Research in the News

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

Top 10 Medical Research Issues and Trends to Watch in 2015
January 15, 2015

Our friends at FasterCures prepared this list of the top 10 medical research issues and trends to watch in 2015, first appearing in The Huffington Post. Each one of these issues impacts children with cancer.

(Huffington Post) - At FasterCures, we are always thinking about ways to innovate the medical research system so that better treatments and cures get to patients faster. Read below to find out the top 10 issues and trends that we are watching this year:

  1. Crossing the aisle for cures. Kicking off 2015 will likely be the release of a draft of the 21st Century Cures legislation the U.S. House Energy & Commerce Committee has been developing under the leadership of Chairman Fred Upton and Representative Diana DeGette. The process itself has been a rare bright spot of comity and bipartisanship in the Congress, and a positive undertaking by members and staff to address a number of the entrenched systemic challenges in medical research that have become status quo. Will it make it through the wringer that it takes for an idea to become a bill and then a law? Perhaps, if patient groups and advocates like what they see and encourage their members to move it along.
  2. PDUFA VI: Regulations for patients? Also kicking off in 2015 will be the process of discussions leading to a new Prescription Drug User Fee Act (PDUFA), due to be passed in 2017. PDUFA V introduced some important initiatives to improve the regulatory process; what add-ons will the new round of negotiations bring? We'll be looking for continued improvement in the patient-focused drug development process that began with PDUFA V (specifically the inclusion of patient perspective in the assessment of desired benefits and tolerable risks, not just the understanding of disease and adequacy of existing treatments), a commitment to the sustainability of existing accelerated approval mechanisms like "breakthrough therapy designation," and more focus on the Food and Drug Administration's (FDA) regulatory science needs.
  3. Making the value case for new therapies. There was plenty of good news in FDA's unofficial year-end report, beginning with the fact that FDA approved a record 41 new drugs and biologics in 2014, the most since 1996, giving new options and hope to millions of patients. This included the highest number of orphan drugs and new biologics ever approved in one year, and 43 percent of new approvals were first-in-class drugs. While these numbers seem to bode well for the state of innovation - FasterCures' Senior Fellow Bernard Munos suggested earlier this month that we are on the cusp of a hyper-innovation age - how will the payer marketplace react? There may be a lot of very costly new drugs coming at us. Forbes's Matt Herper noted that the most important immunotherapy drugs just approved will carry price tags of $150,000 per patient per year. How will the value these new therapies deliver to patients be defined and evaluated? There is wide agreement that the current paradigm of uncertainty about how innovation will be paid for cannot be sustained, but will stakeholders be willing to align their interests with those of patients?

Read more 

Early Progress Seen in Pediatric Immunotherapy
December 2014

(Clinical Oncology News) - Heralded as a game-changer in adult oncology, immunotherapy is reshaping pediatric oncology too, but in very different ways.

"Unlike our adult [oncology] colleagues who are looking at immunotherapy as possibly their only effective therapy, [in pediatrics] we've got an approach that we need to integrate into chemotherapy, radiation and surgery. That gives us a lot to be excited about," said Paul M. Sondel, MD, PhD, the Walker Professor of Pediatrics and Human Oncology at the University of Wisconsin, Madison.

"This is really a paradigm shift for the field," said Dr. Sondel's colleague Christian Capitini, MD, an assistant professor of pediatric hematology, oncology and bone marrow transplantation. "We've had the same three modalities for decades. There's never really been a fourth option for kids once these modalities fail."

Although the key studies presented at this year's annual meeting of the American Society of Clinical Oncology (ASCO) looking at immunotherapy in pediatric populations were preliminary Phase I studies, Drs. Sondel, Capitini and other experts said the research demonstrates that two types of immunotherapies - chimeric antigen receptor (CAR) therapy, also called adoptive T-cell therapy, and monoclonal antibodies - are revolutionizing treatments for children with cancer.

