International MPN News, Science & Opinion

Clinical trial? Karen’s choice.

KAREN NEW

This is my wife, Karen:  On Valentine’s Day she was diagnosed with leiomyosarcoma.  Seven months later, she died.

With the help of compassionate doctors, she entered a clinical trial as an in-patient at NIH in Bethesda.  It helped kill her.

The beginning was all innocence. The ending was a horror show too often repeated.

 After failure of traditional chemo and radiation, Karen tried everything from shark cartilage to flax oil. She was young, the mother of young children. We finally found a doctor in Houston brewing antineoplastons from mare’s piss. He claimed extraordinary success. We found a forming clinical trial of the potion and begged the physicians to get us on it.  They did  That was the good, the hopeful part.

I left Karen after the first week in the round the clock care of the NIH mother ship staff in  Bethesda. When I got back to California she called. Her sarcoma showed signs of increasing. She had been summarily discharged and needed to come home immediately. I was frantic making long distance arrangements getting her a flight, getting her to the DC airport, getting her home.  A few days later, she died.

Records of that trial simply show the patient discontinued treatment.

This ability of sponsors and trial investigators to manipulate data, to interpret results selectively is far from the worst abuse of clinical trials. Even the failure of the great majority of trial sponsors simply to report results — as required — isn’t the worst abuse.  The thick curtain of secrecy and silence drawn around clinical trial data by sponsors and FDA regulators alike guarantee we will be kept ignorant of the results of human experimentation done in the name of science.  Infantilization of patients queuing up for treatment in wildly delusional hopes of a cure,  that’s the worst, most shameful, abuse.

Even after the death of MPN patients and the suspension of Sanofi’s fedratinib clinical trial, the FDA stalwartly refused to honor two Freedom of Information Act requests on the grounds of “privacy” regulations. To this day few of us understand why investigators never knew of the drug-related deaths on that trial and continued to dose patients for months.

Clinical trial is not a walk in the park. Not for sponsors, not for investigators, not for staff and not for regulators. The work is detailed and time-consuming. And there is a lot of it.

Monitoring Case Report Forms (CRFs), answering to Institutional Review Boards and Data Monitoring Committees, managing Contract Research Organizations,  assuring monitoring of clinical trial procedures across multiple sites. All that is in addition to attending to patients who are part of the clinical trial cohort to assure compliance with the study protocols and monitor drug effects.

But for us, for patients, it’s more than work. It’s a flat out risk that amounts to a gamble with our health. We have few justifications to enter a clinical trial. It’s a risk that requires us to travel to the trial site, to keep records, to take drugs with unknown impacts, to incur expense, to leave our regular doctor for the care of an institutional physician and staff. And after all the danger, stress and inconvenience we have no reason on earth to believe the trial — unlike 92% of others currently in progress — will result in an approved drug. 

And even if that rare result emerges, we have no guarantee the drug will be effective for us.

There may be a limited place for clinical trial among the options weighed by MPN patients. But unaware of the intent, structure, and real risk of these trials. we pin our hopes on unrealistic expectations of outcomes.

In a New York Times article questioning whether or not clinical trials work, Donald A. Berry, a professor of biostatistics at M. D. Anderson, contends “The failures of the last 20 or so Phase 3 trials testing drugs for Alzheimer’s disease could have been predicted based on the lackluster results from Phase 2. Still, the payoff for a successful Phase 3 trial can be so enormous that drug makers will often roll the dice — not on the prospect that the therapy will suddenly work, but on the chance that a trial will suggest that it does. (Emphasis added.)

Consider this: Currently there are over 200,000 clinical trials listed in www.clinicaltrials.gov. Under 8% make it through to file for FDA approval of a new drug application. 92% will fail in the end.

And all the time we are engaged in the Clinical Trial Lottery, our symptoms progress and our condition worsens.

The Clinical Trial Lottery has very high odds against success. Hundreds, perhaps thousands of compounds are developed and purified before animal testing begins. And then there are three phases an Investigational New Drug must successfully navigate before applying for approval to the FDA.  Phase I: Safety. Phase II: Safety, dosing and efficacy. Phase III: Safety, efficacy, side effects.

