Research in the News

Following are some of the most recent, significant findings, as reported by media across the country.

Adult Cancer Drugs Show Promise Against an Aggressive Childhood Brain Tumor

(Digital Journal) - The quest to improve survival of children with a high-risk brain tumor has led St. Jude Children's Research Hospital investigators to two drugs already used to treat adults with breast, pancreatic, lung and other cancers. The study was published today online ahead of print in the journal Cancer Cell.

Researchers demonstrated that the drugs pemetrexed and gemcitabine killed cells from mouse and human brain tumors, called group 3 medulloblastoma, growing in the laboratory. Medulloblastoma is diagnosed in about 400 children annually in the U.S., making it the most common pediatric brain tumor. Of the four distinct medulloblastoma subtypes, patients with group 3 medulloblastoma have the worst prognosis.

Used together, pemetrexed and gemcitabine doubled life expectancy of mice with human group 3 medulloblastoma, compared to untreated mice. When pemetrexed and gemcitabine were combined with two chemotherapy drugs currently used to treat pediatric medulloblastoma, the mice lived even longer.

The drugs were identified by screening the St. Jude library of 7,389 compounds looking for ones that targeted group 3 mouse tumor cells rather than normal mouse brain cells. The library included all 830 drugs U.S. Food and Drug Administration (FDA) approval. Pemetrexed and gemcitabine emerged as the top candidates, based in part on their ability to kill group 3 medulloblastoma tumor cells at concentrations that researchers showed were safe and achievable in patients...Read more 

New Ewing Sarcoma Research Center at Texas Children's Hospital Made Possible by Virani Family

This article features CureSearch Young Investigator Jason Yustein, MD, PhD. To learn more about his research click here.

(Bio News Texas) -The Texas Children's Cancer Center has opened a new space to develop more effective diagnostics and therapeutic approaches for Ewing Sarcoma in chldren. The goal of the new Faris D. Virani Ewing Sarcoma Center is to help find the cure to the rare childhood disease, in which cancer cells are found in bones or soft issues.

Made possible by a $2 million gift from the Virani family, the center will be directed by Dr. Jason Yustein, assistant professor of pediatrics at Baylor College of Medicine, and will honor Asha and Farid Virani's son Faris. During the event that has celebrated the Virani family's gift, Faris, who has been fighting Ewing sarcoma, presented Yustein with a piggy bank containing $1,000.

Over the past 30 to 40 years, only marginal advancements have been made in the care and management of patients with Ewing sarcoma due to the lack of understanding of its biology. "There has been a lack of new chemotherapeutic agents introduced for patients with this disease and current treatments are extremely intensive and often have both short- and long-term side effects that can negatively impact lifestyle and quality of life for these patients," said Yustein...Read more 

Novel Approaches and Mechanisms of Immunotherapy for Glioblastoma

This article was published by CureSearch Young Investigator Meenakshi Hegde, MD. To learn more about her research click here.

(Discovery Medicine) - Abstract: Glioblastoma (GBM) is the most aggressive primary brain tumor. Combination therapy with surgery, radiation, and chemotherapy is not curative at present and carries a significant risk of toxicity. Advancements in the knowledge of tumor biology and tumor microenvironment have led to the development of novel targeted therapies for glioblastoma. In the past 15 years, a vast amount of pre-clinical data has been generated for glioblastoma immunotherapy. Translating these promising results into the clinic is, however, still an evolving process. Early clinical trials have demonstrated the feasibility and safety of several such approaches in patients with recurrent as well as newly diagnosed glioblastoma. Both passive as well as active immunotherapeutic modalities have also shown potential clinical benefit in at least a subset of these patients. This brief review discusses 'why' and 'how' various types of immunotherapies are being employed to treat glioblastoma.