Explaining the potential advantage of immunotherapeutic techniques with respect to resistance, Dr. Capitini said, "Even if you have a new chemotherapy drug for kids who have relapsed, these new drugs tend to act in a similar pathway to some other drug they got before. But when you talk about immunotherapy, resistance to chemotherapy does not mean resistance to immunotherapy. It's a totally new tool in your armamentarium, so it gives a lot of people new hope." Read more 

Young Adults with ALL Have Better Survival with Pediatric Regimens

(The Oncology Report) - Teens and young adults don't like being treated like children, but they should make an exception when it comes to acute lymphoblastic leukemia therapy, because pediatric ALL regimens are associated with significantly better event-free and overall survival among patients with ALL from the ages of 16 to 40 years.

Those findings come from a clinical trial 14 years in the making, in which 296 adolescents and young adults (AYA) with ALL were treated with an intensive pediatric chemotherapy combination regiment rather than a less-intensive adult regimen. At 2-year follow-up, the rate of overall survival (OS) was 78%, with the median overall survival not yet reached, and the event-free survival (EFS) rate was 66%, reported Dr. Wendy Stock from the University of Chicago Medical Center.

In contrast, EFS rates among AYA treated with adult regimens have historically ranged from 35-40%, Dr. Stock said at a press briefing at the annual meeting of the American Society of Hematology.

"These data really started 14 years ago at this ASH meeting when we presented data showing that young adults ages 16 to 20 who were treated on adult cooperative group studies in the United States fared much worse than the same age group who were treated on pediatric studies," she said. Read more 

Innovations in Treating Pediatric CNS Tumors Offer Promise for Young Patients

( - While pediatric central nervous system (CNS) tumors are relatively rare — with about 3,000 new cases each year in the U.S. — they present a unique challenge. The implications of treatment must be weighed carefully as significant side effects can linger into adulthood. The good news is advancements in treatments are offering patient families more options.

There is no known cause for most pediatric CNS tumors, which include a variety of types (medulloblastomas, astrocytomas and others), but the working theory revolves around an underlying genetic origin in many cases. Standard treatment often involves surgical resection, although some tumors may be in locations that are not amenable to resection and only a biopsy is possible. Following surgery, chemotherapy, and in some cases radiation therapy, are advised. These options, however, carry potential side effects, which may impact speech, cognition and emotional development in this young patient population.

Over the last 10 years, we have begun to better understand the molecular and genomic signatures of these tumors and are developing more targeted treatment approaches. In particular, by delving into how the cell regulates itself, we may be able to use different agents to target specific pathways to turn off certain cell functions, such as shutting down or blocking the pathways that allow cell growth or division.

Perhaps, the biggest change occurring in neuro-oncology at present is risk stratification. By identifying patients at low, standard and high risk for recurrence, it is possible to adjust treatments accordingly. If recurrence is less likely, the patient likely needs less therapy, reducing the chance of long-term implications. For example, radiation near the pituitary gland can cause endocrine problems, while chemotherapy can cause complications relating to memory and higher executive functioning. Read more 

Childhood Cancer Treatments Can Result in a Host of Chronic Illnesses

(The Sydney Morning Herald) - Standing at the doorway you are struck by the mess. Coffee mugs, paper bills and a crushed Fanta can lie scattered amongst discarded clothes. Look more closely and you see that the mess is concentrated in an area served by an oversized television, a laptop computer and a fraying beanbag. The coffee table in the centre of the disarray is smothered with brochures, its only visible part rimmed by aging coffee stains in concentric circles.

At first glance it looks like a university student's den, the kind that makes a visiting parent shudder in dismay. But the inhabitant of this flat is neither indulged teen nor tertiary student. Jade is a childhood cancer survivor, who at age 42, has lived here for the past decade.

As the chaos of her flat suggests, Jade is not coping well with everyday life. Her problems are both physical and psychological. She is one of several hundred childhood cancer survivors in Victoria who are living with the long term health effects of the treatment that so many years ago, saved their lives.

