Research Highlights - Promise Winter 2015

Research Highlights - Promise Summer 2014

Gene therapy provides life-changing relief from hemophilia
People with severe hemophilia B, a bleeding disorder, are at risk of serious health problems and early death

The power of phone calls for protecting the heart
Despite the potential benefits, many survivors fail to get recommended checkups on schedule.

Science meets art
The Art of Science pairs St. Jude scientists with local artists.

Two genes are double trouble in Ewing sarcoma
New secrets have been revealed by a detailed study of Ewing sarcoma, a cancer of the bone and soft tissue.

Launching a three-pronged attack against Ewing
St. Jude scientists have discovered a promising new triple-drug therapy for Ewing sarcoma

First Lady visits St. Jude
First Lady Michelle Obama visits St. Jude.

A new connection between diet and disease
Changes in the human gut have been linked to diseases ranging from cancer to inflammatory disorders.

Protecting fertility in boys with cancer
Chemotherapy saves lives, but often at a price. For men who were treated during childhood with certain drugs, that price can be fertility.

Survivors’ Day reunites St. Jude patients, caregivers
More than 350 individuals returned to the hospital for Survivors’ Day 2014.

Gene therapy provides life-changing relief from hemophilia

People with severe hemophilia B, a bleeding disorder, are at risk of serious health problems and early death. To manage their disease, they endure lifelong injections of a blood clotting protein.

Now, gene therapy developed at St. Jude, University College London (UCL) and the Royal Free Hospital has transformed life for 10 men with severe hemophilia B.

Years after receiving a single DNA treatment, the men continue to produce their own clotting factor with minimal side effects. This treatment has significantly decreased their reliance on injections. Some now participate in sports like soccer without worrying about bleeding.

“The results so far have made a profound difference in the lives of study participants by dramatically reducing their risk of bleeding,” said Andrew Davidoff, MD, St. Jude Surgery chair. “This study provides the first clear demonstration of the long-term safety and efficacy of gene therapy.”

Injections of blood clotting protein can cost $250,000 a year. The researchers estimate that overall spending on injections of the missing protein has declined more than $2.5 million for study participants. The findings were published in the New England Journal of Medicine.

The power of phone calls for protecting the heart

Certain treatments for childhood cancer, while effective, can lead to serious heart problems later in life. For childhood cancer survivors, regular heart checkups can save lives. Yet despite the potential benefits, many survivors fail to get recommended checkups on schedule.

St. Jude researchers and their colleagues worked with 472 at-risk survivors to try a new approach. Results from the study showed that two brief phone counseling sessions from a nurse practitioner, added to a written care plan, doubled the chances of survivors getting recommended heart screenings.

“This intervention offers a model for how to motivate other cancer survivors to be more proactive about their health,” said Melissa Hudson, MD, of St. Jude Oncology. Researchers are now working on ways to implement the approach more widely.

The findings were published in the Journal of Clinical Oncology.

Science meets art

bearded man in front of painting at science meets art display

Trey Oguin of St. Jude Immunology discusses one of the images on display at the fourth annual Art of Science reception in Memphis. This collaborative project pairs St. Jude scientists with local artists. Researchers meet with their artist partners to share and discuss biomedical images from their research, which the artists use as inspiration for their works of art.

Two genes are double trouble in Ewing sarcoma

New secrets have been revealed by a detailed study of Ewing sarcoma, a cancer of the bone and soft tissue. Mutations in two genes appear to work together to drive cancer growth and reduce the chances a patient will survive. Mutations in the genes, STAG2 and TP53, were previously linked to Ewing, but their combined impact on survival was unknown.

“This is an important step in developing more effective diagnosis and treatment,” said Jinghui Zhang, PhD, of St. Jude Computational Biology. New clinical trials are being planned at St. Jude to test therapies that may be effective for Ewing patients with these mutations.

The work, published in the journal Cancer Discovery, was a collaboration between the St. Jude – Washington University Pediatric Cancer Genome Project and the Institut Curie-Inserm through the International Cancer Genome Consortium.

Launching a three-pronged attack against Ewing

St. Jude scientists have discovered a promising new triple-drug therapy for Ewing sarcoma. This is good news, as survival rates have been stalled for nearly two decades, and remain dismal for patients whose disease has spread or reappeared after treatment.

In laboratory studies, many tumors treated with the triple-drug therapy disappeared and did not return. The therapy consisted of two drugs currently used to treat Ewing sarcoma, plus experimental drugs called PARP inhibitors that interfere with a cellular process called DNA repair. Clinical trials are now being planned to evaluate the therapy in patients.

The findings were published in the journal Cell Reports.

First Lady Michelle Obama visits St. Jude and takes a selfie with a young male and a young female patient

First Lady visits St. Jude

During a visit to St. Jude, First Lady Michelle Obama poses for a selfie with patient Courtney Davis. As part of her historic visit, Mrs. Obama toured the hospital; spoke with patients, families and staff members; and answered a variety of lighthearted and thoughtful questions from patients during a question-and-answer session. Mrs. Obama joins Nancy Reagan, Barbara Bush and Hillary Rodham Clinton as First Ladies who have visited St. Jude.

A new connection between diet and disease

The human gut is home to trillions of bacteria and other microbes, known collectively as the intestinal microbiome. Changes in its makeup have been linked to diseases ranging from cancer to inflammatory disorders, though how has been poorly understood.

Intriguing results from St. Jude scientists now shed light on this connection. They found that diet-induced changes in the gut microbiome can alter susceptibility to an autoinflammatory bone disease. The key connection appears to be an immune molecule called IL-1beta, which promotes inflammation and is influenced by the dietary changes.

“These results are exciting because they help to explain how environmental factors like diet can influence susceptibility to autoinflammatory diseases,” said Thirumala-Devi Kanneganti, PhD, of St. Jude Immunology. “While multiple lines of evidence have suggested that diet can impact human disease, the scientific mechanism involved was a mystery.”

The findings were published in the journal Nature.

Protecting fertility in boys with cancer

Chemotherapy saves lives, but often at a price. For men who were treated during childhood with drugs called alkylating agents, that price can be fertility (see related article).

A St. Jude study of 214 such men revealed that over half of them had abnormal sperm levels years after their cancer treatment, with a quarter producing no sperm at all. The researchers used the data to determine the cumulative chemotherapy dose that poses a strong risk to male fertility. The information should help guide decisions for young patients who may want to have biological children of their own someday.

“Fertility preservation is important to patients and families, which is why at St. Jude we provide fertility counseling and preservation for active patients,” said Daniel Green, MD, of St. Jude Epidemiology and Cancer Control. “Until now, however, there was little information to guide clinicians, families and patients trying to assess the risk alkylating agents will pose to sperm production years in the future.”

The study appeared in the journal Lancet Oncology.

Survivors’ Day reunites St. Jude patients, caregivers

Survivors with Melissa Hudson, MD

More than 350 individuals returned to the hospital for Survivors’ Day 2014. Survivors and their families received updates and information about survivorship during a series of workshops. Participants also attended a panel discussion in which survivors shared their stories and received answers to questions about battling childhood cancer. Melissa Hudson, MD, Cancer Survivorship Division director (second from right), visits with survivors who now work on the hospital’s campus: (from left) Gabby Salinas of Chemical Biology and Therapeutics, Shane Glover of Biomedical Engineering, and Miguel Betances of ALSAC.

Abridged from Promise, Winter 2015

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