Introduction: Glioblastoma (GBM) is the most common primary brain tumor in adults with the annual incidence of over 17,000 in the United States (Grossman et al., 2010; Omuro and DeAngelis, 2013). Despite the considerable improvements made in the conventional therapy for glioblastoma over the recent years, prognosis remains extremely poor with a median survival of 18 to 21 months (Finlay et al., 1995; Grossman et al., 2010; Johnson et al., 2012). Complete tumor resection is difficult owing to the diffusely infiltrative nature of the tumor (Grossman et al., 2010). Concomitant and adjuvant chemotherapy with the alkylating agent temozolomide along with radiation has only been shown to improve median survival by 2.5 months (Stupp et al., 2005). In the past decade, substantial amount of progress has been made in dissecting the glioblastoma biology in relation to its microenvironment as well as the host immune system. This has paved the way for researchers to explore novel targeted immunotherapeutic approaches that have the potential to improve cure rates with minimal toxicities, due to sparing of the surrounding normal brain structures. Here we review the advances made in some of the passive and active immunotherapeutic strategies for glioblastoma...Read more 

Infants with leukemia inherit susceptibility

(Washington University in St. Louis) - Babies who develop leukemia during the first year of life appear to inherit an unfortunate combination of genetic variations that can make the infants highly susceptible to the disease, according to a new study at Washington University School of Medicine in St. Louis and the University of Minnesota.

The research is available online in the journal Leukemia.

Doctors have long puzzled over why it is that babies just a few months old sometimes develop cancer. As infants, they have not lived long enough to accumulate a critical number of cancer-causing mutations.

"Parents always ask why their child has developed leukemia, and unfortunately we have had few answers," said senior author Todd Druley, MD, PhD, a Washington University pediatric oncologist who treats patients at St. Louis Children's Hospital. "Our study suggests that babies with leukemia inherit a strong genetic predisposition to the disease."

The babies appear to have inherited rare genetic variants from both parents that by themselves would not cause problems, but in combination put the infants at high risk of leukemia. These variants most often occurred in genes known to be linked to leukemia in children, said Druley, an assistant professor of pediatrics...Read more 

New Promising Information to Treat, Cure Childhood Leukemias

(Science World Report) - Researchers from the UT Health Center may have pinpointed a protein that could potentially play a key role in the development of pediatric AML-promising new information to treat and cure possible childhood leukemias.

Background information from the study notes that AML begins when maturing cells start to grow into different kinds of blood cells. For this, the cancerous cells grow and proliferate in an abnormal way as they fail to develop into normally functioning white blood cells. The high levels of a protein known as WTAP can contribute to abnormal cell behavior, which could harm the effectiveness of treatments for this type of leukemia.

For Sanja Bansal, Ph.D., and researchers at Greehey Children's Cancer Research Center at The University of Texas Health Science Center at San Antonio, they worked to "knock down" leukemia cells via a technique known as WTAP expression in AML cells. The results yielded successful findings, according to background information from the study.

"Knocking down this protein, WTAP, greatly suppressed proliferation and induced differentiation," said Hima Bansal, Ph.D., senior research associate at the Health Science Center and lead author of the paper, via a press release. "It took care of both problems."...Read more 

Child's PTSD Risk Not Increased By Cancer Diagnosis

(Oncology Nurse Advisor) - Despite being diagnosed with life-threatening illnesses, childhood cancer patients are no more likely than their healthy peers to develop post-traumatic stress disorder (PTSD).

According to the a study from carried out at St. Jude Children's Research Hospital in Memphis, Tennessee, young cancer patients were also more likely than children who experience other stressful events to report having benefited from the experience. Reported benefits included developing greater empathy and growing closer to family and friends. The research appears in the Journal of Clinical Oncology (2014; doi:10.1200/JCO.2013.49.8212).

The study included 255 St. Jude patients who were between ages 8 to and 17 years when their cancer was diagnosed. Based on self-reported patient symptoms, researchers concluded that 2.8%, or 7 patients, met the criteria for a diagnosis of PTSD either when the study was conducted or in the prior to the study timeframeast. The PTSD was cancer-related in two 2 patients. In the other five 5 patients, the anxiety disorder was linked to a drive-by shooting, Hurricane Katrina, or other stressful events.