For those who do not make a complete recovery from childhood cancer, a line can never be drawn under this episode in their early lives. In Jade's case the treatment has left her with a range of health problems, including severe hormonal imbalance and impaired cognitive function, the last a result of the aggressive radiotherapy that advanced her cure. Read more 

Classifications of Gene Mutations in Children's Cancer May Lead to Better Treatments

(Drug Discovery & Development) - Oncology researchers studying gene mutations in the childhood cancer neuroblastoma are refining their diagnostic tools to predict which patients are more likely to respond to drugs called ALK inhibitors that target such mutations. Removing some of the guesswork in diagnosis and treatment, the researchers said, may lead to more successful outcomes for children with this often-deadly cancer.

"Some mutations are more important than others," said Yaël P. Mossé, MD, a pediatric oncologist at The Children's Hospital of Philadelphia, and a co-leader of the new study published online today in the journal Cancer Cell. "By integrating biochemistry into our clinical strategies, we can better match a patient's specific ALK-mutation profile with an optimum treatment." Mossé is also an assistant professor of Pediatrics in the Perelman School of Medicine, University of Pennsylvania.

"Understanding the specific mutations that trigger signals in cell receptors to stimulate cell growth will help us identify biomarkers for specific subtypes of neuroblastoma," said study co-leader Mark A. Lemmon, PhD, professor and chair of Biochemistry and Biophysics at Penn. Lemmon's research focuses on cell receptors in cancer. Read more 

Unlocking the Secrets of Pediatric Cancers Can Help Doctors Treat Children and Adults

(Huffington Post) - The fact that childhood cancer is, thankfully, a rare disease belies the fact that it is the leading cause of disease-related death in U.S. children, age 1-19. The fact that it is a rare disease also belies the fact the number of people with a direct stake in expanding research into pediatric cancer is quite large and extends well beyond the small number of children with cancer and their families. Not only are the combined life-long contributions of children cured of cancer enormous, but understanding cancers of young children could also hold the key to understanding a broad range of adult cancers. The time is ripe to allocate more resources, public and private, to research on pediatric cancer.

In an age of increased understanding of the genetic basis of diseases, one thing is striking about many childhood cancers. They are relatively "quiet" cancers, with very few mutations of the DNA. The young patients haven't lived long enough to acquire the large number of mutations that create the background "noise" associated with years of living. This makes it much easier to pinpoint the relevant genetic abnormalities in a young child's cancer.

Add to this the growing realization that biology, including how various tumors use common "pathways," not the malignancy's location in the body, is a major factor in how the cancer responds to treatment. Thus, a mechanism that's relatively easier to observe in the cancers of young children could help scientists understand cancers in adults in which the same mechanism is hidden amid the clutter of mutations acquired over a longer life. Unlocking the secrets of a rare pediatric brain tumor, for instance, could help unlock the secrets of lung cancers that share the same biology -- and provide an elegantly narrow focus in the search for more effective treatments of both. This turns on its head the current conventional paradigm of discoveries in adults slowly making their way to children. Read more 

Clearing Up Myths and Misconceptions About Clinical Trials

(Memorial Sloan Kettering Cancer Center) - In our last blog post about clinical trials, medical oncologist Paul Sabbatini shed light on how phase I trials are conducted at Memorial Sloan Kettering and how to know if you should consider participating in one. In this Q&A, we asked Dr. Sabbatini to demystify common myths and misconceptions surrounding clinical trials.

Myth: Clinical trials are only appropriate for people who have exhausted all of their standard treatment options. 

That is one of the most common misconceptions people have about clinical trials. It's true that we often offer clinical trials when the standard of care hasn't resulted in clinical benefit, or when there's no standard of care and the therapies being used aren't yielding good results. However, it's often appropriate to explore clinical trial options earlier in your course of treatment, even when you still have other standard therapies available.

For example, some patients may be eligible for a clinical trial in which we try to improve a standard drug by adding a new agent to it. In this way, it's possible to receive the standard of care with the hope that the addition of a novel strategy will result in greater benefit. Others may be able to try a promising new drug and then go back to standard therapies, which are always available if it wasn't successful.

