DIPG News

CONGRESSIONAL TESTIMONY

This Testimony is Embargoed Until Thursday, February 2nd at 9:00 AM

Please visit: http://waysandmeans.house.gov/committeesubmissions/ and submit your comment for the record. There are many of us out there who have suffered as the result of losing our child and then having a thief take our kids’ social security numbers. Please consider submitting a comment.

FROM: Jonathan Eric Agin, Esq.
TO: House Ways and Means Committee, Subcommittee on Social Security
DATE: February 2, 2012
RE: H.R. 3475

Good morning Mr. Chairman and members of the Committee. Thank you for allowing me to be here today to testify on this issue of vital importance.

My family’s story begins with the diagnosis of our amazing daughter, Alexis Gina Agin, with a terminal brain tumor at the age of two on April 10, 2008. Ultimately, this terrible disease took her life on January 14, 2011, just two weeks shy of her fifth birthday. Alexis was and is my hero. Fighting valiantly until the end, she has inspired thousands around the world with her journey.

In 2010, my wife and I travelled with Alexis up and down the East Coast trying several experimental treatments, in a desperate attempt to save her. With each trip, Alexis’ medical bills mounted. When it came time to file our 2010 taxes, compiling all of our receipts for the medical expenses was time-consuming and emotionally draining. Accordingly, my wife and I, through our accountant, filed with the IRS for an extension. In October 2011, after completing the difficult and grueling task of finalizing our 2010 taxes, I received a telephone call from our accountant advising us that someone had already filed a tax return for 2010 using Alexis’ social security number. Beyond being completely stunned at that very moment, we were advised that we would not be able to file an electronic return. Instead, our accountant would have to complete the paper forms and file them in the traditional manner. More importantly for purposes of HR 3475, he told us that we ultimately would have to prove that our deceased daughter was, in fact, our daughter. In situations involving this type of criminal fraud, the IRS credits the first filer and presumes that the initial filing is accurate.

That same day, we reached out to the community of grieving cancer parents that we have come to know since April 2008 and told them what had happened. With incredulous amazement, we learned within a single hour of no fewer than fourteen other families whose children had died and also had experienced the additional travesty of their child’s social security number being stolen. Clearly we were not alone. We then learned through our own research and from other parents that this is, in fact, a very widespread issue impacting parents who lose a child due to any and all reasons imaginable.

Not surprisingly, when I first learned that Alexis’ social security number had been fraudulently used, I wanted to know how someone could have found it. Within a matter of seconds on the internet, I was able to locate her complete social security number and other personal identifying information, including her birth and death dates, on several websites intended for genealogical research. I immediately contacted one of the services, who directed This Testimony is Embargoed Until Thursday, February 2nd at 9:00 AMme to the service’s outside counsel. When I asked the attorney to remove my daughter’s personal information from the website, he advised me that the service was within its legal rights to display the information and that it refused to remove her social security number. The attorney cited as support for their position a 1980 consent judgment between the United States Government and a private citizen, Ronald Perholtz. It was at that point that we truly realized how significant this problem is, and more importantly, how the federal government is partly to blame. It is my belief that the federal government is responsible for providing identity thieves the information required to commit this costly crime. By affording widespread access to this type of information, the federal government provides the perfect platform for the commission of this crime.

The common denominator in this story is the Death Master File (hereinafter “DMF”). The Social Security Administration makes the DMF available to the National Technical Information Service (NTIS) of the Department of Commerce, who then sells the DMF to private and public sector customers, including government agencies, financial institutions, investigative entities, credit reporting organizations, genealogical researchers and other industries. Some of these purchasers, namely organizations hosting websites aimed at facilitating genealogical research, then make available the DMF for free to the public at large. It therefore is available to nearly anyone and perpetuates identity theft and fraud against the federal and state governments at astronomical levels. As a taxpayer and parent of a child who passed away from cancer, I am outraged at the most private information of our children being made commercially available. Not only is this a significant invasion of my child’s privacy, but it adds to the tremendous grief that my wife and I live with on a daily basis and will continue to live with for the rest of our lives. While it may seem trivial to some, Alexis’ social security number is one of the only things that we have left of her identity. Thus, the theft of it robbed us of something truly priceless.

Due to an ongoing media probe and public pressure, the IRS for the first time recently responded to inquiries on this issue, and estimated that there were approximately 350,000 fraudulent tax filings in 2010. According to IRS officials, these fraudulent filings claimed $1.25 billion in refunds.1 The cost to the federal government to investigate and prosecute that magnitude of fraud could be spent in much better ways, including research to fund cures for our children.

