What is a clinical trial?

Children’s Oncology Group (COG)

The tremendous progress made in pediatric cancer cure rates over the past 50 years came about because treatments for all childhood cancers have been continually improved through research protocols organized internationally by the Children’s Oncology Group (COG), an association of institutes dedicated to research in pediatric oncology.

• Children’s is among the top 10 hospitals in COG for numbers of children enrolled with newly diagnosed cancer treated each year.
• Nearly 90 percent of our eligible patients participate in clinical trials.
• With greater than 100 open COG studies, the majority of which are clinical trials, patients at Children’s are able to receive the newest treatment options available.

Clinical trial on neuroblastoma

Children’s recently participated in a clinical trial for patients with neuroblastoma, a common solid tumor in children.

During the trial, 226 patients with neuroblastoma were randomly assigned to receive standard treatment, or standard treatment plus immunotherapy. The immunotherapy targets the neuroblastoma cancer cells as well as other substances that stimulate the patient’s own immune cells to attack cancer cells. This trial was concluded a year earlier than expected. The early results showed that patient survival without experiencing a relapse of the disease was 20 percent higher in the patients in the immune therapy group compared to the standard treatment group.

As a result, Children’s is currently one of 30 COG centers participating in a new study that is researching the side effects of this immunotherapy to determine whether it should become the new standard of care for all high-risk neuroblastoma patients.

Clinical Trials for Young Adults with Pediatric Cancers

According to the National Cancer Institute, sometimes teens and young adults with pediatric cancers do better with treatment approaches tailored to young children rather than those designed for older adults. Furthermore, clinical trials are an important treatment option for cancer patients of all ages, and often yield better results for patients who participate than those who do not. At Children’s, 15- to 19-year-old teens and young adults are enrolled in clinical trials at four times the national average for the same age group. The information from these trials is already influencing the way adult oncologists treat young adults with acute lymphoblastic leukemia.

Therapeutic Advances in Childhood Leukemia (TACL)

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 bortezomib 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.

International Pleuropulmonary Blastoma (PPB)/DICER1 Registry

In 1986, Children’s hematologist/oncologist Jack Priest, MD, founded the International Pleuropulmonary Blastoma (PPB)/DICER1 Registry to track pleuropulmonary blastoma cases worldwide. PPB/DICER1  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/DICER1 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/DICER1. It identified a mutation more likely to produce PPB/DICER1 or other related developmental cancers. While the discovery has major implications for families with a child diagnosed with PPB/DICER1, it could also be a breakthrough for cancer research in general. The search for the cause of PPB/DICER1 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/DICER1 information about the risk factors for this disease.

More on the actual study

The PPB/DICER1 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/DICER1 patients and their families.

PPB/DICER1 has a strong genetic component. Individuals with this mutation are more likely to develop PPB/DICER1 or other related developmental problems. Current studies show that about 40 percent of PPB/DICER1 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/DICER1 or related childhood tumors. The scientists say finding this variant form of a gene in some PPB/DICER1 families is a first step to understanding why PPB/DICER1 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/DICER1 and adults with other tumor types. While the discovery has major implications for families with PPB/DICER1, it could also be a breakthrough for cancer research in general.

Hemophilia Treatment Centers (HTC)

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.

The International Ovarian and Testicular Stromal Tumor Registry

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 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

Contact our registry coordinator, Anne Harris: 612-813-5861, [email protected]

The International OTST Registry is a collaboration of Children’s 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.

The Cancer and Blood Disorders Program at Children’s Minnesota works with other top children’s hospitals to gives your child local access to a network of national clinical trials for the treatment of brain tumors.

Children’s Minnesota is a member of the Pacific Pediatric Neuro-Oncology Consortium (PNOC), which is made up of 18 children’s hospitals dedicated to improving the outcomes for children with brain and spinal cord tumors.  They conduct clinical trials of new therapies for children with brain tumors with the goal of improving outcomes by creating new individualized treatments from the latest findings in cancer biology.

Our involvement in cooperative groups such PNOC as allows us to offer innovative treatment options that might not be available at most centers.

The brain tumor team at Children’s Minnesota participates in select open clinical trials through St. Jude Children’s Research Hospital. In partnership with St. Jude’s, we are able to provide our patients with innovative therapeutic clinical trials aiming toward higher cure rates and decreased toxicity. Collaborative St. Jude clinical trials offered at Children’s Minnesota includes treatment for pediatric and young adult brain tumors.