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Targeted Medicine: Healthcare's Next Big Thing


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http://www.fool.com/investing/high-grow ... thing.aspx


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On the surface, it seems crazy: Eliminate a portion of the patient population as potential customers for a drug.

But scratch just below the surface, and you'll see that drug makers are crazy like a fox. By targeting a smaller patient population made up of people more likely to benefit from a given drug, drugmakers can both increase the likelihood of getting a drug onto the market, and sell it for a higher price.

Individuals aren't averages

When companies report clinical trial data, we typically get the average response for the entire patient group. However, within that group there's usually a wide range of responses, from "did really well" to "sort of did better." If the drug is the only one available, then that range doesn't really matter -- after all, for those at the low end of responses, some response is better than no response.

But as the number of drugs that can treat a given disease begins to grow, it becomes increasingly difficult to beat what's out there. In other words, how can the new drugs differentiate themselves? One way to do this is to just target a subset of those with the disease. Then the drugmaker might be able to show efficacy that's good enough to get the drug on the market.

Building a better mousetrap

This is already being done to some extent. Perhaps the most famous of all targeted therapeutics is Genentech's (NYSE: DNA) Herceptin, which inhibits the HER2 receptor. Unfortunately, there are some breast cancers that don't have high levels of the HER2 protein, and consequently don't respond to the drug. By testing for HER2 overexpression before administering treatment, doctors can limit the patient population to just those who will respond to the drug.

Here's another example: Rule Breakers pick Exelixis (Nasdaq: EXEL) is testing its oncology compound, XL647, in women or Asians or non-smokers who have lung cancer. Seems like an odd combination, but XL647 is an inhibitor of the EGF receptor (a protein that helps the tumor grow), and sensitivity to EGF receptor inhibitors has been linked to mutations found in those classes of patients. By limiting the population, Exelixis is increasing the chances for the drug to work well enough to compete against drugs like Genentech's Avastin or ImClone Systems' (Nasdaq: IMCL) and Bristol-Myers Squibb's (NYSE: BMY) Erbitux, if the latter is approved to treat lung cancer.

Bystander growth

It's not just drug companies that will benefit from targeted medicine. Diagnostic test makers will, too, because patients will need to be tested to determine whether the drug is right for them. Insurance companies should be willing to shell out for the test because the testing could save big bucks by avoiding payment for drugs that don't work.

For instance, Invitrogen (Nasdaq: IVGN) has a new test that's able to detect HER2 expression in order to tell whether Herceptin will be effective in fighting the cancer.

These tests don't just check to see if a drug works, they can also test to see if a drug is likely to cause adverse reactions. For instance, Myriad Genetics (Nasdaq: MYGN) has a test to determine whether patients will have an adverse reaction to the chemotherapy treatment 5-FU/capecitabine. Another example in this class is patients' sensitivities to blood thinner warfarin, for which the FDA now recommends testing.

Targeting better returns

How can investors get in on the new era of targeted or personalized medicine? Keep a keen eye out for drugs in pipelines that target smaller groups of patients. That may look odd, but the drugs are likely to do better than me-too drugs that target larger groups, while not working any better than what's currently available.

Complementing that with a well-selected group of diagnostic test makers could result in some seriously high returns.

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(The Motley Fool, By Brian Orelli, August 1. 2008)


The information contained in these articles may or may not be in agreement with my own opinions. They are not posted as medical advice of any kind.

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Thanks for the article.

This article supports the development of bio marker testing. Are current FDA approval processes and standards keeping up with developments in this field? I am at a specially designated cancer care center and there has been no mention of screening for bio markers to facilitate treatment matching. Seems this mutation testing should already be best practices and not just available to those with big bucks.

During the height of AIDS activism we had focus groups developing expertise, monitoring progress, and pushing on specific areas of treatments. This included the anti-retrovirals, but also the viral load testing and other bio markers. Seems we need the equivalent in lung cancer....we may already have it. If we do, how we doing in the bio marker testing area?

AIDS activists worked closely with the pharmaceutical companies and bio marker development companies, often in concert pushing the FDA for faster approval and access. Activists also worked on pricing issues so that new drugs, bio markers would be

reasonably priced. Mixed results on the pricing issues.

