-
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 & LONDON–(BUSINESS WIRE)–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.
– ENDS –
Notes to Editors
About GINAKIT2
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.
About ASGCT
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).
Contacts
Cell Medica
Chris Nowers, CEO
+44 20 7554 4070
Kevin
S. Boyle Sr., CFO
+1 832 581 4476
[email protected]
Instinctif Partners (Media enquiries)
Tim Watson/Sue Charles
[email protected]
+44
20 7457 7861
