News & Events
1. Translational Medicine is vital and needs support. The definition of translational medicine is taking what has been discovered in the lab and moving it rapidly to patients. The hallmark of translational medicine is that it seeks to measure in manner what was measured in the laboratory to assure that the new drug behaves as its designers intended.
2. Translational medicine and scientific research needs our support. Why? In 2000, the NIH funded 1 grant out of 4 applications. In 2007, the NIH cut funding substantially and now funds 1 grant out of 12 applications. Clinical research has historically been under funded and this has only widened the gap.
3. Immunotherapy is using the immune system to fight cancer. The immune system is able to recognize tumors but is unproductive unless re- educated and this can be accomplished using different methods of gene therapy.
4. Tumors grow within an environment, including the stroma and the vasculature. Tumors are moving targets and evasive. However, "the vasculature is not as smart as the tumor," and unwittingly accommodates tumor growth. In the last 2 years the vasculature has been identified as a potential "achilles heel" of the tumor and can be targeted by new therapies for controlling and preventing tumor growth.
5. Gene therapy is a mechanism to introduce new controls ("switches") into the tumor. The understanding of which "switches" to use is the subject of current research; the switches may be proteins, an antibody or a modified leukocyte to activate the immune system to control the tumors.
6. Clinical trials using this methodology are underway in treating both breast cancer, including its 6 different subtypes, and ovarian cancer. To access information about which gene therapy trials are currently completed, active or are recruiting patients, one should go to About cancer gene therapy, Clinical trials.
There are currently about 485 gene therapy trials for all types of cancer. Clinical trials are conducted within strict regulatory guidance of the FDA.
Phase I - Safety, 15-40 patients to determine the maximum safe dosage of a drug. Interestingly, most drugs are most effective at less than the maximum toleration amount.
Phase II - 40-400 patients (sometimes randomized) and addresses primarily whether the drug will work as well as provide additional safety data.
Phase III - 100 -1000 patients. Effectiveness and safety, with comparison to the current standards of care.
7. Gene therapy is improving patient's lives now. There are now many patients who are living longer because of treatment with gene therapy.
8. The understanding of key molecular targets, using gene therapy, that are expressed on tumors will eventually lead to prevention, such as the gardasil vaccine which targets a very specific HPV virus.
9. Gene therapy, especially, immunotherapy is a reality.
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