Navigation Links
New drug found effective against rare form of basal cell skin cancer
Date:6/6/2012

New York, NY (June 6, 2012) A clinical study has demonstrated that a new drug, a targeted molecular therapy called vismodegib (trade name Erivedge), can dramatically shrink basal cell skin cancers and prevent the formation of new ones, in patients with basal cell nevus syndrome (BCNS). This rare genetic condition causes dozens, and sometimes hundreds or thousands, of skin cancers on each patient's body. The primary treatment option is surgical removal. These study results are significant as they indicate the possibility of an alternative treatment with oral medication; although side effects remain a consideration.

The phase II clinical study, led by researchers at NewYork-Presbyterian Hospital/Columbia University Medical Center (NYPH/CUMC) and Children's Hospital of Oakland Research Institute (CHORI), was published today in the online edition of the New England Journal of Medicine.

"In its current formulation, vismodegib is appropriate only for BCNS patients with very large numbers of basal cell skin cancers. Still, this is a huge step forward, pointing to the day when we can offer every one of these patients an alternative to repeated surgery, which can be disfiguring and burdensome," said study co-leader David R. Bickers, MD, the Carl Truman Nelson Professor and chairman of dermatology at CUMC and director of dermatology at NewYork-Presbyterian Hospital/CUMC. The study was co-led by Ervin H. Epstein, Jr., MD, a senior scientist at CHORI.

The study is the first to evaluate vismodegib in patients with BCNS. Forty-two patients were randomized to receive either vismodegib (taken orally) or a placebo, for a maximum of 18 months. Overall, the study tracked more than 2,000 existing surgically eligible basal cell skin cancers (SEBs) and documented 694 new SEBs, on the 42 patients.

Patients taking vismodegib experienced an average of 2.3 new SEBs, compared with 29 for patients in the placebo group. Among patients taking the drug, the diameter of clinically significant skin cancers decreased an average of 65 percent, compared with 11 percent among controls. In light of these findings, the independent data and safety monitoring board appointed to oversee this trial recommended switching all patients into the treatment group.

"In many patients, we observed a dramatic reduction in the size of the lesions within one to two months," said Dr. Bickers.

BCNS, also called Gorlin syndrome, encompasses multiple defects that involve the skin, nervous system, eyes, endocrine glands, and bones. The hallmark of BCNS is the appearance of basal cell carcinomas, a slow-growing form of skin cancer, at or around puberty.

BCNS has been linked to mutations in a gene called PTCH1. PTCH1 is the primary inhibitor of a signaling pathway called sonic hedgehog, which helps ensure proper segmentation of the developing embryo. At birth, PTCH1 activity causes most sonic hedgehog signaling to cease. When PTCH1 is mutated, however, sonic hedgehog signaling continues postnatally. The result can be abnormal cell growth and proliferation, setting the stage for tumor formation.

Vismodegib was designed specifically to achieve targeted inhibition of the sonic hedgehog pathway, which is implicated in many types of cancer. The drug is manufactured by Genentech, Inc., of South San Francisco, CA.

Earlier clinical trials found vismodegib to be an effective treatment for metastatic and recurrent locally advanced basal cell skin cancer. On January 30, 2012, the Food and Drug Administration approved vismodegib for use in adults with these conditions who cannot be treated with surgery or radiation.

"Vismodegib is a new treatment option for those with extensive disease, particularly those whose lives may be devastated by the disease and the need for repeated treatment," Dr. Bickers said. However he would not recommend vismodegib for all BCNS patients. The side effects, which include loss of taste and appetite, weight loss, muscle cramps and hair loss, occur in nearly half of patients. Though not life threatening, they can be debilitating. Since patients would have to take vismodegib indefinitely, it is not a suitable alternative to surgery for patients with sporadic basal cell cancers.

"The purpose of the study was to see whether, by using this targeted molecular therapy, we could match the performance of a surgeon, and in many ways, we could," Dr. Bickers added. "The challenge now is to see if we can lessen the adverse effects while achieving the same therapeutic benefits by modifying the dosing schedule or perhaps by alternating drug treatment with other modalities such as photodynamic therapy, which can be effective for smaller lesions."

