Create Account | Sign In: Author or Forum

 
 
News  |  Journals  |  Conferences  |  Blogs  |  Articles  |  Forums  |  Twitter    
 

 Headlines:

 

Category: Oncology | Pharmacy | Research | News

Back to Health News

New Drug Effective for Rare Genetic Skin Cancer: Studies

Last Updated: June 06, 2012.

 

In early research, fewer tumors developed in patients with basal cell nevus syndrome

Share |

Comments: (0)

Tell-a-Friend

 

  Related
 
In early research, fewer tumors developed in patients with basal cell nevus syndrome.

By Barbara Bronson Gray
HealthDay Reporter

WEDNESDAY, June 6 (HealthDay News) -- When a clinical trial is stopped abruptly just eight months after its start, it's either very good or very bad news.

In the case of a study on a skin cancer drug, the results were so impressive that the trial's independent data and safety monitoring board decided to offer the drug immediately to the study participants who were taking placebos.

The drug, vismodegib (Erivedge), was approved in January by the U.S. Food and Drug Administration for people with aggressive, large basal cell carcinoma that had spread to the lymph nodes or other body systems. The researchers wanted to test the oral medication against a disfiguring form of skin cancer called basal cell nevus syndrome, a rare genetic condition.

Researchers followed 41 patients with basal cell nevus syndrome and found that those taking vismodegib got an average of slightly more than two new cancers, while those not taking the drug developed 29.

The study was reported in the June 7 New England Journal of Medicine.

"This is one of the first clinical trials that show the drug can be used in prevention," said Dr. Jean Tang, a co-author of the study and assistant professor in the department of dermatology at Stanford University School of Medicine.

Tang said the positive effects of the drug are visible within a month after starting to take it. "This is a life-changing drug for these patients," she said.

Basal cell nevus syndrome typically starts at puberty. The condition can involve hundreds of basal cell lesions, often requiring many surgical and nonsurgical procedures to treat.

Basal cell carcinoma of the skin is the most common cancer worldwide, and its prevalence is increasing. There are about 2.1 million new cases of non-melanoma skin cancer treated in the United States every year, according to journal background information. Some 750,000 of these are cases of basal cell carcinoma. Basal cell nevus syndrome -- also known as Gorlin syndrome-- occurs in fewer than 1 percent of them.

The study tracked more than 2,000 existing basal cell skin cancers and documented 694 new lesions in the study group.

The research also provides new evidence about a key genetic pathway in the development of basal cell and other cancers. Vismodegib targets what is called the "hedgehog-signaling pathway." (It was named after mutant mouse embryos in early studies that looked like hedgehogs.)

The pathway directs cell growth in embryos and also regulates adult stem cells involved in maintaining and regenerating tissue. If the pathway malfunctions, it can result in basal cell carcinoma and other cancers.

"The research trial demonstrates proof of the impact of the hedgehog-signaling pathway in basal cell cancers," Tang said. The findings may have broader relevance to treating other types of basal cell skin cancer, she said.

The drug's side effects, including muscle cramps, changes in taste perception, weight loss, hair loss and fatigue, can be debilitating. In order to reduce their impact, the researchers are testing whether dosing the drug intermittently -- two months on, two months off -- will reduce the symptoms while still being at least 90 percent effective. They're also testing reduced dosages to better understand how to strike the right balance, Tang said.

At this point, vismodegib costs $250 a day, Tang said. The drug's maker, Genentech, contributed nearly $1 million to support the research, including the costs of patient travel, office visits and biopsies, she said.

A second, related study in the same journal issue found that some patients responded to vismodegib for locally advanced and metastatic basal cell carcinomas. This study also received funding from Genentech.

Dr. John Lear, a consultant dermatologist at the Manchester Royal Infirmary in England, wrote an editorial on the studies. "It is a landmark day for patients with basal cell carcinoma," he said in an interview. "The next step is to develop topical applications and injections that could minimize side effects while effectively preventing and treating lesions."

More information

To learn more about basal cell nevus syndrome, visit the U.S. National Library of Medicine.

SOURCES: Jean Tang, M.D., assistant professor, department of dermatology, Stanford University School of Medicine; John Lear, M.D., consultant dermatologist, Manchester Royal Infirmary, Manchester, England; June 7, 2012, New England Journal of Medicine

Copyright © 2012 HealthDay. All rights reserved.


Previous: Exercise Appears to Ease Nerve-Damage Pain in Rat Study Next: New Medicine Might Fight Drug-Resistant TB, Study Says

Reader comments on this article are listed below. Review our comments policy.


Submit your opinion:

Name:

Email:

Location:

URL:

Remember my personal information

Notify me of follow-up comments?

advertisement.gif (61x7 -- 0 bytes)
 

Are you a Doctor, Pharmacist, PA or a Nurse?

Join the Doctors Lounge online medical community

  • Editorial activities: Publish, peer review, edit online articles.

  • Ask a Doctor Teams: Respond to patient questions and discuss challenging presentations with other members.

Doctors Lounge Membership Application

 
     

 advertisement.gif (61x7 -- 0 bytes)

 

 

Useful Sites
MediLexicon
  Tools & Services: Follow DoctorsLounge on Twitter Follow us on Twitter | RSS News | Newsletter | Contact us
Copyright © 2001-2014
Doctors Lounge.
All rights reserved.

Medical Reference:
Diseases | Symptoms
Drugs | Labs | Procedures
Software | Tutorials

Advertising
Links | Humor
Forum Archive
CME | Conferences

Privacy Statement
Terms & Conditions
Editorial Board
About us | Email

This website is certified by Health On the Net Foundation. Click to verify. This site complies with the HONcode standard for trustworthy health information:
verify here.