More MS news articles for August 1999

Girl will get experimental transplant

OHSU approves a stem cell procedure for Mollie Hauck, 9, that is intended to cure her juvenile rheumatoid arthritis

Thursday, August 26, 1999

By Oz Hopkins Koglin of The Oregonian staff

Nine-year-old Mollie Hauck is about to become a pioneer.

The round-faced little Canby girl is slated to be one of the first patients in the nation to have a stem cell transplant in hopes of curing an autoimmune disease.

Mollie has severe juvenile rheumatoid arthritis, an inflammatory disorder in which the immune system turns against the body, stunts growth and causes pain. That's why she is at least a head shorter than friends her age and why she walks slowly with a limp. Her body is bloated because of the medicines she's had to take since she was 3 years old. Periodically, she has to have multiple injections in her joints, as many as 26 at a time, but she is not getting better.

Juvenile Rheumatoid Arthritis

What it is: Arthritis refers to a group of diseases that cause pain, swelling, stiffness and loss of motion in the joints. Juvenile rheumatoid arthritis strikes children 16 or younger and affects more than 70,000 children in the United States. It can cause deformity, stunted growth, painful joint swelling and, in severe cases, can contribute to organ failure and death. Girls are three times more likely than boys to have it. What causes it: It is an auto-immune disorder, which means the body mistakenly identifies some of its own cells and tissue as foreign and begins to attack them.

Treatment: There is no cure for rheumatoid arthritis, so the main goal in treating children is to preserve physical functioning and maintain a good quality of life. Most children with the disorder need medication and physical therapy.

Information: For more information about juvenile rheumatoid arthritis, call the Arthritis Foundation at 503-222-7246 or, outside of Portland, 800-283-3004.

This week brought new hope to Mollie: Doctors at Doernbecher Children's Hospital at Oregon Health Sciences University told her parents, Sam and Kathy Hauck, that key OHSU clinical review boards have approved a long-awaited stem cell transplant.

The procedure, a variant of a bone marrow transplant, probably will take place in mid- to late September, said Dr. Theodore Moore, director of the Pediatric Stem Cell Transplant program at Doernbecher.

Moore hopes the procedure will re-educate Mollie's immune system and prevent it from destroying her joints. In addition, the procedure could become an option for patients with autoimmune diseases who have tried other treatments without success.

"Mollie is a true pioneer for other children with autoimmune disease," Moore said.

Traditionally, a bone marrow transplant is performed to fight cancer or to correct rare genetic deficiencies. Doctors came across the idea of transplantation as a possible therapy for autoimmune diseases when they noticed that patients receiving the treatment for cancer sometimes got relief from symptoms of their autoimmune diseases, such as multiple sclerosis.

Last November, researchers at Northwestern University and Medical School in Chicago reported that after the procedure, a small group of patients had short-term relief from symptoms of multiple sclerosis, lupus or rheumatoid arthritis, all autoimmune diseases.

Interest in the technique accelerated in February when Dr. Nico Wulffraat and his colleagues in Utrecht, the Netherlands, reported that four juvenile rheumatoid arthritis patients had decreases in joint swelling and pain and no longer needed to take drugs after a transplant.

Mollie's treatment will closely follow the method used in the Netherlands, although in recent contacts with the researchers there Moore learned that of the 20 juvenile rheumatoid arthritis patients who have been treated with transplants, one died of cardiac arrest and two of organ failure related to multiple causes.

Mollie's mother said the family understands the risks.

"We have been really honest with her," Kathy Hauck said. "On all of this we have been upfront on how sick she will be and what could happen. She has been in hospitals enough she is not afraid. Her goal is to grow six feet tall and be able to run and play."

For the procedure, doctors will force stem cells from Mollie's bone marrow into her blood, then collect the cells and store them. Stem cells are the immature cells that can form all the different types of cells in the bloodstream. Some time later, she will enter the hospital and over about nine days she will get combination antibody, chemotherapy and radiation therapy to wipe out her immune system. After Mollie has a day to recover, her stem cells will be thawed and infused back into her.

The following two to three weeks will be the wait for the new marrow to "take." During this time, she may be at risk for bleeding, anemia or infection. Because the stem cells are her own, she will not have to take anti-organ-rejection drugs. When her blood count is normal, she can go home.

The quest for a stem cell treatment began about a year ago when Mollie's doctors in Kaiser Permanente's health plan and the Children's Hospital & Regional Medical Center in Seattle realized they were running out of treatments for her. They asked Moore to explore the possibility of the experimental transplant.

Juvenile rheumatoid arthritis is different from rheumatoid arthritis in adults because adults have lifelong symptoms, but symptoms disappear in more than half the affected children. However, some children, such as Mollie, have severe, unrelenting disease.

Even though the transplant is experimental, Kaiser Permanente has approved it and will pay Mollie's expenses, according to Dr. David Tilford, a Kaiser physician.

"Of the thousands of children I have seen with arthritis, Mollie's arthritis is definitely in the worst 1 percent," said Dr. David Sherry, Mollie's rheumatologist at Children's Hospital in Seattle. "If anything is going to keep her from having totally destroyed joints, it will be the transplant."
 

You can reach Oz Hopkins Koglin at 503-221-8376 or by e-mail at ozkoglin@news.oregonian.com.