Welcome!

News Feed Item

Engineered Immune Cells Produce Complete Response in Child with an Aggressive Pediatric Leukemia

--CHOP Oncologist Achieves Potent Anticancer Effect with Engineered T Cell Therapy on First Use in Children--

ATLANTA, Dec. 9, 2012 /PRNewswire/ -- By reprogramming a 7-year-old girl's own immune cells to attack an aggressive form of childhood leukemia, a pediatric oncologist has achieved a complete response in his patient, who faced grim prospects when she relapsed after conventional treatment. The innovative experimental therapy used bioengineered T cells, custom-designed to multiply rapidly in the patient, and then destroy leukemia cells. After the treatment, the child's doctors found that she had no evidence of cancer.

To view the multimedia assets associated with this release, please click: http://www.multivu.com/mnr/50777-chop-engineered-immune-t-cell-therapy-anticancer-effect-in-children

(Photo: http://photos.prnewswire.com/prnh/20121209/MM25935)

Pediatric oncologist Stephan A. Grupp, M.D., Ph.D., of The Children's Hospital of Philadelphia, and colleagues from the University of Pennsylvania presented updated results of the clinical trial involving these engineered cells at the American Society of Hematology (ASH) annual meeting today in Atlanta. Grupp is the director of Translational Research for the Center for Childhood Cancer Research at The Children's Hospital of Philadelphia, and a professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

Grupp's research builds on his ongoing collaboration with Penn scientists who originally developed the modified T cells as a treatment for B-cell leukemias. The Penn team reported on early results of a trial using this cell therapy in adult chronic lymphocytic leukemia (CLL) patients in August of 2011. Carl H. June, M.D., of the Perelman School of Medicine at the University of Pennsylvania, leads this research group, which is presenting new data at the ASH meeting showing that nine of 12 patients with advanced leukemias in the clinical trial, including two children, responded to treatment with CTL019 cells.

One of the nine responding patients is the 7-year-old with acute lymphoblastic leukemia (ALL). Grupp and Penn colleagues adapted the treatment to combat ALL, the most common childhood leukemia, and also the most common childhood cancer. Although physicians can cure roughly 85 percent of ALL cases, the remaining 15 percent of such cases stubbornly resist treatment.

The CTL019 therapy, formerly called CART19, represents a new approach in cancer treatment. T cells are the workhorses of the immune system, recognizing and attacking invading disease cells. However, cancer cells fly under the radar of immune surveillance, evading detection by T cells. CAR T cells (chimeric antigen receptor T cells) are engineered to specifically target B cells, which become cancerous in certain leukemias, such as ALL and CLL, as well as types of lymphoma, another cancer of the immune cells.

CD19 is a protein found only on the surface of B cells. By creating an antibody that recognizes CD19, and physically connecting that antibody to T cells, the researchers have created a guided missile that locks in on and kills B cells, thereby attacking B-cell leukemia.

In using the CTL019 treatment in his pediatric patient, Grupp found that the very activity that destroyed leukemia cells also stimulated a highly activated immune response called a cytokine release syndrome. The child became very ill and had to be admitted to the intensive care unit.

Grupp and his team decided to counteract these toxic side effects by using 2 immunomodulating drugs that blunted the overactive immune response and rapidly relieved the child's treatment-related symptoms. These results were effective enough that this approach is now being successfully incorporated into CTL019 treatments for adults as well.  

The immunomodulating drugs did not interfere with the CTL019 therapy's anti-leukemia benefits, which have persisted 6 months after the infusion of cell therapy. This persistence is essential, because the engineered T cells remain in the patient's body to protect against a recurrence of the cancer.

"These engineered T cells have proven to be active in B cell leukemia in adults," said Grupp. "We are excited to see that the CTL019 approach may be effective in untreatable cases of pediatric ALL as well. Our hope is that these results will lead to widely available treatments for high-risk B cell leukemia and lymphoma, and perhaps other cancers in the future."

"This type of pioneering research addresses the importance of timing when considering experimental therapies for relapsed patients," added Susan R. Rheingold, M.D., one of the leaders in the Children's Hospital program for children with relapsed leukemia.  "To ensure newly relapsed patients with refractory leukemia meet criteria for options like CTL019, we must begin exploring these innovative approaches earlier than ever before.  Having the conversation with families earlier provides them more treatment options to offer the best possible outcome."

In August 2012, Novartis acquired exclusive rights from Penn to CART-19, the therapy that was the subject of this clinical trial and which is now known as CTL019.

For more information about the  engineered T cell clinical trial at the University of Pennsylvania, or interviews with Penn Medicine lead researcher Carl June, M.D., or investigators David Porter, M.D., or Michael Kalos, Ph.D., contact Holly Auer, cell: 215-200-2313, or holly.auer@uphs.upenn.edu.

"CD19-Redirected Chimeric Antigen Receptor T (CART19) Cells Induce a Cytokine Release Syndrome (CRS) and Induction of Treatable Macrophage Activation Syndrome (MAS) That Can Be Managed by the IL-6 Antagonist Tocilizumab (toc)"
--Abstract #2604 presented, Sun., Dec. 9, 2012, 6 p.m. ET; Hall B1-B2, Level 1, Building B--

About the Cancer Center at The Children's Hospital of Philadelphia: The Children's Hospital of Philadelphia cares for more children with cancer than any other general pediatric hospital in the United States. Its large basic and clinical research programs are particularly strong in pediatric neuro-oncology, neuroblastoma, leukemia and lymphoma, and sarcomas. Of all pediatric institutions, Children's Hospital enrolls the most patients in national clinical trials, working in close collaboration with national organizations such as the Children's Oncology Group. Physicians at Children's Hospital have had pioneering roles in developing international standards for diagnosing and treating neuroblastoma, and in developing programs for survivors of childhood cancer.