Myth: I must give up standard therapy in order to participate in a clinical trial. 

Some clinical trials contain standard therapy as a part of the study.

Also, there are a whole host of nontherapeutic studies that people may not be aware of, many of which can be done while receiving standard therapy. For example, you may have the opportunity to have your tumor analyzed and archived in a tumor registry, get involved in a study that evaluates data gathered from periodic questionnaires, or participate in a psychosocial trial in which different approaches to counseling are evaluated. Read more 

Lymphoseek Approved for All Solid-Tumor Cancers

(MedPage Today) -Approval of the Lymphoseek system for detecting sentinel lymph nodes has been extended to cover all solid-tumor cancers, its manufacturer said Wednesday.

The FDA is also permitting the radiolabeled tracer system to also now be used with or without lymphoscintigraphy, according to Navidea Biopharmaceuticals.

Previously, Lymphoseek had been approved in conjunction with melanomas, breast cancers, and head and neck tumors.

The product uses a technetium-99 labeled tracer to identify lymph nodes serving areas near primary tumors, allowing oncologists to select for excision and analysis those nodes most likely to harbor emigrating cancer cells. The tracer is called tilmanocept, and it binds to CD206 receptors in lymph nodes. Read more 

T-Cell Therapy Puts Leukemia Patients in Extended Remission

(New York Times) -An experimental therapy has brought prolonged remissions to a high proportion of patients who were facing death from advanced leukemia after standard treatments had failed, researchers are reporting.

The therapy involves genetically programming cells from the patient's own immune system to fight the disease.

The research included 30 patients: five adults ages 26 to 60, and 25 children and young adults ages 5 to 22. All were severely ill, with acute lymphoblastic leukemia, and had relapsed several times or had never responded to typical therapies. In more than half, the disease had come back even after a stem-cell transplant, which usually gives patients the best hope of surviving. Their life expectancy was a few months, or in some cases just weeks.

Six months after being treated, 23 of the 30 patients were still alive, and 19 of them have remained in complete remission.

The study, by researchers at the Children's Hospital of Philadelphia and the Hospital of the University of Pennsylvania, is being published in The New England Journal of Medicine. Read more 

How I Manage: Survivorship Issues After Allogeneic Hematopoietic Cell Transplantation

(Clinical Oncology News) - Allogeneic hematopoietic cell transplantation (allo-HCT) is a potentially curative treatment for a number of diseases. Advances in transplantation practices and the development of supportive multidisciplinary centers have led to patients living longer after transplant than ever before. The majority of deaths occur within the first 2 years, after which 10-year survival is approximately 80% to 90% and brings with it a number of late effects that can cause significant patient and health care burden.

The most common late-onset causes of death after allo-HCT include recurrent disease, chronic graft-versus-host disease (GVHD), infection, cardiovascular and pulmonary complications, and secondary malignancy. Additionally, comorbidities such as chronic pain, fatigue, insomnia, sexual dysfunction, memory loss, mood changes, vision and dental complications, and financial stressors cause significant distress for patients. Increasing numbers of long-term survivors, especially those with difficult or no access to a long-term survivorship clinic, return to their local general hematologist or primary care physician (PCP) for follow-up. This article will address topics that both transplant and general physicians should be aware of in managing long-term survivors of allo-HCT.

Should allo-HCT recipients receive more rigorous screening for malignancy than the general population? 

Due to prior conditioning chemotherapy regimens and total body irradiation, allo-HCT survivors are at high risk for a secondary leukemia, post-transplant lymphoproliferative disorder, and new solid tumors, at a 2- to 4-times increased incidence compared with age-matched controls. Particularly higher incidences exist for secondary oral cancers and thyroid cancers, especially in patients with chronic GVHD or prior irradiation therapy, respectively. Late-effects guidelines published in 2012 by the Center for International Blood and Marrow Transplant Research emphasize the importance of regular age-appropriate cancer screening, which should be stressed to both patients and their primary physicians. Additionally, earlier mammography is recommended for women with a history of total body irradiation, starting at age 25 years or 8 years after radiation. Regular examination of the oral cavity and thyroid should be performed. Due to their increased incidence of skin cancers, transplant patients should be educated on the importance of sunscreen protection and regular skin examination. Ongoing clinical trials seek to determine whether there are particular genetic defects or lifestyle factors that increase the risk for secondary malignancy (eg, NCT00949052 is active and recruiting). Read more 

