Clinical Trial Check-In (February 2015) — Still NED!

Despite the phantom scan snafu (flew to Denver without my scans being scheduled) and a crazy busy schedule, this clinical trial trip to Denver has been great. I avoided the worst of the snowstorm, learned my new North Face Thermoball jacket keeps me toasty in subzero windchill, met my new great nephew Tate AND lung cancer advocate Kimberly Ringen (at different times), and participated as a patient advocate on the University of Colorado Cancer Center SPORE (an NCI-funded translational cancer research program).

Oh, yeah, I also had a clean PET-CT scan and brain MRI. STILL No Evidence of Disease, 26 months and counting.

My pulmonary embolism (diagnosed in December, but first appeared on scans in July 2014) is still present, but slightly smaller. Evidently the warfarin is working. My port certainly behaved well.

Did I mention I’m STILL NED?

The Phantom Scan

My bimonthly clinical trial appointment is this coming week. I typically fly from Seattle to Denver on Sunday, have my labs and scan on Monday, see my doctor Tuesday, and fly home Wednesday.  Scanxiety has a new twist for me this time around. I discovered late Friday that Monday’s scan doesn’t exist in the University of Colorado Hospital’s online schedule. Of course, it was too late in the day to contact anybody at the cancer center … continue reading

EPatients on the Front Lines: Precision Medicine, the FDA, and Me

On February 19, 2015, I was an invited patient advocate speaker at the 11th Annual Moores Cancer Center Industry/Academia Translational Oncology Symposium. My topic, “EPatients on the Front Lines:  Precision Medicine, the FDA, and Me,” explained how cancer research could move faster and be more successful if researchers, pharmaceutical companies, and the biotech industry would collaborate with patients early in the trial design process.

You can see my slides here:

Edit May 7, 2015:  UCSD posted the video of my speech

Here’s the text of the speech, along with the links on the slides.

