HOUSTON & LONDON--(BUSINESS WIRE)--Cell Medica announces the treatment of the first patient world-wide to receive CMD-501, an autologous CAR-NKT therapy targeting pediatric neuroblastoma. This is the first time an engineered NKT cell therapy has been used in humans. Cell Medica is a clinical-stage biopharmaceutical company that is transforming the treatment of solid and hematological cancer by developing the next generation of CAR therapies.
This open-label Phase 1 study, GINAKIT2, is being carried out in collaboration with both Baylor College of Medicine (BCM) and Texas Children’s Hospital.
Dr. Andras Heczey, Principal Investigator, Assistant Professor, Pediatrics-Oncology at Baylor College of Medicine and Physician-Scientist, Texas Children’s Cancer Center commented: “Dosing the first patient with this novel CAR-NKT therapy is an important milestone for all pediatric patients with neuroblastoma. CAR-NKTs may offer an exciting new therapeutic option for these patients and potentially for others with solid and hematological cancers. I am extremely grateful to the patients and families participating in this ground-breaking study.”
Chris Nowers, Cell Medica’s CEO, said: “We believe that our CAR-NKT platform has a unique profile, with a potential to target solid and hematological tumors, as well as the possibility of a subsequent allogeneic “off the shelf” CAR-NKT therapy that could address some challenges of current autologous CAR-T therapies. This study marks an important step forward for Cell Medica and we are proud to be leading the development of this innovative class of next generation CAR therapies with our colleagues at BCM and Texas Children’s.”
Innovative CAR-NKT Platform
CMD-501 is based on Cell
Medica’s novel CAR-NKT platform, a next-generation technology of
engineered immune cells with enhanced functions for the treatment of
hematological and solid tumors, utilizing the unique properties of NKT
cells, a specialized type of innate lymphocytes, sharing properties of T
and NK cells. CMD-501 is the initial study from Cell Medica’s CAR-NKT
pipeline and utilizes an autologous approach. The patient’s own NKT
cells are genetically engineered with a CAR targeting GD2, a molecule
expressed on the surface of nearly all neuroblastoma cells. In
collaboration with its partners at BCM and Texas Children’s, Cell Medica
designed this CAR-NKT cell therapy to also 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.
Dr. Leonid Metelitsa, Professor of Pediatrics, Hematology-Oncology, Baylor College of Medicine and Co-Director, Neuroblastoma Program, Texas Children’s Cancer Center added: “It has been a great pleasure leading the multi-disciplinary team in the development of this versatile CAR-NKT platform. NKT cells effectively traffic to the tumor site, so expressing tumor-specific CARs in these cells ensures delivery to the site of disease for maximum efficacy. We’re now exploiting another natural feature of NKT cells, their lack of allo-reactivity and we are developing allogeneic, “off the shelf”, therapies that will further harness the unique advantages of NKT cells.”
First-in-Human Study
GINAKIT2 is a first-in-human,
dose escalation evaluation of CMD-501 in children with relapsed or
refractory (R/R) high-risk neuroblastoma, (NCT03294954).
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 BCM investigators, Drs. Heczey and
Metelitsa.
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Notes to Editors
About Neuroblastoma and GD2
Neuroblastoma is a
cancer of the sympathetic nervous system which can occur in the chest,
neck, abdomen and adrenal glands, and can metastasize to the bone marrow
and other organs. Children with low or intermediate risk neuroblastoma
can be cured through surgical intervention and/or chemotherapy, however,
at least half of all children with neuroblastoma have high risk disease,
which often requires combined surgical, radio-, immuno-, and
chemotherapy, in addition to autologous stem cell transplantation.
Patients with relapsed/refractory high-risk neuroblastoma have one of
the deadliest types of childhood cancer and a poor prognosis, with
median survival of 1-3 years.
GD2 is a molecule expressed on tumors of neuroectodermal origin, including almost all neuroblastomas, and a substantial fraction of small cell lung cancer and melanoma, with restricted expression on normal tissues, making it a good target for CAR-NKT cell therapy.
About CMD-501
CMD-501 is an innovative autologous
product in which NKT cells are genetically engineered with a CAR
targeting GD2. NKT cells are a subset of T lymphocyte 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 Cell Medica
Cell Medica is a clinical-stage
biopharmaceutical company focused on transforming the treatment of solid
and hematological cancer by developing next generation CAR-T therapies.
Developing both allogeneic and autologous therapies, the company’s
revolutionary platform engineers chimeric antigen receptors (CARs) on
Natural Killer T cells (NKTs), a subset of T lymphocytes. A robust
pipeline is being created in partnership with the Baylor College of
Medicine, Texas Children’s Hospital and University College London.
Headquartered in London, the company also has facilities in Houston and
Zurich. For further information, please visit www.cellmedica.com
About Baylor College of Medicine
Baylor
College of Medicine 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 20th 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
and Twitter.
