TARGETED CYTOTOXIC GENE THERAPY FOR HUMAN PAPILLOMAVIRUS INFECTION

Human papillomavirus (HPV) is the most common sexually transmitted infection in the world, particularly among young adults. As many as 24 to 40 million men and women are infected with HPV in the U.S. alone and 0.5 to 1 million new HPV infections occur each year. HPV infects the epithelial surfaces of skin or mucosa leading to genital warts and squamous epithelial lesions. A persistent HPV infection can lead to cervical cancer, the leading cause of cancer-related deaths in women worldwide. At least 80% of squamous cell carcinomas of the cervix and vagina harbor HPV, with type 16 being the most common. Current treatments for HPV-associated lesions rely on painful removal of the HPV-infected tissue and do not treat the infection per se. Thus, there is an urgent need for developing treatment strategies for HPV infections that would prevent its progression to cancer. The researchers at UCSF have designed a novel HPV-specific cytotoxic gene therapy vector for the treatment of HPV-related condylomas or dysplasias. Using a specific promoter that responds to a HPV-specific transcription factor, our investigators demonstrated that in cell culture this method specifically eliminates cells expressing early HPV genes such as HPV16, 18,11 and/or 6. This treatment strategy has minimal toxic effects on HPV-negative cells and does not require an immune response. Therefore, this treatment would be particularly beneficial for immunocompromised HIV+ individuals.

This approach represents an important therapeutic advance for the treatment of cervical and anal intraepithelial lesions, and would be particularly useful for the treatment of pre-cancerous lesions before they progress to invasive cancers. An efficient delivery system would allow elimination of the reservoir of HPV from infected individuals, preventing recurrence. The optimization of gene delivery methods such as topical targeting using liposomes as well as testing for the safety and efficacy in animal models is underway and can be accomplished in routine experimentation.

If you would like to receive further information about this technology and potential licensing opportunities, please contact:

Sunita Rajdev, Ph.D.
Licensing Officer
(415) 353-4470 phone
(415) 348-1579 fax
Sunita Rajdev

Reference: OTM Case #SF00-047