Childhood Cancer and the Promise of Gene Therapy

gene therapy

At its most basic level, gene therapy is a powerful technique for correcting mistakes (called mutations) in DNA of human cells. Lately, the therapy has been gaining traction as a potentially life-saving treatment for children with an array of inherited rare blood and immune disorders, as well as certain cancers. Gene therapies are being carefully tested in clinical trials, using improved, safer DNA delivery methods to reduce the risk of complications: some patients in earlier trials developed leukemia when treated with previous “vectors” – engineered viruses that transfer copies of normally functioning genes into cells of ill patients.  So far, …

Continue reading

Progress in the Treatment of Childhood Leukemia

Kimberly Stegmaier

Although treatments for childhood cancer patients are improving, cancer remains the leading cause of death by disease in children. Doctors and researchers are also focused on decreasing the toxicity of these treatments, which can have side effects years after a child finishes treatment. “The war against childhood cancer is hardly over,” says Kimberly Stegmaier, MD, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “We need to do better.” Stegmaier, who focuses her research on identifying new drug targets and new drugs for leukemia, Ewing sarcoma, and neuroblastoma, recently discussed advances in childhood cancer treatment in a Science, Innovation, …

Continue reading

What Is AT/RT Cancer?

SMALL_Mark_Kieran_SOG_5446_14

An atypical teratoid rhabdoid tumor (AT/RT) is a very rare and fast-growing tumor of the central nervous system. AT/RT is part of a larger group of malignant tumors called rhabdoid tumors, which are found outside the brain, in the kidneys, liver and other locations. AT/RT is most commonly found in the cerebellum and brain stem, which control most of the body’s basic functions. Since AT/RT grows very rapidly, symptoms can develop quickly over days or weeks. Increased head size often occurs in infants due to swelling inside the brain. Other common symptoms of AT/RT include headaches, especially upon waking in the …

Continue reading

You Are Not Alone: Supporting Siblings of Childhood Cancer Patients

When Phoebe Clark was 14-months old, she had little knowledge of what was happening to her brother, Harry. The 5-year-old had been diagnosed with a brain tumor and was undergoing multiple surgeries and radiation therapy at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. But a few years later, when Phoebe was 5-years old, her brother suffered a brain bleed and was back in the hospital. Suddenly, the hospital and the Jimmy Fund Clinic were not only at the center of Harry’s world, they were a major part of Phoebe’s life, too. “You really started to see the impact of what …

Continue reading

Isolation Patients Bond During Treatment

Isolation patients Shannon and Zack bond during treatment

This post originally appeared on the Jimmy Fund blog. By Erica Equi “People would say ‘I feel bad for her’…I’m the same person I ever was, but better, stronger,” says 14-year-old Shannon Curley, reflecting on the time she spent in isolation treatment at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “I wouldn’t change anything. I’ve met so many amazing people and learned some important things.” In December 2012, Shannon, a middle school basketball star and Billerica native, was diagnosed with acute bilineal leukemia. Due to the rarity and nature of her leukemia, doctors recommended an aggressive treatment plan. After three …

Continue reading

What’s New in Pediatric Brain Tumor Treatment?

Mark Kieran, MD, PhD

As one of the most difficult cancers to treat, childhood brain tumors are the leading cause of cancer-related deaths in children under age 10. However, researchers are making more progress than ever before. “Over the last 10 years there has been a lot of excitement about new treatments for pediatric brain tumors,” says Peter Manley, MD, a pediatric neuro-oncologist with Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and director of the Stop & Shop Family Pediatric Neuro-Oncology Outcomes Clinic. “We’re looking at brain tumors on a molecular level to find potential targeted therapies that can not only treat the cancer, …

Continue reading

Do Childhood Cancer Survivors Have a Higher Risk of Developing Cancer Later in Life?

SOG_4905_09

During cancer treatment, patients are often focused on just one thing: getting healthy. But for survivors, particularly of childhood cancer, it’s also important to learn about staying healthy after treatment, including prevention of new cancers that may occur later in life. We spoke with Lynda Vrooman, MD, associate medical director of the David B. Perini, Jr. Quality of Life Clinic at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center: Do childhood cancer survivors have an increased risk for developing a second cancer later in life (not just a recurrence)?  There are many factors that need to be taken into account when …

Continue reading

A Better Way to Care for Seriously Ill Children and Their Families

Joanne Wolfe, MD, MPH

This post originally appeared on WBUR’s Cognoscenti blog.  By Joanne Wolfe, MD, MPH How is it that, in this day and age, a talented teenager treated for lymphoma emerges cured but with a life-threatening eating disorder? How is it that, in our nation’s capital, a boy dying at home from neuroblastoma experiences excruciating pain in his final moments? How is that, when we develop new drugs to treat children with cancer, we do not, at the same time, routinely and in a standardized manner ask them how they are feeling? As a pediatric oncologist and palliative care physician, I was alarmed by stories like …

Continue reading

Childhood Cancer Patients Mark Hospital Departure with Bubbles

KC_20150508_BubbleParade-13_15

Avery McAvoy’s last day in the hospital was a long-awaited milestone, but all the 2-year-old cared about was how it ended: with bubbles. After 12 months of treatment for neuroblastoma at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, much of which was spent on the inpatient oncology and hematology unit at Boston Children’s Hospital, Avery participated on a Friday afternoon last month in what has become a beloved discharge ritual for clinical staff and patient families. Lining both sides of the corridor outside her room, nurses and other caregivers cheered and blew bubbles as Avery and her parents walked past …

Continue reading

Can Kids Get Melanoma? What Parents Need to Know

Yawkey Center for Cancer Care healing garden.

Although melanoma is more commonly found in adults, childhood and adolescent melanoma affects approximately 300 children in the U.S. each year. Thankfully, the incidence rate has started to decrease in recent years. While melanoma is the least common type of skin cancer in adults, skin cancer in children is almost always melanoma. The biggest increase in melanoma incidences has been in girls ages 15-19, possibly because girls are more likely than boys to sunbathe and use tanning beds. Because melanoma often appears differently in children than in adults, doctors and parents sometimes overlook it or misdiagnose it as a different skin …

Continue reading