This incidence of PTSD was comparable to with rates reported in community samples of children without cancer and a similar group of 101 healthy peers recruited for the study. The prevalence, however, contrasts sharply with previous reports from other investigators who identified cancer-related PTSD as a widespread problem. Those estimates suggested that 20% to 35% of childhood cancer patients would develop PTSD...Read more 

High Long-Term Survival of Most Common Pediatric Brain Tumor, Less When Radiation Was Used

(Science Codex) - The first comprehensive, large-scale cohort study of the long-term survival of children treated for low-grade gliomas, the most common pediatric brain tumor, finds that almost 90 percent are alive 20 years later and that few die from the tumor as adults. However, children who received radiation as part of their treatment had significantly lower long-term survival rates than children who were not radiated, researchers from Dana-Farber/Boston Children's Cancer and Blood Disorders Center report. These findings stand regardless of whether surgeons could successfully remove a child's entire tumor or only part of it, suggesting that the radiation itself may explain the difference.

The findings were published online by the journal Pediatric Blood and Cancer.

Delivery of radiation to children's developing brains has been linked to a number of adverse long-term effects, including cognitive development and endocrine function. While a number of major hospitals, including Dana-Farber/Boston Children's, have almost eliminated radiation in treating low-grade gliomas, the extent to which other institutions employ radiation varies, according to the study's senior author, Peter Manley, MD, of the Brain Tumor Center at Dana-Farber/Boston Children's...Read more 

Learn more about radiation therapy here.

Antipsychotic Drug Fights Cancer

(Oncology Nurse Advisor) - In a prime example of finding new uses for older drugs, studies in zebrafish show that a 50-year-old antipsychotic medication called perphenazine can actively combat the cells of a difficult-to-treat form of acute lymphoblastic leukemia (ALL). The drug works by turning on a cancer-suppressing enzyme called PP2A and causes malignant tumor cells to self-destruct.

The findings suggest that developing medications that activate PP2A, while avoiding the psychotropic effects of perphenazine, could help clinicians make much-needed headway against T-cell ALL (T-ALL), and perhaps other tumors as well.

The study was led by A. Thomas Look, MD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center, and reported the in the Journal of Clinical Investigation...Read more   

Gene for Most Common Childhood Cancer Identified

(University of Washington) - An aberrant gene has been found to cause the most common childhood cancer in the world, pre-B cell acute lymphoblastic leukemia or ALL.

The gene, PAX5, has long been known to be involved in ALL. The new study indicates a mutation in the gene alone is sufficient to eventually cause the disease. ALL affects nearly 3,000 children and teenagers in the United States each year.

The discovery should make it possible to screen for the gene in families with a history of the disease and suggests new strategies for treating the disease, said Dr. Marshall Horwitz, professor of pathology and of medicine at the University of Washington. He is a co-author of the new study...Read more   

Childhood Cancer Survivors Had Higher Modifiable Cardiovascular Disease Risk Factors

(dailyRx News) - Childhood cancer is very rare. And when it does strike, most kids live through it. The lifesaving treatments affect young bodies though, and those effects can show up later in life.

Adults who were treated for cancer as children were more likely to develop cardiovascular disease than the general population, a new study has discovered.

The researchers examined the incidence of and modifiable risk factors for cardiovascular disease in childhood cancer survivors...Read more   

Proton Therapy Offers New, Precise Cancer Treatment for Children With High-Risk Neuroblastoma

(Medical Xpress) – Proton therapy, using high-energy subatomic particles, may offer a precise, organ-sparing treatment option for children with high-risk forms of neuroblastoma. For patients in a new study of advanced radiation treatment, proton therapy spared the liver and kidneys from unwanted radiation, while zeroing in on its target.

"As survival rates improve for children with neuroblastoma, we need to reduce treatment-related long-term toxicities," said study leader Christine Hill-Kayser, M.D., a radiation oncologist in the cancer center at The Children's Hospital of Philadelphia (CHOP). "Proton beam therapy offers precise targeting with less radiation exposure to healthy tissue."

Hill-Kayser and colleagues published their study online June 4, 2013 in Pediatric Blood & Cancer...Read more   

Pregnancy Possible for Many After Childhood Cancer

(Reuters Health) – Despite research indicating that women who had cancer as girls have difficulty getting pregnant, a new study suggests that most can conceive, though it might take longer than usual.

