Molecular
Targeted TherapyCecily's Oil
A Pioneering Effort in Targeted Drug Therapy for Lung Cancer
True cutting-edge practitioners are pushing forward with existing drugs ("officially" approved for other illnesses and/or other cancers) that have already been shown to be safe and potentially effective for lung cancer treatment, using them as "make due" drugs for the "cancer cocktail" since they have shown positive results from limited testing in labs and humans ... and their relative safety is well-established.
It is tragic that there are so very few "Targeted Drugs" (ie-Cocktail components) currently approved by the FDA for lung cancer, since it is the number one cancer killer. Of course a major reason for this state of affairs is that lung cancer is quite a virulent disease, and creating and testing such drugs is not an easy task --- however there are also other reasons:
- If a drug approved as safe for another cancer or illness is demonstrated to have a potential effect on lung cancer, it will still take a period of years and years of testing before the FDA will "officially" approve it for that type of cancer. Although legally such drugs can be used by any doctor "off label", few are willing to risk their careers by doing so.
-When a new drug is about to undergo testing, the drug manufacturer will first explore whether there is an illness that the drug will work with that affects only 200,000 or less Americans . This is defined by the FDA as a "rare disease." Receiving approval for a "rare disease" places the drug under the domain of the "Orphan Drug Act," with all sorts of tax breaks and cash incentives to the manufacturer. Then later, perhaps years later, it can go through approval for other illnesses that it already seemed likely to be useful for. [For legitimate development of drugs for rare diseases, the Orphan Drug Act is a Godsend. But it is abused.]
-Lung Cancer is one of the most difficult cancers to treat. Unless a drug is specifically designed for lung cancer (the profit potential for success in this area is enormous), all things being equal the company will go after the form of cancer that is likely to show the best-looking statistics in test results. This leads to an increase in the company's stock value, and increases the likelihood of FDA approval.
Drugs that were most immediately under consideration for "Cecily's Oil" included many agents that had shown in various trials to be safe and have positive effects on some lung cancers; not all drugs would be administered simultaneously --- a very complex schedule of dosing was worked out for maximum synergy and to avoid any contraindications with Chemotherapy, which would be administered concurrently:
The initial list of candidate cocktail components for "Cecily's Oil":
Thalidomide (officially approved for leprosy)
Targretin (has shown in studies to be effective in some lung cancers)
Tamoxifen (typically for breast cancer, but some lung cancers (usually in female patients) also respond; has also been shown to re-sensitize cancer cells to Platin drugs at high doses a day before, during and after chemo)
Iressa (FDA approved for 3rd line lung cancer use; to have been used by Cecily in the First Line, but NOT taken the day before, during, and after chemo)
COX-2 Inhibitor* (effective in slowing 70% of NSC Lung Cancers. Oral COX-2 inhibitors can be hard on the stomach --- an I.V. COX-2 inhibitor is approved in Europe; it is being studied by the FDA in America.) *although recent research of the COX-2 Inhibitor Vioxx suggests that long-term use of Vioxx for over 12-18 months may increase the risk of heart attack, if a given strain of cancer cells shows vulnerability to COX-2 Inhibitors, months of such use to attempt remission in the case of a deadly illness is none-the-less surely justified.
Honokiol (a non-toxic herbal derivative shown in mouse tests to have powerful anti-tumor properties on many human cancer cell lines)
Gleevec (designed for a form of adult Leukemia; in the lab, it has shown potential for Lung cancer for reasons not fully understood --- the target that it is designed to hit doesn't exist in Lung cancer, but it seems to non-the-less render lung cancer cells more vulnerable to chemo ; it is also now showing enormous possibilities for Ovarian cancer)
Erbitux (showing promise for lung cancer, but not made available to Cecily in time, even after FDA approval)
Avastin (showing promise for lung cancer, but not made available to Cecily in time, even after FDA approval)
Tarceva (was finally approved in November 2004 after many, many years of testing ... and lack of availability to Cecily; it is technically approved only for NSC Lung cancer, but has already shown synergy when cocktailed with Avastin in testing on various cancers. A cocktail of Thalidomide, Avastin, Tarceva and Honokiol may be a potent anti-angiogenesis formula, but has yet to be tried. Tarceva is possibly not to be used on the day before, during and after a Platin drug).
In Cecily's first Targeted Cycle, a Cox-2 Inhibitor, Thalidomide and Tamoxifen were added to the Chemo combination of Gemcitibine + Carboplatin --- not the first chemo choice according to the FDA Standard Protocol, (it is believed that Gemcitibine also acts as a re-sensitizer to cancer cells that have developed an immunity to Carboplatin). This formula put the brakes rapidly on the cancer progression --- yielding a 2/3rd decrease in the rate of acceleration (ie- a home run) in a single 3 week cycle! Iressa (scheduled around chemo), Targretin, and High Dose Tamoxifen (8x normal dosage the day before, during and after chemo, to re-sensitize the cancer cells to platin drugs) were added to "Cecily's Oil" in the next cycle.
Cecily was indeed a Pioneer. And had secretion of fluids collecting beneath the lower lobes of the the lungs been successfully stopped, (cancer cells in fluids are immune to chemo and targeted drugs), who knows what future this new pioneering effort might have held. Cecily unquestionably established its promise; for that the world owes her a great debt of thanks.
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Footnotes:
The only Targeted drugs actually approved for Lung Cancer are Iressa and Tarceva, and they are not approved as first line treatments. However scheduling these drugs to not be used on the day before, during and following chemo should in principle allow them to be used as first line agents.
A biopsy test showed that the cancer cells were likely to be most sensitive to a "triplet" combination of Chemotherapy drugs: triplets are not even listed in the "FDA standard protocol" for lung cancer treatment.
The following are labs that test biopsy samples to guide the Oncologist in helping to determine which drugs show the most promise against the unique cancer cells found in any given patient, and to also rule out with near-certainty which drugs will not work. Unlike the only 4 regimens that are part of the "FDA Standard Protocol" for lung cancer, these labs will test up to 30 different combinations of reagents in an effort to find a "personalized" regimen. Comparatively few Oncologists make use of this process today. Most will likely say that the test is "not accurate" or is not "foolproof."
Testing a tumor sample is very informative in guiding a cutting-edge Oncologist's judgment in trying to send a patient into remission, and for the longest possible time. But most insurance carriers won't pay for the test. And most Oncologists wouldn't have the experience necessary to read and interpret the lab report.
Bath Cancer Research (Bath, England)
Anti-Cancer drugs for lab experimentation purposes can be purchased from:
http://www.sigmaaldrich.com/Area_of_Interest/Life_Science/Cancer_Research.html
by authorized users.
CUTTING-EDGE CANCER INFO
Finding
a 'true' Cutting Edge Oncologist
The
Early Formula for "Cecily's Oil"
The
Need For New Drug Approval Laws
60
Minutes Story: The FDA and Politics
AP:
FDA Silences Internal Critics