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Cancer, Clinical Trials

Clinical trials: My next good chance

Clinical trials: My next good chance

We’ve partnered with Inspire, a company that builds and manages online support communities for patients and caregivers, to launch a patient-focused series here on Scope. Once a month, patients affected by serious and often rare diseases will share their unique stories; the third in our series comes from cancer patient/blogger Linnea Duff.

On April 5th, I quietly noted the seventh anniversary of the day my world turned upside down. For two years a persistent cough and shortness of breath had been attributed to adult onset asthma. It was only when I developed pneumonia that a chest CT scan was ordered, revealing a 5-cm mass on my left lung. A biopsy confirmed the unthinkable; I had an uncommon variety of non small cell lung cancer.

I was 45 years old and had never smoked. The only thing I knew about my diagnosis was that the overall five-year survival rate for lung cancer was only 15 percent. Determined to beat those odds, we transferred my care to Massachusetts General Hospital, where we hoped I’d have access to the most current research in the treatment of lung cancer.

Despite removal of the entire lower lobe of my left lung as well as adjuvant chemotherapy, my cancer recurred almost immediately. Three years after the initial diagnosis my health began to decline rapidly. Another therapy proved useless and my cancer was now deemed terminal. The situation seemed hopeless when a new biopsy revealed that I was positive for a mutation of the ALK gene. Given the chance to enroll in a phase I clinical trial for an experimental therapy that targeted the newly identified ALK mutation, I jumped at the chance.

And then an amazing thing happened. While I had hoped the experimental drug (crizotinib) would extend my life for several months, I ultimately spent almost three years on the trial. Six months ago, I entered my second phase I clinical trial for yet another ALK inhibitor (LDK 378).

Clinical trials, which remain under-referred as well as underutilized (enrollment of cancer patients hovers around 5 percent), have become my personal lifeline. I no longer view clinical trials as a last resort, but rather as my next good chance.

I am fortunate to have been able to participate in these trials at the same hospital where I receive my care, but I know patients who travel great distances to gain access to experimental therapies. These patients generally share my perspective; we are aware of the risks and understand that not every clinical trial will be effective in treating our individual cancers. And yet, we embrace the opportunity to enroll. Perhaps our quality of life will be enhanced and our lives extended.

No matter the outcome, we’ve been given the opportunity for not just another avenue of treatment, but to do something meaningful. By participating in a clinical trial, we are paving the way for other patients; the experimental therapies of today may become the gold standards of tomorrow.

Linnea Duff, of Amherst, NH, is a married mother of three who has been battling NSCLC (stage IV) for over seven years. She blogs at life and breath: outliving lung cancer.

6 Responses to “ Clinical trials: My next good chance ”

  1. Dee Says:

    As a 7 year stage 3 ovarian cancer survivor who participated in a clinical trial I agree with you 100% -“we are paving the way for other patients; the experimental therapies of today may become the gold standards of tomorrow.”

  2. Ninjacat1 Says:

    It was march of this year I was diagnosed with sclc and have signed on for any research studies. Being done. My son callers it a second class citizen as it is primarily blamed on smoking. Which I have been for many years. I feel good about getting through and beyond this challenge in my life. Positive thoughts and gibes from all welcome and my faith continues to carry me through. None of us has an experation stamped on the bottom of our feet

  3. Carmen Gonzalez Says:

    Kudos to SCOPE for partnering with Inspire to give expression to the many patient voices around the country. As Linnea’s story attests, it is through science that the promise of better treatments is possible. I happen to work for a consultancy that helps pharma recruit patients for clinical trials, but long before I had this job I knew of the importance of clinical research. I even volunteered for a study as a healthy patient that was investigating the use of a vaccine to fortify the immune system and insulate recipients of organ donations from infectious disease risk. I thought that was an important initiative to support, and someday hope to enroll in another study. If we as healthy and unhealthy patients rethink our role to advance drug and device development through study volunteering we might just accelerate the day when the promise of science becomes real.

  4. Carolyn Thomas Says:

    Linnea, your dramatic story deserves to be read by all patients, no matter what the diagnosis – and this is particularly true for women.

    “Clinical trials .. remain under-referred as well as underutilized.” Because women are not yet equally represented in medical research, our health care professionals have had to assume that diseases and conditions affect both women and men in the same way. Medical research has focused on the ‘bikini approach‘ to womenÂ’s health: breasts and reproductive organs.

    Heart research is a great example of this gender inequality: although women comprise 53% of patients with cardiovascular disease, in clinical trials they represented only 29% of subjects with coronary artery disease. See also: “Cardiac Research: Where Did All The Women Go?” –

  5. Gregory D. Pawelski Says:

    Only 8% of new drugs entering Phase I trials ever make it to marketing and this percentage is even lower for cancer drugs, because current drug testing is inefficient, with many drugs failing late in development, with these expensive failures owing, in large measure, to ineffective drugs and poor patient selection (lack of prognostic and predictive markers for response to therapy). Little progress has been made in identifying which therapeutic strategies are likely to be effective for “individual” patients, not average populations. Identifying markers that can predict response to a particular drug remains a great challenge.

    The one-size-fits-all paradigm is crumbling as individual patients with unique biological features confront the results of the blunt instrument of randomized clinical trials. Few small laboratories, start-up companies and early-stage biotech firms have the resources to marshal multi-million dollar clinical trials to test new therapies. This may in part be why advances in cancer therapy are moving so slowly forward. The barriers to entry are insurmountable, causing many good ideas to fall by the wayside for the lack of the hundreds of millions of dollars required to achieve FDA approval.

    Were academic clinical investigators incentivized to achieve greater clinical successes, there would be fewer failed Phase II and Phase III trials. Contrary to the business world where success is rewarded, academic clinicians receive the same compensation for every patient treated, whether the intervention is successful or not. This has the unintended consequence of encouraging physicians to accrue patients to clinical trials with no focus on effective therapies. While it may be gratifying to the trialists to have successes, they receive the same compensation for their failures. Academic clinical investigators need skin in the game.

    Using novel combinations of targeted agents, cell function analysis is years ahead of the clinical trial process. Cancer patients evaluated in cell-based functional profiling labs with favorable profiles can respond to some of the newest drugs, many of which have already completed Phase I of clinical trials. It’s a shame that they aren’t allowed to help accelerate the drug development process with the pharmaceutical companies and the FDA to put these drugs to a formal, real-time test. The scientific method of assay validation and clinical evaluation, based on using real-time, real patient data, under real-world conditions, to guide medical evidence.

    Patients like Linnea should have received “active” drugs, the first time around, and doctors should be allowed to give them, using techniques that match patients to “active” therapies. I believe human tumor primary culture analyses may be the answer to this dilemma. In fact, it could be so instructive and should be incorporated into clinical trials for these new targeted agents. Until the controlled, randomized trialist approach has delivered curative results with a high success rate, the choice of physicians to integrate promising insights and methods like the assays, remains an essential component of this kind of research and treatment technology.

  6. Bev Goldner Says:

    Thank you for taking part in this trial. I am thrilled that you had the results you did and grateful. In Feb 2013 I was diagnosed with stage IV lung cancer at the age of 48, a single Mother of four children. I too was a non-smoker and your story mirrors my own. I have been on crizotinib now for over a year with excellent results and anticipate needing the next step also. So again, thankyou for helping me and others to be able to benefit from this incredible drug. It would not be available without you. I wish you continued success with your treatment.


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