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, …

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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, …

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I Have Metastatic Breast Cancer: What’s My Prognosis?

Rachel Freedman, MD, MPH

By Rachel A. Freedman, MD, MPH Metastatic breast cancer generally means that the cancer has spread beyond the breast and nearby lymph nodes under the arm. For approximately 10 percent of women with breast cancer, the disease has metastasized when they are first diagnosed, but metastatic disease can also occur when cancer returns after previous treatment. The prognosis is not the same for all metastatic breast cancer patients and can vary tremendously based upon multiple factors, including your breast cancer subtype (such as estrogen receptor [or ER] status and human epidermal growth factor receptor [or HER2] status), the degree of …

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Do All BRCA Mutations Come with the Same Cancer Risk?

Gentic testing for breast and ovarian cancer patients.

Women born with mutations in the genes BRCA1 or BRCA2 have an increased risk of developing breast and ovarian cancer, but the degree of increase depends on a variety of factors. Not all mutations within these genes raise the risk equally. A study published earlier this year tracked breast and ovarian cancer occurrences over a 75-year period in 31,000 women who had inherited mutations BRCA1 or BRCA2. The researchers found that mutations at either end of the BRCA1 gene increased the risk of breast cancer more than the risk of ovarian cancer. A group of mutations that occur in the middle …

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Why I Ride: Dr. Christopher Sweeney

Team Shawmut PMC

Since 1980, more than 88,000 cyclists have taken to Massachusetts’ roads for the Pan-Mass Challenge (PMC) to raise funds for cancer research and patient care at Dana-Farber. Among the riders are many patients, their family members, and their doctors. Christopher Sweeney, MBBS, medical oncologist in Dana-Farber’s Lank Center for Genitourinary Oncology, is one of them. We recently spoke with Dr. Sweeney about what motivates him to ride. Why did you start riding the PMC? In addition to treating patients with genitourinary cancers, particularly prostate and testicular cancer, I do clinical and translational research, with a focus on trying to find …

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Solving Puzzles with Cigall Kadoch

SMALL_SOG_1102_15

Growing up in the San Francisco area, Cigall Kadoch, PhD, had a passion for puzzles. The daughter of a Moroccan-born, Israeli-raised father and a mother from Michigan who together developed an interior design business, Kadoch excelled in school and pretty much everything else. Above all, she loved to solve brain-teasers. In high school, however, Kadoch came up against a problem that defied solution. Breast cancer took the life of a beloved family caretaker who had nurtured her interests in science and nature. “I was deeply saddened and very frustrated at my lack of understanding of what had happened,” recalls Kadoch, …

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Approval of Targeted Lung Cancer Drug Iressa Culminates Long Research Trail

precision medicine

The Food and Drug Administration’s approval of the drug Iressa® for a form of metastatic lung cancer represents a return to prominence for the compound that launched the era of targeted therapy in lung cancer – even if that wasn’t clear at the time of its original clinical trial in patients. The FDA approved Iressa (gefitinib) as a first-line treatment for patients with metastatic non-small cell lung cancer (NSCLC) whose tumor cells harbor specific mutations in the gene EGFR. “The approval of Iressa is important because newly diagnosed patients with EGFR-mutant lung cancer now have more treatment options,” said Pasi A. …

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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, …

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Science Illustrated: How Is Immunotherapy Used to Fight Cancer?

Immunotherapy, including the vitally important discovery of PD-1/PD-L1, is one of the most promising areas of cancer research today. One immunotherapy strategy  is to use checkpoint inhibitors to “take the brakes off” the immune system and unleash an attack on cancer cells. Learn about PD-1 and PD-L1, and how immunotherapy can be used fight cancer:

Know Your Surroundings: How Cancer Treatments Can Keep Cells From Supporting Tumors

By Eric Bender Multiple myeloma is a poster child for recent advances in treatment: In the past decade, the U.S. Food and Drug Administration (FDA) approved no fewer than nine treatments for the blood cancer, and several more drug approvals appear to be near. Not coincidentally, multiple myeloma is also a popular target that researchers use to study the interactions of tumor cells and their “tumor microenvironments” — the non-cancerous cells, molecules and blood vessels that surround and often support the malignant cells. “These new myeloma drugs are all based on understanding how the tumor cells interact with other cells …

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