 (PTCH1) gene. To date, we have detected 73 mutations inPTCH1 and ten of them (14  %) were suspected splicing mutations. Eight out of the ten mutations were localized near the splice donor site. Five mutations were localized within the invariant GT-AG splice site, whereas the other five mutations occurred outside the invariant GT-AG site including the last exonic nucleotide. Whe n the transc…

Source: Familial CancerCategory: Cancer & Oncology Source Type: research

ConclusionAlthough alopecia is a well‐known adverse event associated with the administration of Hh inhibitors, data associated with the appearance and/or clinical severity of alopecia and the treatment efficacy of Hedgehog inhibitors are limited. The Hedgehog pathway plays an important role in the normal cycling of the hair follicles in adults and, therefore, the pathomechanism of Hedgehog inhibitor‐induced alopecia is considered unique for this drug class. Based on the fact that Hh inhibitor resistance is associated with partial reactivation of the Hh pathway, it would not be illogical to suggest that reversal of Hh i…

In conclusion, utilizing genomic sequencing we have detected a novel SUFU mutation in a child with multiple cancers and family history consistent with mutations in the Sonic Hedgehog-Patched-Gli pathway. In the absence of genotype/phenotype specific guidelines, Gorlin syndrome recommendations including intensive brain MRI are being followed and given the paucity of data related to outcome for targeted therapies in children with pathogenic SUFU mutations further investigations are warranted.Citation Format: Michael F. Walsh, Megan Harlan, Jennifer Kennedy, Jacob Musinsky, Michael LaQuaglia, Zsofia Stadler, Stephan Gilheeney…

Source: Cancer ResearchCategory: Cancer & Oncology Authors: Tags: Applied and Clinical Genomics Source Type: research

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