In addition, it is worth noting that the federal government discloses far more information than is required under the 1980 settlement. In June 2008, the Inspector General of the Social Security Administration issued a critical report detailing how publication of the DMF has resulted in the breach of citizens’ personally identifiable information.2 The report concludes that the Social Security Administration “discloses far more detailed personal information in the DMF than required under the original consent judgment that resulted in the creation of the DMF. Under the terms of the agreement, SSA was to compile a list that identified deceased numberholders’ SSNs, surnames and dates of death. However, SSA expanded the information published in the DMF to include the decedent’s date of birth, first and middle name, and last known residential state/zip code.”3 The report’s conclusion is simple: less information should be released, and greater efforts at accuracy and protection must be taken.

Significantly, Ronald Perholtz, the man who filed the lawsuit that led to the 1980 consent judgment resulting in creation of the DMF, sought release of the information to help reduce fraud. Specifically, he wanted the information as a tool for pension companies to identify theft of pension benefits. Soon after learning the DMF was created, Mr. Perholtz learned that the DMF frequently listed the social security numbers of people who were not, in fact, dead.4 Now, he believes that changes need to be made in order to stop this type of fraud. Indeed, Mr. Perholtz stated that he is willing to renegotiate the original settlement as he feels so strongly that the DMF is being abused.5

H.R. 3475 is a solution to a significant problem that affects not only grieving parents, but also every family who loses a loved one. It also is a solution to a problem that was never anticipated, and would eliminate dissemination by the federal government of extraneous information that it is not required to release. This additional information, along with the readily accessible nature of individuals’ social security numbers, has provided identity thieves an avenue to commit this crime and defraud the taxpayers and government.

Those who argue that the release of this information is critical to combat fraud and conduct genealogical research fail to understand that this Bill is not intended to prevent or limit the lawful use of Social Security Numbers or genealogical research. First, I would say to any individual conducting genealogical research, why do you need to know my daughter’s social security number? Why should it be publicly available to anyone with a computer? What purpose does her full social security number, along with her birth and death dates, address, and other personal identifying information have for your familial research? The clear answer is that it has absolutely no purpose. Alexis didn’t die a long time ago—she died last year. While it may be difficult to find information about your ancestors from generations ago, it should not be hard to confirm your familial connection (or lack thereof) to someone born just six years ago.

More importantly, this Bill will not prevent credit bureaus and financial institutions from fulfilling their charge of protecting us from fraud. To the contrary, because access to this information will be more restricted, these institutions will be more empowered knowing that the potential incidence rates of identity theft and fraud will be curtailed. Potentially far fewer instances of fraud against lawful citizens will be committed, thus reducing the amount of investigation necessary. The intent of this legislation is not to limit or prohibit financial institutions from investigating fraud; rather it is to prohibit the widespread publication and easy access of personal information that is utilized by criminals to defraud the government. As for hospitals and other institutions who claim to utilize the DMF to determine if their patients are deceased, I submit that there are other far less destructive methods to make such determinations and conduct your research. Again, this Bill is not aimed at those who have a legitimate need for access to individuals’ social security numbers and they will continue to have access to this information.

I have been told that in most cases, the government does not have the resources to prosecute this crime. It either is too costly, or the government simply does not have the ability to track and punish those who are stealing from it and taxpayers alike. If this crime was prevented, to the best extent possible upfront with this simple measure, there would be little concern regarding the cost to prosecute as more resources could be made available, stiffer penalties proscribed and additional deterrents fully understood.

In closing, this is not a victimless crime. My daughter is a victim. She was victimized twice. Once by the cancer that stole her from this earth, and then by a cold-hearted criminal who stalked her and utilized her death for profit. It disgusts me to no end to know that someone prayed upon my daughter’s death for his or her own gain. It is an added insult for a grieving parent. It is nothing short of a despicable crime and the release of Alexis’ complete social security number and other personal identifiers to the general public facilitated this crime. But this simply is not an emotionally charged issue, as some argue. Fraud is not something that we simply should accept as a necessary consequence of easy access to information. Yes, security breaches will always be possible regardless of the measures that we put into place. But when there is a simple fix to a significant problem that affects all taxpayers, the fix should be taken seriously and enacted with haste. It is time that this loophole is closed and this legislation is the manner in which to accomplish this aim. Nothing short of this will accomplish the task. It is simple and to the point, and in this era when our government is struggling to find ways to save money for the taxpayer, it is a very easy fix with little to no consequences or repercussions to citizens of this country.

Thank you Mr. Chairman and distinguished members of the Committee.
Jonathan Eric Agin

1 Dale McFeatters, Govt. shares blame as ID theft worsens, available athttp://bostonherald.com/news/opinion/op_ed/view/2011_1105govt_shares_blame_as_id_theft_worsens (November 5, 2011).

