Profile of vismodegib and its potential in the treatment of advanced basal cell carcinoma

Muzafar A Macha1, Surinder K Batra, Apar Kishor Ganti

  • 1Department of Biochemistry and Molecular Biology, University of Nebraska Medical Center, Omaha, NE, USA.

Insights

Basal cell carcinoma (BCC), the most common human cancer, is now treatable with vismodegib, a targeted therapy. This drug inhibits the mutated hedgehog pathway crucial for BCC development, showing significant patient responses.

Area of Science:

  • Oncology
  • Dermatology
  • Molecular Biology

Background:

  • Basal cell carcinoma (BCC) is the most prevalent human malignancy.
  • Understanding BCC's molecular pathogenesis has enabled targeted therapy development.
  • The hedgehog signaling pathway is frequently altered in BCC.

Purpose of the Study:

  • To review the role of the hedgehog pathway in BCC development.
  • To detail the pharmacology and clinical activity of vismodegib.
  • To discuss vismodegib as a targeted therapy for advanced BCC.

Main Methods:

  • Literature review of hedgehog pathway in BCC.
  • Analysis of clinical trial data for vismodegib.
  • Pharmacological review of vismodegib's mechanism of action.

Main Results:

  • Hedgehog pathway mutations are nearly universal in BCC.
  • Vismodegib, a hedgehog pathway inhibitor, demonstrates significant efficacy.
  • Targeted therapy offers a new treatment modality for advanced BCC.

Conclusions:

  • The hedgehog pathway is a critical driver of BCC.
  • Vismodegib represents a successful targeted therapy for advanced BCC.
  • Further research into pathway-specific treatments is warranted.

Related Concept Videos

Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Skin Cancer01:30

Skin Cancer

Skin cancer is a type of cancer that occurs when there is an abnormal growth of skin cells, usually triggered by damage to the DNA within the skin cells. It is primarily caused by exposure to ultraviolet (UV) radiation from the sun or artificial sources like tanning beds. Skin cancer is the most common type of cancer worldwide, and its incidence continues to rise.
Basal Cell Carcinoma (BCC): BCC is the most common type of skin cancer, accounting for about 80% of cases. It typically develops in...
Mitogens and the Cell Cycle02:38

Mitogens and the Cell Cycle

Mitogens and their receptors play a crucial role in controlling the progression of the cell cycle. However, the loss of mitogenic control over cell division leads to tumor formation. Therefore, mitogens and mitogen receptors play an important role in cancer research. For instance, the epidermal growth factor (EGF) - a type of mitogen and its transmembrane receptor (EGFR), decides the fate of the cell's proliferation. When EGF binds to EGFR, a member of the ErbB family of tyrosine kinase...
Treatment Resistant Cancers02:56

Treatment Resistant Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...