The bringers of hope



       
   

Glossary

Bone marrow: the soft, fatty tissue inside bones. The human body contains many stem cells, cells that can differentiate into different cell types and whose job is to repair and maintain the cells in the part of the body where they are found. Stem cells produced in the bone marrow are responsible for the production of red blood cells, white blood cells, and platelets.

Bone marrow transplantation: replacing diseased or damaged bone marrow stem cells with healthy ones. The intense chemotherapy and radiation needed to destroy cancer cells also destroy the sensitive bone marrow cells, which must be replaced. In autologous transplants, some of the patient's own stem cells are removed, either from circulating blood or directly from the marrow, then frozen and stored before chemotherapy and/or radiation. Returned when treatment is over, the cells resume producing healthy, normal blood cells. In the more common allogeneic transplants, the only difference is that the cells to be transplanted come from a donor. Identical twins are the best genetic matches (syngeneic transplants), followed by siblings and other family members; but suitable unrelated donors can sometimes be found through a national bone marrow registry.

Graft-versus-host disease: a life-threatening condition in which the transplanted donor cells (the graft) attack the patient's body (the host).

 
       

The bone marrow transplant team is turning despair over a full range of cancer cases into real hope.

Even in the best of cases, patients who have had bone marrow transplants face a risk of rejection after transplant.

Debbie Barth, however, experienced rejection before hers.

Barth had been seriously ill for two years. A bone marrow transplant was her only hope. When she was rejected for a bone marrow transplant by the Atlanta hospital where she had been treated for those two years, she was terrified. She knew the end of her life could be near.

"I was at the end of my rope, and they wouldn't even take me," says Barth, who was subsequently treated at Winship and now is happy to be enjoying life for the first time in years. "I don't know what I'd have done if I hadn't found Winship and Dr. Waller [Winship oncologist Edmund Waller]. He gave me hope. He gave me a new life."

Many patients like Barth, who had been told she was too high-risk for a transplant, arrive at Winship's Bone Marrow and Stem Cell Transplantation Center having exhausted all other options and time. Some have been turned away from other bone marrow treatment centers because their cases are extremely complicated. Some treatment facilities turn patients away simply because their prognoses are not good. 

Winship's bone marrow transplant (BMT) program is for all Georgians, with physicians fiercely committed to treating patients and to advancing the science that supports that treatment. That commitment is not limited by a patient's age, severity of illness, or type of insurance coverage.

"We are the tertiary referral center for the most complicated cases in the Southeast," says Sagar Lonial, an international expert on multiple myeloma, one of the cancers that is often treated with a transplant.

In addition to taking on the most complicated cases of blood cancers, Winship physician-scientists are leading the medical advances that have changed how these cancers are treated, including the development of new drugs, combinations of drugs, and ways to minimize risk of rejection, or graft-versus-host disease, after a transplant. Another Winship strength is its focus on an individualized treatment plan tailored to each patient's case. "We are disease-focused and not just transplant-focused," says Lonial. 

In addition, the Winship bone marrow transplant team has led the way in establishing best practices from which those individualized plans are developed. 

"Others follow guidelines. We set guidelines," says Lonial.

He's not exaggerating. For example, Winship patients benefit from more than 60 ongoing clinical trials related to these diseases, with 17 open or soon to open, that focus specifically on transplant. Winship also is the leading accruer of patients in a national clinical trial headed by Emory cardiologist Arshed Quyyumi that examines the effect of bone marrow stem cells on recovery after heart attack. The study tests whether such transplants will improve healing and blood flow within the heart. The team consistently publishes studies in leading scientific journals that push forward the knowledge not only about transplantation but also the diseases for which it is used as treatment. For example, Lonial was one of only three clinicians worldwide to be awarded with an Innovator Award from the Multiple Myeloma Research Foundation last fall. He also is one of three members of that group's steering committee. 

Lonial and his team, including Jonathan Kaufmann and Ajay Nooka, have identified eight genetic subtypes of the disease and also have worked on virtually every new drug approved for treatment of myeloma in the past 10 years—two of which have been approved in the past year alone. 

The group sets guidelines—and changes how diseases such as myeloma, leukemia, and lymphoma are treated—based on the depth and breadth of their research. Equally important, they translate that research into patient care. In fact, the BMT program is one of the best examples of how patient care is shaped and informed by the high-level research that happens within an NCI-designated center and a research university.

