Courtesy of NorthJersey.com
Dr. Burton Appel remembers when he wrote a college letter of recommendation for a patient. Not an uncommon request for most physicians. But this one is particularly noteworthy for Dr. Appel.
When the now college sophomore was three years old, Dr. Appel treated him for leukemia. The boy, Peter Bernhard, 19, of Waldwick, has been in remission for years and is considered cured of the disease.
It is such a pleasure to see him grow and develop into the young man he is,” said Dr. Appel, attending physician in the Division of Pediatric Hematology/Oncology (Children’s Cancer Institute), Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center, Hackensack.
Bernhard, who sees Dr. Appel once a year for follow ups, said the treatment process was upbeat thanks to Dr. Appel.
“He even helped make it fun. He’d race me down the halls,” Bernhard, who attends Stockton University said. “He’s not just my doctor. He’s also my friend.”
A diagnosis of pediatric leukemia, or other childhood blood disorders, can be devastating news to a family. However, hospitals, such as HackensackUMC, offer advanced methods of treatment in an area of the facility designed specifically for children and their families. Hospitals, including HackensackUMC and Englewood Hospital and Medical Center, in Englewood, also help children deal with the emotional impact of various types of medical testing, exams and treatments.
The research team at HackensackUMC’s Children’s Cancer Institute has access to more than 100 clinical trials. The Joseph M. Sanzari Children’s Hospital provides medical and surgical pediatric care in more than 30 specialties, including blood and marrow transplantation, cardiology and pediatric cancer and blood disorders. Dr. Appel said he helps children from newborn to the young adult age of 22.
Blood Disorders in Children - Progress and Hope
Blood disorders, such as leukemia, starts in the blood forming tissue. The cancer cells overcome and replace normal blood and marrow cells (www.cancercenter.com). Blood carries oxygen, vitamins and other necessities to our tissues. When compromised, patients can experience such symptoms as easy bruising, fatigue, fever, chills and frequent infections.
Leukemia is considered the most common cancer in children and teens, according to the American Cancer Society. Other blood disorders in children include various types of anemia, when patients have fewer healthy red blood cells than normal.
In general, the treatment for acute lymphocytic leukemia (ALL) in children has advanced and changed enormously since the 1960s, Dr. Appel said. Chemotherapy medicines can now cure more than 80 percent of children with ALL, compared to about four percent in the 1960s. About three out of four types of childhood leukemia are acute lymphocytic leukemia, with the remaining being acute myelogenous leukemia (AML).
Basic treatments for leukemia include various types of chemotherapy. One procedure, called photophoresis, can filter a patient's blood to help reduce the side effects of a bone marrow treatment, Dr. Appel said.
The high cure rates can be attributed to the many clinical trials performed nation-wide, Dr. Appel said. New drug combinations are tested on patients, where they can be refined and perfected. Dr. Appel said parents often find it reassuring that treatments have been tested on thousands of children in similar situations.
"That's really how progress is made. They are building on the successes of the past," Dr. Appel said. "Childhood leukemia is a treatable and curable disease."
Keeping Life Normal for Pediatric Patients
Hospital visits can be overwhelming for children, their siblings and their families. Creating an atmosphere that is supportive, while child friendly, is key.
Englewood Hospital and Medical Center, for example, features a Child Life Program. The program helps ease anxiety and stress before medical procedures or hospitalization for children and their families. A certified child life specialist works with children to explain all the procedures he or she may undergo at the hospital.
Courtesy of City Of Hope Hospital
Most people know that the U.S. Food and Drug Administration must approve a drug before it reaches the market or a hospital room. But very few people outside of the medical field know what happens before the drug gets to the FDA.
Clinical trials (or clinical studies) are medical studies that focus on establishing the safety and effectiveness of new, potentially beneficial drugs by testing them on increasingly large groups of human subjects. These trials can involve new medications, treatments, devices and even psychological therapies. It’s a years-long process that involves constant study and research before the new drug is ever presented to the FDA.
At City of Hope, at least 400 clinical trials are going on at any given time. To help illuminate the clinical trial process for patients, here are five facts you may not have known.