Inoperable Tumor Removed from Five Year Old, Thanks to 3D Printing

( - A team of surgeons at the Hospital Sant Joan de Déu in Barcelona, Spain has successfully completed a "highly complex" surgery to remove a tumor in a 5 year old boy, thanks to 3D printing.

The child was diagnosed with neuroblastoma, one of the most common cancers in children. It's an "extremely aggressive" tumor that forms in nerve tissue and is diagnosed primarily in children during the first five years of life. The treatment involves surgical removal of the tumor, combined with chemotherapy and/or radiotherapy.

However the tumor is very 'difficult' to remove because there are many blood vessels and arteries surrounding it. This means that surgeons must proceed with "extreme precision" to extract the tumor cells without damaging the arteries and endanger the patient's life.

According to Jaume Mora, head of the operation, the boy has been well treated and they have controlled the disease, but that led to the creation of a tumor in his stomach.

"We tried the surgery twice but we failed because we could not access," said Mora Wednesday in a press conference. But "instead of surrendering we tried to find a solution", he said.

In such a case, it is important to test the operation in advance. In collaboration with Foundation CIM Technology center at the Polytechnic University of Catalonia (UPC), the team has made ​​a prototype that reproduced exactly the tumor using 3D printing. Read more 

Risk-based Strategy Outlines New Approach to Treatment in Pediatric Soft Tissue Sarcomas

(Healio) - A risk-based treatment strategy categorized pediatric patients with nonrhabdomyosarcoma soft tissue sarcomas into distinct prognostic groups while utilizing less radiotherapy at lower doses, according to findings from the Children's Oncology Group study ARST0332 presented at the ASCO Annual Meeting.

"Nonrhabdomyosarcoma soft tissue sarcomas comprise about 4% of childhood cancer, but they have been very poorly studied during the past 4 decades with only three prospective clinical trials being conducted in North America," Sheri L. Spunt, MD, MBA, professor of pediatrics in hematology and oncology at the Lucile Salter Packard Children's Hospital, said during a presentation. "As a result, there is no clear standard of care for most of these patients. However, retrospective studies have suggested that outcome is predicted by tumor grade, size, the extent of surgical resection and the extent of disease — similar to adults with soft tissue sarcoma."

Spunt and colleagues sought to evaluate outcomes in 551 patients with a median age of 13.7 years with newly diagnosed nonrhabdomyosarcoma soft tissue sarcomas.

Patients were assigned to one of four risk-based treatment categories. Arm A included patients who underwent surgery with grossly excised low-grade tumors or a ≤ 5 cm widely excised high-grade tumor; arm B included those with a ≤ 5 cm marginally resected high-grade tumor; arm C included those with a > 5 cm grossly resected tumor ± metastases; and arm D included those with a > 5cm unresected tumor ± metastases. Read more 

Screening Childhood Cancer Survivors for Heart Problems

(Harvard School of Public Health) - One of the first studies to analyze the effectiveness of screening survivors of childhood cancer for early signs of impending congestive heart failure (CHF) finds improved health outcomes but suggests that less frequent screening than currently recommended may yield similar clinical benefit. The researchers, in a study published in the Annals of Internal Medicine, utilized a simulation-based model to estimate the long-term benefits associated with routine screening.

The study's findings suggest that the current CHF screening guidelines for survivors of pediatric cancer should be re-examined. The current guidelines recommend that survivors treated with chemotherapy agents known to affect long-term heart health be screened as often as every year, with a schedule dependent on their level of CHF risk. The new study suggests that screening survivors less often may be nearly as effective in detecting heart disease early. Some survivors might be better served by a different method of screening than the one currently used.