Scientific advances

Patients who come to Winship for a bone marrow or stem cell transplant may have a hematologic cancer such as leukemia, lymphoma, or multiple myeloma that is destroying the cells in the bone marrow responsible for producing new blood cells. Other patients have aggressive cancers that have relapsed or become unresponsive to standard treatment or have severe autoimmune disorders that are unresponsive to intensive drug therapy. Some, like Barth, have aplastic anemia. Winship is there for all of them.

It's immensely rewarding, says BMT Center Director Waller, "to be able to turn despair to hope, by providing a personalized therapy with the potential for cure."

Since Emory's first bone marrow transplant in 1979, the Winship program has grown steadily in scope, depth, and breadth of clinical trials and experience. Winship BMT physicians expect to reach a major milestone this year—the 4,000th bone marrow transplant. A team of eight physicians, more than 40 specially trained nurse practitioners and physician assistants, and other staff provided 346 bone marrow transplants last year alone, making Winship one of the country's 10 highest-volume adult BMT centers. In addition, these Winship physicians are conducting clinical trials that result in improvements to protocols that other treatment centers follow, making Winship a clear leader in the state and the Southeast. Just last fall, thanks to Waller, the world's leading BMT experts gathered at Winship for an intense exchange of ideas for improvements to the BMT process. 

Winship also is Georgia's broadest, most diversified BMT program, treating the full range of conditions for which transplant is used. In addition to being designated a national center for excellence by various insurance carriers, Winship is recognized nationally as a referral center of excellence for complex and rare cancers—and for its long-term success in transplanting patients with more complicated illnesses than those accepted for transplantation elsewhere. 

Winship constantly is striving to make transplants more successful, with fewer complications, as demonstrated by the following examples.

winship cancer doctors

Choosing the best cells.

Using stem cells from donor bone marrow — the most common form of BMT—is a balancing act. The healthy donor cells attack and kill cancer cells. But these same donor cells (called the graft) sometimes regard the recipient's body (called the host) as foreign. They therefore mount an immune response resulting in problems ranging from dry mouth and skin rash to severe infection and organ damage. A good match lowers the risk of this graft-versus-host disease. There is no risk for autologous transplants using a patient's own stem cells and very little risk for transplants from an identical twin. However, even with treatment that suppresses the unwanted immune response, the chance of graft-versus-host disease rises to 30% to 40% for allogeneic transplants using cells donated by siblings or family members and 60% to 80% for unrelated donors, according to the National Institutes of Health (NIH).

"We can't control what disease the patient has or whether he or she has an identical twin, brother or sister, or other great tissue match," says Waller, "but increasingly we can control which cells we transplant." In earlier studies using a mouse model of leukemia, he and Emory colleagues discovered that precursor bone marrow cells that produce a rare subset of bone marrow cells called plasmacytoid dendritic cells (pre-pDCs) help activate transplanted cells to attack the leukemia while minimizing the immune overactivity that causes graft-versus-host disease. When Emory received a $2 million grant to characterize grafts given to 550 cancer and autoimmune disease patients nationwide and to analyze the success of their transplants accordingly, preliminary analysis showed similar improvement in transplant success for patients who received more pre-pDCs from their bone marrow donor. Results were published in the New England Journal of Medicine, with Waller as a major author. Taking these clinical results back to the mouse lab, the team is unraveling the answers to exactly how transplanting more pre-pDC increases the chance of cure. 

Amplifying cells in the lab. 

Most transplants use 100 billion bone marrow cells or more. What if, instead of painstakingly picking out highly desirable cells, you could just multiply those you want, like the magic brooms in the Sorcerer's Apprentice? It's no fairy tale, says Waller. Last year, with support from Winship, Children's Healthcare of Atlanta, the Aflac Cancer & Blood Disorders Center, and the Emory School of Medicine, Jacques Galipeau, an expert in helping the body repair or regenerate cells, tissues, and organs, launched a new Emory Personalized Immunotherapy Center (EPIC). The center is designed to create new, personalized cellular therapies for Emory patients facing medical catastrophes including cancer, autoimmune disease, and various heart, lung, and neurologic problems. EPIC scientists already are involved in removing and amplifying specific bone marrow cells found to suppress the immune system, then working with Winship's BMT team to return them to young patients with Crohn's disease. Now, a grant from the Georgia Tech/Emory Center for Regenerative Medicine is enabling the team to test a new method of increasing the numbers of pre-pDC cells in the graft before transplantation. Next step? A clinical trial to see if transplantation of large numbers of these cells can control the overactive immunity that causes graft-versus-host disease and graft rejection. 