2 Office of the Inspector General, Social Security Administration, Personally Identifiable Information Made Available to the General Public Via the Death master File, Audit Report A-06-08-18042 (June 2008).

3 Id. at 6.

4 Thomas Hargrove, Social Security ‘Death File’ designed to fight fraud but now aids it, available at www.scrippsnews.com (November 14, 2011).

5 Id.

Cancer sequencing initiative discovers mutations tied to aggressive childhood brain tumors

St. Jude Children’s Research Hospital – Washington University Pediatric Cancer Genome Project provides first evidence linking cancer to mutations in genes involved in DNA organization

Researchers studying a rare, lethal childhood tumor of the brainstem discovered that nearly 80 percent of the tumors have mutations in genes not previously tied to cancer. Early evidence suggests the alterations play a unique role in other aggressive pediatric brain tumors as well.

The findings from the St. Jude Children’s Research Hospital – Washington University Pediatric Cancer Genome Project (PCGP) offer important insight into a poorly understood tumor that kills more than 90 percent of patients within two years. The tumor, diffuse intrinsic pontine glioma (DIPG), is found almost exclusively in children and accounts for 10 to 15 percent of pediatric tumors of the brain and central nervous system.

“We are hopeful that identifying these mutations will lead us to new selective therapeutic targets, which are particularly important since this tumor cannot be treated surgically and still lacks effective therapies,” said Suzanne Baker, Ph.D., co-leader of the St. Jude Neurobiology and Brain Tumor Program and a member of the St. Jude Department of Developmental Neurobiology. She is a corresponding author of the study published in the January 29 online edition of the scientific journalNature Genetics.

DIPG is an extremely invasive tumor that occurs in the brainstem, which is at the base of the skull and controls such vital functions as breathing and heart rate. DIPG cannot be cured by surgery and is accurately diagnosed by non-invasive imaging. As a result, DIPG is rarely biopsied in the U.S. and little is known about it.

Cancer occurs when normal gene activity is disrupted, allowing for the unchecked cell growth and spread that makes cancer so lethal. In this study, investigators found 78 percent of the DIPG tumors had alterations in one of two genes that carry instructions for making proteins that play similar roles in packaging DNA inside cells. Both belong to the histone H3 family of proteins. DNA must be wrapped around histones so that it is compact enough to fit into the nucleus. The packaging of DNA by histones influences which genes are switched on or off, as well as the repair of mutations in DNA and the stability of DNA. Disruption of any of these processes can contribute to cancer.

Researchers said that the mutations seem unique to aggressive childhood brain tumors.

“It is amazing to see that this particular tumor type appears to be characterized by a molecular ‘smoking gun’ and that these mutations are unique to fast-growing pediatric cancers in the brain,” said Richard K. Wilson, Ph.D., director of The Genome Institute at Washington University School of Medicine in St. Louis and one of the study’s corresponding authors. “This is exactly the type of result one hopes to find when studying the genomes of cancer patients.”

The results are the latest from the PCGP, an ambitious three-year effort to sequence the complete normal and cancer genomes of 600 children with some of the most poorly understood and aggressive pediatric cancers. The human genome includes the complete set of instructions needed to assemble and sustain human life. The goal is to identify differences that explain why cancer develops, spreads and kills. Researchers believe the findings will provide the foundation for new tools to diagnose, treat or prevent the disease.

For this study, researchers sequenced the complete normal and cancer genomes of seven patients with DIPG. “The mutations were found at such high frequency in the cancer genomes of those seven patients that we immediately checked for the same alterations in a larger group of DIPGs,” Baker said. When researchers sequenced all 16 of the related genes that make closely related variants of histone H3 proteins in an additional 43 DIPGs, they found many of the tumors contained the same mistakes in only two of these genes.

Of the 50 DIPG tumors included in this study, 60 percent had a single alteration in the makeup of theH3F3A gene. When the mutated gene was translated into a protein, the point mutation led to the substitution of methionine for lysine as the 27th amino acid in this variant of histone H3 protein. Another 18 percent of the DIPG patients carried the same mistake in a different gene, HIST1H3B.

Researchers are now working to understand how mutations in H3F3A and HIST1H3B impact cell function and contribute to cancer. Earlier research provides some clues. The lysine that is mutated is normally targeted by enzymes that attach other molecules to histone H3, influencing how it interacts with other proteins that regulate gene expression, Baker said. Mutations in the enzymes that target histone H3 have been identified in other cancers, but this is the first report showing a specific alteration of histones in cancer.

H3F3A and HIST1H3B were also mutated in other aggressive childhood brain tumors, glioblastoma, that develop outside the brain stem. Of 36 such tumors included in this study, 36 percent carried one of three distinct point mutations in the genes. The alterations included another single change in the makeup of H3F3A not found in DIPGs.