Researchers from the U.S. and Canada found that female childhood cancer survivors tended to take longer to conceive than their sisters, but nearly two-thirds of the infertile survivors eventually did get pregnant.

"The main message counters what some people have thought, which is if you had cancer you won't be able to get pregnant or have children," said Dr. Lisa Diller, the study's senior author, from the Dana-Farber/Boston Children's Cancer and Blood Disorders Center...Read more   

First National Clinical Trial for Incurable Children's Cancer Launched by Van Andel Institute

( -- GRAND RAPIDS, MI – The first national clinical trial ever approved to study all incurable children’s cancers is underway at Van Andel Institute.

Of the 10,000 pediatric cancer cases diagnosed each year, about 25 percent are considered incurable even with aggressive treatments. The study will analyze the molecular makeup and genetic mutations of individual tumors to create a targeted treatment plan for each patient.

"We are very excited to have this study at Van Andel Institute," said Dr. Giselle Sholler, the pediatric oncologist leading the research...Read more   

Scientists Find Mutation Driving Pediatric Brain Tumors

( -- BOSTON—A type of low-grade but sometimes lethal brain tumor in children has been found in many cases to contain an unusual mutation that may help to classify, diagnose and guide the treatment of the tumors, report scientists at Dana-Farber Cancer Institute.

The researchers led a study of pediatric low-grade gliomas, samples of which were collected through an international consortium organized by brain tumor specialists at Dana-Farber/Children's Hospital Cancer Center. Their findings are being published online by the Proceedings of the National Academy of Sciences (PNAS) the week of April 29.

Low-grade gliomas are the most common type of pediatric brain tumors, diagnosed in about 1,000 young patients annually in the United States. There are about 30 distinct types of these tumors, which arise from specialized cells called glia in the brain. Low-grade gliomas are generally slow-growing, said Keith Ligon, MD, PhD, a senior author of the study, but they behave unpredictably...Read more   

Retinoblastoma More Invasive at Diagnosis in Eacial, Ethnic Minority Children

( -- When the eye cancer retinoblastoma is diagnosed in racial and ethnic minority children whose families don't have private health insurance, it often takes a more invasive, potentially life-threatening course than in other children, probably because of delays in diagnosis, Dana-Farber/Children's Hospital Cancer Center (DF/CHCC) researchers will report at the 26th annual meeting of the American Society of Pediatric Hematology Oncology being held in Miami, April 24-27.

By analyzing data and tumor samples from 203 children across the United States who had been treated for retinoblastoma, the investigators found that the disease was more invasive at diagnosis in patients who were non-white, Hispanic, uninsured, or covered by Medicaid. Researchers now need to explore why the disease tends to be diagnosed later in such children and how those delays can be eliminated, said the study authors.

Retinoblastomas are tumors that develop during childhood in the light-sensitive retina at the back of the eye. There are about 350 new cases diagnosed each year in the United States. Treatment may require surgical removal of the affected eye and, if the disease...Read more   

Inherited Genetic Variations Have a Major Impact on Childhood Leukemia Risk

(PRNewswire-USNewswire) -- Humans have between 20,000 and 25,000 genes that carry instructions for assembling the proteins that do the work of cells. Work led by St. Jude Children's Research Hospital found that children who inherit certain variations in four particular genes are at much higher risk of developing acute lymphoblastic leukemia (ALL).

The study also showed that Hispanic patients were more likely than patients of European or African ancestry to inherit high-risk versions of two of these genes. ALL rates are known to be higher among Hispanic children than those of European or African ancestry, this discovery points to at least one reason for that difference.

Each person's genome includes two copies of each gene, one from each parent. Thus, individuals could inherit up to eight high-risk versions of the four genes tied to an increased ALL risk. In this study, researchers found that having more than five copies of the risk genes resulted in a nine-fold greater risk of developing ALL in childhood than inheriting no more than one copy...Read more   

Drugs Aim to Make Several Types of Cancer Self-Destruct

Saturday, December 22 (New York Times) For the first time ever, three pharmaceutical companies are poised to test whether new drugs can work against a wide range of cancers independently of where they originated — breast, prostate, liver, lung. The drugs go after an aberration involving a cancer gene fundamental to tumor growth. Many scientists see this as the beginning of a new genetic age in cancer research. 

