About My Health Careers Internship MedBlogs Contact us
Medindia LOGIN REGISTER
Advertisement

Clinical Trial Result Shows Promise for CAR T-cell Therapy in Treating Leukemia

by Anjali Aryamvally on February 6, 2018 at 11:30 AM
Font : A-A+

Clinical Trial Result Shows Promise for CAR T-cell Therapy in Treating Leukemia

CAR T-cell therapy, tisagenlecleucel, is a landmark personalized treatment for a high-risk form of acute lymphoblastic leukemia (ALL). Recent results from a global clinical trial reveal that children and young adults continued to show high rates of durable complete remission of the disease. The study published in New England Journal of Medicine also suggests that most side effects were short-lived and reversible.

Based on previous findings, the U.S. Food and Drug Administration (FDA) approved tisagenlecleucel--brand name Kymriah--in August 2017 for children and young adults up to 25 years of age with B-cell ALL that is refractory or in second or later relapse. It was the first-ever FDA-approved gene therapy, as well as the first FDA-approved CAR-T cell therapy.

Advertisement


The updated results report new data from the Novartis-sponsored ELIANA trial, conducted in 25 centers in 11 countries worldwide.

Chimeric antigen receptor T (CAR-T) cell therapy genetically modifies a patient's immune cells to make them seek out and kill leukemia cells. The approach was developed by a team led by Carl June, MD, of the Perelman School of Medicine at the University of Pennsylvania. That team collaborated with Children's Hospital of Philadelphia (CHOP), the first institution to use the therapy in children with leukemia.
Advertisement

The current study includes longer-term follow-up of safety and efficacy data in 75 patients aged 25 and younger.

"This expanded, global study of CAR T-cell therapy gives us further evidence of how remarkable this treatment can be for our young patients in whom all other treatments failed," said lead author Shannon L. Maude, MD, PhD, a pediatric oncologist at CHOP and Assistant Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. "Our data show not only can we can achieve longer-term durable remissions, and longer-term survival for our patients, but that these personalized, cancer-fighting cells can remain in the body for months or even years, effectively doing their job."

In the 75 children and young adults who received the CAR T-cells, 61 (81 percent) had a response to treatment, defined as complete remission with or without complete recovery of blood counts, after at least three months of follow-up. Relapse-free survival in those 61 patients was 80 percent at six months after treatment, and 59 percent at 12 months. Overall survival in all 75 patients was 90 percent at six months and 76 percent at 12 months. The survival rates, say the authors, are evidence that the early response rates were durable. In the absence of CAR T-cell therapy, patients with multiple relapsed or refractory B-cell ALL face a poor prognosis.

Accompanying the durable responses, the CAR T-cells persisted in the patients for as long as 20 months, with a median persistence of 168 days at the point at which data were analyzed for this study. All patients in the trial received a single infusion of the CAR T-cells.

In line with earlier studies of CAR T-cell treatment, the most common treatment-related side effect was a severe flu-like condition called cytokine release syndrome (CRS), involving high fever, muscle pain, neurologic symptoms, and circulatory and respiratory problems. Nearly half of the 75 patients received intensive care unit treatment to manage CRS, but the effects were treatable and transient.

The lead investigator of this Phase 2 trial was Stephan A. Grupp, MD, PhD, Director of the Cancer Immunotherapy Frontier Program at CHOP.

"One of our more challenging questions - 'can we manage the serious side effects of CAR T-cell therapy?' - was asked, and answered in this global study," said Grupp, the Section Chief of Cell Therapy and Transplant at CHOP, and a Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. "Some of our patients get very sick, but we showed that most toxic effects can be short-lived and reversible, with the potential for our patients to achieve durable, complete remissions. That's a pretty amazing turnaround for the high-risk child who, up until now, had little chance of surviving."

The research team is pursuing ongoing clinical trials, studying ways to mitigate serious side effects and overcome relapse.



Source: Eurekalert
Advertisement

Advertisement
News A-Z
A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
News Category
What's New on Medindia
Black Pepper as Preventive Measure Against Omicron
FODMAP Diet: A Beginner's Guide
Smallpox
View all

Medindia Newsletters Subscribe to our Free Newsletters!
Terms & Conditions and Privacy Policy.

More News on:
Leukemia Clinical Trials Clinical Trials - The Past and The Future Clinical Trials - Different Phases of the trial Chronic Myeloid Leukemia Acute Myeloid Leukemia Acute Lymphoblastic Leukemia Reiki and Pranic Healing Multiple Myeloma Diet and Nutrition for Healthy Weight Loss 

Recommended Reading
CAR T-Cell Therapy
CAR T-cell therapy is a type of gene therapy where the patient's T-lymphocytes are genetically ......
CAR T-cell Therapy Safe and Effective in Treating Pediatric Leukemia
Treating pediatric acute lymphoblastic leukemia with CAR T-cell therapy, a feasible global option .....
Novel Therapy Yields High Responses in Treatment-Resistant Chronic Lymphocytic Leukemia
A high percentage of chronic lymphocytic leukemia patients saw their tumors shrink after an ......
Mortality Risk Higher Among Acute Lymphoblastic Leukemia Patients
The survival rates of people with acute lymphoblastic leukemia living in socioeconomically deprived ...
Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is the cancer of white blood cells characterized by excess lympho...
Acute Myeloid Leukemia
Acute myeloid Leukemia, more popularly known by its abbreviated form AML, is a fast- evolving leukem...
Chronic Myeloid Leukemia
Chronic myeloid Leukemia is one of the most common types of blood cancer. It is characterized by ex...
Clinical Trials
Clinical trials are experiments that yield useful information to clinicians if a particular treatmen...
Clinical Trials - Different Phases of the trial
Clinical trials serve as a vital component for improving the treatment of medical conditions as they...
Clinical Trials - The Past and The Future
Many of the drugs that are used today have been discovered by chance or often by mere serendipity...
Diet and Nutrition for Healthy Weight Loss
Correct diet and a planned exercise regime is the mantra of healthy and sustainable weight loss....
Leukemia
Encyclopedia section of medindia gives general information about Leukemia...
Multiple Myeloma
Multiple Myeloma caught public attention when model turned actress Lisa Ray, who worked in Deepa Meh...

Disclaimer - All information and content on this site are for information and educational purposes only. The information should not be used for either diagnosis or treatment or both for any health related problem or disease. Always seek the advice of a qualified physician for medical diagnosis and treatment. Full Disclaimer

© All Rights Reserved 1997 - 2022

This site uses cookies to deliver our services. By using our site, you acknowledge that you have read and understand our Cookie Policy, Privacy Policy, and our Terms of Use