The histone H3 genes, however, were not mutated in any of the 252 other childhood tumors researchers checked for this study. The list included the brain tumors known as low-grade gliomas, medulloblastomas and ependymomas plus other cancers outside the brain and nervous system. The H3 changes have not been reported in any other cancers, including adult glioblastoma. “This suggests these particular mutations give a very important selective advantage, particularly in the developing brainstem and to a lesser degree in the developing brain, which leads to a terribly aggressive brain tumor in children, but not in adults,” Baker said.

“This discovery would not have been possible without the unbiased approach taken by the Pediatric Cancer Genome Project,” Baker said. “The mutations had not been reported in any other tumor, so we would not have searched for them in DIPGs. Yet the alterations clearly play an important role in generating this particular tumor.”

The study’s first authors are Gang Wu, Alberto Broniscer and Troy McEachron, all of St. Jude. The study’s other corresponding authors are Jinghui Zhang and James Downing, both of St. Jude. The other study authors are Charles Lu, Li Ding and Elaine Mardis, all of Washington University; and Barbara Paugh, Jared Becksfort, Chunxu Qu, Robert Huether, Matthew Parker, Junyuan Zhang, Amar Gajjar, Michael Dyer, Charles Mullighan, Richard Gilbertson and David Ellison, all of St. Jude.

The research was funded in part by the PCGP, including Kay Jewelers, a lead project sponsor; the National Institutes of Health, the Sydney Schlobohm Chair of Research from the National Brain Tumor Society; the Cure Starts Now Foundation, Smile for Sophie Forever Foundation, Tyler’s Treehouse Foundation, Musicians Against Childhood Cancer, the Noyes Brain Tumor Foundation and ALSAC.

St. Jude Children’s Research Hospital
Since opening 50 years ago, St. Jude Children’s Research Hospital has changed the way the world treats childhood cancer and other life-threatening diseases. No family ever pays St. Jude for the care their child receives and, for every child treated here, thousands more have been saved worldwide through St. Jude discoveries. The hospital has played a pivotal role in pushing U.S. pediatric cancer survival rates from 20 to 80 percent overall, and is the first and only National Cancer Institute-designated Comprehensive Cancer Center devoted to children. It is also a leader in the research and treatment of blood disorders and infectious diseases in children. St. Jude was founded by the late entertainer Danny Thomas, who believed that no child should die in the dawn of life. Join that mission by visiting www.stjude.org or following us on www.facebook.com/stjude and Twitter@StJudeResearch.

Washington University School of Medicine
Washington University School of Medicine’s 2,100 employed and volunteer faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Children’s hospitals. The School of Medicine is one of the leading medical research, teaching and patient care institutions in the nation, currently ranked fourth in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Children’s hospitals, the School of Medicine is linked to BJC HealthCare.
St. Jude Media Relations Contacts

Summer Freeman
(desk) (901) 595-3061
(cell) (901) 297-9861
summer.freeman@stjude.org
Carrie Strehlau
(desk) (901) 595-2295
(cell) (901) 297-9875
carrie.strehlau@stjude.org.

Washington University Media Relations Contact

Caroline Arbanas
(cell) (314) 445-4172
(desk) (314) 286-0109
arbanasc@wustl.edu

http://www.stjude.org/baker-DIPG

A soo-weet collaboration between
entrepreneurship, philanthropy, and BBQ

Hogs for the Cause founders Becker Hall and Rene Louapre
with their inspiration, Ben.

This post is dedicated to the memory of Ben Sarrat Jr. for the movement he inspired.

The formula for the first annual pig roast was simple: a few friends, a pig, and a keg. However, when Hogs for the Causefounders Becker Hall and Rene Louapre met Ben Sarrat, Jr., they were inspired to use their pig roast at the Fly to raise money for a family friend in need.

With that newfound motivation and drive, the duo has turned what was an idea for a small, pork infused gathering with friends into a highly anticipated festival that continues to triple in size each year.

In true New Orleans form, Hall and Louapre go back to childhood. Having grown up together, they always considered each other as brothers, and now play the roles of business partners with equal ease. Part of their success can be traced to the evident camaraderie and balance between the businessman and the lawyer, but their conviction and motivation has turned the young professionals into moonlighting entrepreneurs and philanthropists.

In 2009, the two friends and culinary aficionados came up with the idea of hosting a pig-roast, a tailgate tradition from Hall’s alma mater at the University of South Carolina, and a surprisingly new concept to New Orleans.

