It’s about 7:30 Friday morning, and I’m just back from a run. I chug a 16-ounce bottle of low-fat Quik chocolate milk, because physiologists say it may boost post-workout recovery (and it’s yummy). As soon as I stop sweating, I’ll change my shirt.
Then my wife and I will take our 14-year-old daughter about 20 clicks around the Beltway, from our home in Alexandria to Falls Church, for a consultation with a hematologist/oncologist.
It’s an unsettling experience, hearing from your primary care physician that the results of a blood test indicate “something’s off” with your child’s blood count. In her case, both her white blood cells and platelets (blood-clotting cells) are “mildly, moderately low.”
That she’s also showing a bit of a rash is actually a good thing: It’s a sign that after a period of pretty intense fluctuations in her body temperature (it got as high as 103.8 F), her immune system is fighting back against a viral infection.
Aside from the fever, she’s also complained of exhaustion and lethargy, and that her muscles feel weak. She has no appetite. My kid’s a year-round swimmer, a soccer player, and a newbie to cross-country running. She’s a worker who demands of herself, athletically and academically.
The test for mononucleosis was negative. Results for mycoplasma pneumonia are still pending. There are other uncertainties that prompted our doctor to refer us to the specialist.
She also advised against using Dr Google to try to narrow down the problem.
Too late.
After all, that’s what I do. I’m a researcher and a writer. And I’m a father and a husband before those things.
It’s more difficult to put the brakes on my natural curiosity when the matter becomes as personal as this.
The latest innovation in pediatric oncology has taken the cancer survival and cure rate from 10% in the 1950s to early 1960s to about 80% across all stages and disease categories.
Thankfully, we never go to this stage of the process, because our hematologist/oncologist called with the good news that our daughter’s blood test revealed that her white blood cell and platelet counts had returned to normal.
But based purely on numbers, kids like my daughter are usually diagnosed with acute lymphocytic leukemia (ALL), which accounts for 75% of pediatric leukemia cases.
“Acute” means it’s a fast-moving type of leukemia, as opposed to chronic.
“Lymphocytic” or “lymphoblastic” refers to a leukemia that starts in the lymphoid cells, which make different types of white blood cells.
Leukemia that starts in myeloid cells – which lead to red blood cells, platelets, or white blood cells known as granulocytes – is called “myeloid” (or myelogenous/myeloblastic) leukemia.
According to the Cancer Research Institute, several types of immunotherapy are in various stages of testing for leukemia treatment. These categories include adoptive cell therapy, monoclonal antibodies, therapeutic vaccines, adjuvant immunotherapies, cytokines, and checkpoint inhibitors.
Within the past 12 months, experimental tests of a particular type of checkpoint inhibitor have shown remarkable success — Juno Therapeutic (NASDAQ:JUNO) T-cell therapy.
In clinical trials at Seattle Children’s Hospital, the Fred Hutchinson Cancer Research Center, and hospitals in Philadelphia, New York, and Memphis, the cancer goes away in 90% of patients with ALL that’s returned and resists regular drugs.
The usual rate of remission under these circumstances is lower than 10%.
The use of human T-cells is the most radical approach among several that use the human body’s immune system to fight cancer. According to Juno, it “has the potential to revolutionize the way cancer is treated.”
Different checkpoint inhibitors developed by Merck (NYSE:MRK) and Bristol-Myers Squibb Company (NYSE:BMY) have been used to treat skin and lung cancers.
Juno’s approach to leukemia involves genetically engineering a patient’s own T-cells to recognize and attack cancer cells. Its first treatment is a chimeric antigen receptor T-cell, or CART, known as JCAR015.
Such cell-based therapies could be effective, regardless of the type of previous treatments patients have experienced. And they may have few side-effects other than a strong fever.
Novartis AG (NYSE:NVS) is developing a similar therapy to treat kids with ALL, while Kite Pharma (NASDAQ:KITE) is working on one for patients with a type of non-Hodgkin’s lymphoma.
According to Roots Analysis and The Alliance for Regenerative Medicine, the oncology market for cell therapies is expected to reach $30 billion by 2030, and more than 600 therapies were in clinical trials at the end of 2015.
Juno, a $3.4 billion company that IPO’d in December 2014, has a long way to go to get past the “proof of principle” phase: Tests of its T-cell therapy so far have been small, “with no control groups, no comparisons, but also no other explanation than T-cells for why the cancer disappears,” according to MIT Technology Review.
Juno’s Phase II clinical trial of JCAR015 was placed on “clinical hold” by the U.S. Food and Drug Administration (FDA) after two patient deaths in July.
“The fatalities occurred after the addition of chemotherapy drug fludarabine to the pre-condition regimen,” reports The Fly. Juno proposed to continue the trial using JCAR015 with a different chemotherapy drug, cyclophosphamide, in the pre-conditioning step.
In early July, the company submitted a Complete Response to the Clinical Hold to the FDA, including a revised patient informed consent form, revised investigator brochure, and a revised trial protocol, and the “clinical hold” was removed on July 12.
During the company’s second-quarter conference call, CEO Hans Bishop said that due to the clinical hold, Juno has pushed back the anticipated approval of JCAR015 from 2017 to 2018.
Juno reported a second-quarter revenue gain of 120% to $27.6 million, due in large part to a payment from partner Celgene Corp (NASDAQ:CELG) as part of 10-year, $1 billion deal announced in 2015. Celgene plans to commercialize Juno’s cancer and autoimmune disease research.
But the company posted a second-quarter loss of $64.6 million ($0.64 per share), versus a $29.6 million loss, or $0.35 per share, a year ago.
Its research and development spend surged to $72.1 million, up from $23.9 million a year ago.
Juno shares took a big hit following the FDA’s clinical hold in July, falling from north of $40 to around $27.
But the FDA also granted the JCAR015 therapy “breakthrough” designation, which means it could receive approval after just one large clinical trial. It’s still on track for approval on an accelerated basis.
Juno’s major potential economic advantage – commercializing personalized cellular treatments – is intact.
Get Smart
“This instantiation of Watson, dubbed Watson for Oncology, is an artificial intelligence system that has access to millions of pages of medical textbooks and journal articles. Oncologists at Memorial Sloan Kettering Cancer Center trained the system to provide appropriate treatment recommendations by giving it descriptions of patients and then telling Watson the correct treatment.
“[It’s] the same way we would train a young doctor by showing how our specialists would treat a certain cancer in a certain person,” said Dr. Mark Kris, a Memorial Sloan Kettering lung cancer specialist who’s leading the hospital’s collaboration with IBM.”
Smart Investing,