Clinical Trials

In 2011, the St. Baldricks's Foundation, a charity dedicated to raising money for childhood cancer research, awarded Kris Ann P. Schultz, MD, a pediatric oncologist in the cancer and blood disorders program at Children's Hospitals and Clinics of Minnesota, a $330,000 grant to establish the International Ovarian and Testicular Stromal Tumor (OTST) Registry.

The OTST Registry is the nation's first registry to study rare ovarian and testicular tumors that occur in children and adolescents. The registry hopes to collect tumor and blood samples to determine what causes ovarian and testicular stromal tumors, who is at risk and what therapies work best for treatment – with the ultimate goal of increasing survival rates and reducing late effects.

The registry collects cases of ovarian and testicular sex cord-stromal tumors.

Examples of ovarian tumors eligible for the registry include:

  • Sertoli Leydig cell tumors
  • Juvenile granulosa cell tumors
  • Gynandroblastomas
  • Sex cord-stromal tumor with annular tubules
  • Undifferentiated sex cord-stromal tumors

Examples of testicular tumors eligible for the registry include:

  • Juvenile granulosa cell tumors
  • Leydig cell tumors
  • Sertoli tumors
  • Sex cord-stromal tumor with annular tubules
  • Undifferentiated sex cord-stromal tumors

In addition, the registry can help facilitate central pathology review and DICER1 testing. By collecting cases and studying these rare tumors, investigators hope to find ways to treat ovarian and testicular stromal tumors as well as other more common cancers.

For more information about the OTST Registry, call or email the registry coordinator, Anne Harris: 612-813-5861, This e-mail address is being protected from spambots. You need JavaScript enabled to view it.

The International OTST Registry is a collaboration of Children's Hospitals and Clinics of Minnesota, Children's National Medical Center and Dana Farber Cancer Institute.

The International Ovarian and Testicular Stromal Tumor Registry is funded by St. Baldrick's Foundation, Hyundai Hope on Wheels and the Pine Tree Apple Tennis Classic, a local charity dedicated to children's cancer research.

Children's oncologists participate in the Pediatric Blood and Marrow Transplant Consortium (PBMTC) that is made up of clinicians and scientists from around the world engaged in the area of Hematopoietic Stem Cell Transplantation. The purpose of the PBMTC is to engage in scientific and educational activities related to the use of hematopoietic stem cells in the treatment of diseases of children and adolescents.

In 1986, Children's hematologist/oncologist Jack Priest, MD, founded the International Pleuropulmonary Blastoma (PPB) Registry to track pleuropulmonary blastoma cases worldwide. PPB is a rare pediatric lung cancer, occurring in 30 to 50 children in the U.S. each year. Today, we continue to gather information from patients diagnosed with PPB from around the world under the direction of Children's hematologist/oncologist Dr. Yoav Messinger, MD.

In March 2009, results from the genetic research studies were released uncovering clues about PPB. It identified a mutation more likely to produce PPB or other related developmental cancers. While the discovery has major implications for families with a child diagnosed with PPB, it could also be a breakthrough for cancer research in general. The search for the cause of PPB has uncovered important information about how the cancer develops and potentially sheds light on how other cancers form. The research study continues with a focus on designing genetic testing for the specific mutation that will give families with a child diagnosed with PPB information about the risk factors for this disease.

More on the actual study

The PPB Registry staff at Children's worked with colleagues from many institutions from around the country and the world to identify a specific genetic mutation in PPB patients and their families.

PPB has a strong genetic component. Individuals with this mutation are more likely to develop PPB or other related developmental problems. Current studies show that about 40 percent of PPB cases occur in families with a history of the disease or certain other childhood tumors. In contrast, most pediatric cancers occur sporadically, without any familial patterns. This led scientists and doctors to suspect the tumors were caused by an inherited genetic abnormality.

To uncover the role of the mutated gene, the research team studied the genetic makeup of 11 extended families with two or more members having PPB or related childhood tumors. The scientists say finding this variant form of a gene in some PPB families is a first step to understanding why PPB and other conditions may occur in some families. Results of the study may lead to new diagnostic tests and new treatments that may improve survival for children with PPB and adults with other tumor types. While the discovery has major implications for families with PPB, it could also be a breakthrough for cancer research in general.

In 1973, the National Hemophilia Foundation launched a campaign to establish the creation of a nationwide network of hemophilia diagnostic and treatment centers. Today, there are about 141 federally funded treatment centers and programs across the country. Children's is designated as a Hemophilia Treatment Center (HTC) and is the only such program in the region focused solely on pediatrics. This designation ensures that children and adolescents receive comprehensive care from a team of pediatric experts in bleeding and clotting disorders.

The development of comprehensive care over the past 30 years, has greatly improved the quality of life for people with bleeding disorders, helping them to have more independent and productive lives. The treatment center care network has also lowered patients' complications, morbidity and provided cost-effective care.

Children's of Minnesota is a founding member of the cooperative research group, Therapeutic Advances in Childhood Leukemia (TACL), which focuses on developing new treatments for children with a diagnosis of leukemia that recurs after the initial treatment is complete.

Most children with a diagnosis of leukemia have acute lymphoblastic leukemia (ALL), acute myelogenous leukemia (AML), or chronic myelogenous leukemia (CML), and most are cured. However, treatment options are more limited for the children whose disease recurs.

TACL seeks to rapidly develop and carry out clinical trials of promising new drugs for these patients. Children's is one of few hospitals in the Midwest where these clinical trials are available.

The TACL consortium began clinical trials in 2006 to standardize treatments internationally that serve large numbers of children with leukemia. Researchers at TACL member hospitals, working with the pharmaceutical industry, develop new drugs and combinations of drugs to combat recurrent leukemia. TACL's studies test new anti-cancer agents alone or in combination with medications typically used for relapsed leukemia.

Study for relapsed ALL using Bortezomib (Velcade)

TACL recently completed a clinical trial combining bortezomib (Velcade) in combination with standard chemotherapy for patients with multiply recurrent acute lymphoblastic leukemia (ALL) who respond poorly to therapy. Children's hematologist/oncologist Dr. Bruce Bostrom wanted to investigate whether or not agents like bortezomib, which are active in multiple myeloma (adult bone marrow cancer), may be active in childhood ALL. Dr. Bostrom tried bortizomib in combination with dexamethasone (Decadron) in a patient with an encouraging response. This led to the TACL trial, chaired by Children's hematologist/oncologist Yoav Messinger, MD, which proved to be the most successful trial ever for patients with multiply relapsed ALL. Plans are underway to study bortezomib with standard chemotherapy in patients with a first relapse of ALL.