Shortly after they decided to add a philanthropic component to their roast, they met Ben. The young son of a family friend had been diagnosed with DIPG, an incurable brain tumor that cannot be removed because of its location on the brain stem, typically giving those who are diagnosed only one year of survival after detection. Hall and Louapre were taken by Ben’s enthusiasm and courage and decided to use their pig roast to raise money to help him and his family. Within a month, Hogs for the Cause was incorporated and registered as a 501c(3) non-profit organization.

“Meeting Ben was a really profound moment for us both, and immediately changed everything,” Hall says of this pivotal moment that inspired their endeavor. “Even with such terrible circumstances, he still continued to have a genuine enthusiasm for life. At that point, we decided to do whatever we could to help Ben have the happy childhood he deserved.”

In their first year, Hall and Louapre, along with 250 friends, raised $10,000 to help support Ben’s family with their expenses. However, they knew they could do more.

The week before their second Hogs for the Cause, Ben tragically lost his battle to DIPG. However, his story only reinforced Hall and Louapre’s mission and drive to continue their fundraising for other families. In its second year, Hogs for the Cause drew in 2,500 people and raised $30,000, with a large portion of it going to support families like the Sarrats.

Hogs for the Cause’s mission is to alleviate some of the financial burden families face when a child is being treated for pediatric brain cancer. The money raised goes toward grants that help cover the peripheral expenses that insurance does not otherwise pay for, such as travel and lodging expenses incurred during cancer treatment.

Hall and Louapre filled a void of funding resources for pediatric cancer outreach services, as well as another void that New Orleans was ironically missing: pork cook-offs. In 2011, Hogs for the Cause moved from the Fly to City “Pork,” where they raised $100,000 for their cause, along with the help of 45 teams battling it out with their pork creations and 7,000 attendees.

And that was only year three.

This year, within three weeks of registration opening, 60 spots were filled with teams of pork enthusiasts, amateur cooks, award-winning BBQ experts, and renowned chefs and restaurant owners. Besides their genuine enthusiasm to support the cause, the teams participate for the opportunity to prove their culinary skills and compete for the ultimate title of Grand Champion. To them, the pork cook-off is merely the grand finale of a year spent practicing recipes and practicing witty twitter banter and intimidation between the teams. (I train my stomach’s pork capacity by reading their twitter feeds during this time. I recommend you do the same.)

The participants all compete in several categories, including Ribs, Whole Hog, Butt, Porkpourri, Grand Champion (who must Compete in three of the four aforementioned categories), Best Booth and Presentation, Fundraising Champion, Best Sauce, and Fan Favorite, which gives the fans a voice and opportunity to vote through the Hogs for the Cause iPhone app.

While Hogs for the Cause founders are grateful for the loyalty and enthusiasm of all the competitors, the team members continue to gain recognition for their participation in the competition as well. After winning last year’s Porkpourri category, Chef Adam Biderman credited his participation in Hogs for the Cause for the inadvertent loyal following he built before opening The Company Burger last August.

This year, Biderman will be back to defend his title and continue his support to the cause, along with other local chefs, including Aaron Burgau of Patois, Drew Dzejak of The Windsor Court’s Grill Room, Chefs Allison and Slade Rushing from MiLa, and Le Petite Grocery’s Chef Justin Devillier, just to name a few.

Hogs for the Cause is March, 24th 2012

Hogs for the Cause is March, 24th 2012

Six bands have also been confirmed to perform on the NOLA Brewing stage this year, including Trombone Shorty & Orleans Avenue, Voice of the Wetlands All-Stars, 2012 Grammy nominated artist Marcia Ball, The Stooges Brass Band, Mississippi Rail Company, and Austin, Texas’ The Gourds.

While the event now launches the beginning of festival season in New Orleans and continues to grow significantly each year, Hall and Louapre still find that the greatest reward is being able to help their grant recipients.

“The greatest feeling in the world is walking into a room and delivering to a family a sign of hope,” said Louapre about his motivation to continue the growth of the annual cook-off. “These are families with odds stacked against them. Their child is fighting against a brutal killer, and to be able to deliver just a small dose of help — a reassurance that people care and a big hug of humanity — makes it all worth it.”

The entrepreneurial-minded team attributes the growth of their brand to their conviction and passion to help the families in need. And, when asked about where they see Hogs for the Cause in five years, they both agreed on being at the forefront of funding sources for pediatric brain cancer outreach services. Louapre added, “The cook-off will have 125 teams, there will be two stages of live music, and Becker and I (who have known each other our whole lives and are almost brothers) will still be fighting over minor details as we continue to make Hogs better.”

Hall’s vision includes taking the fundraising to a national level with cook-offs across the country.