Contact: Rachel Salis-Silverman
The Children's Hospital of Philadelphia
Cell: (267) 970-3685
Salis@email.chop.edu

SOURCE The Children's Hospital of Philadelphia

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
Niagara Networks exhibited at the 19th International Cloud Expo, which took place at the Santa Clara Convention Center in Santa Clara, CA, in November 2016. Niagara Networks offers the highest port-density systems, and the most complete Next-Generation Network Visibility systems including Network Packet Brokers, Bypass Switches, and Network TAPs.
Zerto exhibited at SYS-CON's 18th International Cloud Expo®, which took place at the Javits Center in New York City, NY, in June 2016. Zerto is committed to keeping enterprise and cloud IT running 24/7 by providing innovative, simple, reliable and scalable business continuity software solutions. Through the Zerto Cloud Continuity Platform™, organizations can seamlessly move and protect virtualized workloads between public, private and hybrid clouds. The company’s flagship product, Zerto Virtual...
WebRTC is about the data channel as much as about video and audio conferencing. However, basically all commercial WebRTC applications have been built with a focus on audio and video. The handling of “data” has been limited to text chat and file download – all other data sharing seems to end with screensharing. What is holding back a more intensive use of peer-to-peer data? In her session at @ThingsExpo, Dr Silvia Pfeiffer, WebRTC Applications Team Lead at National ICT Australia, looked at differ...
Without a clear strategy for cost control and an architecture designed with cloud services in mind, costs and operational performance can quickly get out of control. To avoid multiple architectural redesigns requires extensive thought and planning. Boundary (now part of BMC) launched a new public-facing multi-tenant high resolution monitoring service on Amazon AWS two years ago, facing challenges and learning best practices in the early days of the new service. In his session at 19th Cloud Exp...
For basic one-to-one voice or video calling solutions, WebRTC has proven to be a very powerful technology. Although WebRTC’s core functionality is to provide secure, real-time p2p media streaming, leveraging native platform features and server-side components brings up new communication capabilities for web and native mobile applications, allowing for advanced multi-user use cases such as video broadcasting, conferencing, and media recording.
With major technology companies and startups seriously embracing IoT strategies, now is the perfect time to attend @ThingsExpo 2016 in New York. Learn what is going on, contribute to the discussions, and ensure that your enterprise is as "IoT-Ready" as it can be! Internet of @ThingsExpo, taking place June 6-8, 2017, at the Javits Center in New York City, New York, is co-located with 20th Cloud Expo and will feature technical sessions from a rock star conference faculty and the leading industry p...
"Plutora provides release and testing environment capabilities to the enterprise," explained Dalibor Siroky, Director and Co-founder of Plutora, in this SYS-CON.tv interview at @DevOpsSummit, held June 9-11, 2015, at the Javits Center in New York City.
Security, data privacy, reliability and regulatory compliance are critical factors when evaluating whether to move business applications from in-house client hosted environments to a cloud platform. In her session at 18th Cloud Expo, Vandana Viswanathan, Associate Director at Cognizant, In this session, will provide an orientation to the five stages required to implement a cloud hosted solution validation strategy.
SYS-CON Events announced today that CA Technologies has been named "Platinum Sponsor" of SYS-CON's 20th International Cloud Expo®, which will take place on June 6-8, 2017, at the Javits Center in New York City, New York, and 21st International Cloud Expo, which will take place in November in Silicon Valley, California.
The security needs of IoT environments require a strong, proven approach to maintain security, trust and privacy in their ecosystem. Assurance and protection of device identity, secure data encryption and authentication are the key security challenges organizations are trying to address when integrating IoT devices. This holds true for IoT applications in a wide range of industries, for example, healthcare, consumer devices, and manufacturing. In his session at @ThingsExpo, Lancen LaChance, vic...
SYS-CON Events announced today that delaPlex will exhibit at SYS-CON's @CloudExpo, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. delaPlex pioneered Software Development as a Service (SDaaS), which provides scalable resources to build, test, and deploy software. It’s a fast and more reliable way to develop a new product or expand your in-house team.
Wooed by the promise of faster innovation, lower TCO, and greater agility, businesses of every shape and size have embraced the cloud at every layer of the IT stack – from apps to file sharing to infrastructure. The typical organization currently uses more than a dozen sanctioned cloud apps and will shift more than half of all workloads to the cloud by 2018. Such cloud investments have delivered measurable benefits. But they’ve also resulted in some unintended side-effects: complexity and risk. ...
The explosion of new web/cloud/IoT-based applications and the data they generate are transforming our world right before our eyes. In this rush to adopt these new technologies, organizations are often ignoring fundamental questions concerning who owns the data and failing to ask for permission to conduct invasive surveillance of their customers. Organizations that are not transparent about how their systems gather data telemetry without offering shared data ownership risk product rejection, regu...
When you focus on a journey from up-close, you look at your own technical and cultural history and how you changed it for the benefit of the customer. This was our starting point: too many integration issues, 13 SWP days and very long cycles. It was evident that in this fast-paced industry we could no longer afford this reality. We needed something that would take us beyond reducing the development lifecycles, CI and Agile methodologies. We made a fundamental difference, even changed our culture...
More and more brands have jumped on the IoT bandwagon. We have an excess of wearables – activity trackers, smartwatches, smart glasses and sneakers, and more that track seemingly endless datapoints. However, most consumers have no idea what “IoT” means. Creating more wearables that track data shouldn't be the aim of brands; delivering meaningful, tangible relevance to their users should be. We're in a period in which the IoT pendulum is still swinging. Initially, it swung toward "smart for smart...