1. Clinical trials require approval before commencing.
Clinical trials are only approved once an institutional review board, or independent ethics committee, has weighed the potential benefits against the possible risks and determined the testing worthy. An institutional review board or independent ethics committee pores over all materials related to the study before and during the trials.
Often years of preclinical work has gone into researching and experimenting on isolated living cells, while zeroing in on potential benefits, appropriate dosage ranges, problems and side effects, and the most effective ways of applying the drug, before human subjects can become involved.
2. There are different types of trial subjects.
Often, existing patients with a specific condition or particular set of characteristics, such as age, gender, or stage of disease, will volunteer to participate in a clinical trial. In other instances, trials will make use of volunteers who are otherwise healthy, depending on the type of drug or device being tested.
Patients already under medical care may choose to participate because the trial gives them access to new treatments not available to others. Many volunteers sign on because they want to help advance science and medicine toward new cures. All participants must go through a process of informed consent with the trial’s principal investigators before it starts.
3. Clinical trials are run by special investigators.
Medical professionals who are trained in the “Good Clinical Practice” guidelines designed to safeguard subjects and assure proper collection of data can become investigators on clinical trials. By participating, investigators can position themselves to discover new developments and stay on the front edge of emerging medical discoveries, especially in their fields of expertise.
They can have an impact on advancements, while authoring papers and articles that push their field forward. Investigators are often paid for their work, unlike the subjects of clinical trials.
4. There are four distinct phases of a clinical trial.
Phase 1 focuses on safety by keeping the sample group small, from 20 to 80 people. It’s an initial evaluation of how the human body handles a new drug, while investigators take note of safe dosage ranges and any side effects.
In phase 2, the experiment group grows to 100 to 300 subjects, with more of a focus on effectiveness, though safety is always being evaluated.
During phase 3, investigators increase the sample size to 300 to 3,000 subjects and lengthen the amount of time the drug is taken, while the same safety and efficacy parameters are studied.
Phase 4 requires an even larger sampling group, with a focus on long-term effectiveness and safety, as well as how the new drug compares with other similar drugs and their costs, before approval is sought from the FDA.
5. Only a small percentage of new drugs ever hit the market.
The review process takes anywhere from six to 12 months, depending on whether the new drug is only a minor improvement on an existing therapy, a major advancement or a completely new treatment. Right now, the National Institutes of Health service ClinicalTrials.gov lists more than 215,000 clinical studies currently in progress around the world.
Ultimately, the FDA only approves about 10 percent of the drugs that went through clinical trials in the U.S.
Courtesy of ABC News
Cancer is never an easy diagnosis but it is an especially hard blow when given to a child. Cancer is the leading cause of childhood death by disease in the United States, with 13,500 new diagnoses each year according to the American Cancer Institute. One out of every 300 boys and one out of every 333 girls will develop cancer before their 20th birthday, according to the American Society of Clinical Oncology.
Experts say there is cause for optimism in the fight against childhood cancer. National Cancer Institute statistics show that in the U.S. the combined, an overall five-year cancer survival rate for children under 19 with cancer has increased from 62 percent in the mid-1970s to 84 percent today. For the most common type of childhood cancer, acute lymphoblastic leukemia, the cure rate is now over 90 percent.
"We've made amazing progress on pediatric cancers in just one generation. It's the biggest success story in cancer right now," said James Downing, M.D., co-chair of the American Association for Cancer Research's special conference on pediatric cancer; and scientific director at St. Jude Children's Research Hospital in Memphis.
Downing said one of the reasons such great strides have been made in pediatric cancer is that treatment protocols for younger patients tend to be vastly different from adult protocols. With adult cancer, there is a tendency to take breaks in treatment and back off if it gets too intense, he said. But many pediatric oncologists are aggressive about having their patients push through treatment until there is a cure -- sometimes for up to three years without a break.
Another reason for the high success rate is that up to 60 percent of pediatric cancer patients are treated as part of a research trial, compared to just 5 percent of adults, Downing said. Such trials offer access to cutting-edge treatments and a chance for oncologist to confer with a team of oncologists, other specialists and researchers.
