Why bone marrow transplantation?
Bone marrow transplantation is part of standard treatment for certain types of diseases, including acute myeloid leukemia and treatment-resistant acute lymphoblastic leukemia. More than 3,000 transplants have been performed here since 1982, making St. Jude one of the largest pediatric programs in the world. While bone marrow or stem cell transplants can offer a cure to children, the complexity of the procedure comes with significant risk. Researchers are working tirelessly to improve outcomes for patients as well as to make the potential cure accessible to more people.
The promising efforts of this talented team have the potential to save thousands of lives in the future.
For example, historically, in order to re-ceive a bone marrow transplant, patients had to have a marrow or stem cell donor whose blood matched theirs in several key markers. Less than 60 percent of patients will have a matched donor—usually a sibling or someone found through a registry of potential donors. St. Jude pioneered what is known as the haploidentical transplant, meaning the transplant could be performed even if the donor was only partially matched—opening the door for parents to become marrow or stem cell donors for their child.
Research from St. Jude showed that bone marrow transplant survival more than doubled in recent years for young, high-risk leukemia patients. The greatest improvements in survival were patients who had haploidentical donors. These findings are truly a game-changer for children with leukemia: Bone marrow transplants are now possible for children without matched donors—who otherwise had no hope for a cure.
The remarkable advancements in this field over the past 30 years have also paved the way for other cellular therapies. These exciting, pioneering efforts—including immunotherapy, gene therapy and the use of gene editing—are widely believed to be the next frontier in medicine.
Here’s a glimpse of some of the research efforts currently underway:
- Researchers at St. Jude are currently working in our laboratories to create an immunotherapy treatment for acute myeloid leukemia, which is very rare in children and has a very poor prognosis. This type of experimentation involves quite a bit of trial and error because of the many steps involved in the process, to determine if a treatment is not only effective but also safe for children. Before this work can be translated into a clinical trial, scientists must figure out the ideal structure of CAR-T cells to use in the treatment, as well as how to best prepare a patient in advance of treatment, and how to avoid inducing a dangerous reaction in patients. They are hopeful that a clinical trial could open at
St. Jude by the end of 2019.
- St. Jude researchers are studying how immunotherapy could be used to treat patients with solid tumors. Unfortunately, outcomes for children with recurrent or metastatic solid tumors are poor. St. Jude scientists are investigating the use of a particular form of CAR-T cell to save these children? While this work is currently taking place in labs, the ultimate goal is to move the research to the clinic.
- Scientists are investigating ways to genetically engineer CAR-T cells to treat children with brain tumors. Brain tumors are devastating—even when cured, children suffer from terrible long-term effects from the cancer and its treatments. Researchers are hoping to use gene editing to fix a problem in the patients’ DNA and then return the cells to fight the tumor. Using a process called “knock out and knock in,” the team is experimenting with using gene editing technologies to silence the problematic gene and insert something better into the cell.