While they might already be sparring about the exact coordinates of their five-year benchmark, it’s unmistakable that their brotherly dynamic has helped them surpass even their own expectations. So, who’s to say it won’t all be possible?

The country’s largest cook-off and nationally renowned charity based out of New Orleans is merely just a baby step toward what they can accomplish together. What’s in store for March 24, 2012? Meatier competition, a new music lineup, larger location in City “Pork,” an adult “porkade,” frolf (frisbee-golf) games, a new iPhone app, and bigger fundraising goals.

To learn more, purchase tickets, and make a donation, visit their website, and follow them on Twitter and Facebook for all the updates and witty pork puns. Download the iPhone app to learn more about Hogs for the Cause, stay updated with the competition, vote for fan favorite, and purchase tickets.

Pre-Sale General admission tickets for $10, as well as ticket packages that include unlimited food and beer, judging privileges, and access to the “Boss Hog” tent, are available now and can be purchased online or on the iPhone App.

http://www.nola.com/nolavie/index.ssf/2012/01/a_soo-weet_collaboration_betwe.html

 

By Adriana Lopez

PATIENT GROUPS AND RESEARCHERS JOIN FORCES TO SPEED TREATMENTS FOR RARE PEDIATRIC BRAIN TUMOR

Four Organizations Fund International Research Consortium to Improve Lives of Children Suffering From Diffuse Intrinsic Pontine Glioma (DIPG)

Accelerate Brain Cancer Cure (ABC2), CureSearch for Children’s Cancer, The Cure Starts Now Foundation and The Lyla Nsouli Foundation for Children’s Brain Cancer Research today announced their collaborative funding to support groundbreaking research aimed at dramatically improving the lives of children suffering from Diffuse Intrinsic Pontine Glioma (DIPG) – one of the most devastating pediatric cancers.

Together, the four organizations have committed $229,000 to support the work of the DIPG Preclinical Consortium, the only international scientific group focused on preclinical development of targeted therapy combinations for DIPG. The goal of the research is to test and then move the most effective therapy forward to early phase clinical trials in the next 18 – 24 months.

Children with DIPG have a uniformly dismal prognosis with a median survival of 9 months. A DIPG tumor grows amidst the nerves in the pons (middle) of the brain stem, and therefore cannot be surgically removed. Radiotherapy provides only temporary improvement of symptoms. No chemotherapy has ever proven effective. Novel therapies are desperately needed. “The scientific community has truly rallied around this cause. The mandate for a novel therapeutic approach was born in the Children’s Oncology Group brain tumor committee under the bold leadership of Dr. Amar Gajjar. With the consortium co-leadership of clinical trialist Maryam Fouladi and the accountability to DIPG patients and their family, this program is moving unexpectedly quickly towards its goal,” says Charles Keller, MD, Associate Professor and leader of the Pediatric Cancer Biology Program, Pape’ Family Pediatric Research Institute in the Department of Pediatrics at Oregon Health & Science University.

“If we succeed, it will be because families that have donated their children’s tumor gave us this opportunity. We are reminded every day that the cultures we study are parent-directed Legacy Gifts of the most selfless kind from children who current therapy could not save (the brain stem being vital to life; therefore, tumor donation can only occur at autopsy). What ABC2 , CureSearch for Children’s Cancer, the Lyla Nsouli Foundation, and The Cure Starts Now have done to make our consortium possible, and so quickly, is unprecedented – and greatly appreciated,” adds Dr. Keller.

The research project entitled, “Rapid Preclinical Development of a Targeted Therapy Combination for DIPG” was launched with initial support from The Cure Starts Now Foundation. Two additional European labs were added to the project with funding from The Lyla Nsouli Foundation for Children’s Brain Cancer Research (based in London, UK).

The funding from ABC2 and CureSearch for Children’s Cancer added a cutting-edge functional genomics component that will prioritize potential new drug targets. “We are proud to support this multi-national team of researchers in their efforts to rapidly develop effective drugs to treat children suffering from DIPG,” said Max Wallace, CEO of ABC2. “By combining forces with our non-profit partners, ABC2 looks forward to leveraging the resources and expertise of all the organizations to improve the lives of children with cancer.” John Lehr, president and CEO of CureSearch for Children’s Cancer echoed Wallace’s comments saying that “developing new drug targets is an integral step to providing children with DIPG a better prognosis. CureSearch is committed to funding research in rare cancer types so that one day, all children will be guaranteed a cure.” Keith Desserich, Chairman and Co-Founder of The Cure Starts Now Foundation added, “innovative strategies such as Dr. Keller’s and his collaborators will help lead to a revolution in cancer care for all. Not only by focusing on rare cancers like DIPG can we offer hope to children fighting this disease, but we also learn vital skills that may ultimately lead to a cure for all cancers; and in this way, this may be the start of a truly homerun strategy in cancer cures.”

