Innovations in Stem Cell Transplant Allow Parent to Become Donor
Cameron Dykstra was born without an immune system. The stem cells in the infant’s bone marrow did not produce functioning lymphocytes -- cells that play a crucial role in fighting disease. But within a week of coming to the University of Chicago Medicine Comer Children’s Hospital, his father’s donated blood stem cells – purified using a novel technique -- were transplanted to Cameron where they took over the task of defending the infant’s body against harmful bacteria and viruses.
The baby’s parents, Rich Dykstra and Emily Evans, brought four-month-old Cameron to the Comer Emergency Department on October 12, 2007, concerned about projectile vomiting and crackling in his lungs. Using sophisticated genetic tests, doctors quickly diagnosed Cameron with severe combined immunodeficiency syndrome (SCID), commonly known as "bubble boy disease." He had already contracted pneumocystis pneumonia and was gravely ill when he arrived at the hospital.
Severe combined immunodeficiency syndrome is a rare disorder that strikes mostly boys and is characterized by a genetic defect in the body’s lymphatic system. Lymphocytes develop from the blood stem cells of the bone marrow. In children with SCID, the lymphocytes are absent or nonfunctional and their bodies cannot fight infection. Without a stem cell transplant, children with SCID usually die from infections in their first or second year of life.
"Cameron’s case was an emergency. The sooner we did the transplant, the sooner he would be protected from infections," says John Cunningham, MD, chief of the Section of Pediatric Hematology/Oncology and a stem cell transplant expert. "Because he didn’t have any siblings, who are sometimes a full match, we determined his father was the best possible stem cell donor."
Recent innovations in stem cell transplant techniques enable scientists at the University of Chicago and a few other centers around the country to purify the stem cells of parents (who are "half" matches for their children) making them acceptable for donation. In the past, stem cells donated from parents often rejected their new environment, resulting in graft vs. host disease (GVHD). A serious complication, GVHD can cause skin rashes, diarrhea and liver damage. Purification significantly reduces the risk of GVHD and other complications of stem cell transplant. This unique approach, currently being studied in Dr. Cunningham’s laboratory, is improving success rates for many children, including those who require stem cell transplants to treat leukemia, lymphoma or other diseases.
In the 1970s and 1980s, SCID patient David Vetter spent his early childhood in an isolation bubble necessary to protect him from germs and infections. The world watched as doctors attempted to cure the "boy in the bubble" with the first unmatched stem cell transplant. David did not survive the transplant, but his life in the bubble contributed much to the scientific understanding of severe combine immunodeficiency syndrome.
"We learned a lot from David Vetter’s treatment and have come a long way in the decades since that time," says Dr. Cunningham. "Stem cell transplant, even from half-matched and unmatched donors, is now curing these children and allowing them to live normal lives."
Following transplant, Cameron was in the hospital for five months. Experts in several pediatric subspecialties including hematology/oncology, gastroenterology and dermatology observed and treated him for rashes, fevers, diarrhea and other conditions that could be signs of or caused by GVHD. Infectious diseases specialists monitored him for infections. Physical, occupational and play therapists worked with Cameron to help him along developmentally since his movements and social interactions were restricted in his isolation room.
In March 2008, Cameron was healthy enough to go home. He has regular outpatient visits, but has been hospitalized only once since his discharge. At each visit, he is measured and he has been consistently growing taller and gaining weight.
"Cameron’s father’s stem cells are vigorous and his lymphocytes are functioning normally," reports Dr. Cunningham one year after discharge from the hospital.
The toddler’s mom, Emily, agrees: "Those 72 million stem cells that Cameron got from Rich worked! Sometimes I wonder how we made it through. But Cameron is a very strong-willed child and a fighter. Now he is a busy little boy. He is non-stop, all day long."
Cameron Dykstra’s Pediatric Medical Team
Hematology/Oncology/Stem Cell Transplant