A Cancer Doctor Weighs In On CAR-T, Precision Medicine And Pricing Debates


“Yesterday’s historic FDA approval of the first engineered T-cell treatment for cancer, Novartis’ Kymriah (tisagenlecleucel), was accompanied by inevitable questions about how the product would be priced. In the end, Novartis set the price at $475,000, which was lower than many analysts had predicted, considering the treatment is designed to cure some forms of acute lymphoblastic leukemia (ALL)—and in clinical trials it did just that for most patients.”

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The CAR T-Cell Treatment: Will It Work for Solid Tumors?

Chimeric antigen receptor (CAR) T-cell therapy is a new, immune system-based cancer treatment that has garnered recent media attention. In a clinical trial, CAR T-cell treatment left no signs of tumors in 70% to 90% of children and adults with the aggressive blood cancer acute lymphocytic leukemia (ALL). ALL is almost always fatal, and the results observed with CAR T-cell treatment are nothing short of spectacular. Continue reading…

New Ways to Treat Solid Tumours

Editor’s note: Recent research has uncovered a potential new way to fight some cancer types. The key is a protein called Eph3A, which is made by the cells of blood cancers and solid tumors. Researchers made a new drug called KB004 to target and kill cells with Eph3A. The drug is currently being tested in a clinical trial with volunteer leukemia patients.

“An international team of scientists has shown that an antibody against the protein EphA3, found in the micro-environment of solid cancers, has anti-tumour effects.

“As EphA3 is present in normal organs only during embryonic development but is expressed in blood cancers and in solid tumours, this antibody-based approach may be a suitable candidate treatment for solid tumours…

“Currently, KaloBios Pharmaceuticals is testing the anti-EphA3 antibody KB004 in a multi-centre Phase I/II clinical trial in Melbourne and the US in patients with EphA3 expressing blood malignancies: AML, MDS and myelofibrosis.”

Down Syndrome Ups Risk for Infection-Related Death in Pediatric ALL

“Researchers have identified Down syndrome as a major risk factor for infection-related mortality among pediatric patients with acute lymphoblastic leukemia (ALL) enrolled in the Medical Research Council UKALL 2003 trial.

“ ‘Our analysis demonstrated that a significant proportion of sepsis deaths occurred during induction and during periods of neutropenia,’ wrote researchers led by David O’ Connor, MD, of Imperial College London. ‘Thus increased awareness of the potential for septic complications is required when caring for patients during these high-risk periods.’ ”

“The currently reported rates of treatment-related mortality in trials of patients with ALL is between 2% and 4%, with the most common cause being infection. With ALL having such high survival rates in low-risk patients, reducing infection-related mortality is an important goal for improving outcomes with children with this disease, according to O’Connor and colleagues.”


MRD May Guide Augmented Post-Remission Treatment in Pediatric ALL

Editor’s note: In a recent study, scientists compared two different post-remission treatments for children and adolescents with acute lymphoblastic leukemia (ALL) who had only a small number of cancerous cells left in the body (minimal residual disease, MRD) after initial treatment (remission induction therapy). The scientists found that patients who received an augmented post-remission treatment, compared to the standard one, experienced better outcomes. However, these patients also had worse side effects. Ongoing clinical trials will reveal more about improved post-remission treatment and reduction of side effects.


“Children and adolescents with acute lymphoblastic leukemia who had minimal residual disease at the end of remission induction therapy demonstrated improved outcomes with augmented post-remission therapy compared with standard treatment, according to study results.

“However, patients who received augmented therapy experienced higher rates of adverse events, results showed.”

Pediatric ALL Protocols Have Low Risk of Late Effects

Bone marrow from a 3-year-old ALL patient

“Long-term outcomes of patients treated for pediatric acute lymphoblastic leukemia (ALL) with modern treatment protocols are good, with an overall low risk for serious long-term side effects, according to the results of report from the Childhood Cancer Survivor Study cohort.

“ ‘This data will be useful for oncologists counseling newly diagnosed patients, and provides reassurance that the ‘devastating’ diagnosis of ALL can often have good short- and long-term outcomes,’ study author Paul C. Nathan, MD, of the Hospital for Sick Children, University of Toronto, told Cancer Network.

“With survival rates of childhood ALL at about 90%, researchers and clinicians have to focus not only on curing the disease, but on the quality of that cure and its long-term adverse effects.”

Image: “Bone marrow from a 3-year-old ALL patient”

FDA Grants Breakthrough Designation to Personalized Immunotherapy for ALL

“The FDA today granted breakthrough therapy designation for CTL019, an investigational personalized immunotherapy, for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia, according to a press release issued by Penn Medicine.

“CTL019 (Novartis), developed by the University of Pennsylvania, is the first personalized cellular therapy for the treatment of cancer to receive this classification.

“In early-stage clinical trials conducted at the Hospital of the University of Pennsylvania and Children’s Hospital of Philadelphia, 89% of patients with ALL who were not responding to conventional therapies achieved complete remission after treatment with CTL019.

“ ‘Our early findings reveal tremendous promise for a desperate group of patients, many of whom have been able to return to their normal lives at school and work after receiving this new, personalized immunotherapy,’ Carl H. June, MD, director of translational research in the Abramson Cancer Center of the University of Pennsylvania. ‘Receiving the FDA’s Breakthrough Designation is an essential step in our work with Novartis to expand this therapy to patients across the world who desperately need new options to help them fight this disease.’ ”

Editor’s note: This story describes a new leukemia treatment called CTL019, which boosts a patient’s own immune system to fight cancer. CTL019 treatment is personalized for each patient, since it involves altering a patient’s immune system cells to attack tumor cells. It has been tested in volunteer patients in clinical trials, and has shown promising results for adults and children with relapsed or refractory acute lymphoblastic leukemia (ALL). The U.S. Food and Drug Administration (FDA) has now granted breakthrough therapy designation for CTL019, meaning that review and approval will be accelerated so that the drug can more quickly reach patients outside of clinical trials.