Great uncertainties remain, but such drugs could mean new treatments for rare, neglected cancers, as well as common ones. Merck, Roche and Sanofi are racing to develop their own versions of a drug they hope will restore a mechanism that normally makes badly damaged cells self-destruct and could potentially be used against half of all cancers...Read more 

Blood Cancer Studies Assess Genetics, Modified Treatment To Improve Outcomes, Reduce Toxicity

Research identifying genetic factors that affect survival of patients with blood cancers and evaluating the effectiveness of modified treatment strategies to improve outcomes while reducing toxicity were presented at the 54th Annual Meeting of the American Society of Hematology (ASH).

While the cancer research community has seen many significant therapeutic advances over the last decade, only recently have investigators identified how patients' individual genetic makeup influences their short- and long-term response to therapy, demonstrating that while the disease may respond positively to therapy, the patient may not... Read more 

Drugmakers Nudged by FDA to Find Childhood Cancer Cures

Tuesday, December 4 (Yahoo) Four experimental cancer treatments that may one day help gravely ill children are facing review by U.S. pediatric advisers who have been working for years to find ways to develop more drugs for this underserved population.

The medicines from GlaxoSmithKline Plc (GSK), Amgen (AMGN) Inc., Threshold Pharmaceuticals Inc. (THLD) and Boehringer Ingelheim GmbH have the potential to treat children with a variety of tumors and fast-spreading leukemia. The pediatric advisers to the Food and Drug Administration, in a meeting today, will discuss how best to test the medicines in sophisticated clinical trials in kids that may eventually lead to marketing approval... Read more 

Existing Drugs Offer New Treatment Options for High-Risk Childhood Leukemia Subtype

MEMPHIS, Tenn., Aug. 13, 2012 /PRNewswire-USNewswire/ -- Scientists have identified new genetic alterations underlying a high-risk subtype of the most common childhood cancer that could be effectively targeted with existing leukemia therapies.

The study focused on a subtype of acute lymphoblastic leukemia (ALL) known as Philadelphia chromosome-like ALL (Ph-like ALL). This subgroup accounts for as much as 15 percent of childhood ALL that is associated with a high risk of relapse and a poor outcome. The genetic changes driving the disease were previously unknown for about half of all patients with Ph-like ALL. The work identified new alterations in genes that regulate how cells grow and proliferate. St. Jude Children's Research Hospital investigators led the research, which appears in the online edition of the journal Cancer Cell... Read more 

Key Mutations Discovered for Most Common
Childhood Brain Cancer

Sunday, July 22 (Boston Business Journal) BOSTON -- Researchers at Dana-Farber/Children's Hospital Cancer Center (DF/CHCC) and several collaborating institutions have linked mutations in specific genes to each of the four recognized subtypes of medulloblastoma, the most common malignant brain tumor of children. The discovery, reported July in the journal Nature, provides doctors with potential biomarkers for guiding and individualizing treatment and reveals prospective therapeutic opportunities for countering this devastating malignancy.... Read more 

Big Progress in Treating Side Effects of Adults
Who Had Cancer as Children

Tuesday, July 17 ( -- As childhood cancer treatments have become more successful, more and more patients are surviving to adulthood. In fact, the National Cancer Institute estimates that one in nearly every 500 adults in the United States between ages 20 and 45 is a survivor of childhood cancer.

With longer survival, there’s more chance that young patients will experience the late effects of their cancer treatments as they get older.... Read more 

Study Shows Worse Outcomes for Young Adults With Acute Lymphoblastic Leukaemia: Presented at ASCO

TUESDAY, June 5 ( CHICAGO -- A phase 3 study of adolescent and young adult patients with high-risk acute lymphoblastic leukaemia showed more relapses and worse survival in the older patients.... Read more 

Childhood Cancer Survivors Face High Breast Cancer Risk

MONDAY, June 4 ( -- Children diagnosed with cancer remain at risk from a variety of serious, long-term complications even years after doctors pronounce them cured.

