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Cancer Stories: The Art of Oncology - Compassion and Compassionate Use, by David J. Einstein

Compassion and Compassionate Use, by David J. Einstein

Cancer Stories: The Art of Oncology

09/30/19 • 29 min

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One oncologist provides his take on responding to "compassionate use" requests

TRANSCRIPT

The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care, and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience, and conclusions. The mention of any product, service, organization, activity, or therapy should not be construed as an ASCO endorsement.

[DR. LIDIA SCHAPIRA] Welcome to JCO's Cancer Stories-- The Art of Oncology, brought to you by the ASCO Podcast Network, a collection of nine programs covering a range of educational and scientific content, and offering enriching insight into the world of cancer care. You can find all of the shows, including this one, at podcast.asco.org.

[GREG GUTHRIE] "Compassion and Compassionate Use," by David J. Einstein. The email came down to this. This patient is running out of options, and he and his family are starting to think outside the box. Two months prior, the sender was my co-fellow. Now, after a blissful vacation month entirely unplugged from oncology, he and I were junior attending physicians, launched into the dual frontlines of inpatient and outpatient oncology.

He was staffing the consult service, I the inpatient oncology service, both of us simultaneously trying to keep our backburner outpatient clinics and research projects from boiling over. He was letting me know about my newest admission, a patient with advanced choriocarcinoma, which was ostensibly within my area of expertise of genitourinary cancers.

During years of residency training, we had learned to eyeball patients and differentiate sick from not sick-- or really, those who might be imminently dying from those who were OK for now. As oncology fellows, we recognized a new group-- the patients who seemed to be imminently dying on paper, but in person looked amazingly normal. The patient referred to in the email was such a patient-- an energetic engineer in his 50's, with a more than slight Boston accent, despite his years in California and Texas, and plenty of Red Sox gear to match.

On paper, he was in dire shape, with treatment-resistant advanced cancer and increasing toxicities of treatment. The patient and this family had taken on his disease with a battle mentality that works for some and makes others cringe. After multiple rounds of chemotherapy, resections, and radiation treatments failed to cure his disease, he moved on to the high-dose chemotherapy with autologous stem cell rescue that is the standard of care in this setting. If some chemotherapy is good, more is better.

However, he eventually exhausted his supply of stem cells, still without having achieved remission. Time to think outside the box, turning to his identical twin brother as a source of new stem cells. The patient proceeded to a last-ditch effort at high-dose chemotherapy, this time with syngeneic transplantation. It worked, for approximately three months.

Despite the most intensive efforts, his disease kept bouncing back. At this point, it was clear that chemotherapy would only transiently suppress his cancer, but not cure it. Meanwhile, his treatments left him with hearing loss severe enough to require hearing aids, poor kidney function, foot numbness, and bone marrow that, even on imaging, looked exhausted, plus prolonged cytopenias to prove it. What could be done?

This patient and his family had crossed the country in search of an outside the box treatment that might offer him the benefit that stand treatments had not. We are used to patients looking online for new diets and complementary medicines. We understand that this is a way to seek an active part in their care, when they otherwise feel like passive recipients.

The patient and his family had gone further, identifying a clinical trial available at our hospital that they desperately wanted to join. But the trial was in a different tumor type. And moreover, he would not be a candidate for any of the trial, given his accumulated treatment-related toxicities.

Undaunted, his sister found an online poster from our peer institution across the street, describing a novel compound used to treat a patient with his disease, with the resulting and seemingly miraculous remission. Again, we are accustomed to the patient who brings in the latest online research to an appointment. Sometimes it is not scientifically valid. Sometimes it is valid, but not applicable to the situation. And occasionally, it is both valid and applicable, and actually does result in a treatment change.

As I scrutinized the information the patient's sister provided, it actually seemed both possibly valid and applicable. I could find no obvious toxicity that precluded use of this drug. And yet, it was e...

09/30/19 • 29 min

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