ForPatients and Families
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, according to the US National Cancer Institute, 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.
Engineered NKT Cell Therapies for Cancer
Cancers are usually characterized by rapid cell growth. The earliest cancer drugs were derived from agents used in chemical warfare. These drugs, now known as chemotherapy, were designed to kill all fast-growing cells. Unfortunately, many normal cells in the body grow fast, so chemotherapy drugs are often non-specific and 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. 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 several advantages:
- The engineered NKT cells have a specific targeting molecule, termed a chimeric antigen receptor or CAR, which helps the cells find and kill cancer cells.
- The engineered NKT cells have a prolonged life and can replicate in the patient, providing long term protection against newly developing cancer cells, leading to longer responses and potentially preventing relapses.
- In addition to the CAR, the engineered NKT cells have natural tumor fighting ability thanks to the invariant T cell receptor present on these cells, which recognizes and directly kills some tumors and also kills inhibitory cells within the tumor, leading to immune activation against the tumor.
- The engineered NKT cells can home to sites of solid tumors, which have historically been difficult to treat with other immune cellular therapies.
Neuroblastoma is a cancer occurring in children of the sympathetic nervous system which can occur in the chest, neck, abdomen and adrenal glands, and which can metastasize (spread) 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 hematopoietic stem cell transplantation, as described by a recent news interview with a neuroblastoma patients mother and Dr. Heczey at Texas Children’s Hospital
GD2 is a molecule expressed on almost all neuroblastomas, and on some other tumors, 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 NKT cell platform technology in combination with genetically engineered CARs. A clinical trial is currently open to recruitment at Baylor College of Medicine and Texas Children’s Hospital, Houston, Texas, USA.
In addition, travel assistance is available to qualifying families, through a grant awarded by Alex’s Lemonade Stand Travel Care program
Patients & families interested in participating in the neuroblastoma trial can register at https://www.neuroblastomastudy.com/ and a member of the clinical study team will contact you about next steps.
See our press release regarding the first dosed patient here.
Diffuse Large B Cell Lymphoma
Lymphoma is a cancer of a part of the immune system called the lymph system. There are many types of lymphoma. One type is Hodgkin lymphoma. The rest are called non-Hodgkin lymphomas (NHLs). NHLs begin when a type of white blood cell called a T lymphocyte or a B lymphocyte, becomes abnormal. The cell divides again and again, making more and more abnormal cells. These abnormal cells can spread to almost any other part of the body.
Diffuse large B-cell lymphoma (DLBCL) is the most common type of NHL worldwide, accounting for up to one-third of patients with newly diagnosed NHL. DLBCL is an aggressive (fast-growing) NHL that affects B-lymphocytes. B-lymphocytes (or B cells) are a type of white blood cell that make antibodies to fight infections and which are an important part of the lymphatic system. Although it can occur in childhood, the occurrence of DLBCL generally increases with age, and most patients are over the age of 60 at diagnosis. DLBCL can develop in the lymph nodes or in “extranodal sites” (areas outside the lymph nodes) such as the gastrointestinal tract, testes, thyroid, skin, breast, bone, brain, or essentially any organ of the body. It may be localized (in one spot) or generalized (spread throughout the body). Despite being an aggressive lymphoma, DLBCL is considered potentially curable, although many patients will respond to initial therapy and then experience a relapse at a later date.
Acute Lymphocytic Leukemia and Chronic Lymphocyte Leukemia
Leukemias are cancers that start in cells that would normally develop into different types of blood cells. Most often, leukemia starts in early forms of white blood cells. There are several types of leukemia, which are divided based mainly on whether the leukemia is acute (fast growing) or chronic (slower growing), and what type of blood cell that it comes from. Knowing the specific type of leukemia helps doctors better predict each person’s prognosis (outlook) and select the best treatment.
Acute lymphocytic leukemia (ALL) is also called acute lymphoblastic leukemia. “Acute” means that the leukemia can progress quickly, and if not treated, would probably be fatal within a few months. “Lymphocytic” means it develops from early forms of lymphocytes, a type of white blood cell. ALL starts in the bone marrow (the soft inner part of certain bones, where new blood cells are made). Most often, the leukemia cells invade the blood quickly. They can also sometimes spread to other parts of the body, including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles (in males). ALL can occur in children and adults; ALL in children is often curable but is more difficult to treat in adults. Patients with relapsed or refractory ALL generally have limited effective treatment options.
Chronic lymphocytic leukemia (CLL) is the most common leukemia in adults. It is a type of cancer that starts in cells that become certain white blood cells (called lymphocytes) in the bone marrow. The cancer (leukemia) cells start in the bone marrow but then go into the blood. In CLL, the leukemia cells often build up slowly. Many people do not have any symptoms for at least a few years. But over time, the cells grow and spread to other parts of the body, including the lymph nodes, liver, and spleen. There are new targeted agents that are safe and effective in many cases of CLL, but some patients relapse after treatment with these new molecules and these patients have limited treatment options.
CD19 is a molecule expressed on B lymphocytes and their precursors of the immune system. CD19 is also expressed on most DLBCL, ALL and CLL tumor cells. CD19 is a good target for CAR-NKT cell therapy because it is widely expressed on these tumors. Because it is expressed on normal B cells, the CAR-NKT cell therapy is expected to kill the normal B cells as well as the tumor. Luckily the function of normal B cells can be replaced temporarily with infusions of antibodies (which normal B cells make). After the tumor is killed by the CAR-NKT cells and the CAR-NKT cells dissipate from the body, then the normal B cells grow back.
We are developing CMD-502 for the treatment of relapsed, refractory
DLBCL, ALL and CLL. CMD-502 is an allogeneic (off the shelf) cell therapy targeting CD19, which utilizes our NKT cell platform technology in combination with genetically engineered CARs. An advantage of the off the shelf CAR-NKT product is that the therapy will be immediately available to sick patients and they will not need to undergo a collection of their own cells followed by a several week long manufacturing process, which is the standard, traditional process for autologous immune cell therapies. We are planning to open a clinical trial of CMD-502 in conjunction with our partners at Baylor College of Medicine in Houston, Texas, USA see our pipeline for current status.