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Ray of Hope Article Published

Article by K.Michelle Scott published in "Your Health" from the Medical Association of Georgia, number 1 volume 1, Spring 1997

Results of Grady Memorial Hospital, Emory University collaboration on bone marrow transplants for sickle cell anemia sufferers are encouraging

Six years ago, researchers initiated experimental bone marrow transplantations in 22 children throughout the United States and Europe who suffered from sickle-cell disease. Today, scientists . say 16 of those children are cured of the disease. Imagine that each day your new-born daughter must receive medication regularly to reduce the risk of meningitis and pneumonia-causing bacterial infections until she’s 5 years old. Once she becomes a toddler, doctors say your daughter is 300 times more likely to have a stroke than a healthy child her age. You know that with each stroke comes the threat of permanent damage to her speech, vision and learning abilities.

To reduce the risk of strokes, your daughter begins monthly transfusion therapy. These blood transfusions reduce the risk of stroke by 90%. Unfortunately, the transfusions also cause an unhealthy buildup of iron, for which she receives a shot each day. As she grows older, your daughter begins to complain of pain and swelling in her hands and feet. She also develops ulcers around her ankles, and of blood and oxygen to vital tissue and she suffers from excruciating "pain crises" The pain occurs when sickle shaped red blood cells block the flow of blood and oxygen to vital tissue and organs. Over time, life-threatening tissue and organ damage is likely to occur.

Your daughter is a victim of sickle-cell disease. Fortunately, researchers have recently identified hydroxyurea (a drug approved for the treatment of cancer) to be an effective pain medication for sufferers of the disease. Unfortunately, hydroxyurea can only be used by adult sufferers experiencing severe bouts of pain. Researchers have also determined that the drug may pose the risk of leukemia and other side effects. And while hydroxyurea may be an effective medication for the pain, it’s no cure for the deadly disease.

However, after years of painstaking research, scientists may have found a lead to the cure they’ve been looking for. Following a four-year study, researchers recently determined that bone marrow transplants present a promising cure for sickle-cell disease. Unfortunately, the transplant procedure has its drawbacks. It’s extremely risky, reserved for the very sickest of patients and costs nearly $200,000.

Despite the high risks and high costs, bone marrow transplantation currently offers the only ray of hope that a cure exists for many sufferers of sickle-cell disease who, like your daughter, have an average life expectancy of 42 to 48 years.

The bone marrow link

Sickle-Cell disease is an inherited blood disorder which causes red blood cells to become hard, sticky and crescent-shaped. Because the blood cells are awkwardly shaped, they tend to pile up and block the blood vessels. When blood vessels are blocked, the blood and oxygen supply to vital tissue and organs is blocked. This blockage causes the pain, strokes, organ damage and, ultimately, death associated with the disease.

The link between bone marrow and sickle-cell disease is simple. Bone marrow makes red blood cells. The bone marrow of healthy individuals produces normal red blood cells — soft, smooth and round. But some individuals have bone marrow which contains sickled blood cells, resulting in sickle-cell disease. In their search for a cure to the disease, scientists conducted their study to determine if replacing unhealthy bone marrow (which produces sickled cells) with healthy bone marrow (which produces normal red blood cells) would cure a sickle- cell patient.

The researchers established three strict criteria in the selection of patients for their study. The patients had to be younger than 16 and severely ill as a result of the disease. Also, the young patients had to have a brother or sister whose tissue type matched theirs; these siblings would become bone marrow donors. Finally, the researchers selected 22 patients, 3 to 13 years old, all of whom suffered a serious complication or chronic pain episodes from the disease.

In preparation for the bone marrow procedure, the young patients had to undergo a series of radiation and chemotherapy treatments to kill off their own bone marrow and suppress their immune system. Although these treatments were often painful, they were necessary to ward off a complication (graft vs. host disease) in which the donor’s bone marrow attacks the recipient’s.

Now, some six years since the procedure was initiated, researchers report that 16 of the children treated in their pilot study are alive and show no evidence of sickle-cell disease. The Sickle-Cell Disease Foundation of America Inc. (SCDAA) reports that one patient died from a brain hemorrhage three months after the procedure; a second child died a year later from complications related to graft vs. host disease, and four children rejected the transplant and their sickle-cell disease recurred.