But Downing said he thinks the biggest advances in treatment have come from the sequencing of the human genome.
"DNA sequencing of tumors helps us define the mutations that underlie pediatric cancer to help us attack cancers where we are not yet winning and will be a major catalyst to make progress in the next several years in how to treat those cancers," he said.
Downing admitted there are some unique challenges when treating young ones stricken with cancer.
Most cancer drugs were developed for use with older, more mature bodies so proper dosages and side effects can be tricky, he said. The cancers seen in children are vastly different from the ones seen in adults, so they aren't always as completely studied or well understood. And it's also difficult to know whether the chemotherapy, radiation and surgery children receive might lead to health problems later in life.
"Our challenge is to cure them so they can reduce long-term complications and live a normal life," he said.
As part of ABC Health's focus on children this month, we're holding a tweet chat today at 1 p.m., ET, on conquering childhood cancer. The chat is moderated by Dr. Richard Besser, our chief health and medical correspondent and we'll have researchers, clinicians and loved ones of cancer patients from all over the country tweeting their thoughts about this topic.
Courtesy of US News
This year alone, the American Cancer Society projects that 14,660 new cases of cancer will be diagnosed in children and adolescents. About 1,850 children and adolescents die from cancer annually – making it one of the leading causes of death in this age group. However, an increasing proportion of kids now survive cancer.
“If you look at all diagnoses, all ages, all stages, the cure rate is over 80 percent,” says Dr. Jean M. Tersak, an oncologist and director of the Survivorship Program at Children's Hospital of Pittsburgh of UPMC. For many kids with leukemia, survival rates exceed 90 percent. But childhood cancer survivors routinely face substantial health challenges from treatment as they grow older, which profoundly affect their quality of life and can hasten death. Oncologists say these so-called late effects range from chemo leading to heart problems and infertility to radiation affecting mental development and causing secondary cancers. So top hospitals around the country that treat cancer in kids have increasingly been focusing on not only curing with care, but reducing so-called late effects from treatment.
As part of that effort, some hospitals are participating in large-scale research like the National Institutes of Health-funded Childhood Cancer Survivor Study, which uses data from more than 14,000 survivors of childhood cancer who were diagnosed between 1970 and 1986, plus more than 10,000 survivors diagnosed between 1987 and 1999.
“It’s the only way we’ll ever understand the cost of the cure,” says Tersak of Children’s Hospital of Pittsburgh participation in research including the Childhood Cancer Survivor Study. The study has found that survivorship outlooks have improved for some who’ve beaten childhood cancer: “Looking at patients that were diagnosed between 1970 and 1986 versus patients diagnosed between 1987 and 1999 – that latter group has a lower incidence of second cancers and heart failures and other late effects – based on things we learned with the first group, and things that we changed in therapy,” Tersak notes.
She sees reduction of the use of radiation therapy to the brain as the biggest advancement in reducing late effects, including cognitive deficits that can hold kids back in school and reduce their professional options when they enter the workforce as adults. In addition, clinicians are looking at using more targeted radiation therapies – as in adults – that zap cancer cells, while damaging less of the normal healthy cells. Oncologists are also experimenting with using newer combinations of chemo drugs that reduce side effects, including heart problems, later. Moreover, doctors are seeking to anticipate late effects before administering treatment like chemo. At Children’s Hospital of Pittsburgh, some patients are being placed on a beta blocker medication commonly used to treat high blood pressure to see if that might reduce cancer treatment-related problems with heart function. That’s being done as part of another national study through the Children’s Oncology Group, which conducts research for children, adolescents and young adults with cancer. “We’ll follow those patients over time to see if that medication has made a difference,” Tersak says.
Though initial evidence shows advancements are making a difference in reducing late effects, experts say much more still remains to be done. According to the Childhood Cancer Survivor Study, by age 50, more than half of survivors have experienced a severe or life-threatening health condition, compared to only 19 percent of siblings. These can range from heart failure to a secondary cancer diagnosis. And the study finds the “health gap” continues to widen with age.