# Slide Speech
1 Title Thank you for inviting me to speak to you today.  I’m going to share a view of precision medicine from the patient’s perspective.  If I seem a bit tense, blame it on the PET scan I’ll have 4 days from now.  I’ll post the speech on my blog tomorrow, so you don’t have to take notes.
2 Genome Scarf This is the legacy of an epatient.  It’s a genome scarf. It represents the chromosome 18 base pair sequence of colonrectal cancer patient Jay Lake.  Jay was a prolific science fiction author and my friend.
3 Genome Scarf and Jay pic Jay was example of an epatient:  a patient who is Equipped, Engaged, Empowered, and Enabled, whether or not they’re online. In 2011, after several surgeries and chemo regimens, Jay was running out of options.  Friends told him about genomic sequencing and helped him research clinical trials. The science fiction community crowdsourced the funding to sequence and analyze Jay’s personal and cancer genomes. Jay shared his data with NIH researchers for his immunotherapy trial, and with Harvard’s open-source Personal Genome Project.
4 My journey-Diagnosis Like Jay, I’ve pursued cutting-edge scientific research in hopes of living longer with metastatic cancer. I was diagnosed with Stage 3a non-small cell lung cancer in May 2011.  I never smoked anything – except a salmon.
5 My Journey-Progression 1 After a month’s delay to treat pneumonia, I had concurrent chemo and radiation. My primary tumor and lymph nodes all responded. Two months later, a PET scan found a new hotspot on my collarbone. A biopsy confirmed my cancer had progressed.  Since I had severe radiation pneumonitis, my oncologist recommended a break from treatment. In the next three months, I grew a 3-inch mass on my collarbone.
6 My Journey-Progression 2 I had more chemo, followed by more radiation. A new scan showed all the known tumors were gone or dead. BUT … I had two new tumors in my other lung.  I now had metastatic lung cancer. Whenever I stopped treatment, I had a new tumor within two months.  My oncologist told me I would be on chemo for the rest of my life.
7 My journey-Patient as Participant However, I wasn’t just a recipient of care. The information I learned in the Inspire online lung cancer community enabled me to become an interactive participant.  From other epatients, I learned to ask for my data, including radiology and pathology reports.  I also learned more extensive molecular testing was available at other facilities, and arranged to have my slides sent to the University of Colorado Hospital for a 10-oncogene panel. Unfortunately, all tests were negative.
8 My Journey-ROS1 & Trial Here’s where the tone of my story changes.  An online patient told me I fit the profile of patients who had the ROS1 translocation–relatively young, adenocarcinoma, neversmoker, triple negative for the most common mutations.  He sent me the journal article of early trial results.  After my second progression, I contacted University of Colorado again, and learned they had recently developed a ROS1 test.  I gave permission to use my remaining slides.  When I learned my cancer was ROS1 positive, I enrolled in the crizotinib trial in Colorado.
9 My journey-NED Thanks to precision medicine and the online lung cancer community, I’ve had  No Evidence of Disease for over two years. I’m not cured, but life is relatively normal for now–if you ignore the scanxiety every 8 weeks.  I chose to enroll in a trial for treatment in hopes of better option than chemo forever.
10 Smart Patient LC Trials Chart Epatients are very interested in the treatment options available in precision medicine trials, but sometimes we have trouble finding the right ones. New trial finders–like this format created with input from epatients–can help patients find the right treatment at the right time.
11 Purpose of Clinical Trial For clinicians, researchers, pharmaceutical firms, and industry, clinical trials are scientific experiments.  For epatients, clinical trials are treatment. Clinical trials are hope. By collaborating with epatients early in the design process, clinical trials not only can recruit more patients–they also move cancer research forward in ways that are meaningful to patients. Here are some examples.
12 Life Raft Group One of the earliest examples of patient involvement in clinical trial design comes from the Life Raft Group.  In the year 2000, gastrointestinal stromal tumor patients involved in the early Gleevec trials began sharing their experiences online in ACOR. Now Life Raft Group has the largest patient-generated clinical database in the world, and is driving research on GIST genome sequencing and drug screening.
13 LMS Direct Research Foundation Another example of patient-driven research is the Leiomyosarcoma Direct Research Foundation.  LMS is very rare–only 4 people in 1 million have it.  In 2004, over 800 of those patients were members of an ACOR online support group.  One group member read a journal article about a GIST molecular study, and emailed the researcher to ask “What would you need to study LMS?” The answer was “tissue samples”  Patients recruited 500 donors from the online group, collected  slides from clinics, deidentified them, and gave them to the researcher. The Stanford lab has since identified several molecular subtypes of LMS as well as potential drug targets, and published nine journal articles in its first four years.  Key elements of this successful research collaboration were a motivated online patient network and a researcher who listened to those patients and trusted them as collaborators.
14 TLS protocol crowdsourcing Technology is providing new ways to incorporate the patient voice.  In December 2012, the FDA cleared an Investigational New Drug Application (IND) for a multiple sclerosis therapy.  What’s remarkable is that the clinical trial protocol was the first ever developed with the aid of global crowdsourcing. That helped define primary and secondary endpoints, inclusion/exclusion criteria, and remote monitoring strategies for tracking patients.
15 ALCMI Young Lung Study 1 Patient networks and online technologies are also driving research for the most deadly cancer: lung cancer.   Currently 3-6 thousand newly-diagnosed lung cancer patients in the USA are under the age of 40, typically athletic never smokers.  The patient-founded Addario Lung Cancer Medical Institute designed a study of the somatic and germline mutations that might be driving the cancer in these young patients. The study is unique in that it allows patients to enroll either at a study site or online. It also provides genomic profiling data and treatment recommendations to patients as well as physicians.
16 ALCMI Young Lung Study 2 Because this trial was created in response to patient-identified needs and included the patient voice in all phases of trial development, it accrued 30 patients in the first two weeks.
17 Petition to FDA Patients, clinicians, and researchers can also collaborate on regulatory issues that impact clinical trials.  While working with a laboratory director at the University of Colorado, Dr. Dara Aisner, I realized that patients like me who had a genomic cancer variation might be unable to access essential testing under the FDA’s proposed regulations for laboratory developed tests.  By collaborating with medical professionals, I was able to help lung cancer advocacy groups submit comments to the FDA, and draft an online petition that collected over 700 signatures in less than three days. You can still sign the petition, by the way.
18 CTTI The Clinical Trials Transformation Initiative, which seeks to increase the quality and efficiency of clinical trials, recognizes the patient voice must be included when defining the precision medicine landscape.
19 Where to Find Epatients If you’re interested in finding epatients for collaboration, there are many places you can look for them.  Here’s where they may be hiding.

20 Obama Quote When President Obama announced the Precision Medicine Initiative, he said:”Patient advocates are not going to be on the sidelines. It’s not going to be an afterthought. They’re going to help us build this initiative from the ground up.”  He recognized the importance of including patient voices early in the design process. To be successful in the age of precision medicine, oncology researchers must collaborate with patients.
21 Thank You I hope I’ve encouraged further collaboration between cancer epatients, researchers, and industry. It will create faster paths to cancer cures.  Thank you for inviting me to share an epatient perspective at this symposium.

Call to Action: Proposed FDA Regulations Could Limit Cancer Patient Access to Life-Saving Therapies

This article first appeared on my February 1, 2015, blog for Cure Today Magazine.  Reprinted here with permission.

As posted in a recent CURE article, the US Food and Drug Administration (FDA) has proposed draft regulations titled “Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs).”  The FDA should withdraw this proposed framework because it could limit cancer patient access to potentially life-saving therapies.

