12/5/2018 by Share Your Story

Episode 12 – Innovations from the American Society of Hematology

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Topics: American Society of Hematology’s 60thAnnual Meeting / Mark Winland #RisingTuesday


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The American Society of Hematology’s 60thAnnual Meeting ends today in San Diego. I am going to start by fully admitting that I am an innovation geek. In my prior advocacy life, I spent most of my time sitting behind the camera while some of the greatest minds in medicine were interviewed. One of the reasons I am so passionate about what I do is that the innovation yet to be discovered holds an enormous amount of hope for not only individual patients, but for us as the nation leading the world in innovation.

Out of ASH this week there is a lot of good news for blood cancer patients and sickle cell disease patients. Today we are going to talk about some of those highlights.

[from the Editor: Okay readers, there’s a lot of information here. If you want the science-y stuff, click on the highlighted “press release”. If you just want to pass this by, go to “What does this all mean?”

The CAR-T studies:

CAR-T is a type of treatment in which a patient’s T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells.

From a Gilead press release:

[from the press release] “With aggressive cancers such as refractory large B-cell lymphoma, our primary goal is to extend the lives of patients,” said Sattva S. Neelapu, MD, ZUMA-1 Co-Lead Investigator and Professor, Department of Lymphoma/Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center. “Outcomes with traditional standard of care for this highly refractory patient population have been extremely poor. Nearly 40 percent of patients in ZUMA-1 remain in response and half of the patients are still alive after at least two years of treatment with Yescarta.”

From a Novartis press release:

[in English, from the press release] “This two-year analysis is an exciting milestone for the field, as it is the longest follow-up data for a multicenter CAR-T cell trial for those patients who have failed to respond to other treatment options,” said Stephan A. Grupp, MD, PhD, Director of the Cancer Immunotherapy Program and Section Chief of Cell Therapy and Transplant at Children’s Hospital of Philadelphia, and a Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. “Seeing that the majority of responding patients from ELIANA are still in remission for this long after a one-time infusion further establishes Kymriah as a truly transformative treatment option.”

[In English, from the press release]: “Stephen J. Schuster, MD, the Robert and Margarita Louis-Dreyfus Professor in Chronic Lymphocytic Leukemia and Lymphoma Clinical Care and Research in the University of Pennsylvania’s (Penn) Perelman School of Medicine and director of the Lymphoma Program at the Abramson Cancer Center says, “For physicians treating this patient population, duration of response and a consistent safety profile are incredibly important data points, and the findings from this updated analysis further instill confidence in the continuing potential of Kymriah in the treatment of these patients.”

From a Bluebird Bio press release:

[In English, from the press release:] “Patients with multiple myeloma often undergo multiple cycles of treatment because there is currently no known cure for this aggressive cancer,” said Nina Shah, M.D., University of California, San Francisco…. “The early clinical data from this Phase 1 study show manageable safety findings, and most patients in this initial group achieved an objective response. Future study is needed to assess durability of response at the current dose, as well as safety and activity at higher doses of bb21217.”

You can  read more about the CAR-T studies here.

From an Amgen press relsease:

BCMA is a protein linked to multiple myeloma. Amgen Inc, updating the first trial of its bispecific antibody for multiple myeloma, said on Monday seven out of 10 patients given the second-highest dose of AMG420 responded to the drug, including four with no detectable cancer. Six patients were still responding at 7.5 months of follow-up, according to research presented in San Diego at the annual meeting of the American Society of Hematology (ASH). AMG420 has been given fast track approval by the FDA. Other companies exploring this link: Celgene, bluebird bio, and Johnson & Johnson

from the Cancer Network of the Oncology journal:

  •  Enhanced Genomic Models are created when researchers find new ways to identify and hopefully treat subsets of patients in need of better treatments. For B-cell lymphoma patients, researchers developed an enhanced genomic model combining clinical factors and select gene mutations in order to predict better overall survival.

from a Bluebird, bio press release:

  • LentiGlobin Gene Therapy brought some good news for Sickle Cell Disease (“SCD”) patients. SCD is a serious, progressively debilitating and life-threatening genetic disease. SCD results from production of abnormal sickle hemoglobin (HbS), which leads to sickled red blood cells (RBCs) and hemolysis. It is estimated that SCD affects 100,000 Americans.

[in English, from the press release] “After patients with sickle cell disease were treated with LentiGlobin they began to produce gene-therapy derived HbA T87Q, which was associated with lower levels of sickling hemoglobin, the type of hemoglobin that damages red blood cells,” said David Davidson, M.D., chief medical officer, bluebird bio. “These clinical findings were consistent with results in newly developed exploratory assays used to evaluate patient samples that demonstrated reduction of HbS in most red blood cells, and a reduction in sickling comparable to sickle-trait, suggesting the potential for LentiGlobin to fundamentally improve the underlying red blood cell pathology responsible for the clinical consequences of sickle cell disease.”

What does all this mean?

We need more.

Yes, we pay for that innovation in higher prices that are a direct result of increased scientific innovation, length of patent, and a supply chain rebate system that makes your head spin. Our prices reflect the free medicine given away around the world and the price fixing imposed by socialized nations. At the very least, our leaders and the industries themselves need to figure out how to innovate in insurance and payment delivery design so as not to burden patients who need the health care system to work for them in ways that are not sustainable.

But for all the money the biopharmaceutical industry spends on innovation, we are sorely lacking as a nation in developing new delivery and payment models to accommodate this development. Everyone wants to throw innovators under the bus, but from where I sit, innovation is the only thing that is going to save us. Insurance companies create nothing. Pharmacy Benefit Managers create nothing. They are Tony Soprano shaking everyone down for their piece of the action and patients who are left unable to gain access are the ones paying the ultimate price.

As a full disclosure, we do receive funding for our programs from the pharmaceutical industry, but they in no way influence our content or what we talk about on this podcast or on our website. Each show is solely at the discretion of Patients Rising.


Mark Winland

Meet Mark Winland on Instagram. Mark is a U.S. Marine whose health is deteriorating because California is forcing him to taper off the pain medicine that allows him function and quality of life.

They eventually got Mark on the right dose of pain medication. “It worked great. I was working every day. No more surgeries. No need to up my medicine. Just right for 4 years.” Then the state of California forced Mark’s doctor to begin tapering down his medicine.

In a short period of time they reduced Mark’s pain medicine by 41%. Now he’s only working 2 or 3 days a week instead of full time. His pain is not controlled. Mark is the victim of a culture of political expediency that has forgotten the goal of pain management; to improve the function and quality of life of people in pain.

Read more about Mark here.



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