Pediatrics In The News

August 1, 2018
UT Southwestern Scientists Identify New Mechanisms Underlying Pediatric Kidney Cancer

Connecting two previously unrelated insights about the formation of pediatric kidney cancer, researchers at UT Southwestern Medical Center have uncovered the means by which the cancer continues to grow, providing potential targets for more effective treatments in the future.

Wilms tumor is the most common cancer of the kidney in children. Typically, the disease is treated with surgery, radiation, and chemotherapy. This combination is effective for many patients but has numerous side effects, and a cure remains elusive for those with aggressive disease. This situation has driven investigators at UT Southwestern to look for more effective and less toxic ways to treat Wilms tumor.

Previously, pediatric investigators from the nationally recognized Kidney Cancer Program at UT Southwestern’s Harold C. Simmons Comprehensive Cancer Center identified a new molecular subset of Wilms tumors driven by recurrent mutations at “hot spot” residues in genes of the microRNA (miRNA) processing pathway (Rakheja et al., Nat Comm, 2014). A miRNA is a tiny RNA that reduces the production of specific proteins in cells. Nevertheless, it was unclear exactly why impairment of miRNA function caused Wilms tumors. 

Dr. James Amatruda, Associate Professor of Pediatrics, Molecular Biology, and Internal Medicine, along with Dr. Kenneth Chen, Instructor of Pediatrics, steered the study published in Genes and Development. Read more

June 28, 2018
Researchers Discover New Vulnerability in Deadly Form of Lung Cancer

Researchers at the Children’s Medical Center Research Institute at UT Southwestern (CRI) have discovered a new metabolic vulnerability in small cell lung cancer (SCLC) that can be targeted by existing drug therapies.

SCLC is a deadly and aggressive form of lung cancer with few therapeutic options and an incredibly low five-year survival rate of 5 percent. Researchers at CRI believe the key to finding new therapies for this disease lies in better understanding the metabolism of SCLC.  Cancerous cells reprogram their metabolic pathways to grow and spread rapidly through the body. In some forms of cancer, cancer cells become highly dependent or “addicted” to specific metabolic pathways as a result of genetic mutations. Identifying these pathways can lead to new treatment options. Read more 

June 22, 2018
Sendelbach, de Lemos Honored with Regents' Outstanding Teaching Awards

Cardiologist Dr. James de Lemos, Professor of Internal Medicine, and pediatrician Dr. Dorothy Sendelbach, Assistant Dean of Undergraduate Medical Education at UT Southwestern, have been recognized with the UT System’s highest educational honor, the Regents’ Outstanding Teaching Awards, for their academic prowess and mentoring talent.

With their selection as recipients of 2018 Regents’ Outstanding Teaching Awards (ROTA), UT Southwestern now has more than 40 faculty who have been honored with the Regents Teaching Award, which recognizes outstanding teaching, mentoring and personal commitment to students and the learning process. 

“Both Dr. Sendelbach and Dr. de Lemos are exemplary scholars and educators, and we are truly delighted that the Board of Regents have recognized their commitment to our students and other learners. Their dedication and talents as teachers makes it possible for UT Southwestern to prepare the future physicians and scientists who will improve the health and well-being of our community and those beyond,” said Dr. Daniel K. Podolsky, President of UT Southwestern Medical Center, who holds the Philip O’Bryan Montgomery, Jr., M.D. Distinguished Presidential Chair in Academic Administration, and the Doris and Bryan Wildenthal Distinguished Chair in Medical Science. Read more

June 5, 2018
A Popular Aquariam Fish May Hold Answers to How Tumors Form in A Childhood Cancer

Muscle precursor cells called myoblasts are formed during normal fetal development and mature to become the skeletal muscles of the body...

Cancer researchers at UT Southwestern Medical Center developed a zebrafish model for the childhood cancer. To do this, Dr. James Amatruda's lab inserted the human PAX3-FOXO1 gene into the DNA of zebrafish. Using this new transgenic zebrafish, the researchers showed that the fused-gene DNA causes rhabdomyosarcoma that is similar to the human disease. They found it does this by turning on another gene, HES3, which leads to overproduction of the skeletal muscle precursor cells and allows for PAX3-FOXO1+ cells to survive during development instead of dying. Read more

May 23, 2018
Children's Research Institute at UT Southwestern professor named Howard Hughes Medical Institute Investigator

Dr. Ralph DeBerardinis, Professor at the Children’s Medical Center Research Institute at UT Southwestern (CRI), today became the University’s newest Howard Hughes Medical Institute (HHMI) Investigator.

Dr. DeBerardinis is Chief of the Division of Pediatric Genetics and Metabolism at UT Southwestern and an attending physician at Children’s Health. He is also a Professor of Pediatrics and member of the Eugene McDermott Center for Human Growth and Development at UT Southwestern. His selection makes him one of a select group of 19 distinguished biomedical scientists named Howard Hughes Medical Institute Investigators this year and brings to 15 the number at UT Southwestern, which leads the state in HHMI investigators. HHMI is a philanthropic organization created to advance basic biomedical research and science education for the benefit of humanity. Read more

March 29, 2018
Pediatric cancer drug shows 93 percent response rate

A first-of-its-kind drug targeting a fused gene found in many types of cancer was effective in 93 percent of pediatric patients tested, researchers at UT Soutnwestern's Simmons Cancer Center announced.