This is one of the first human cancer therapies based on determining the genetic mutations in a particular tumor pathway (in this case, sonic hedgehog) and inhibiting the pathway directly. The findings may have broader relevance to treating other types of basal cell skin cancer (the most common form of cancer in adults), which are caused by similar dysregulation of the sonic hedgehog pathway.


'/>"/>

Contact: Elizabeth Streich
estreich@columbia.edu
212-305-3689
Columbia University Medical Center
Source:Eurekalert

Related medicine news :

1. One-third of antimalarial medicines sampled in 3 African nations found to be substandard
2. Michael J. Fox Foundation Awards $1 Million to Drive Critical New Research Tools and Technologies in Parkinsons Drug Development
3. Highmark Foundation Awards $120,000 to the American Heart Association
4. Stuttering Foundation Hails New Research
5. Autism Science Foundation Offers IMFAR Grants to Autism Stakeholders
6. Alzheimers Foundation of America Applauds Social Security for Speeding Disability Benefits for Early-Onset Alzheimers Disease
7. Corbett Accel Healthcare Group Mourns Passing of Founder Frank J. Corbett
8. Reliv Foundation Donates $450,000 In Nutritional Supplements to Haitian Relief Efforts
9. Kaiser Foundation Health Plan and Hospitals Report Fourth Quarter and Fiscal Year 2009 Financial Results
10. Instaflex Announces Proceeds Agreement with Arthritis Foundation
11. AMERICAN IDOL to Benefit the UN Foundation Fund for Haiti Thursday, February 25, on FOX
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/20/2017)... MT (PRWEB) , ... January 20, 2017 , ... Spectrum Aquatics Launches New ADA Portable ... saving deck space. Since the lift is mounted on wheels, it can be wheeled out ... lock down kit, to fasten to the deck. "We have transformed the feedback from customers ...
(Date:1/20/2017)... ... January 20, 2017 , ... Doctor C LLC, a company based ... ECRM trade show to continue the marketing and distribution of its product, The Right ... providing 400 percent better absorption than traditional vitamin C supplements. At the trade show, ...
(Date:1/20/2017)... ... 2017 , ... International Protein, a company based out of Australia that focuses ... January ECRM trade show in Hilton Head, SC. , International Protein was founded ... a line of products that would elevate her fitness regime. At this ECRM trade ...
(Date:1/20/2017)... , ... January 20, 2017 , ... “Mary Magdalene: Grace ... mysterious life of the woman who witnessed Jesus Christ firsthand. “Mary Magdalene: Grace is ... who spent her career as an educator interacting with countless women who had little ...
(Date:1/20/2017)... (PRWEB) , ... January 20, 2017 , ... ... vPEP ® Oscillating Positive Expiratory Pressure (OPEP) device, was featured in a study ... Doug Pursley, MEd, RRT-ACCS, FAARC, “Analysis of Three Oscillating Positive Expiratory Pressure ...
Breaking Medicine News(10 mins):
(Date:1/21/2017)... , Jan. 20, 2017  Faruqi & Faruqi, LLP, ... Inc. ("KemPharm" or the "Company") (NASDAQ: KMPH ) ... Company and certain officers and directors and underwriters of the ... seek the role of lead plaintiff. The lawsuit ... District Court for Johnson County on ...
(Date:1/20/2017)... Research and Markets has announced the addition of ... OTC Amplifiers, Diagnostic Instruments), Sales Volume, Company Analysis and Forecast ... ... Sales Volume, Company Analysis and Forecast to 2022 provides a ... The growing prevalence of hearing impairment coupled with an ...
(Date:1/20/2017)... -- Stock-Callers.com today evaluates the following Drug Manufacturers ... Nordisk A/S (NYSE: NVO ), Sucampo Pharmaceuticals Inc. ... (NASDAQ: PTX ). These stocks belong to the ... 2017, finishing near its session lows. As per a NASDAQ ... shares of health care companies in the S&P 500 also ...
Breaking Medicine Technology: