Clinical trial or "Gold Standard" treatment: How on earth does one decide?

I have been reading this wonderful sight since April 14th, the day that my dad told me that a mass was found in his lung, and I am finally ready to post.

My 78 year old dad, a long time smoker, went to the ER on April 13th for pain in his midsection and hip area (later determined to be bone mets). A CT scan found a 4 x 5 cm mass in his right lung. A needle biopsy confirmed it and a PET scan showed spreading to his shoulder joint, pelvis and ribs. Stage IV, NSCLC

He has had two visits with oncologists and we have unanimously chosen one, just this past week. He offered the following 2 treatment options to my dad (along with the possibility of radiation for pain relief):
1) the "2009 Gold Standard" treatment of Carboplatin andTaxol +/- Avastin by IV, once every 3 weeks for 3-4 cycles.
2)Clinical trial of Sutent pill, for patients over 70 years of age. My Dad had prostate cancer within the 5 year limit that the study states but his PSA is so negligible that he was accepted into the study regardless.

The oncologist that we selected sent him for a brain scan two days ago to check for brain mets. I was wondering why on earth neither his pulmonologist nor the first oncologist he met with never mentioned it. I hate all of of this time wasting as I feel that time is ticking away. No results from the brain scan yet.

So, now decisions have to be made. How on earth do we know what to do??? My dad is not in the best of shape and is in alot of pain for his bone mets. Obviously, the trial, with its pill administration and older population, is enticing but it is hard to turn away from a treatment referred to as the 'gold standard'.

I am grateful that he was accepted into this trial as it provides him with choices, but then again, having to decide is so, so hard. I feel that time is against us (his dr. would only say that he guesstimates 3-6 months survival time without treatment; with treatment, he said that it is too variable to speculate and that the first 3 months of treatment are vital). So, any advice for a daughter whose head is spinning???

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That is a tough decision. Have you read anything about the assay test you can send in to a Dr Nagourney in Calif? There has been some buzz about it in the past few days. You could send in a sample of your dad's tumor and they can test it against several chemotherapies and find out in the lab what your dad's tumor cells are sensitive to. There was a recent post about this under the discussion "my story" by Ghanbidge.

I have read some about sutent, and I know it is approved for kidney and some type of stomach cancer or some sort of gastrointestinal cancer. Is your dad guaranteed to get sutent or do some of the participants get a placebo? That is an important thing to know.

Good luck with your decision. I am sure you will get some good advice from this site.

Sue M

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Hi, and welcome, so sorry you had to find us, but there are alot of caring, knowledgeable caregivers and surviors here that will hopefully offer you help and suggestions.
My husband who is much younger than your father, age 43, was diagnosed Jan this year. Stage IV NSCLC with mets to his brain. What a shock, and I understand the need to do sometype of treatment ASAP.
Because my husband had mets to his brain the onc sent him for radiation first, agressive 15 to his brain, and 10 to his lung.
2 weeks after radiation he had a PET and the new was good, he only had a little spot light up in his other lung, but the primary tumor which we were told was orginally about the size of a grapefruit, had shrunk buy 50 percent.
Next they started the chemo, with the same drugs they are offering your Dad, he was able to have one infusion, on the 2nd he had a really bad reaction to TAXOL, turned beet red, could not breathe, so that one was out, also shortly after he had bad nosebleeds, even had to be hospitalized for them, you can read my journals about them, so the drug Avastin was then out, because the onc felt it was causing the bleeding, now he is on Gemzar and Carboplatin, having CT scans next week.
With your fathers age, and otherall health I would seriously consider whatever treatment the onc feels would have the least side effects, and gives him the best results. The radiation my husband had made him feel better, when it was over. Not in as much pain, I guess because of the shrinkage, his overall sideeffects were being more tired and achy feeling all over. But his age is on his side. Everyone reacts differently to treatments, some have horrible side effects, others not bad. I would really look into the trial, what is it, and would he get the actual drug or a placebo? I know I have rambled on, but the more you learn, ask questions to all the doctors, and what is most important is keeping your father from not having too much pain, and what quality of life he has left is the best he can have. Good luck with whatever you decide, your family is added to my prayers, Karen

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Conventionally, oncologists rely on clinical trials in choosing chemotherapy regimens (the so-called gold standard). But the statistical results of these population-based studies might not apply to an individual. For many cancers, more than one standard treatment exists. There is rarely a situation where there is only one form of therapy. Physicians select drugs based on their personal experience, possible side effects and the patient's condition, among other factors.

The system is overloaded with drugs and underloaded with wisdom and expertise for using them. As Sue M hinted at, pre-testing might help find the best option, and/or save from fruitless additional treatment. What would be more beneficial is to test pharmacodynamic endpoints with the ability to measure multiple parameters in cellular screens now in hand using flow cytomety. Using a systems biology approach could be vastly more beneficial.

Joining forces with collaborations between surgeons, medical oncologists and radiation oncologists can result in better outcomes for cancer patients. While patients may need any one of a wide array of doctors, each may handle only one aspect of care. Patients need a "team" of doctors, with one to step forward to serve as quarterback, and have easily accessible electronic medical records.

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I have to agree that sending a biopsy to Dr. Nagourney in California might be best, that site is www.rationaltherapeutics.com just click on it, read it and call them, they will explain, insurance does cover it some do and some don't. But it could work best in your decision. If the standard gold dose won't kill his cancer than that will rule that one right out for you, and a decision will be much easier.
Go for it. God Bless you. Sandy

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In choosing which treatment to pursue, I would also ask a lot of questions regarding side effects and their impact on quality of life. At the stage your father has been diagnosed, the aim of treatment is not curative, but rather to ease pain and perhaps prolong life. Traditional chemo is very difficult, and a lot of people have a very hard time with it (being older and in poor health can make it harder as well).
I am currently enrolled in a clinical trial and have found the side effects so much more manageable than the traditional chemo I received (cisplatin/taxotere)--so this is just another thing to consider. Best of luck, Linnea

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Thanks so much for your thoughts and advice. It is great to have the knowledge of others who are experiencing this same fight against the same cancer. I am so intrigued by the Rational Ther. concept. Totally makes sense and makes me wonder why it is still so limited. Has anyone spoken to their oncologists about it? I wonder if they themselves have found it reliable and worth the expense. One thing that puzzles me though...my dad had a needle biopsy already that his insurance covered (at great expense, I am sure). Would an insurance company pay for yet another biopsy solely for this purpose? I read that very few insurances cover the cost of the RT labwork itself. Mmmm...I would LOVE to have this labwork performed for my dad. Maybe I will check and see what Dad's dr. thinks of it and whether its a good thing for my Dad.
Thanks again!

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I would think having additional support of drug patient-specific activity as determined by extensive laboratory pre-tests could very well bolster an argument to the insurance company in seeking payment. It amazes me not only that some private insurance carriers don't like to pay for such tests but that they don't empharically mandate it as a requirement for obtaining chemotherapy reimbursement against "ill-directed" treatments. Many of these drugs cry out for validated clinical biomarkers to help set dosage and select people likely to respond. And it could be vastly more beneficial to measure the net effect of all processes (systems) affecting the cell instead of just identifying individual molecular targets.

The pre-test screen doesn't change tumor biology, it reveals it. It is a reasonably good prognostic indicator for clinical outcomes with different drugs, in a disease where there is an abundance of otherwise equally-effective choices. The tumors of different patients have different responses to chemotherapy.

The cell is a system, an integrated, interacting network of genes, proteins, and other cellular constituents that produce functions. One needs to analyze the systems' response to drug treatments, not just one or a few targets (pathways/mechanisms). There are many pathways/mechanisms to the altered cellular (forest) function, hence all the different "trees" which correlate in different situations. Improvement can be made by measuring what happens at the end (the effects on the forest), rather than the status of the indivudal trees.

You can experiment with trial-and-error treatment or you can take the time to find the most effacious combination of drugs matched to your tumor cells. The evidence supporting a doctor's use of empiric-therapy may be less solid than the evidence supporting assay-directed therapy. If you have serious interest in fighting your cancer logically, have your oncologist call the medical directors at these labs to further identify this protocol, and at the same time, educate him/her about the merits of cell-based assay-directed therapy, what the rationale is and what the data which support what is being considered.

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You are in an impossible situation. There is no right answer for you. I was faced with the same dilemma 16 years ago. The so-called gold standard was not so golden back then so I opted for the clinical trial. It didn't work so I went to the Carboplatin andTaxol (Avastin wasn't invented yet) That didn't work either. We finally tried combinations and luckly found some drugs that did work.
My point is this:
Everyone is different and there is no guarantee that what works for someone else will work for you.
There are many more drugs available and different combinations that may work for you so you need a doctor with an open mind who will keep on trying to help you.
My Onc told me that medicine is as much an art as it is a science, you will need an artist to help you through this maze.
Good Luck to you and your father.

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You are so correct giaccino. Everyone is different and there is no guarantee that what works for someone else will work for you.

A cancer patient has their own unique individuality. A chemo-induced gene mutation can happen when the original chemo received does not work. The cancer comes back. When it does this, the cancer comes back more aggressively.

Primary tumors are highly heterogeneous that each cell within it is likely to have a unique genomic signature at the level of mutation. They will genomically be different to the cells in the primary tumor.

The mutagenic effects (changes in form) of chemotherapy on a genetically-unstable tumor, drives the tumor into a state of more aggressive behavior.

You might kill off a whole lot of cancer, only to cause a mutation in the remaining cancer, such that the remaining cancer behaves in a more aggressive fashion (like when chemotherapy on an ER+ cancer brings about a remission, but in the process also causes any remaining cancer to become ER-).

Cancers that are a product of these genetic mutations release cells from the usual controls of proliferation and survival, making them so much harder to fight it. Following this mutation, the cancer cells acquire the ability to proliferate without the normal restraints.

When the first-line treatment is unsuccessful, the cancer can return in a more dangerous drug resistant form. It would seem more prudent to invest the time in using diagnostic technologies for detecting cancer growths, as well as the properties of cells that are destined to metastasize.

Reference: Cell Function Analysis

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Hi, I faced a similar "fork in the road" decision when I was dx with Stage IV lung cancer and brain mets last Sept. I panicked because I thought I had to start taking anything asap. But fortunately I was able to consult with Dr. Nagourney of Rational Therapeutics immediately, and was convinced that taking another week to have my tumor tested (it's called an EVA-PCD assay test) would at least give me some piece of mind that the treatment we decided on would be the most effective. What no one ever mentions is that even the "gold standard" of chemo is still a guessing game in that all drugs don't always work on everyone the same. The assay test allows all combinations of drugs to be tested on a tumor sample outside of your body to determine which will be the most effective. This can save your dad wasted or difficult chemo treatments - something that people who are already in fragile health have a hard time tolerating. I was lucky - I have a genetic mutation that responded well to Tarceva & Avastin. 8 months later, my tumors are under control and I have never felt ill from treatments. The brain mets are common metastases from primary lung cancer. Mine were small and I had them blown away with non-invasive stereo-surgery. It was pretty freaky, but the radiation oncologist assured me that the brain mets "were the least of my worries"! It's true that the prognosis for Stage IV lung cancer is pretty bad, but there is plenty of reason for HOPE, especially that the quality of his life can be helped with the right treatment. You just need to feel comfortable that his cancer journey gets started on the right foot. I agree with Sandi - it won't hurt to give Rational Therapeutics a call (562/ 989-6455). If you ask for Sheri, she is lovely and can answer your questions as to whether or not they think they can help your dad. BTW, my assay test cost $3500, but was fully paid for by my insurance. Your oncologist may not be supportive of assay tests (older versions didn't work as well as the new ones), but if you think it's for you, go with it! I really don't know what the downside would be - at the very least it can give you some peace of mind. All my best to you and your dad!

Pat

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It IS a tough decision. I have always heard if you can get into a clinical trial you should -- but I'm not sure why. Supposedly because anything new can't be worse than chemo which only helps so many and only for so long...

BUT, the old gold standard worked for me. In less than six months it wiped out a 7cm x 9cm mass, mets to lymph nodes liver other lung and right hip.

BUT -- that's just me. And I'm only 52.

I would like to point out that if the doc is convenient enough that that gold standard chemo is given by some doctors more often in lower doses to help with side effects. I thought 6 months or 6 treatments was the 'gold standard' and that's what I had -- low dose 3 weeks on 1 week off for 6 months; each 3-week session = to what others sometimes get in 1 week.

Good luck with the choice. I don't know anything about this particular clinical trial so I can't make a judgement on that, just take your time and don't feel TOO rushed. A third opinion can't hurt either (but boy can't all this information confuse you???)

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Get the assay done by all means! It is another piece of badly needed data to help you make a decision. I didn't know about it when I was diagnosede with Stage aIIIb NCLSC 5 cm tumor (with no mets), and was faced with the same choice as your Dad.. In my case, I wasn't really presented with a choice--I was told flat out that "the Gold Standard treatment isn't going to do you any good, so you'd better join my trial."

My wife and I are not stupid, and immediately sought a second opinion. The team we found did a thorough analysis of the available data and told me that the sensible course of action was to do the Gold Standard treatment, which was the drugs suggested for your Dad plus extensive radiation. This I did. It has been 2 1/2 years since diagnosis and my scans show NED (no evidence of disease).

I'm not saying that your Dad should necessarily do the Gold Standard treatment. Rather, my point is GET A SECOND OPINION! Thesecond opinion I got saved my life.