Most cancer drugs are targeted to specific organs or locations in the body. Larotrectinib is the first cancer drug to receive FDA breakthrough therapy designation for patients with a specific fusion of two genes in the cancer cell, no matter what cancer type. The research appears in The Lancet Oncology.

“In some cancers, a part of the TRK gene has become attached to another gene, which is called a fusion. When this occurs, it leads to the TRK gene being turned on when it’s not supposed to be and that causes the cells to grow uncontrollably. What’s unique about the drug is it is very selective; it only blocks TRK receptors,” said lead author Dr. Ted Laetsch, Assistant Professor of Pediatrics and with the Harold C. Simmons Comprehensive Cancer CenterRead more

February 28, 2018
MMK Foundation makes $1 million gift to Children's Health, UT Southwestern Medical Center

The MMK Foundation, founded by Mark and Marcia King, has pledged $1 million to be split equally between Children’s Health and UT Southwestern Medical Center for the purpose of enhancing the pediatric hospital’s neonatal ICU, furthering groundbreaking research at UT Southwestern and providing unrestricted funds for the critical needs of children. Read more

February 21, 2018
Drug targeting mutant cancer gene is highly effective, durable

A drug targeting a gene fusion that occurs in lung, colon, and other cancers was effective in 75 percent of patients of all ages in clinical trials, and an even higher percentage of pediatric patients, researchers at UT Southwestern’s Simmons Cancer Center announced. The drug is being given fast-track consideration by the Food and Drug Administration.

The drug, larotrectinib, targets TRK-fusion, which can occur in lung, colon, thyroid, and many other types of cancer, said Dr. Ted Laetsch of UT Southwestern’s Department of Pediatrics and the Harold C. Simmons Comprehensive Cancer Center. Dr. Laetsch led the pediatric arm of the clinical trial for the study, which appears in the New England Journal of MedicineRead more

February 14, 2018
CAR-T clinical trial enrolling multiple myeloma patients

UT Southwestern Medical Center is one of nine exclusive sites in the country enrolling multiple myeloma patients for a clinical trial of the CAR-T “living drug” therapy for cancer. CAR-T therapy (chimeric antigen receptor T-cell therapy) is an innovative immunotherapy that uses a re-engineered version of the patient’s own immune cells to fight cancer. A UT Southwestern cancer researcher who was among the first patients to be treated, has now been cancer-free for two years.

As one of just 49 in the country designated a Comprehensive Cancer Center by the National Cancer Institute and the only one in North Texas, UT Southwestern’s Harold C. Simmons Comprehensive Cancer Center is offering both treatments and clinical trials in several critical areas:

  • Dr. Ted Laetsch, Assistant Professor of Pediatrics, led the only clinical trial site in the Southwest for a CAR-T treatment for acute lymphoblastic leukemia (ALL). Results from the trial resulted in FDA approval for the first CAR-T therapy, and Dr. Laetsch is now treating ALL patients who are 25 and under with this CAR-T therapy at the Pauline Allen Gill Center for Cancer and Blood Disorders at Children's Health.
  • In November, the FDA approved a CAR-T treatment for lymphoma, and UT Southwestern physicians soon will be offering this treatment to patients through the Bone Marrow Transplantation/Hematologic Malignancies Clinic at the Simmons Cancer Center.
  • As new uses for CAR-T continue to be explored, leadership and guidance on how and when to use these treatments also is needed. Dr. Ankit Kansagra, Assistant Professor of Internal Medicine, is co-leader of the Global CAR-T Initiative, a group of physicians who are meeting to draw up guidelines for CAR-T use. Read more 

February 1, 2018
Simmons Cancer Center researchers part of historic CAR-T breakthrough

A historic study involving researchers from UT Southwestern’s Harold C. Simmons Comprehensive Cancer Center demonstrates the effectiveness of CAR-T therapy, which uses genetically modified immune cells to treat acute lymphoblastic leukemia (ALL) in children and young adults. The research appears in the New England Journal of Medicine. Dr. Ted Laetsch served as the lead investigator for the only clinical trial site in the Southwest for the CAR-T trial. Read more 

January 24, 2018
Amid ADHD spike, doctors urge closer look at sleep issues

Amid a steady rise in the number of children diagnosed with ADHD, debate is brewing whether the condition may be a sleep disorder....“If adults don’t get enough sleep, they’ll appear sleepy,” says Dr. Syed Naqvi, a pediatric sleep expert at UT Southwestern’s Peter O’Donnell Jr. Brain Institute. “Children don’t do that. They show ADHD-like behavior instead – hyperactive or inattentive.” Read more