Given those challenges, cancer survivors and pediatric oncology experts say that for someone facing a new cancer diagnosis, its critical not to put off talking about life after cancer – even if it may seem counterintuitive to do so. “We … start talking about life after cancer from the very beginning,” says Dr. Karen Burns, clinical director of the Cancer Survivorship Center at Cincinnati Children’s Hospital Medical Center and an assistant professor of pediatrics at the University of Cincinnati. This has two benefits, according to Burns: “First, it shows the family that we intend to get them through this.” Though high survival rates still afford no guarantee, clinicians say it’s critical for a child’s mind and body to be future-oriented in treatment. Secondly, the concept of survivorship and late effects is introduced in the beginning. “We cannot control everything and late effects do happen, but knowledge is power and often your greatest ally,” she says.
In clinical practice and research, Burns is exploring ways to improve treatment of long-term cancer survivors with fertility issues and prevent the same issues caused by chemo and radiation for future childhood cancer patients. “Our goal is to address future fertility with every patient and take whatever steps we can to ensure it is preserved, so the child can have their own family one day,” she says. Burns adds that she works closely with the hospital’s adolescent gynecology and urology divisions to deliver the most comprehensive and cutting-edge care in fertility preservation, ranging from sperm banking or testicular tissue cryopreservation for male patients to ovarian tissue cryopreservation for female patients. Still being evaluated as an option, the hope is that these preserved tissues could someday be used by survivors to start a family, where that’s not otherwise possible.
Katie Wilson is now nurse at Cincinnati Children’s Hospital Medical Center, where she underwent treatment for cancer as a teenager.
Along with understanding treatment options, experts say it’s important patients and doctors talk about limitations and the potential for late effects in order to appropriately set expectations. Diagnosed with a rare type of leukemia as a teenager, Katie Wilson now takes medication to maintain her blood pressure and heart rate because of a severe heart condition called cardiomyopathy she developed as a result of chemo. “Because of the heart issue, I’m not able to actually go through pregnancy, because it’s too much of a risk on my heart,” says the 28-year-old of Fort Thomas, Kentucky. As a patient at Cincinnati Children’s – where she’s now a nurse treating oncology patients herself – she says her medical team did a great job early in the process of alerting her to potential late effects of treatment. She and her husband got married four years ago and have been thinking lately about having a family. “We’ve being talking a lot about that with my cardiologist,” she says. However, she says they’ve put things on hold for now, adding that they’re open to adopting.
At the University of Texas MD Anderson Cancer Center in Houston, clinicians use proton beam radiation, a highly targeted therapy, to focus on brain tumors – including in pediatric patients. “If you can focus it in a small area where the tumor is, you may not have the cognitive effects,” says Dr. Cindy Schwartz, division head and chair ad interim of pediatrics at MD Anderson. Experts say cognitive late effects are particularly pronounced in kids since young brains are still developing, and these deficits can affect everything from a child’s ability to learn to occupational prospects later on.
No matter what the long-term cost of the cure is – or the means by which late effects may be reduced – clinicians say it’s critical that patients are monitored long after their cancer treatment is completed to improve the best possible long-term outlook. Approaches differ on how to do that, however, with some research being done on using telemedicine, such that adult survivors don’t have to travel long distances to where they were originally treated as child – and to ease the hand-off to adult doctors. But experts agree patients and their loved ones are best served by seeing clinicians who have full knowledge of their cancer treatment and its potential effects, and that a childhood cancer survivor’s health provider should, at the very least, be in contact with the doctors or institution where that patient was treated.
In the meantime, experts say much more study is still needed to best determine how to care for childhood cancer survivors in the years after they’ve been deemed cancer-free. “We need to continue to try to learn how to support these patients,” Schwartz says – to try to find “better ways … of curing them without harming them in the long run."
When Treatment Makes Kids Feel Sicker Than The Illness Itself, This Program Offers Healing, Nutritious Bites
Courtesy of the Huffington Post
Danielle Cook’s oldest son was only 11 years old when he was diagnosed with stage three Hodgkin’s lymphoma, a type of cancer that affects the immune system. “There was a huge sense of powerlessness and great sadness,” remembers Cook, a mother of three who worked for years as a cooking demonstration instructor in the Washington, D.C., area.