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Thank you all so... much for your input. I am always amazed that so many of you take the time to put your thoughts into words, just to benefit another. I know that some of you are really not feeling well, battling both the symptoms of the beast and the treatments, so I feel truly blessed by your concern.

My dad told me last night that he and his dr. have decided that he will enter into the Sutent trial. I am still looking into the assay test; just not sure that I can convince my dad to undergo another biopsy, etc. I am educating my brothers (4) on the assay test to see if they can be of help.

Question: since Sutent (the clinical trial drug) is already used with other cancers, does anyone know whether Rational Therapeutics would have access to it in order to test it against my dad's cells?

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My wife has stage 4 lung cancer, is 62, at present she has just finished her 5th chemo, Taxotere, Carboplatin, Avastin, every three weeks. We were told after the 2nd infusion that her tumor had shrank, the cancer in the pleural cavity was "either calcified, so tiny, or gone because it did not"light up" on the PET Scan." At this point I had to ask the oncologist, "where do we go from here?' I was told to the 6th infusion, wait 6 weeks and take another CT and PET and see where we are. Well, she qualifies for a clinical trial that is ideally suited to her stats, which I believe is called Lupraxin, which supposedly is a very important new vacine through Rush in Chicago, or she could go on Avastin as a maintenance program, but not both. BUT, the clinical trial is a double bind clinical trial meaning half get a placebo. Talk about a a decision. If we were to chose the clinical trial she could end up hoping and receiving no treatment whatsoever. Talk about a guinea pig, how utterly cruel. Make sure your clinical has no such restrictions.

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It was discovered that endothelial cells (cells that form the walls of blood vessels) are present in tumor microclusters and anti-angiogenesis drug effect upon these cells can be assessed.

At a critical point in the growth of a tumor, the tumor sends out signals to the nearby endothelial cells to activate new blood vessel growth. Endothelial cells are the source of new blood vessels and have a remarkable ability to divide and migrate.

Functional tumor cell analysis (which Rational Therapeutics and Weisenthal Cancer Group have the most extensive technological knowledge) has a morphological endpoint which allows for visualization of both tumor and microvascular cells and direct assessment of both anti-tumor and anti-microvascular drug effect. It can simultaneously test for direct anti-tumor activity and for anti-vascular activity within the three dimensional tumor cell clusters (this is what makes this technology vastly far superior than technology from over twenty years ago).

Anti-angiogenesis treatment (like Sutent and Nexavar) is somewhat like chemotherapy. Both are forms of systemic treatment. This means that both treatments use drugs that travel throughout the body to have their effects. Many doctors now consider these drugs to be the best treatments when systemic therapy is needed. But anti-angiogenesis drugs do not work the same way chemotherapy does. Because of this, they differ in terms of how well they work.

Chemotherapy drugs work by attacking cells in the body that divide quickly. This is why they work against cancer cells. But they can also harm other cells that divide quickly, such as those in the bone marrow, the skin, and in the mouth and intestines. This can lead to serious side effects like low blood cell counts (which can cause fatigue, infections, and bleeding), hair loss, mouth sores, nausea, and diarrhea.

Unlike chemotherapy drugs, anti-angiogenesis drugs do not target these normal cells. They act where new blood vessels are forming, so for the most part, anti-angiogenesis drugs tend to have milder side effects than chemotherapy drugs.

But anti-angiogenesis drugs can have their own side effects. While they're not as common or severe as those from chemotherapy, they can still be serious, or even life-threatening. Because only a few anti-angiogenesis drugs are being used, it's not yet clear if the effects seen so far will be seen with all of these drugs.

Photomicrographs in the functional tumor cell assay have been able to show if clones of tumor cells accumulate or don't accumulate the drugs. If the cells don't accumulate, they will not get killed by the drugs.

The short term future of cancer therapeutics is combinations of "conventional" and "targeted" agents for all solid cancers.

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Hi Brenpasta,

Good question about testing the Sutent. Rational Therapeutics tests only FDA approved drugs in various combinations. Again, I would just give them a call - they will be able to answer you over the phone and are really helpful.

I was so panicked when I got my diagnosis that I would have taken anything anyone gave me right then and there in the hospital, but I am so thankful I took the time to wait a few extra weeks. I had a pretty extensive wedge biopsy done, but even with that I was in and out of the hospital in 3 days.

Gpawelski is the most knowledgeable person I have read on the web when it comes to the efficacy of the assay tests. I can only tell you from my personal experience that I believe getting a second opinion from Dr. Nagourney and having the assay test which identified the right drugs saved my life, or at the very least gave me a better quality of life and certainly peace of mind.

Good luck to you and your dad on his journey!

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