Hello! This has been a very busy beginning to my term as President. And I am happy to say how well things are moving along. Our board is enthusiastic and eager to work on new projects.

First, a quick note about myself. I am the mother of a 9 year old SDS patient who received a successful BMT 3 � years ago. He was diagnosed at 13 months at a military treatment facility.  I am a Navy wife and volunteer with the boy scouts and at school. I am looking forward to volunteering with SDSF and making a difference with families, the community and medical fields. Feel free to contact me at [email protected].

Plans for this summer's family conference at Camp Sunshine are almost complete. We think it will be our best year yet! Our fundraising chair, Ed Turkaly, is working with a family in NC on a golfing fundraiser. And an exciting media production project is in the works, with Ed Bickel our Creative Media Chair,

SDSF is excited to have received letters of intent for research studies and have been reviewed. Successful applicants have been asked to submit full proposals to be vetted by our Medical Advisory Board. We hope to be able to make an announcement regarding this in the near future.

Have a fun and safe summer!

Christine James, President

The date for SDS week at Camp Sunshine this summer is July 13-18th. Are you joining us? Is this your first time attending? Here is what to expect during your stay.

Camp Sunshine hosts 40 families in suite style rooms which can sleep 6. All units have a full bath, a small refrigerator, and microwave. There are also laundry facilities available, if the need arises. No TVs here! This is a time to step away from your chaotic life, relax and mingle. Make new friends, listen to the experts, learn from other parents' experiences and overall have a good time. Towels and linens are provided by SDSF.

Children are divided into age specific groups. And each group has a designated room with age appropriate toys and activities. Even the youngest of visitors is taken care of in a lovely nursery. Yes, they do change diapers! Volunteers, however, are not permitted to dispense medications. The children will be brought to you if it is time for them to get their medicine. There are more than enough volunteers to entertain and care for the children in a safe environment. The children have so much fun at Camp Sunshine that we have seen children not wanting to leave their volunteers for a meal with their parents, or leave Camp at the end of the week.

Adults and parents will participate in some ice breaking activities, attend medical specialist lead conferences, and discussion groups. There will also be time for one on one discussions with the doctors if you have any specific questions. The doctors do eat in the dining room with the families. Invite them to your table. Get to know the experts.

This year we are excited to have a large group of doctors volunteering their time to attend Camp:

Dr. Akiko Shimamura, Hematology, Seattle

Dr. Lauri Burroughs, BMT, Seattle

Dr. Matthew Giefer, GI, Seattle

Dr. Kasiani Myers, BMT, Cincinnati

Dr. Katherine Baum, Neuro-psych, Cincinnati

Dr. Johnson Liu, Hematology, New York

Dr. Monica Bessler, Hematology, Philadelphia

Dr. Peter Kannu, Bone & Dental, Toronto

Dr. Janet McGill, Endocrinology, St. Louis

Maura Donahue, School Psychologist, California

Nancy Cincotta, Psycho-Socialworker, Camp Sunshine

The staff at Camp Sunshine runs a tight ship. During the week many activities are planned; a masquerade dance, talent show, parent/adult gourmet dinner and karaoke, crafts, open pool time, and free time. If you have room, pack a small costume for your little one. If you don't Camp has a large costume closet. You are sure to find something in there. Does your child have a talent? A few families in the past have performed as a group. During free time take a walk to the lake and paddle boat or canoe. Or take in a game of mini-golf. There is plenty to do. Sorry, no swim diapers in the pool.

We are looking forward to meeting new families and reconnecting with returning friends. Safe travels!

See you at Camp Sunshine!