"It is important to monitor survivors so we can reduce the late effects of treatment whenever possible, but we may be asking them to be tested too often, which burdens both individuals and the health care system," says senior author Lisa Diller, MD, chief medical officer of Dana-Farber/Boston Children's Cancer and Blood Disorders Center. "We think it is worthwhile to review the current CHF screening guidelines."

"Our findings suggest that there is a long-term benefit in screening survivors at elevated risk for CHF," says lead author Jennifer Yeh, PhD, of the Center for Health Decision Science at Harvard School of Public Health. "Yet less frequent screening than currently recommended may be reasonable when other factors are considered. We hope these results can help inform the ongoing discussion about screening childhood cancer survivors." Read more 

Mayo Clinic Trial: Massive Blast of Measles Vaccine Wipes Out Cancer

(Star Tribune) - Stacy Erholtz was out of conventional treatment options for blood cancer last June when she underwent an experimental trial at the Mayo Clinic that injected her with enough measles vaccine to inoculate 10 million people.

The 50-year-old Pequot Lakes mother is now part of medical history.

The cancer, which had spread widely through her body, went into complete remission and was undetectable in Erholtz's body after just one dose of the measles vaccine, which has an uncanny affinity for certain kinds of tumors.

Erholtz was one of just two subjects in the experiment and the only one to achieve complete remission. But the experiment provides the "proof of concept" that a single, massive dose of intravenous viral therapy can kill cancer by overwhelming its natural defenses, according to Dr. Stephen Russell, a professor of molecular medicine who spearheaded the research at Mayo.

"It's a landmark," Russell said in an interview last week. "We've known for a long time that we can give a virus intravenously and destroy metastatic cancer in mice. Nobody's shown that you can do that in people before."

Until now.

The research, published online Wednesday in the journal Mayo Clinic Proceedings, represents a "benchmark to strive for and improve upon," according to an accompanying editorial by Dr. John C. Bell of the Centre for Innovative Cancer Research in Ottawa...Read more 

Chemist Turns Software Developer After Son's Cancer Diagnosis

( - A scientist's ambitious plan to create an early detection system for eye cancer using people's home cameras is coming along.

Last fall, we told you about Bryan Shaw's scheme. He believes parents' cameras can reveal whether their baby has leukocoria, a white glow coming from the pupil when you shine a light in their eyes. This so-called white eye can be an early sign of retinoblastoma, a rare form of eye cancer.

Children with the disease develop tumors on their retinas at the back of their eyeballs. Shaw wants to make software that will recognize this leukocoria.

This isn't Shaw's field. He's a chemist at Baylor University. But he got involved with leukocoria detection when his newborn son was diagnosed with retinoblastoma in 2008. He and his wife, Elizabeth, had seen leukocoria in baby pictures when Noah was 3 months old, but at the time the photos were taken, they hadn't realized what was going on.

Naturally enough, after his son's diagnosis, Shaw's first thoughts were to do whatever it took to save his son's life....Read more 

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 children. 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   

Our Disproportionate Focus on Adult Over Pediatric Cancer Research

( - Danny and other children with cancer could benefit sufficiently from more research into pediatric-specific treatments. While adult-oriented chemotherapy is proven to help cure cancer, it has innumerable side effects. Even though the treatments are approved for children, they are also the same used for adults, just at a lower dose.

But there is not enough funding for childhood cancer, specifically. The National Cancer Institute, a government organization, provides funding for researchers, but only 10 percent of them can move forward with their findings due to budget cuts. Most of the financial support researchers receive is from philanthropists.

In the meantime, research that could benefit children on an individual level stays in the lab, and doctors prescribe the same regimens that can be successful, but can also hurt the patient in several ways. Researchers say they are working hard to discover new theories and treatments, but they feel they are being held back.

"Ninety-six percent of grants don't get funded," said Dr. William Carroll, researcher and director of the cancer institute at New York University. "There's no doubt there's less funding available, and it's driving people out of the field." Read more 

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