Thanks, Hank

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During the height of AIDS activism we had focus groups developing expertise, monitoring progress, and pushing on specific areas of treatments.

Yes, Hank, that is exactly what is needed. There is a lack, or so it would seem, of activism in the area of lung cancer and bio marker use.

We are at a cancer center and I have yet to hear anything about bio markers for cancer being a pretreatment procedure.

Maybe I missed something, or maybe it is used for those with deeper pockets?

When HIV/AIDS was being ignored, there was a national and communal outcry. There was also much media attention to the eventual change around in attitude. I remember when all of that occurred (in the mid-eighties).

To have bio markers to evaluate effectiveness of treatment in place for everyone with cancer would be a change in thinking.

That would need initiation from the medical community and the heath insurance providers. Since it is a matter of money, that may be the stickler.

Coming to the realization that to use drugs/medications for those for whom they would work actually would save money, and negate useless regimens for those who are treated with them.

Change does not occur easily in our healthcare system.


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I understand and agree with the underlying theories supporting more specific targeting, but when the actions of the pharmaceuticals are based on their first priority of making megabucks, I have to wonder how many of us are dying for their dollars.

This is along the same lines as their developing a new drug that might work as 2nd line chemo, but they will only test it (clinical trials) as a first line. In the meantime, those of us who could possibly stay alive if it were available as a 2nd line are dying so that the pharmaceuticals can go for larger market. :(


PS This is another aspect of our nation's obeisance to the almighty dollar that I hope to address upon my return (See http://www.lungevity.org/l_community/viewtopic.php?p=367076#367076 for more about Carole the Good Time Girl)

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I also worry about the tests themselves. What if they are not accurate?

Someone might miss out on something that might be of some benefit.

There is an awful lot to do in that area of medicine.

Recently, when I came back from my brother's funeral, Bill and I took our family to a long-time favorite Italian restaurant. The owner, a young woman (around early fifties) came in to tell Bill and me that she was just diagnosed with lung cancer.

She didn't know what kind. That just floors me. Doesn't want to know what kind? She said it was the kind that is "definitely related to smoking" (SCLC?). I don't know.

In the past, over the last few years, when we were discussing Bill's lung cancer, she said that she believed "they were not coming up with a cure in order to keep the money shovelling into the system."

I would hate to think that there is any truth in that.


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  • 2 weeks later...
she believed "they were not coming up with a cure in order to keep the money shovelling into the system."

In my Hi, Barb.

In my not at all humble opinion, that's about the stupidest thing I've ever heard (although not knowing what kind of lung cancer you have also ranks right up there :().

My own thinking has more to do with the fact that the pharmaceuticals are loaded with megabucks, leaving no explanation as to why they can't run limitless clinical trials for these new drugs.

I realize that they have to answer to the FDA to a certain extent, but that doesn't explain why I can't be given a drug that may have a 78% chance of slowing or stopping progression of my cancer according to their earlier tests.

And then we have the FDA telling me the pharmaceuticals can't give it to me because it might kill me (based on fact that the pharmaceuticals haven't proved it mightn't) when I'm dying already! Arrrrggggh!

Affectionately and with my sick sense of humor still intact,


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Hi Carole,

BTW, I don't think you have a sick sense of humor. To me :roll: , it's quite normal.

The woman who told me that gem lost a waitress at the restaurant last year due to breast cancer that had metastasized to her lung after 20 plus years of thinking she had been cured. I think there is a lot of depression going on in her thinking.

After a 30-year "war" having been declared on cancer (during the Nixon Administration), it's not unusual for people to wonder and become jaded.

She did tell me that she had the lung cancer "heavily linked to smoking." She was probably talking about SCLC. She is very scared, and that is understandable.

People face this disease in any number of ways - sometimes, even stupidly. Wisdom isn't always in the forefront when someone is frightened. She probably has no support in place.

Your assessment of whether or not a chemotherapy drug is allowed via the FDA is exactly as is mine.

Let us hope the "powers that be" will change their minds about that in future. I hope so.

Love you (and whatever you do, Carole, don't stop the humor),


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