Curing Bubble Babies
Dateline: 02/25/99
Doctors have successfully treated babies born with severe combined immune deficiency (SCID), a fatal and rare immune disease, through a unique form of bone marrow transplant. Ninety-five percent of these transplants, performed within the first four months of life, are successful. SCID occurs in approximately 1 in every 500,000 births and is particularly devastating, leaving its sufferers with virtually no immune system. As a result, most patients succumb to any number of infections within the first year of life. One highly publicized case was David Vetter, depicted on TV and in the press as the "Bubble Boy," who lived in a germ-free environment for almost twelve years.
During the transplant, T cells are removed from the donor marrow lessening the chance of graft-versus-host disease. Since T cells are not present, the marrow is less likely to recognize its new host as "foreign," and recipients don't have to take antirejection drugs. The probability of a match rises significantly. Similar to a technique discussed in The Mismatch, a full genetic match is no longer necessary.
Doctors found that an early transplant greatly reduces the possibility of infection both during and after the transplant and allows SCID babies to develop a healthy and fully functioning immune system before any opportunistic infections can set in. After the transplant, any follow-up can be treated on an outpatient basis eliminating the necessity for germ-free environments and chemotherapy.
The procedure is also cost effective. The cost of the transplant is minuscule compared to what could be spent later in the child's life for a transplant that will likely be unsuccessful.
Doctors note that early diagnosis is the key. Babies born with the disease need to be treated during that critical first few months for the procedure to be successful. Even with the small window of time for successful treatment, babies are rarely screened though the estimated cost for screening is only $50. Researchers are hopeful that with the success of the new technique, testing will become more pervasive.
What do you think? Might other immune disorders be cured by early intervention? If so, how much of this technique do you think is applicable? Come over to the Biology Forum and share your thoughts, opinions, and feelings. 'Til next time...
For related information, see:
Immune System
Imagine trying to defend a fortress that is constantly under attack from outside invaders. Now you have an idea of the immense task that our natural defense system must undergo to keep us healthy.