Cook, who now also works as a holistic nutritionist, relentlessly looked for answers in food. After months of making special recipes, she saw her son go from a “worn, depressed, tired kid to a healthy adolescent,” she says.
Drawing from her experience, she founded Happily Hungry, a program that consists of cooking workshops geared towards hospitalized kids battling cancer and other illnesses.
Courtesy of The Children's Hospital of Philadelphia
Oncology researchers have discovered that an abnormal fused gene that drives pediatric brain tumors poses a triple threat, operating simultaneously through three distinct biological mechanisms — the first such example in cancer biology.
This finding potentially offers triple benefits as well — more accurate diagnoses, clues for more effective treatments, and new insights into molecular processes underlying other types of cancer.
Gene rearrangement offers a precision medicine approach
“The gene rearrangement we investigated offers a great candidate for a precision medicine approach in improving treatment for children with this type of brain tumor,” said study co-leader Adam C. Resnick, PhD, a neuro-oncology researcher in the division of Neurosurgery at The Children’s Hospital of Philadelphia (CHOP). “Our research exemplifies the transformative power of large multi-institutional research collaborations in sharing and empowering data from new diagnostic technologies.”
Resnick’s co-study leaders were Keith L. Ligon, MD, PhD, and Rameen Beroukhim, MD, PhD, both of Dana-Farber Cancer Institute, with co-authors from nearly 20 centers in five countries. Co-first authorship was shared between the CHOP and Dana-Farber laboratories, with Payal Jain, a University of Pennsylvania graduate student, leading the efforts at CHOP.
The study appeared online in Nature Genetics.
The scientists investigated pediatric low-grade gliomas (PLGGs), a varied group collectively representing the most common pediatric brain tumor. Drawing on samples gathered by the Children’s Brain Tumor Tissue Consortium and a consortium at Dana-Farber, as well as additional, previously uncurated datasets, they analyzed the genomes of 249 such tumors — the largest amount of data available for PLGGs.
Being classified as “low-grade” does not mean the tumor’s effects on children are mild, said CHOP neurosurgeon Phillip Storm, MD, a study co-author: “Survival rates are better than in higher-grade tumors, but there may be serious consequences for a patient’s long-term quality of life, including hormone disruptions, blindness, or coma, depending on the tumor’s location. Often the tumor is in a location where it can’t be surgically removed.”
Among the 249 samples were 19 tumors classified as angiocentric gliomas, which occur in the brain’s temporal lobe. In virtually all the angiocentric gliomas, the researchers found that two genes, MYB and QKI, had fused together to express an abnormal, cancer-driving fusion protein.
The MYB-QKI fusion gene promotes tumor formation through three simultaneous mechanisms. Unlike normal proteins expressed by the MYB genes, the rearranged gene expresses truncated, constitutively active proteins that give rise to cancer. Secondly, enhancer regions in or near QKI’s DNA move in proximity to MYB during the fusion event. This causes abnormal expression of the fusion protein in brain tissues, leading to a feedback loop that drives cell proliferation. Finally, the fusion gene disrupts QKI’s protective role as a tumor suppressor.
These disease-causing events provide new examples of how genomic dysregulation interacts and synergizes with epigenomic dysregulation. Epigenomic dysregulation comprises complex changes in gene expression not caused by changes in the DNA sequence of protein-coding elements.
Implications for clinicians
The current research, added Resnick, has important implications for clinicians. Identifying the MYB-QKI fusion gene as a defining event in angiocentric glioma may allow oncologists to better diagnose this subtype of tumor, guiding them toward directed therapies less likely to overtreat or undertreat children. Furthermore, although there do not appear to be existing drugs to target the abnormal MYB protein, there are potential drugs that may be effective against the type of epigenomic dysregulation seen in these tumors.
In addition, he said, “Now that we better understand the three mechanisms involved, we may be better able to craft our treatment strategies against any of those mechanisms.”