Emory leads in transplants

Through the combined efforts of Emory University School of Medicine and the Georgia N.I.H. Comprehensive Sickle Cell Center at Grady Health System, two Georgia children participated in the initial transplantation study. Since that time, Emory University Hospital has become a leader in pursuing this revolutionary treatment for the disease. According to Dr. James Eckman, an Emory University professor of medicine and director of the Grady-based center, doctors at Emory recently performed a third transplant procedure on a young Georgia patient and are in the process of preparing for a fourth procedure. Eckman said, "Emory was one of the first participating unit centers. To date, it has performed more bone marrow transplants [for sickle-cell victims] than any other center."

So far, all Georgia participants in the procedure have been Grady patients who were screened for selection by Eckman’s center. Once a patient qualifies, the procedure is conducted at Emory University Hospital. The success rate for Georgians has been encouraging.

"I would say the first two patients are definitely cured. The third patient appears to have been cured. To be certain, we’ll need to do a six-month follow-up on the third patient:’ said Eckman.

Not suitable for all

Despite these promising results, a recently published SCDAA report suggests that the bone marrow procedure, as it is, is not the cure they’ve been waiting for. According to the report, "If the bone marrow transplantation is successful, it is a cure

"However, SCDAA cites several drawbacks to the treatment which indicate that much more work needs to be done before claiming that the procedure is a general cure for the disease) the report said.

In addition to the high costs, bone marrow transplantation is an extremely risky procedure. "It’s reserved for the sickest of patients who have a perfect tissue-matched sister or brother as the marrow donor. It’s estimated that only 20 percent of African American patients have a suitable sibling donor) reports SCDAA. Secondly, the Association cites the possibilities of infertility and growth retardation and the potential development of graft vs. host disease and cancer as possible risks of the procedure.

Eckman agrees that bone marrow transplantations are risky and that precautions must be taken in selecting patients forte procedure. "Bone marrow transplantation is a very aggressive form of therapy with a high morbidity and mortality rate. The chemotherapy and radiation treatments required for the procedure also present later complications: 10 percent of the children die from complications. It’s not a treatment for all children with sickle-cell," he said.

While researchers acknowledge tat much work needs to be done to determine the yet undiscovered risks of the procedure, they also realize that, to date, bone marrow transplantation is the most promising treatment towards finding a cure for the disease. And for the 16 children who have been declared cured of sickle-cell as a result of the treatment, bone marrow transplantation is a godsend.

Early detection is best

According to the National Heart, Lung and Blood Institute, nearly 80,000 people suffer from some form of sickle-cell disease. And contrary to popular belief, sickle-cell disease is not restricted to persons of African descent. The Sickle Cell Foundation of Georgia (SCF of GA) reports that although most cases of sickle-cell disease occur among blacks and Hispanics from the Caribbean, it also affects people of Arabian, Greek, Italian, Turkish and southern Asian ancestry. The exposure of this myth is important, since early detection of the disease may prolong a child’s life.

In 1987, a National Institute of Health panel recommended that all babies be screened for sickle-cell disease at birth. However, not all states heed their warning. According to SCF of GA, "To date, some 30 states are testing newborns for sickle—cell disease While most sickle-cell sufferers still await a general cure for the disease, there are precautions which should be taken to ease their wait. According to D. Jean Brannan, executive director of SCF of GA, early detection offers sufferers the best opportunity to begin treatment for symptoms of the disease. "There are nearly 600 abnormal hemoglobin types:’ says Brannan, "of which sickle-cell disease is one. Everyone should be screened and learn the results of their screening:’

In addition to hemoglobin screenings, SCF of GA offers a variety of support services to improve the quality of life for victims of sickle-cell. As well as educational assistance involving tutoring and scholarships, the Foundation also hosts an annual summer retreat at Camp New Hope for children with sickle-cell disease.

For more information about the disease, treatments and support programs, victims and their families are encouraged to contact the 24-hour information line of Georgia’s N.I.H. Comprehensive Sickle Center at Grady Health System at (404) 616-3572 or the Sickle Cell Foundation of Georgia at 1-800-326-5287. .

For More Information Contact:

Sickle Cell Information Center
P.O.Box 109, 80 Butler Street Atlanta, GA 30335
Tel: 404-616-3572
FAX: 404-616-5998
Internet: aplatt@emory.edu

Send mail to aplatt@emory.edu with questions or comments about this web site.
Copyright © 1997 Sickle Cell Information Center
Last modified: January 29, 1998