Drs. Lauri Burroughs and Akiko Shimamura, experts in the field of hematopoietic cell transplantation (HCT) and bone marrow failure, along with their colleagues at the Fred Hutchinson Cancer Research Center and Seattle Children's Hospital, are investigating a new HCT approach for the treatment of patients with Shwachman-Diamond syndrome. At the current time, successful cures for the bone marrow (hematopoietic) complications of Shwachman-Diamond syndrome have been attained only after HCT. However, patients with Shwachman-Diamond syndrome are more sensitive to the toxicities from standard bone marrow transplant regimens which can result in serious complications including organ damage. Outcomes between different reduced intensity conditioning regimens may vary. To address this serious problem, the doctors at Fred Hutchinson Cancer Research Center and Seattle Children's Hospital have developed a HCT procedure that uses a new combination of reduced intensity chemotherapy drugs, Treosulfan and Fludarabine, for patients with Shwachman-Diamond syndrome. Importantly, several studies in Europe have shown that similar conditioning regimens using Treosulfan dramatically reduce the risk for transplant related complications and therefore increase survival.

Dr. Burroughs and colleagues are conducting the first prospective clinical trial in the United States using treosulfan for HCT conditioning in patients with non-malignant diseases. To date over 50 patients have been received a transplant on this clinical trial of which 7 had underlying high-risk marrow failure disorders including 3 patients with Shwachman-Diamond syndrome. All seven patients with marrow failure disorders are alive and doing well including the 3 patients with Shwachman-Diamond syndrome who all have 100% donor engraftment and are cured of their underlying bone marrow (hematopoietic) complications. Importantly, this clinical trial uses all stem cell sources including bone marrow, peripheral blood, and cord blood thus allowing nearly all patients a chance at a potential cure.

Treosulfan has also been used in HCT for patients with myelodysplastic syndrome (MDS) and acute myelogenous leukemia (AML) and has been shown to have low toxicities and decreased transplant related mortality. Therefore, patients with Shwachman-Diamond syndrome who have developed MDS or AML are candidates for transplantation using Treosulfan-based conditioning.

The Seattle Cancer Care Alliance (SCCA) is a comprehensive medical center that unites the expertise of the Fred Hutchinson Cancer Research Center with two other internationally renowned institutions - Seattle Children's Hospital and the University of Washington Medical Center. The Fred Hutchinson Cancer Research center has a long history and commitment to advancing the field of hematopoietic cell transplantation and bone marrow failure. Patients receive state of the art multi-disciplinary medical care and are provided with excellence in nursing care, child life, pharmacy and psychosocial support which are all vital in the care of patients. No matter what treatment is given, every patient receives specialized attention and support to help them and their families through a difficult time. Our nurse coordinator, Kathleen McGregor, will work with your family to assist with travel and logistics including lodging, schooling, insurance issues, and family support.

Dr. Burroughs and Dr. Shimamura will be attending the Shwachman-Diamond syndrome family camp - Camp Sunshine this July 2014. If you have any questions regarding the clinical trial, transplantation, or Shwachman-Diamond syndrome please contact Dr. Lauri Burroughs at [email protected]. or Dr. Akiko Shimamura at [email protected].

Nearly two years ago, my husband, Nathan, and I were in a position no parent wants to find themselves in. We learned our two-year-old son, Alexander "Xander", who had been diagnosed with SDS at 8 months of age, needed a bone marrow transplant.

Because of the expertise at Seattle Children's Hospital, we opted for a transplant there rather than near our home in Kansas City. At the time, they were one of the few centers in the nation involved in the drug trial of the new chemo drug Treosulfan. When we weighed the risks and potential benefit of using Treo-which is overall less toxic to the body than its predecessor Busulfan-it was a no-brainer: we'd do the transplant 2,000 miles from home.

As a parent, I loved always feeling like I was a part of the team treating Xander. During his second hospitalization, the team pressed for me to allow them to place an NG-tube. While I recognized why they wanted to do it, I put my foot down: absolutely not. Xander had had a tube for seven months as a baby, and not only was it a road I didn't want to go down again, but it had made him a lazy eater for a time. When I laid out my reasoning, they quickly backed off, and I was grateful they respected my opinion as well as my knowledge of my son.

Although doing Xander's transplant half-a-continent away from my home was definitely not ideal, if I had it to do over again, I wouldn't hesitate to spend five-and-a-half months there again.

Greenville, NC

Will you be in the Greenville, NC area in July?  Please join the Price Family golf fundraiser:  Swinging for Shwachman Diamond.