Finally, he concluded, “the study expands our current understanding of cancer, by focusing attention on the multiple mechanisms occurring simultaneously, and bringing into relief how gene fusions may give rise to epigenomic dysregulation. Gene fusions occur in many other cancers in both children and adults, so our findings may apply more broadly to other cancers.”
Courtesy of The Children's Hospital of Philadelphia
Pediatric oncologists from The Children’s Hospital of Philadelphia (CHOP) have investigated techniques to improve and broaden a novel personalized cell therapy to treat children with cancer. The researchers say that a patient’s outcome may be improved if clinicians select specific subtypes of T cells to attack diseases like acute lymphoblastic leukemia (ALL) and lymphoma.
“Our main finding is that younger T cells are critically important in T cell immunotherapy,” said pediatric oncologist David M. Barrett, MD, PhD, at The Children’s Hospital of Philadelphia. “Collecting and expanding these cells could increase the number of children with cancer who could benefit from this innovative treatment.”
The study appeared Jan. 6 in Science Translational Medicine.
Barrett collaborated with Stephan A. Grupp, MD, PhD, and Jessica Perazzelli, both of CHOP; and first author Nathan Singh, MD, a resident in the Perelman School of Medicine at the University of Pennsylvania. Grupp has received national attention for his work in using reprogrammed T cells to treat children with relapsed or refractory ALL. The current research reports on insights and laboratory techniques that may advance this treatment.
The T cell therapy, developed with Barrett’s and Grupp’s collaborators at the Perelman School of Medicine at the University of Pennsylvania, is a form of immunotherapy — manipulating the body’s own immune system. Specifically, the scientists modify T cells, the workhorses of the body’s immune system, to attack B cells, other immune cells that become cancerous in specific cancers such as ALL. The researchers first extract a patient’s own T cells and reprogram them to hunt down and eliminate B cells after those modified T cells are returned to the patient.
Barrett and colleagues followed 50 child and adolescent patients in a clinical trial of B-cell cancers at CHOP, of whom 38 had ALL and 12 had non-Hodgkin’s lymphoma (NHL). The study team measured immune system markers and fully characterized their T cell populations once a month for six months following a patient’s initial diagnosis.
Scientists already knew that T cells evolve after they become active, but the current study fully characterized the T cell subtypes in the context of cell therapy. The study team found that early-lineage T cells, classified as either naïve T cells (newly minted cells) or stem central memory T cells (self-renewing, highly proliferative cells) were the most effective in immunotherapy. Those early-lineage T cells also expanded best in the laboratory, before they were returned to each patient for T cell therapy.
Significantly, early-lineage T cells were also more vulnerable to chemotherapy than older cells.
These findings, said Barrett, have the potential to change clinical management in T cell immunotherapy. “In newly diagnosed patients, it may be preferable to collect their T cells much earlier than currently done, before chemotherapy, or between chemotherapy cycles, instead of after a patient relapses,” he said. “We could keep the patient’s early-lineage T cells in reserve, in case the patient needs them later.”
In addition, the study team showed that adding the signaling proteins interleukin-7 and interleukin-15 to T cell cultures expanded stem central memory cells in samples from both ALL and NHL patients.
Finally, the study team found that ALL patients had a different mixture of T cell subtypes than did NHL patients. ALL patients had higher levels of early lineage T cells, with greater expansion potential, than did NHL patients. Barrett added that further research needs to focus on improving outcomes for children with NHL.
The study’s funders were the Leukemia and Lymphoma Society, Weinberg Funds, Cookies for Kids’ Cancer, and a Stand Up to Cancer-St. Baldrick’s Pediatric Dream Team Translational Research Grant.
Hunting Rare Cancers to the Ground
Courtesy of Boston Children's Hospital
As we’ve seen this week on Vector, some rare childhood cancers such as medulloblastoma and neuroblastoma are starting to give up their molecular secrets, raising the possibility (and in medulloblastoma’s case, the reality) of precision treatments. Many cancers, though, are so rare that there aren’t even cell lines in which to study them. Yet they could hold important insights. The first tumor suppressor gene, Rb, was discovered in retinoblastoma, a cancer affecting a mere 500 U.S. children each year.
