Gene Therapy

FDA 101: Human Gene Therapy,” Consumer Update, U.S. Food and Drug Administration (FDA), February 26, 2008.

What is gene therapy?” Genetics Home Reference, Your Guide to Understanding Genetic Conditions, National Institutes of Health, April 14, 2008.

Human Gene Therapies: Novel Product Development Q&A with Celia M. Witten, Ph.D, M.D.,” Consumer Update, U.S. Food and Drug Administration (FDA), October 15, 2007.

Genes – What We Knew, Know, and Hope to Learn,” National Institute of General Medicine Sciences (part of the National Institutes of Health), November 2006.

From Genes To Personalized Medicine,” National Institutes of Health Fact Sheet (PDF Document Download).

Cellular & Gene Therapy,” Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA), July 26, 2007.

Curing Cancer With Viruses,”, May 11, 2008.

NIH Announces New Initiative in Epigenomics,” National Institutes of Health News (part of the National Department of Health and Human Resources), January 22, 2008.

Epigenomics – A Scientific Illustration of How Epigenetic Mechanisms Can Affect Health,” Office of Portfolio Analysis and Strategic Initiatives (OPASI), National Institutes of Health (NIH), January 24, 2008.

Epigenetics – A Tale of Two Mice,” NOVA ScienceNow, PBS, July 2007.

An Introduction to RNA Interference,” Science & Technology, Sirna Therapeutics.

An Animation of RNA Interference in Action,”, 2006.

Liposomal siRNA – Genetic On/Off Switches That Target Ovarian Cancer Through the Trojan Horse Effect,” posted by Paul Cacciatore, H*O*P*E* Blog, March 25, 2008.

Resources and Links,” W.M. Keck Center for Innovative Cancer Therapies, M. D. Anderson Cancer Center, the University of Texas.

2 thoughts on “Gene Therapy

  1. My name is Susana Marques, I’m 39 years old, and i live in Portugal.
    I had a surgery for ovarian cancer clear cell last April, 2009, was diagnosed stage Ic, and had 6 cycles of carbo taxol. 3 months after finishing the last cycle of
    quemotherapies I have a recurrence of the disease with a 1,5 cm node and
    two 0,6 in the perithonial area.
    As clear cell is a kind of cancer resistant to quemotherapies, I’m looking for new therapies.

    Do you have any information on in China, the medicine Gendicine and gene therapies?

    Also askimg about your opinion on a vacine treatment by Dr. robert Gorter in Cologne, Germany

    Hope to hear from you soon.
    Kind Regards,
    Andreia Marques


    • Dear Susana,

      I am so sorry to hear about your ovarian clear cell cancer (OCCC)) recurrence. Our thoughts and prayers are with you. You are absolutely correct that clear cell ovarian cancer is quite chemoresistant. I will address the two therapies listed by you and cover several additional therapies being used in the U.S.

      Gendicine: As you know, Gendicine is a recombinant adenovirus containing the tumour-suppressor gene p53. The thought is that many cancers have a defective p53 gene. Gendicine uses a virus to infiltrate the cancer cell and provide the cell with a fully functioning p53 gene, which acts to suppress the cancer. Western researchers, however, are concerned about how the drug was tested. In the only English-speaking article published containing clinical trial data, Gendicine combined with radiotherapy showed a threefold effect in complete tumour regression compared with a group on radiotherapy alone (Peng, Z.Hum. Gene Ther. 16, 1016–1027; 2005). The data in that study came from 120 patients, which is far less than for a rigorous Phase III trial in Europe or the United States. I could not find any documented cases of its use against ovarian cancer, regardless of subtype (click here for 2006 Gendicine article). M.D. Anderson Cancer Center tested a similar drug, known as Advexin, and seemed to have similar results to Gendicine against head and neck cancers.

      For a list of clinical trials testing gene therapy against ovarian cancer, click here. Unfortunately, none of these clinical trials are open.

      Cologne Model of Immunotherapy: I am aware of the dendritic cell vaccine created by Dr. Robert Gorter. Dendritic cell vaccines also exist in the U.S. For a list of open ovarian cancer vaccine clinical trials, click here. My concern regarding use of a vaccine for you is that such treatments tend to work better with low tumor burden or to prevent a recurrence. I am not sure that a single vaccine therapy will be potent enough for a patient with recurrent, refractory clear cell ovarian cancer.

      Pathology Testing: You should consider having your tumor sample tested for the following: HER-2 positivity, estrogen positivity, PIK3CA gene mutations (possibly through M.D. Anderson — see below), and percentage of tumor that represents “clear cell” classification or histology (e.g., pure clear cell tumor, or mixed clear cell tumor, etc. to determine what percentage of the tumor is clear cell). If your tumor tests positive for HER-2, it may be possible to use the drug Herceptin. Please note that most OCCC is HER-2 negative. If your tumor is estrogen positive, it may be possible to incorporate anti-estrogen therapies in your treatment. See Working Smarter, Not Harder: Use of Anti-Estrogen Therapy to Battle Recurrent Ovarian Cancer.

