Gene therapy is being tested in the US, but very carefully because of some well-publicized adverse reactions.

Markus mentions the case of a teenager with a rare genetic disorder who received an experimental gene therapy at the University of Pennsylvania in 1999 and died four days later. In 2002 a worldwide clinical trial of gene therapy for children with "bubble boy" disease (born without a functioning immune system) ended after a boy in France developed an illness similar to leukemia; two other French children came down with the same ailment over the next three years, and one died.

On a happier note, scientists from the National Cancer Institute reported last September of using gene therapy to successfully treat advanced melanoma in two patients (though the treatment failed in 15 other patients in the study).

For those of you with access to Lexis/Nexis or Factiva, an article in the Los Angeles Times on Aug. 28, 2006, provided a thorough overview of gene therapy. A short sidebar noted the promise of gene therapy in treating cancer and cited Advexin, a drug using the same p53 gene that is in Gendicine. Advexin was developed by Introgen Therapeutics of Austin, Texas -- the same company that says the Chinese developers of Gendicine basically stole its technology and rushed it to market (an allegation the Chinese deny).

Introgen is working with Colgate-Palmolive to incorporate gene therapy into mouthwashes and gels to treat or prevent oral cancer, the sidebar says. (MD Anderson is currently conducting a clinical trial of a p53 oral rinse for people with dysplasia or SCC in situ.)

If you go to the search engine for the main OCF site (just click on the Search link at the bottom of these pages) and search for Advexin, you'll get five pages of results. According to information provided by the company, "the FDA has selected Advexin for its fast track program to fill an unmet medical need and has designated Advexin for orphan drug use for recurrent head and neck cancer."

So, Kim, things may be moving more slowly than we all would like, but there is certainly no lack of interest by US researchers in gene therapy.

-- Leslie


Leslie

April 2006: Husband dx by dentist with leukoplakia on tongue. Oral surgeon's biopsy 4/28/06: Moderate dysplasia; pathology report warned of possible "skip effect." ENT's excisional biopsy (got it all) 5/31/06: SCC in situ/small bit superficially invasive. Early detection saves lives.