MOLECULAR TARGETED THERAPY --- the "Cancer Cocktail"

A Primer in "Targeted Therapy"

    When it comes to lung cancer, the "LIST OF THE TOP 10 AMERICAN CANCER HOSPITALS" is not necessarily valid --- lung cancer is the "great equalizer" among treatment institutions.

    Many if not most cancer facilities with the finest reputations practice only the basic "FDA Standard Protocol" when it comes to advanced lung cancer (most lung cancers are not discovered until they are already very advanced), offering little treatment beyond a Chemotherapy protocol that still leads to a 75% or greater death rate in advanced lung cancer cases in the first year; reaching 97% within 2 years. The mortality rate beyond 2 years rises so close to 100% with the use of the current "standard protocol" that it cannot be meaningfully calculated. Over 150,000 Americans will die from lung cancer this year, making it the deadliest of all cancers. And lung cancer can strike ANYONE. Only 10% of all lung cancers occur as a direct result of smoking. Current theory suspects that some types of lung cancer may result from a virus.

    With the "standard protocol" so ineffective in giving lung cancer patients much more than extra months, it is hoped that Targeted therapy will help to extend lives by years or to render lung and other cancers treatable, chronic illnesses.

    There are many different kinds of molecular "receptors" (on/off "switches") that reside on the outside of a cancer cell. Some of these switches can cause a cell to spread when turned "on," others can help to shut the cell down.  These switches are controlled by very specific chemicals in the body that bind only to certain receptors --- when a molecular "activator" binds with its designated molecular "receptor," the switch is "on."  When switched on, the  receptors send signals responsible for dividing and spreading the cell --- Targeted drugs "inhibit" this process. Other switches, when turned on, may hinder the process of cancer cells spreading. The goal of Targeted therapy is to design drugs that will "target" specific receptors, binding to the receptor and acting as a permanent safety latch, thus preventing access to the switch --- essentially keeping the switch in a permanent "off" state. With receptors that send a signal to help shut the cancer cell down, the Targeted drug will jam the molecular switch into the "on" state.

above: a cancer cell, with green "receptors" (the switches) protruding from the cell wall. Targeted drugs will will block certain receptors from ever being activated, and others will be targeted to be turned "on" --- this process, it is hoped, will shut down cancer cells when the correct targeted drugs are employed for any given strain of cancer. Today Targeted drugs are hard to get access to, and very expensive.

    Lung cancer cells have lots of switches --- and no single switch is yet known to control the entire fate of a cell. Also, no two cancers are quite alike and the cancers within different people are unique not only by type of cancer strain, but by virtue of which switches are present. And because cancer itself is a mutation, even patients who have the same strain of cancer will none-the-less have different types of switches on the cancer cell walls.  To add to the matter, whether a patient is male or female seems to influence some of the switches that may be present. As a result, in principle Targeted therapy must be uniquely designed for each person. 

    In the actual practice of targeted treatment today, however, it is not feasible to determine exactly which receptors are present in the cancer cells residing in any given patient, nor are there enough different drugs available to cover all the possibilities. But at least the early incarnation of Targeted treatment today is an addition to help chemotherapy battle the cancer and, hopefully, to make it far more manageable ... and possibly non-fatal. It is presumed that when enough targeted drugs exist in the future, chemotherapy will not be necessary at all.

    Today it is like searching for a needle in a haystack to even find a doctor who will risk deviating from the "Standard Protocol," let alone who is self-educated-enough in the current research to effectively practice this treatment; the necessary new knowledge is massive and the bio-chemistry is complex; when Targeted Treatment is finally the standard, today's best Oncologists --- even those also working at the highest research levels --- will likely be required to take time off to attend additional medical school, (even if it is not postured in quite that way at the time). 

    Even more difficult than finding a qualified and willing doctor to offer Targeted treatment, though, is getting access to Targeted drugs. Even as more and more drugs are put into the design phase, the many existing "investigational" Targeted drugs that have shown safety and promise will simply not be made available to doctors and patients for years (outside of "Clinical Trials" being conducted on essentially "already terminal" patients; this in spite of the "Expanded Access Law"  --- most of the time this law doesn't work due to glaring political loopholes). 

    And "Clinical Trials," while vital for future drug development, are not helpful to patients seeking the best possible first-line treatment, since most Trials require a patient to cease taking all other lifesaving drugs in order to participate. These trials are designed primarily to obtain information on the effectiveness of a single new drug (sometimes two combined) using a test subject who is clean of other drugs ---  since previous treatment might result in "tainted" data (by FDA definition), causing the Trial to be unacceptable to the FDA for drug approval. In spite of their value and importance for the future, trials are not fundamentally conducted for the treatment benefit of current patients. 

    Additionally, the very few Targeted drugs starting to become available today are prohibitively expensive for the average person, even with insurance. 

    The cutting-edge cancer doctors today are struggling just to get their hands on the Targeted drugs that seem promising but that are not yet being made available to the general patient population, most frequently turning to drugs that were originally approved for something else, but which have shown to be a possible " make-due" substitute for the Targeted drug needed. A doctor can legally make use of most drugs that are already approved for something else --- this is known as "off-label" use.  Insurance companies will tend to resist covering the costs of "off label" treatment.

    Regardless of how workable drugs are obtained, a cocktail with many Targeted cancer drugs could easily reach an annual retail cost of $100,000 - $200,000, with insurance companies less and less supportive of covering most of the pharmaceutical expense. Eventually the Medical, Insurance and Pharmaceutical systems will have to be re-organized to cope with rapid technological change:  (a) in re-training the front-line practitioners for the 21st Century; (b) in getting promising treatments for fatal diseases out of the lab and into the hands of front-line doctors much, much faster than is currently the case --- today, not in years or months,  and; (c) also in terms of the financial situation. Eventually an acceptable answer for society at large must and will be found. Today it is unaffordable for an average person with an otherwise fatal illness to be treated with Targeted therapy because of cost --- even if a qualified doctor could be located. Immediate planning by all agencies involved is essential to avoid a crisis en-mass of the kind that is today being felt by the pioneers in a system not yet designed for modern treatment options.