Doctors often have no clear consensus for diagnosing and treating rare cancers, and outcomes tend to be poor in both children and adults. Andrew Hong, MD, a postdoctoral fellow in the Broad Institute’s Cancer Program and a pediatric oncologist at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, is part of a research team that wants to fix that.
Armed with recent advances in culture technology, the scientists aim to engineer cell lines for as many rare cancers as they can get samples for — and then interrogate them for therapeutic targets. A proof-of-concept published in Nature Communications last month finds a lot of potential in their approach. Read more on Broad Minded, the Broad Institute’s science blog.
Beyond Appearances: Molecular Genetics Revises Brain Tumor Classification and Care
Courtesy of Boston Children's Hospital
For almost a century, brain tumors have been diagnosed based on their appearance under a microscope and classified by their resemblance to the brain cells from which they are derived. For example, astrocytoma ends with “-oma” to designate that it is a tumor derived from astrocytes. In some cases, especially in children, brain tumors resemble cells in the developing brain and are named for the cells from which they are presumed to arise, such as pineoblastoma for developing cells within the pineal gland or medulloblastoma for developing cells within the cerebellum or brainstem.
In June, the World Health Organization (WHO), which sets the worldwide standard, released an updated brain tumor classification scheme that, for the first time, includes molecular and genetic features.
Going forward, pathologists in hospitals all over the world will diagnose brain tumors not only by their appearance under a microscope, but based on their molecular and genetic signatures. These signatures in some cases establish the underlying mechanisms driving the tumor, and more accurately predict whether the tumor will respond to therapy.
Brain Tumor Classification
A “heat map” showing the four molecular subtypes of medulloblastoma. Now we know what gene mutations underlie these differences in gene expression. (Tenley Archer, PhD, Pomeroy Laboratory) This historic change in brain tumor classification is the result of years of research in labs around the world, including our own.
Not all brain tumors are made the same...
When I began taking care of children with brain tumors at Boston Children’s Hospital in 1991, I quickly realized that some tumors, especially medulloblastomas, had quite varied behavior despite looking very similar under the microscope. About half of the children responded to treatment and are still alive today. But the others progressed and succumbed after variable periods of time, despite the fact that they received identical treatment.
The first clue to this mystery came when my lab discovered in the 1990s that the presence of certain molecules that normally appear during development predict response to medulloblastoma therapy better than the tumor’s appearance under the microscope. In 2002, my lab and colleagues at MIT applied genomics to show that medulloblastomas are actually comprised of subgroups with different molecular profiles and clinical outcomes. The molecular signatures we had found earlier were a manifestation of these subgroups.
Further work by my lab and by colleagues in Toronto, Heidelberg and Memphis further delineated the subgroups. In 2010, we met in Boston, and our consensus — that there are four primary subgroups of medulloblastoma — provided the scientific evidence for the new WHO classification.
Similar schemes are emerging for other types of brain tumors. For example, work that my laboratory did in collaboration with Charles Roberts, MD, PhD, then at Dana Farber Cancer Institute, established that atypical teratoid/rhabdoid tumors (arising in the brain and spinal cord) and other rhabdoid tumors (arising in the kidneys and other sites) all have the same genetic defect.
Collectively, these findings reveal that the molecular features of tumors drive their biological behavior, establishing a basis for precision cancer medicine (see infographic). Using the new WHO diagnostic classification, children with brain tumors all over the world can now receive less radiation and chemotherapy if they have a good molecular prognosis, reducing long-term complications, and intensified treatments if they have a molecular subtype known to not respond to conventional therapy.
The genomic approach is also uncovering molecules that drive tumor growth. These can now be specifically targeted with the goal of further reducing radiation and chemotherapy. In the future, we hope that children will not only survive their cancer, but also have the highest quality of life and grow into successful adults.
MD Anderson, NASA and ILC Dover Partner on Space Suit Art Project to Increase Childhood Cancer Awareness
Courtesy of Yahoo News
HOUSTON, July 8, 2016 /PRNewswire-USNewswire/ -- Today, several of Space City's best-known institutions — The University of Texas MD Anderson Cancer Center, the National Aeronautics and Space Administration (NASA) and ILC Dover — announced a partnership that brings the benefits of arts and science to pediatric cancer patients while increasing awareness of childhood cancer. More than 530 patients, families, and staff members painted original artwork used to create full-sized suits for the Space Suit Art Project.
