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Immunotherapeutics for Pediatric Solid Tumors

09:11 EDT 13 Sep 2018 | NIH

Chimeric antigen receptors (CARs) are hybrid proteins consisting of an antibody binding fragment fused to protein signaling domains that cause T-cells which express the CAR to become cytotoxic.  Once activated, these cytotoxic T-cells can selectively eliminate the cells which they recognize via the antibody binding fragment of the CAR.  By engineering a T-cell to express a CAR that is specific for a certain cell surface protein, it is possible to selectively target those cells for destruction.  This is a promising new therapeutic approach known as adoptive cell therapy.

Anaplastic lymphoma kinase (ALK, CD246) is a tumor-associated antigen that is expressed on the cell surface of pediatric neuroblastomas and some non-small cell lung carcinomas (NSCLC).  This technology from NCI's Pediatric Oncology Branch concerns the development of four (4) CARs, each comprising a different antibody binding fragment to ALK.  The CARs, known individually as ALKCAR15, ALKCAR48, ALKCAR53 and ALKCAR58, can be used in adoptive cell therapy treatment for neuroblastoma and other solid tumors which overexpress ALK or variants thereof. 

NIH Ref. No.: 
E-007-2014
Advantages: 
  • High affinity of the ALKCAR15, ALKCAR48, ALKCAR53 and ALKCAR58 increases the likelihood of successful targeting;
  • Targeted therapy decreases non-specific kiling of healthy, essential cells, resulting in fewer non-specific side-efects and healthier patients
Applications: 
  • Treatment of cancers associated with expression of ALK or variants thereof; Specific cancers include neuroblastoma, NSCLC and other solid tumors
Development Status: 

Pre-clinical (in vivo)

Updated On: 
Jul 19, 2018
Provider Classifications: 
Patent Application: 
61/865,845
Publications: 
Patent Authority: 
US Provisional
NCI
Licensing Contacts: 
Lead Inventor: 
Inventor Lab URL: 
https://med.stanford.edu/profiles/crystal-mackall
LPM FIrst Name: 
John
LPM Last Name: 
Hewes
Inv Is lead: 
LPM Phone: 
240-276-5515
LPM Suffix: 
Ph.D.
LPM Organization: 
NCI - National Cancer Institute
DTDT Classification: 
Therapeutics
DTDT Description: 
Therapeutics
Pat Filing Date: 
2013-11-06
Publication Link: 
https://jitc.biomedcentral.com/articles/10.1186/2051-1426-1-S1-P27
Publication Caption: 
DOI 10.1186/2051-1426-1-S1-P27
Publication Title: 

Orentas RJ, et al. ALK (anaplastic lymphoma kinase, CD246) specific CARs: new immunotherapeutic agents for the treatment of pediatric solid tumors.

Collaboration Sought: 
Yes
Collaboration Opportunity: 

Licensing and research collaboration

Related Invention: 
E-104-2013
E-291-2012
E Number Only: 
E-007-2014
Inventor First Name: 
Rimas
Crystal
Inventor Last Name: 
Orentas
Mackall

Original Article: Immunotherapeutics for Pediatric Solid Tumors

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