Metastatic cancer patients have waited years for the hope that targeted therapies and genomic testing are now giving us. Don’t let the FDA throttle our hope. Knowledge of cancer genomics and proteins is evolving faster than government regulation can move.

Of course, we all want LDTs to be as validated, accurate and clinically relevant as possible. However, we also want the laboratories where these clinical testing services are performed to be able to exercise the flexibility, innovation and medical judgment necessary for good outcomes in thousands of cancer patients.  This isn’t possible with the proposed FDA regulations.

Please sign the petition at, and ask your friends and family to sign. If you or a loved one has benefited from molecular or genomic testing, please say so in the comments.  The petition and its comments will be submitted to the FDA as an official comment. The more signatures we have, the stronger our voice will be.

Here is a specific example of patient harm these proposed regulations might cause, taken from my own journey with metastatic ROS1-positive non-small lung cancer (NSCLC).

I live near Seattle. Because I was able to send my slides to University of Colorado for ROS1 testing, and my slides tested positive for ROS1, I was able to take crizotinib and achieve two years (and counting) of No Evidence of Disease. LDTs for ROS1 have been validated by medical research and have given many patients months or years of extra time.

Under the proposed regulations, some patients might have to travel to a distant or out-of-network medical facility to get the existing ROS1 test and receive treatment for their ROS1 cancer. In addition, some labs might stop offering the test because of the lengthy and cost-prohibitive process to obtain FDA approval. A medically validated test that is currently saving lives may become inaccessible to future lung cancer patients. The proposed FDA regulations would have effectively interfered with the practice of medicine.

Getting the best diagnostic and treatment outcomes from available cancer specimens relies of the practice of medicine, particularly the judgment and skill of pathologists, molecular pathologists and other molecular laboratory professionals.  The use and safety of LDTs can’t be regulated in the same manner as self-contained medical devices such as stents, or commercial test kits that come with pre-defined instructions.  Yet that is how the proposed regulations treat LDTs.

All cancer patients should have access to clinically validated tests that can help decide the best course of treatment.  Please sign the petition to tell the FDA to withdraw its proposed regulations.


A more detailed version of my ROS1 example:

ROS1-rearranged non-small cell lung cancer (NSCLC) testing and treatment with crizotinib are recommended in National Comprehensive Cancer Network guidelines because crizotinib, an FDA-approved drug for ALK-positive NSCLC, “showed marked antitumor activity” against ROS1 NSCLC, with a 72 percent  response rate in clinical trials—one of the highest response rates of any lung cancer drug—and a duration of response that exceeds 17.6 months.  Crizotinib is currently available off-label for ROS1 NSCLC through most insurance companies because of its strong clinical evidence of effectiveness.  Since most cancer treatment facilities do not offer an LDT for ROS1, they send slides to one of several CLIA-approved laboratories for testing, and treat ROS1-positive patients at home with crizotinib. Because no FDA-approved companion test exists for ROS1, any non-FDA approved LDT used to detect the ROS1 rearrangement would fall under the “LDT for Unmet Needs” exemption — IF the LDT is ONLY used for patients within the laboratory’s healthcare system. Therefore, any lung cancer patient whose home clinic does not offer ROS1 testing would have to either send specimens to a laboratory with an FDA-approved ROS1 LDT (none are available at present), or travel for diagnosis and treatment to a facility that offers a ROS1 LDT within its healthcare system–even if the patient’s insurance doesn’t cover that system.  Many patients may not be able to travel to another healthcare facility for ROS1 testing and treatment because they might be too sick, or face work, family, or financial constraints (insurance doesn’t cover travel expenses).  Thus, under the proposed regulations, some future ROS1 patients would not be able to get crizotinib treatment, even though that treatment is available today at their home cancer clinic.

Another example from lung cancer:

Under the proposed regulations, when using an FDA-approved companion test kit (currently available for EGFR and ALK NSCLC), any change in equipment, reagents, or patient specimen type must be submitted to the FDA and obtain the FDA’s approval before it can be offered to patients. Currently patients who have little tumor tissue (a common problem in lung cancer) can sometimes be tested for ALK based on cells obtained from pleural fluid or lymph nodes. Under the proposed regulations, only tumor tissue could be tested for ALK (that’s what the FDA-approved test kit requires) unless laboratories submit their modified ALK LDT for FDA approval. Also, published medical research has demonstrated that sometimes patients test negative for ALK using the FDA-approved test, but those patients may test positive using alternative, validated testing methods (such as genomic sequencing) and respond well to crizotinib. Under the new regulations, those alternative testing methods won’t be allowed unless they obtain FDA approval.  Lung cancer patients who don’t have enough tumor tissue would either go without testing, or undergo risky biopsies in hopes of obtaining enough tumor tissue.

Image credit:  “Researcher looks through microscope (2)” by Rhoda Baer. Licensed under Public Domain via Wikimedia Commons.