Since stem cell treatments first became a possibility in medicine during the 1960s, Duke University Medical Center researchers and physicians have played a major role in advancing their increasingly complex uses.
In recent years, DUMC has worked to create a number of unique methods to apply stem cells to the treatment of cancer and rare diseases.
“Stem cells were once used as a last-ditch effort,” said Dr. Keith Sullivan, director of Duke’s Center for Cancer Outcomes and Research. “But, with Duke’s reputation for the treatment of rare and complex diseases, things are rapidly changing.”
Sullivan is lead investigator of the trial study Scleroderma: Cyclophosphamide or Transplantation, which treats patients suffering from severe forms of the rare skin disease commonly known as systemic sclerosis.
The trial compares stem cell treatment to high-dose drug treatment using cyclophosphamide, a chemotherapy agent used to treat chronic immunosuppression.
Sullivan’s SCOT trial approach, however, is merely one of many at Duke that applies variations to the traditional approach to stem cells, which once required perfect matches with varying results.
Dr. Rebecca Buckley, professor of allergy and immunology in Duke’s department of pediatrics, studies treatments for severe combined immunodeficiency disease, or “bubble boy disease,” which is caused by abnormal changes in the development of the T cells of the immune system.
Buckley has utilized methods that rid bone marrow of T cells, which are regenerated upon implantation.
“Unlike bone marrow [stem] cells requiring perfect matches, our approach doesn’t require a perfect match,” said Buckley, who has utilized stem cell transplants since the 1980s. “The 34 patients we’ve lost over the years were due to viral infection.”
Buckley said methods of “manipulating stem cells to avoid the hindrance of the perfect match” are prevalent among Duke researchers.
“We have one of the largest stem cell reserves in the nation,” Buckley said.
While scientists lobby for increased reserves of available stem cells-through increased blood donation in various forms-approaches to stem cell treatment for cancers and rare diseases are becoming increasingly complex.
Joanne Kurtzberg, director of the Pediatric Bone Marrow and Stem Cell Transplant Program and the Carolinas Cord Blood Bank at Duke, said the use of cord blood stem cells presents growing opportunities for children and some adults suffering from various cancer types and children with rare immune diseases.
Cord blood cells present alternatives to what were once “dead ends” in treatment options for some conditions, she said.
“For patients lacking sufficient bone marrow matches, this is their only choice,” Kurtzberg said. Stem cells from cord blood are currently used to treat patients with cancers, certain genetic blood disorders and metabolic diseases.
Kurtzberg said that while it is likely that many types of stem cells will be used therapeutically over the next few decades, it is important to conduct research to determine the optimal ways to utilize them as therapeutic agents.
“While there are limited options for any one condition, approaches are changing,” she said.