Through innovative technologies and collaboration with leading academic institutions, we aim to harness the natural power of the human immune system for the treatment of cancer.
Despite major advances in the prevention, diagnosis and treatment of cancer, around 14.1 million patients are diagnosed and 8.2 million die each year worldwide. These statistics powerfully illustrate the need for new breakthroughs in the fight against cancer.
Cancers are usually characterized by rapid cell growth. In the early years of cancer research, this rapid growth was used as a marker to recognize cancer cells. Drugs, developed to target and kill all fast-growing cells (chemotherapy), often lead to undesired side effects, such as gastrointestinal toxicity, hematological toxicity and other problems, such as hair loss. In some cases, toxicity from conventional chemotherapy drugs can cause serious harm or even be fatal.
Thanks to major advances in the understanding of the molecular biology of cancers specific cellular features have been discovered that are associated with certain cancers. These discoveries have allowed for the development of targeted cancer therapies, for example engineered T cell products. Targeted cancer therapies generally have fewer side effects compared to older chemotherapies.
We take this tactic even further by using engineered immune cells (NKT cells) to target specific cancer molecules. This approach may bring two advantages:
Cancer cells express molecules either inside the cell or on the surface of the cancer cells that may be used as targets by engineered NKT cells. The challenge for us is to train the engineered NKT cells to recognize and exclusively target the cancer cells and not recognize and attack normal tissues in the patient.
We basically use two different approaches to generate engineered NKT products that can recognize and specifically targeting cancer cells.
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.
GD2 is a molecule expressed on 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.
We are developing CMD-501 for the treatment of high risk, relapsed, refractory neuroblastoma in children. CMD-501 is an autologous cell therapy targeting GD2, which utilizes our natural killer T (NKT) cell platform technology in combination with genetically engineered chimeric antigen receptors (CARs). A clinical trial is currently open to recruitment at Baylor College of Medicine and Texas Children’s Hospital, Houston, Texas.
More information about the Neuroblastoma Trial can be found here https://www.bcm.edu/research/clinical-trials/h-41033
See our press release regarding the first dosed patient here.