Specific Treatment Options

The UCSF Pediatric BMT Program offers a variety of specific treatment options for children whose own bone marrow stem cells are abnormal or defective, who have cancer that may be cured with a bone marrow transplant, or who have a genetic or inherited metabolic disease that affects many organs in the body. These treatment options, or protocols, are all considered research protocols since the patients who participate are treated in a uniform manner, and the results, after being collected and evaluated, may be published in a scientific journal.

Disease protocols

At UCSF, we offer treatment protocols for specific diseases. Among the specific disease protocols are the following:

• leukemia;
• solid tumors and brain tumors;
• high-risk neuroblastoma;
• severe combined immunodeficiency disease (SCID);
• genetic (metabolic) diseases;
• marrow stem cell defects.

Reduced toxicity protocols

Because the risk of death from toxicity in a standard BMT is so great, while BMT remains the best and sometimes only treatment option for some diseases, the Pediatric BMT Program at UCSF has developed novel protocols minimizing toxicity in bone marrow stem cell transplantation. Among the reduced toxicity protocols offered are the following:

• non-myeloablative conditioning;
• busulfan pharmacokinetics;
• in utero transplantation.

Alternative donors

Unfortunately, the majority (>70%) of children with diseases that could be cured with a bone marrow transplant will not have a matched related donor, the optimal donor for most transplants. For this reason, we have developed several clinical programs at UCSF to evaluate the benefit of using alternative donors when an optimal donor cannot be found. The three alternative donor programs are the following:

• Unrelated Donor Program;
• Umbilical Cord Blood Program;
• Haplocompatible Donor Program.

The question often is asked as to whether a relative other than a sibling can be used as a donor for BMT. There is a very small chance (1%) that a parent may be closely matched with his or her child and can be used in the same manner as a matched sibling. In general, the chances that a sufficiently matched relative such as a cousin, uncle or grandparent will be found are so rare that the time and energy required usually do not warrant a search. However, under some circumstances, for example, among certain ethnic groups or when the parents’ families are related, it may be worth a careful extended family search. This will be addressed at the initial consultation evaluation clinic visit.

Fighting cancer post-transplant

Unfortunately, in spite of very intensive chemotherapy and total body irradiation in the conditioning regimens prior to transplant for most cancers, the relapse rate remains very high, anywhere from 10-50%.

We are evaluating potential approaches to reduce the post BMT relapse rate in patients with leukemia or high risk neuroblastoma. One approach for leukemia is to utilize donor T lymphocyte infusions (DLI) at various times post-BMT. This protocol also involves special monitoring for any evidence of early relapse. Future studies will evaluate monoclonal antibodies which have been found to selectively target leukemia cells and cytokines, natural chemicals in the body that manipulate the immune system.

For patients with high risk neuroblastoma, an autologous transplant results in a cure for approximately 40%. However, the relapse rate post-BMT is 50%. In order to reduce this relapse rate children may be eligible for treatment with special monoclonal antibodies that target any remaining neuroblastoma cells with a toxic chemical that is attached to the antibody. This and other similar approaches are in the early stages of evaluation.