About the DIPG Preclinical Consortium
The multi-national consortium is identifying potentially important biological pathways in DIPGs that are readily targetable with currently available molecularly-targeted agents. In addition, the consortium has successfully grown human DIPG tumors from autopsy materials in the petri dish and has developed mouse models of DIPG – a key resource to functionally testing potential therapies.

Since the number of children with this unfortunate disease is limited, and the number of available targeted agents is quite large, the consortiumhypothesizes that it can identify a promising combination of molecularly-targeted agents using functional genomics to prioritize targets. The ultimate goal is to move the most effective single agent or combination therapy forward to early phase clinical trials in the next 18-24 months.

The DIPG Preclinical Consortium team includes:

Charles Keller MD, Kellie Nazemi MD and Nate Selden MD, PhD at Oregon Health & Science University
Oren Becher MD, Duke University Medical Center
Michelle Monje MD, PhD, Stanford University
Maryam Fouladi MD, Cincinnati Children’s Hospital Medical Center
Cynthia Hawkins, MD, PhD, University of Toronto
Xiao-Nan Li MD, PhD, Baylor College of Medicine
Dannis G. van Vuurden MD, MSc, & Esther Hulleman, VU Cancer Center Amsterdam
Jacques Grill, Institut Gustave-Roussy, Villejuif, France

For More Information about the Research Funding Partners:

Accelerate Brain Cancer Cure: www.abc2.org
CureSearch for Children’s Cancer: www.curesearch.org
The Cure Starts Now Foundation: www.curestartsnow.org
The Lyla Nsouli Foundation for Children’s Brain Cancer Research: www.lylansoulifoundation.org

www.abc2.org/article/patient-groups-and-researchers-join-forces-speed-treatments-rare-pediatric-brain-tumor

Dr. Mark Souweidane Performs Successful DIPG Biopsy

Olivia Boccuzzi

On Friday, January 6, Dr. Mark Souweidane performed a DIPG biopsy on Olivia Boccuzzi, who was only 22 months old when she was diagnosed with the disease on September 21 of last year. Olivia’s parents, Enza and Frank, noticed their daughter had a hard time walking and her face was starting to droop. They decided to take her to the emergency room, where the terrible news was confirmed by an MRI. This beautiful young girl who loves dancing like a ballerina, watching Dora the Explorer and playing with 4-year-old brother James, now faced six weeks of radiation and a regimen of Dexamethasone (brand name Decadron), a steroid drug frequently administered to brain stem tumor patients for the swelling and “tightness” of their tumor at the base of their skull. Doctors treating Olivia continued to monitor her until, on December 8, an MRI showed that Olivia’s tumor exhibited a significant amount of necrosis. With these atypical results, her parents consulted with Dr. Mark Souweidane, who agreed to perform the biopsy. This procedure had been done in France but remains a controversial choice in the United States. The operation was a success and after one night in the ICU, Olivia is back home with her family awaiting the results of her groundbreaking biopsy. The Cristian Rivera Foundation wishes to applaud Olivia and her family for their strength and bravery in the face of DIPG and will keep them in our thoughts and prayers as Olivia continues to recover.

Community comes out to support 5-year-old cancer patient

She doesn’t know Zach Cadwalder or have any affiliation with his family, but when Jessica Kane heard about the fundraiser that will benefit the sick 5-year-old, she knew she wanted to be a part of it.

“I’m a teacher, and it’s terrible to see a child sick. So, anything you can do,” she said.

Ms. Kane was one of hundreds who came out to support the Cadwalders after the Green Ridge family found out in November that Zach had diffuse intrinsic pontine glioma – an inoperable brain tumor. The family takes Zach to an experimental clinical trial at the National Institutes of Health in Maryland for treatment.

Family and friends pulled together to make sure the family had what they needed to get by.

“It’s devastating news; he really is the strongest person we know,” said relative Gabrielle Kasaczun, who helped organize the event. “We decided we had to do something to raise money.”

With a large picture of Zach dressed in a purple baseball uniform – his ears poking out from his cap, clutching a bat and flashing a toothy grin – displayed in the middle of St. Mary’s Center on Friday night, hundreds of people came to eat, drink and buy raffle tickets.

The proceeds go toward the young boy’s medical care and travel expenses as Zach undergoes radiation and chemotherapy five days a week. His family said his attitude is as positive as ever.

“He’s very brave. He’s happy and outgoing,” Ms. Kasaczun said of her little cousin.

Zach’s aunt, Lauren Washo, said the family can only be grateful for the overwhelming support shown for the 5-year-old.

“He’s our superhero,” Ms. Washo said.