 The takeaway:  Unless you’re Mother Teresa or simply suicidal, NEVER participate in a Phase I/II dosage-toxicity trial under any circumstances. Stay at home and play with matches and gasoline, Run with scissors. Drink muddy water.  Our health is already compromised. Is there any good reason to play chicken with the gods?

Some of us might justifiably participate in a Phase III trial if we know which drug we will get, what the anticipated result will be and are assured the trial is closely monitored by a physician we trust. Then of course we can be mightily influenced by the carrot held out by Sponsors.  There is an economic selector at work in recruitment for clinical trial. The chance to get an expensive test drug free for the duration of the trial and, in many cases, thereafter is a major inducement for those of us who can’t afford the $10,000 monthly price tag on MPN drugs.

Karen’s choice was stark and instructive. When all our cards are played; When there is no longer hope of recovery from any other means; When we are out of options and near death and somehow want to try one last  hail Mary shot at survival, a clinical trial might make sense.

Take me back to the Contents

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Comments on: "Clinical trial? Karen’s choice." (9)

  1. Elizabeth Goldstein said:

    Had no idea you’d been through this, Zhen. My deepest sympathies on losing
    your lovely wife in such a heartless way.

  2. MarinaPeed said:

    Zhen, your clinical trial experience with your beloved Karen tells us so much. It must be difficult to revisit; you are kind to share with us. Your experience is representative of many families, desperate for helpful treatment. You shine a light on how patients are treated when they quit/drop from a trial, the inconsistent reporting, and the difficulty in getting access to helpful information. Thank you for all the reporting you do for the MPN community. You are a gem and Karen’s memory lives through you and your family. <3

    • Thank you, Marina dear… Karen’s life truly was a blessing for her family and community. I wish you could have known her.

  3. Karen Ulshafer said:

    The name Karen and clinical trial immediately caught my attention. Since my name is Karen and I have looked into a couple of clinical trials for me for myelofibrosis. I have been reading and gathering information thru’ this MPN site for years, now. From early diagnosis of PVera to, now MF. It scares and saddens me emensely to read this story of Karen. How vibrant and young she was. Like most of us, probably looked at the euphoric positive results of a clinical trial to find a drug to “save us” or at least make our disease a chronic one.
    I’m scared as I get older (now 68), that my body is weakening in all other systems, that I am no longer a candidate for stem cell transplant (was not going to take that route unless at very end of desperation), and increase frequency of transfusion and iron overload and fatigue and just feel like giving up. But there is that niggle of hope back there in my mind and tingling thru my body that says, “maybe, just maybe, something will come along in time to change this downward spiral …” Hope is greater now than10 years ago.
    My heart goes out to you for your loss of Karen and the shattered hope of a positive clinical trial and drug.

  4. Rebecca McElroy said:

    Karen looks like such a lovely person. I am so sorry.

  5. Thank you for sharing your story. I am so sorry for your loss.

  6. Marty Prager said:

    Zhen- This was very sad I am so sorry. There is clearly an upside and downside to any medical procedure. My condolences.

  7. So sad for Karen! I know how hard it is to make a decision if your partner is having a lot of trouble, and you do not know what to do! I feel there are Doctors in hematology that can be very helpful, but in a lot of cases I feel that a lot doctors that offer a trial are not exactly sure how to take care of things that involve a trial! This sounds like what happened in Karen’s case. These trials have to be handled by doctors that have a lot of experience with decent outcomes! These new medicines have to have approval by the doctors that are more experienced, and those doctors have to be on board to help extensively in other facilities! If someone is in very serious condition, then the patient has to be told, and if much older, he or she should have a very experienced Doctor! If these experienced doctors want good outcomes on these trials, they need to help out seriously and be very involved! Also the FDA should have a great understanding that older people may not do as well, and provide a very experienced Doctor for care in a trial, if they want better outcomes! THIS IS IMPERATIVE! Patients like these need compassion and a lot of help, and all of the above mentioned, need to get on board!

    My opinion, Regards,
    Trishe

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