Born out of an idea from MD Anderson's Arts in Medicine Program, which helps pediatric patients cope with cancer treatment through art, this project inspired leaders at NASA's International Space Station (ISS) to support the effort with help from astronauts, scientists and engineers. NASA provided patterns for the suits and worked with ILC Dover, a manufacturing and engineering company that develops NASA space suits, to assemble the suits by stitching the hand-painted art pieces together into a wearable replica space suit.
"This project has inspired hope for kids fighting cancer, instilled them with courage and created unity, all while increasing awareness of childhood cancer and the importance of pediatric cancer research," said Ronald A. DePinho, M.D., president of MD Anderson. "We are so proud of this project and grateful for the passion and support we've received from NASA, ISS and ILC Dover. This is a wonderful example of the power of collaboration."
On average, one in 285 children in the US will be diagnosed with cancer before the age of 20. Similar to adults, children going through cancer treatment can experience anxiety and depression. Research shows creative arts therapy benefits cancer patients as mental health and behavioral health are positively impacted. At MD Anderson, art gives patients a sense of control and purpose, makes them more comfortable in the hospital environment, and helps build community among patients and families.
"This collaboration highlights both the knowledge and inspiration that flow from the International Space Station," said Ellen Ochoa, Ph.D., veteran astronaut and director of NASA's Johnson Space Center. "Our astronauts conduct research on board the orbiting National Laboratory that benefit people around the world, including experiments that may inform future cancer research."
Leading the effort to connect science, technology, arts and the human spirit are Ian Cion, director of the Arts in Medicine Program; Nicole Stott, retired NASA astronaut and the first person to paint in space; and David Graziosi of ILC Dover. Their collaboration created the Space Suit Art Project, which demonstrates the transformative power of arts in the healing process through three space suits designed to convey different meanings: Hope, Courage and Unity.
The first suit, HOPE was stitched together from more than 600 hand-painted art pieces created by patients, families and staff at MD Anderson. It represents the hope patients and families have as they go through treatment. Their primary hope is to survive cancer, but it's deeper than survival. The project inspires hope for progress in childhood cancer research, which is consistently underfunded, and hope that childhood diseases like cancer can one day be eliminated. One patient inspired by the Space Suit Art Project and who provided artwork for the first two suits shared his hope.
"Even though my cancer is back after I already survived it twice, working on this project makes the days go faster and reminds me about the importance of hope,' said Jacob, a 17-year-old Ewing's sarcoma survivor. "I'm excited to tell people that my art may go to space, and, one day, I hope to work with the space exploration vehicles at NASA."
COURAGE, the second space suit created with patients at MD Anderson, many of whom were on isolation during their treatment, is meant to demonstrate the courage it takes to be isolated from family and friends during long periods of time. Astronauts face similar isolation during space exploration missions. Stott took a watercolor paint kit on her space missions to remain connected to the world left behind and to help document her experiences.
Creation of the third space suit, UNITY, will be an international collaboration with children's hospitals around the world. The UNITY space suit will represent the global issues surrounding childhood cancers, with a goal to unite others, help spread awareness about childhood cancers and offer hope and courage to cancer patients around the globe.
"The kids and families we've met during this creative journey have shown us all the importance of hope, the power of courage and the strength of unity," said Stott. "All of the partners involved with this project and I hope people will see these works of art and they will be inspired to learn more about the story behind it, which is the need for increased awareness of childhood cancer. There's so much work to be done for pediatric cancer research — we're just trying to do our little part."
The Space Suit Art Project officially launched today at MD Anderson. Ochoa, Stott, patients and families, astronauts and other collaborative leaders of the project joined MD Anderson President Dr. DePinho on stage for the special unveiling of the first space suit, HOPE, and a special announcement that COURAGE will be going into space later this month.
Berni & Murcer, Friends for LifeTM