Broadening the possibilities for transplant. 

At Winship, a large multi-specialty committee reviews each new cancer patient's case, recommending the best possible treatment or combination of treatments, whether chemotherapy, radiation, or transplant; whether transplant using a patient's own cells, donor cells, or umbilical cord blood; or whether a clinical trial offers something needed by a specific patient. As Georgia's only cancer center designated by the National Cancer Institute and as a core site of the NIH-funded Blood and Marrow Transplant Clinical Trials Network, Winship has access to the newest and most promising trials. A fourth of BMT procedures at Winship take place under the umbrella of a national clinical trial. That means hope for some patients for whom no hope had existed. It also means that many patients treated at Winship benefit from treatments that may not be available at nonacademic centers for years. 

For example, Amelia Langston is an investigator in a national phase 3 clinical trial examining whether patients with acute myelogenous leukemia or myelodysplastic syndrome, whose disease is in remission, may do just as well with less intense pre-transplant chemotherapy as they would with much more concentrated, traditional pre-treatment. If "reduced intensity" transplant proves as effective, it would decrease hospitalization and costs, not to mention stress on patients, and would provide another option for patients whose general poor health makes them a poor candidate for traditional high intensity transplant. Langston already has demonstrated that older patients whose cancer is in remission can benefit from the reduced intensity transplant approach.

 Tamara Mobley found Winship when she was referred by a physician at another Atlanta hospital. She had become very ill, very quickly at age 33 in the fall of 2009. She ended up in the emergency room and later learned her diagnosis—multiple myeloma. Without hesitation, the physician told her she needed to go to Winship. 

"He said, ‘if it were my daughter or my wife, I'd send them there,'" recalls Mobley. She was so ill that she was taken by ambulance to Emory University Hospital, where Winship patients receive their tranplants. 

Now able to enjoy her two young children, Mobley says she feels grateful she was referred to Winship. "I totally love Dr. Lonial and Charise," she says, referring to Winship nurse practitioner Charise Gleason. "I feel honored to be under Lonial's care."

William Fuentes of Calhoun also was diagnosed last fall when he was in his early 30s.

"I'd never even heard of it," Fuentes says. His disease manifested itself in his spine, and he was in excruciating pain. Like Mobley, he was brought to Winship by ambulance. The father of two preschoolers was scared. He worried about what would happen to his wife, Sonya, and their children should something happen to him. 

"I used to cry a lot when the children weren't watching," says Fuentes, the manager of a McDonald's. "They were always asking, ‘why are you going to the hospital so much?'"

Fuentes credits the entire treatment team for providing critical emotional and other support.

"I couldn't have asked for better treatment," he says. Social workers made sure gas cards were available when funds ran low. A team of employees from the clinical trials unit came together and provided Christmas gifts for the family. 

"My wife is so thankful. We love it here," says Fuentes.

His doctor is Ajay Nooka, the newest faculty member of the team. As a fellow, Nooka presented posters at last year's annual meeting of the American Society of Clinical Oncology (ASCO), reporting data from his studies that show how special interventions before transplantation can help myeloma patients who have complicated disease.

Like his mentors, Nooka is showing the signature compassion for which the team is so loved.

"He's the nicest man I've ever met," says Fuentes. "He has such integrity. He never changes, no matter who he's around. He treats both his patients and his team well."

Debbie Barth's mother Joanie says a certain word comes to mind when she thinks about her daughter's physician: gratitude.

"I can't tell you how I felt when we got into that room with him that first day," she recalls of Debbie's first appointment with Waller. "Instead of saying there was no hope, he said she had a 50% to 80% chance."

Barth's mom recalls falling silent and then asking Waller: "Can you tell me whether it's closer to 50 or 80?"

Waller looked at his patient's mom and said, "Ms. Barth, she's going to make it."

 "I just started crying and crying because for the first time, we had hope," she says. "When he spoke, the whole room just filled up with hope."