      Secondary Debulking Surgery & Intraperitonal Chemotherapy: Surgery is the best therapy for ovarian cancer assuming it can rid your body of all visible signs of cancer. I do not know if your 1.5 cm node can be removed, but ask your doctor anyway. Also, there is a form of intraperitoneal chemotherapy in the U.S. called “HIPEC” (Hyperthermic Intraperitoneal Chemotherapy). Click here to view a 2009 study.

      Dose Dense Chemotherapy: If additional intravenous (IV) chemotherapy is required consider dose dense therapy. See Extended weekly dose-dense paclitaxel/carboplatin is feasible and active in heavily pre-treated platinum-resistant recurrent ovarian cancer & Modified Chemo Regime Increases Survival In Advanced Ovarian Cancer Patients But Adds Toxicity.

      Dasatinib (Sprycel) Clinical Trials: It appears that the U.S. Food & Drug Administration-approved drug dasatinib (Sprycel) may be effective against OCCC. See the Libby’s H*O*P*E* post entitled, UCLA Researchers Significantly Inhibit Growth of Ovarian Cancer Cell Lines With FDA-Approved Leukemia Drug Dasatinib, dated November 11, 2009, which sets forth hyperlinks within the post to all open ovarian cancer & solid tumor clinical trials currently utilizing dasatinib. Simply click on those hyperlinks and print out the clinical trial summaries for your friend. I believe that there are three open ovarian cancer clinical trials utilizing dasatinib. Unfortunately, I do not believe that UCLA has started its dasatinib clinical trial yet, but the other three clinical trials are currently recruiting. Be sure to review the testing location of each trial to determine whether it is convenient for you. You will also have to review the patient inclusion/exclusion criteria for each trial to determine if you can qualify.

      Use of PI3K Inhibitors: Based on studies conducted by UCLA and M.D. Anderson, researchers have found that approximately 20% to 40% of OCCCs possess a mutation in the PIK3CA gene. See Libby’s H*O*P*E* post entitled, PI3K Pathway: A Potential Ovarian Cancer Therapeutic Target?, dated November 20, 2009. I believe that M.D. Anderson may be testing ovarian cancer patients for this mutation. If your tumor sample tests positive for PIK3CA gene mutation, the use of a PI3K-AKT-mTOR pathway inhibitor should be considered. If you scroll down that post, you will find my response to a comment in which I provide M.D. Anderson contact information regarding this issue. Click here to review that comment. Please be aware that my PI3K Pathway post sets forth hyperlinks in reference footnote #2 to open ovarian cancer and solid tumor clinical trials involving PI3K inhibitors.

      Use of Drugs Designed For Clear Cell Renal (Kidney) Cancer: I recently posted a comment on the Ovarian Cancer National Alliance Message Board (at that addresses this issue. Click here to review my response to a clear cell ovarian cancer question. Although you should read my entire reply (if you scroll down the reply string, “libbyshope” appears as the 10th reply), pay particular attention to my discussion regarding this issue as set forth in paragraph #5 of my reply. This concept can also be discussed with your doctor. The concept here is that at least one study has found that clear cell renal cancer and clear cell ovarian cancer bear a remarkable genetic similarity to one another. In short, the study found that cancers of clear cell classification or histology, regardless of body organ of origin, strongly resemble one another. Thus, the use of renal cancer drugs is within the realm of possibility. This is particularly true for a generally aggressive and chemoresistant form of epithelial ovarian cancer such as clear cell.

      Novel Targeted Gene Therapies Use Diphtheria Toxin To Fight Ovarian Cancer: See Libby’s H*O*P*E* post dated August 10, 2009. At least one clinical trial is currently using this therapy.

      General Ovarian Cancer Clear Cell Information: Susana, I have listed many clear cell ovarian cancer studies on the Libby’s H*O*P*E* “Types of Ovarian Cancer” page. Click here to view that page. Once on the page, simply scroll down to the heading “Clear Cell.” It is important for you to understand that each major subtype of epithelial ovarian cancer (i.e., serous, endometrioid, mucinous, clear cell, and undifferentiated) is distinctly different. Ovarian clear cell cancer is generally aggressive and chemoresistant. So, it is important that you find a doctor that will focus on that fact and review materials and potential treatments that relate to clear cell classification.

      Susana, I realize that there is a lot of information provided above. If you have any additional questions, please feel free to contact me.


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