Navigation Links
New Drug Effective for Rare Genetic Skin Cancer: Studies
Date:6/6/2012

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©2010 ScoutNews,LLC.
All rights reserved  

Related medicine news :

1. Low forms of cyclin E reduce breast cancer drugs effectiveness
2. Gastric Banding Most Effective for Obese Teens
3. Neuroimaging study may pave way for effective Alzheimers treatments
4. China Cord Blood Corporation Warrant Registration Statement Declared Effective by SEC
5. Charging less for more effective treatments could reduce health care costs while improving health
6. Split-course palliative radiotherapy confirmed as effective treatment for advanced NSCLC
7. New drug for kidney transplant recipients effective in humans
8. Two Sepsis Treatments Look Equally Effective
9. Mammogram Plus MRI Seems Cost-Effective in High-Risk Women
10. Mammogram Plus MRI Cost-Effective in High-Risk Women
11. Study Shows Sani-Bag+ to be an Effective Microbial Barrier
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
New Drug Effective for Rare Genetic Skin Cancer: Studies
(Date:6/25/2016)... ... 25, 2016 , ... The temporary closing of Bruton Memorial Library on June 21 due to ... a new, often overlooked aspect of head lice: the parasite’s ability to live away from ... common occurrence, but a necessary one in the event that lice have simply gotten out ...
(Date:6/25/2016)... ... June 25, 2016 , ... On Friday, June 10, Van Mitchell, Secretary of ... award to iHire in recognition of their exemplary accomplishments in worksite health promotion. , ... Workplace Health & Wellness Symposium at the BWI Marriott in Linthicum Heights. iHire was ...
(Date:6/24/2016)... ... June 24, 2016 , ... Marcy was in a crisis. Her son James, eight, ... at his family verbally and physically. , “When something upset him, he couldn’t control his ... it. He would throw rocks at my other children and say he was going to ...
(Date:6/24/2016)... ... June 24, 2016 , ... Topical BioMedics, Inc, makers of Topricin and MyPainAway Pain Relief Products, ... minimum wage raise to $12 an hour by 2020 and then adjusting it yearly to ... value of the minimum wage, assure the wage floor does not erode again, and make ...
(Date:6/24/2016)... ... 2016 , ... Strategic Capital Partners, LLC (SCP) in concert ... capital for emerging technology companies. SCP has delivered investment events and professional ... than a million dollars of capital investment for five companies. The ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... Calif. , June 24, 2016  American Respiratory Labs (ARL), ... is now able to perform sophisticated lung assessments in patients, homes, ... , Inc. Patients are no longer limited to ... EasyOne PRO ® , ARL patients like Jeanne R. of ... in the comfort of her own home. ...
(Date:6/24/2016)... , June 24, 2016 ... Market by Type (Standard Pen Needles, Safety Pen Needles), ... (Insulin, GLP-1, Growth Hormone), Mode of Purchase (Retail, Non-Retail) ... MarketsandMarkets, This report studies the market for the forecast ... to reach USD 2.81 Billion by 2021 from USD ...
(Date:6/24/2016)... Belgium , June 24, 2016 ... today announced the appointment of Dr. Edward ... as a Non-Executive Director, effective June 23, 2016.Dr. ... Compensation and Nominations and Governance Committees.  As a ... will provide independent expertise and strategic counsel to ...
Breaking Medicine Technology: