When the immune system is functioning well, it can identify and eradicate threats to human health. When the hematopoietic (blood) system or the immune system fail to function properly, various life-threatening diseases develop, or the body is vulnerable to attack from without (bacteria, viruses) and within (cancer, autoimmune diseases/disorders, and immune-deficiency diseases). Transplantation of stem cells through myeloablative bone marrow transplantation (BMT), reduced intensity conditioning (RIC) or nonmyeloablative stem cell transplantation (NST) may be the only treatment for such conditions. The purpose of these procedures is to create a competent immune system in the patient, which will eradicate the disease from which the patient suffers.

Each of these medical technologies (BMT, RIC, NST and dendritic cell vaccines) is described below.

 

What are dendritic cell vaccines?
 
Dendritic Cells: the Master Cells of the Immune System

The immune system contains many different players, like an army. The army has two large branches (the innate immune system, which includes natural killer cells and others, and the adaptive immune system, which includes T cells, B cells and the antibodies they produce, and others). Dendritic cells are the master immune cells – the “General” of the immune army – and are responsible for mobilizing the whole immune system to attack pathogens and other threats, including malignant tumor cells. A growing array of novel immune therapies are focused on the dendritic cells – helping to activate them and educate them to mobilize the whole immune system to attack a patient’s cancer cells.

Dendritic Cell Vaccines: restoring the dendritic cells’ natural role

CTCI provides several types of dendritic cell vaccines. These are all “therapeutic” vaccines, which patients take after they already have cancer, rather than preventive vaccines. The vaccines are made from two main ingredients: dendritic cells (master immune cells) plus antigens (biomarkers) of the cancer being treated. The immune cells are obtained through a blood draw, and may be the patient’s own cells or cells from a healthy family member or other donor. The biomarkers are obtained from the patient’s own tumor tissue if such tissue is available. If not, then the biomarkers are obtained from a bank or library, or are produced recombinantly. In cancer patients, the dendritic cells have become impaired and are failing to mobilize the immune system to attack the cancer effectively. Dendritic cell vaccines are designed to activate dendritic cells so that they, in turn, can accomplish this mobilization of the whole immune system.

The vaccines are very simple to administer, generally requiring just an intra-dermal injection under the skin, like a flu shot. Dendritic cell vaccines are non-toxic, as confirmed in a very large and rapidly growing scientific literature and in published reports on clinical trial experience at many institutions in many locations, totaling nearly 2,000 patients.

 
What are BMT, RIC, and NST?
 
Stem Cells: the Foundation of the Blood and the Immune System

The source of all the blood elements and the entire immune system (white and red blood cells, platelets, lymphocytes, monocytes and the entire reticulo-endothelial cells and more) is the stem cell.

Types of Bone Marrow Transplants

Transplants may be autologous (from one's own healthy cells saved before they were damaged, or saved by cryopreservation of cord blood post natally), or allogeneic (from a family member (related) or from some other donor (unrelated)). The kind appropriate for a particular patient is determined by his/her physician based on the type of disease needing treatment, disease status, the medical condition of the patient, and the availability of a donor. Standard BMTs have a severe impact upon the patient, as described below. CTCI’s Medical Director, Dr. Slavin, has pioneered a far less toxic version of BMT, as also described below, which can be provided to a much wider range of patients. One of CTCI’s specialties is providing these milder BMTs.

As described below, the more common and severe BMT procedure is used to restore a patient's immune system after intense (lethal dose of) chemotherapy directed at a serious cancer like leukemia has destroyed the patient’s immune system. The milder version pioneered by Dr. Shimon Slavin does not involve any lethal dose of chemotherapy. It is simply directed at re-establishing an effective immune system by using a donor's healthy immune cells to augment the patient's own immune system, without the donor cells being rejected, to strengthen the patient's own cancer defense response.

Standard BMT Procedures

A fully myeloablative bone marrow transplantation procedure must be performed in a hospital under clean and isolated conditions since the patient's own immune system is destroyed by the process, and the patient has no natural defenses during the period it takes for the transplanted stem cells to create new functioning white blood cells and the immune system. This procedure is difficult for patients and can only be offered to those who can withstand the rigors involved. Myeloablative BMT is generally not offered to patients over age 60.

"Mini Transplant" – A Milder Treatment

This procedure was developed by Professor Shimon Slavin in the 1980s, followed by clinical application of RIC and NST in the 1990s. The nonmyeloablative stem cell transplant (NST) is much easier to tolerate and can be performed on an outpatient basis with few side-effects for the recipient. The procedure can be applied to elderly individuals with no upper age limit and is also suitable for use for patients in less than optimal clinical condition. In children and young adults, fertility is retained. The transplant procedure is not associated with severe side effects such as cataract formation and impairment of growth and development in children. Tests before the procedure may take a number of days, and tests to monitor the effectiveness of the procedure are performed periodically over several months. However, side effects are generally mild and hospitalization for them is infrequent.

 
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