       
 
 

A shared journey back to health

"No matter how dedicated we are to the science and medicine of transplant, we never lose sight of the very human experience it is for our patients and their families," says Amelia Langston, Bone Marrow Transplant (BMT) Program Medical Director. 

It's also intense for Winship clinicians. "Many of our patients are so sick when they arrive, yet display such amazing courage and spirit over the weeks or months sometimes required for recovery, that members of the BMT team form incredible bonds with them, not just as patients but also as people."

For many patients, physical activity is an important part of the journey back to health after transplant, and the BMT team loves being there for the ride—sometimes literally. For example, when Bob Falkenberg celebrated his victory over acute lymphocytic leukemia with a summer-long bicycle ride from Boston to Key West, Langston and nurse practitioner Jessica Thomas took off a weekend to bike some 200 miles beside him. Several of the nurses who had cared for him during his BMT served as drop off/pick up drivers and cheerleaders.

You don't have to cover miles to benefit from the healing potential of doing normal things, says Langston. During Winship's annual Win the Fight 5K, she sees many patients who have completely regained their old lives and others who still struggle but nonetheless cheer on their clinicians and friends, hold their kids' hands, and laugh, "living their lives with sheer grit and determination."

Such spirit inspires the BMT team every day, says Langston, and the 2012 Summer Olympics also inspired her to create the Oncology Olympics. Every day during the international Olympic Games, most recently in London, BMT and other cancer patients, families, and staff gather on the BMT ward at Emory University Hospital to participate in events organized by the BMT nurses such as the hula-hoop marathon, syringe shooting competition, and bedpan shuffleboard. Winship Executive Director Dr. Walter J. Curran Jr. even got in on the fun, "medaling" in the most recent wheelchair race. The patients loved it.

"It shows how much they really care about you here at Winship," says patient Joseph Allen. "It wasn't what I expected at such a big place, but they are here for you doing everything they can for you. It really helps lighten the load."

Langston also uses her experience as a runner to raise money for cancer, whether as part of Winship's own annual 5-K race or in various other events for which she exacts pledges of support for cancer research from friends and colleagues. Last November, she completed the New York Marathon, wearing a shirt signed by many of her patients and bringing in about $3,000 to benefit Be the Match (marrow.org). She and physician Martha Arellano also participate in the Leukemia and Lymphoma Society's Team in Training (teamintraining.org), which prepares novice and veteran athletes alike for endurance running, biking, swimming, and other events to raise money for the fight against blood cancers.

 
         


     
 
match

Be the Match.

Two-thirds of the allogeneic or donor transplants performed at Emory use cells from unrelated donors, strangers who have registered in the national marrow donor program. Swiping the inside of their mouths with a cotton swab provides all the genetic information needed for doctors around the world to search the registry for a tissue match for a patient. If a close enough match is found, the potential donor is asked to donate either bone marrow or stem cells found in circulating blood. 
You can save a life by becoming a bone marrow donor, and it is not a painful procedure. While donors in years past had to typically undergo a surgical procedure, now most do not. If you would like to find out about becoming a donor, go to marrow.org







       
 
 

Dozens of service awards hang on the wall on the eighth floor unit of the Emory University Hospital, where transplants take place.

These awards recognize the nurses and nursing staff on 8E, which show not only the commitment and compassion of the nurses but also a depth experience that cannot be matched elsewhere in the state. Several note 25 years of service or more. Karen Donegan has 35 years of service and Mitzi Smiley 25. BMT nursing director Emily Bracewell has worked with the team for 35 years. Nursing unit clerk Constance Tucker has been there for 25 years. 

The nursing staff at Winship, including nurse practitioners such as Jessica Thomas, Rachel Veldman, and Charise Gleason, and at EUH are key parts of the bone marrow transplant program, patients and physicians say. 

"The knowledge and compassion of our nursing staff is amazing," says Medical Director Amelia Langston. "Our patients never fail to let us know how much they appreciate their nurses, and we in turn appreciate the critical contributions they make to our patients."

The length of service is not only evidence of commitment but also of expertise, Langston says."It means a lot to us as doctors knowing that this nursing staff can handle anything that may happen," she says. "Their knowledge and expert ability strengthens patient care, and, we believe, helps improve patient outcomes."

 
         

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