Cell Medica collaborators, Baylor College of Medicine and Texas Children’s Hospital, present positive early patient data from CAR-NKT neuroblastoma trial at American Society of Gene & Cell Therapy 22nd Annual Meeting

April 30, 2019
  • Early data show highly innovative chimeric antigen receptor-natural killer T cell (CAR-NKT) therapy homing to and shrinking solid tumors in neuroblastoma patients
  • GINAKIT2 Phase 1 trial is evaluating GD2 targeted CAR-NKT therapy, CMD-501, in children with relapsed or refractory (R/R) high risk neuroblastoma

HOUSTON and LONDON – April 30 2019 – Cell Medica, a leader in next-generation cellular immunotherapies for the treatment of cancer, today announced that its collaborators from Baylor College of Medicine and Texas Children’s Hospital presented the latest positive progress in the GINAKIT2 trial for children with R/R high risk neuroblastoma at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Washington, D.C.*

Patients were infused with an autologous CAR-NKT therapy, engineered to target GD2+ neuroblastoma cells. Early data from the first two patients at the lowest dose level (3 x 106 CAR-NKT cells/m2) showed substantial in vivo expansion of CAR-NKT cells and subsequent infiltration of CAR-NKT cells into both the solid tumor mass and bone marrow. There was also strong radiological evidence of extensive tumor regression at 4 weeks post infusion in one patient, with further regression observed at 8 weeks. No significant treatment associated toxicities, including cytokine release syndrome or neurotoxicity, were observed.

The CAR construct used in the GINAKIT2 study was also engineered to induce expression of the cytokine, IL-15. Data from in vivo mouse models, also presented at ASGCT, showed enhanced persistence of the CAR-NKT cells, which is thought to be important for long-term efficacy.

Dr. Andras Heczey, Principal Investigator, Assistant Professor, Pediatrics-Oncology at Baylor College of Medicine and Physician-Scientist, Texas Children’s Cancer Center commented: “Although it is early in the study, we are pleased to see evidence supporting the activity of CAR-NKT cells. We are looking forward to bringing additional patients onto the trial and treating at higher dose levels.”

Dr. Leonid Metelitsa, Professor of Pediatrics, Hematology-Oncology, Baylor College of Medicine and Co-Director, Neuroblastoma Program, Texas Children’s Cancer Center added: “These are early data but, nevertheless, they support the fundamental premise of CAR-NKT cells to effectively home to the site of disease in solid tumors and kill tumor cells. We hope these properties of CAR-NKT cells can be exploited to treat other malignancies and not only using patient-derived cells, but also in the allogeneic (off-the-shelf) setting.”

Chris Nowers, Cell Medica’s CEO, said: “Whilst these data are preliminary, it is exciting to see the first clinical evidence of a CAR therapy based on natural killer T cells reducing a solid tumor in a neuroblastoma patient, especially at the lowest dose level. This study will continue to recruit patients and build a clearer picture of the potential of CAR-NKT therapy. Concurrently we are also finalizing additional studies that will explore the potential of our innovative CAR-NKT platform in an off-the-shelf setting.”

*The data were presented in two oral presentations “Harnessing Natural and Engineered Properties of iNKT Cells for Adoptive Cancer Immunotherapy”, and “NKT Cells Co-expressing a GD2-specific Chimeric Antigen Receptor and IL-15 Show Enhanced In Vivo Persistence and Antitumor Activity Against Neuroblastoma”, given by Dr. Metelitsa and Dr. Heczey respectively.


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Notes to Editors


GINAKIT2 is a first-in-human, dose escalation evaluation of CMD-501 in children with relapsed or refractory (R/R) high risk neuroblastoma (NCT03294954) and is the first ever human trial of a genetically engineered Natural Killer T (NKT) cell therapy. Neuroblastomas occur primarily in children and account for 7-10 percent of all pediatric cancers. Ninety percent of patients are younger than 5 years at diagnosis. R/R high risk neuroblastoma is one of the deadliest types of childhood cancer and the current median survival is around 1-3 years. Almost all neuroblastomas express GD2, which is targeted by CMD-501. This study is supported by a grant from Alex’s Lemonade Stand Foundation (ALSF), awarded to Baylor College of Medicine investigators, Drs. Heczey and Metelitsa.

Find out more at: https://www.neuroblastomastudy.com/

About CMD-501

CMD-501 is an innovative autologous product in which NKT cells are genetically engineered with a Chimeric Antigen Receptor (CAR) targeting GD2.  NKT cells are a subset of T lymphocytes with the cytotoxic and anti-tumor properties of conventional T cells, but with other biological attributes that are expected to improve their ability to attack tumors. GD2 is a molecule expressed on the surface of most neuroblastoma cells.

In collaboration with its partners at BCM, Cell Medica has engineered a GD2-specific CAR construct that is additionally designed to secrete the cytokine IL-15, which has been shown in pre-clinical studies to increase the persistence of CAR-NKT cells and improve their efficacy within the immunosuppressive tumor microenvironment.  CMD-501 is an autologous product, meaning that each patient’s own cells are collected, modified and activated outside the body, and then infused back into the same patient. However, NKT cells also have significant potential for so-called off-the-shelf use, where cells from a healthy donor could be prepared in large quantities in advance and used to treat many different patients. Cell Medica is collaborating with BCM to bring an off-the-shelf CAR-NKT cell product into the clinic in the near future.


The American Society of Gene and Cell Therapy (ASGCT) Annual Meeting provides an international forum where the latest gene and cell therapy developments are presented and critically discussed. As the leading American conference focusing solely on gene and cell therapy, ASGCT’s annual meeting brings together more than 3,400 professionals including scientists, physicians, and patient advocates.

Find out more at: https://annualmeeting.asgct.org/am19

Media registration is open at: https://annualmeeting.asgct.org/forms/media_registration.php

About Cell Medica

Cell Medica is a clinical-stage biopharmaceutical company focused on transforming the treatment of solid and hematological cancers by developing next generation chimeric antigen receptor-natural killer T cell (CAR-NKT) therapies. Developing a portfolio of primarily allogeneic therapies, the company’s revolutionary platform engineers CARs on invariant NKT cells (iNKTs), a subset of T lymphocytes. A robust pipeline spanning both hematological and solid tumors is being created in partnership with Baylor College of Medicine and Texas Children’s Hospital. Headquartered in London, UK, Cell Medica also has facilities in Houston, US and Zurich, Switzerland.

For further information, please visit www.cellmedica.com. Follow Cell Medica on Twitter and LinkedIn 

About Baylor College of Medicine

Baylor College of Medicine (www.bcm.edu) in Houston is recognized as a premier academic health sciences center and is known for excellence in education, research and patient care. It is the only private medical school in the greater southwest and is ranked 16th among medical schools for research and 5th for primary care by U.S. News & World Report. Baylor is listed 21st among all U.S. medical schools for National Institutes of Health funding and number one in Texas. Located in the Texas Medical Center, Baylor has affiliations with seven teaching hospitals and jointly owns and operates Baylor St. Luke’s Medical Center, part of CHI St. Luke’s Health. Currently, Baylor trains more than 3,000 medical, graduate, nurse anesthesia, physician assistant and orthotics students, as well as residents and post-doctoral fellows. Follow Baylor College of Medicine on Facebook (http://www.facebook.com/BaylorCollegeOfMedicine) and Twitter (http://twitter.com/BCMHouston).

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