Now, new research shows that girls treated with radiation for pediatric tumors face a later risk of breast cancer that's six to seven times as high as that of other women.... Read more 

Decoding Cancer: Scientists Release 520 Tumor Genomes from Pediatric Patients

WEDNESDAY, May 30 ( -- Could the answer to cancer lie within our own DNA? Childhood cancer experts are hoping that at least some of what drives pediatric cancers can be gleaned from the genomes of patients and their tumors. That's the thinking behind the Pediatric Cancer Genome Project (PCGP), a three-year, $65 million effort to sequence major pediatric cancers... Read more 

Folic Acid Tied to Lower Child Cancer Risks

WEDNESDAY, May 23 ( -- Rates of two rare childhood cancers declined after the U.S. began requiring grain products to be fortified with the B vitamin folic acid, a new study finds... Read more 

Study Presents Potential New Treatment Options for Pediatric Neuroblastoma Patients

HOUSTON, April 27, 2012 /PRNewswire-USNewswire/ -- A nationwide study led by Dr. Jed Nuchtern, chief of the division of pediatric surgery at Texas Children's Hospital, a pediatric surgeon with Texas Children's Cancer Center and professor of surgery at Baylor College of Medicine, found that the majority of infants with a particular form of neuroblastoma – a childhood tumor that often requires intensive chemotherapy and surgery – excel in their overall progress and survival when the tumor is monitored without surgical resection... Read more 

Childhood Leukemia Survival Rates Reach 90 Percent
Death rates from acute lymphoblastic leukemia dropped substantially, 15-year study finds

MONDAY, March 12 (HealthDay News) -- Children with the most common type of leukemia now have a dramatically better chance of survival, a new study shows.

The researchers found five-year survival rates among children with acute lymphoblastic leukemia (ALL) increased from about 84 percent to 90 percent from 1990 to 2005. Surviving for five years is considered a cure because so few deaths occur past that timeframe... Read more 

New Guidelines for Adolescent and Young Adult Cancer Patients Issued

National Comprehensive Cancer Network guidelines address critical issues for these patients upon diagnosis, during treatment and after therapy... Read more 

NIH Study Links Childhood Cancer to Delays in Developmental Milestones
Language, motor deficits, seen within months of starting treatment

Infants and toddlers who have been treated for cancer tend to reach certain developmental milestones later than do their healthy peers, say researchers at the National Institutes of Health and in Italy... Read more 

Inherited Risk Factors for Childhood Leukemia are More Common in Hispanic Patients
PR Newswire, 1/30/12

MEMPHIS -- Hispanic children are more likely than those from other racial and ethnic backgrounds to be diagnosed with acute lymphoblastic leukemia (ALL) and are more likely to die of their disease. Work led by St. Jude Children's Research Hospital scientists has pinpointed genetic factors behind the grim statistics... Read more 

New Genetic Markers May Tailor Leukemia Treatment - OncologySTAT
Oncology Professional Resource Network, 1/16/12

SAN DIEGO (EGMN) - Novel genetic alterations have been identified in a new subtype of high-risk B-cell acute lymphoblastic leukemia that could be effectively targeted with existing therapies. The subtype, termed Ph-like ALL, was first identified by the Children's Oncology Group in 2009 (N. Engl. J. Med. 2009;360:470-80), and accounts for up to 15% of pediatric acute lymphoblastic leukemia (ALL) cases... Read more 

New Scoring System Devised for Youth With Hodgkin's Lymphoma
Pediatric News, 1/10/12

SAN DIEGO – A simple scoring system identified a subset of young patients with Hodgkin's lymphoma who are predicted to have an event-free survival rate of less than 80%.

The system, known as the Childhood Hodgkin International Prognostic Score (CHIPS), found that four factors were predictive of worse event-free survival: stage IV disease, large mediastinal adenopathy, albumin level of less than 3.5 g/dL, and fever, Dr. Cindy L. Schwartz reported during a poster session at the annual meeting of the American Society of Hematology... Read more 

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