Ms. Kane said working with children as a pre-kindergarten teacher has shown her a lot about the spirit of young kids like Zach.

“They have a wonderful ability to bounce back,” she said. “They have an inner strength you wish adults had sometimes.”

Contact the writer: ksullivan@timesshamrock.com

www.thetimes-tribune.com/news/community-comes-out-to-support-5-year-old-cancer-patient-1.1260755#ixzz1kWWepAQz

 

By Katie Sullivan ksullivan@timesshamrock.com

Milestone Clinical Trial for DIPG Approved

Dr. Mark Souweidane, Director of Pediatric Neurological Surgery, has received FDA approval for a clinical trial for young patients diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG). Dr. Souweidane is the Principal Investigator on the clinical trial, which will use a novel surgical technique (convection-enhanced delivery, or CED) to deliver a tumor-fighting agent directly to the site of the glioma, bypassing the blood-brain barrier that prevents most drugs from reaching these deadly brain stem cancers in children.

The trial will use CED to deliver a therapeutic agent called 124I-8H9 directly to the tumor site using a surgically placed infusion cannula. The agent consists of the 8H9 antibody, which is produced by mice and has been shown to attack many kinds of tumors, combined with the radioactive substance 124I. In studies on other kinds of cancer, 124I-8H9 has delivered a one-two punch, with 8H9 binding to the tumor and 124I killing the cancer cells with radiation. The procedure has been tested safely in animals, but this clinical trial is the first time CED has been used to administer 124I-8H9 to a human brain.

Initial patients will be treated with a small dose of 124I-8H9 and monitored for side effects. As the study progresses, new patients will be treated with increasing doses as Dr. Souweidane monitors the safety and effectiveness of each dosage. The study is expected to last for one to two years, with a minimum of 12 DIPG patients between the ages of 3 and 21 enrolled and monitored.

Dr. Souweidane has been working on DIPG research for more than a decade, and preparing for this clinical trial for much of that time. His dedication to finding hope for his patients has been unswerving, and this trial represents an enormous leap forward in DIPG research.

“This trial is about renewed hope,” says Dr. Souweidane. “It’s a departure from the standard, ineffective, therapy, and has the potential to create a whole new paradigm in brain tumor treatment. Delivering drugs intravenously hasn’t worked because of the blood-brain barrier – to get even a small amount of medicine to the tumor we need high doses of chemotherapy, which is toxic to the rest of the body. But placing the agent outside the blood vessels, directly into the tumor, greatly reduces that toxicity while maximizing the attack on the tumor itself.”

This new approach represents a ray of hope for families facing the diagnosis of DIPG. This rare brain tumor has been uniformly fatal, made all the more heartbreaking by its propensity to strike very young children. The tumors are inoperable due to their ill-defined borders as well as their position in the delicate pons area of the brain stem. Radiation has been the standard approach, but it is not a cure and usually extends life only by a short time. Some 200 to 300 children in North America a year die from DIPG – usually within months of their diagnosis.

Unlike other critical diseases of childhood, DIPG has suffered from a lack of funding that has meant a complete lack of progress in survival rates. Over the past few decades, survival rates for medulloblastoma have reached 70 percent; for acute lymphoblastic leukemia the rate is now 85 percent. Over that same time, survival rates for DIPG have remained essentially at zero, as major foundations directed their support toward more common conditions. It is Dr. Souweidane’s hope that this innovative clinical trial will be a major first step in creating a survival rate for DIPG for the first time.

Dr. Souweidane has been fortunate to have the support of several organizations and groups whose gifts have provided much-needed funding to help him reach this point. He credits the Dana Foundation, The Cure Starts Now, the Cristian Rivera Foundation, the Beez Foundation, the Matthew Larson Foundation, and St. Baldick’s with being instrumental in allowing him to complete the important research needed before this clinical trial could begin.

The DIPG trial will be conducted at both Memorial Sloan-Kettering Cancer Center and Weill Cornell Medical Center, where Dr. Souweidane holds appointments. For more information about the trial, call Dr. Souweidane’s office at
212-639-7056, or visit the complete description of the trial on clinicaltrials.gov.

The fight of her life
Five-year-old battles brain tumor

An ever-smiling Valentina has undergone radiation treatments to shrink her brain tumor. She will undergo an MRI on Jan. 12 to check the status of the tumor.

When a person is diagnosed with a serious medical condition at any age it is devastating news for his or her friends and family. But when the parents of a four-year-old receive word that their child has an inoperable brain tumor, it’s earth shattering.

Unfortunately, that’s exactly what Farmingdale residents Erik and Cristina Bravin were told on July 21. Their daughter, Valentina, was diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG), a tumor located in the middle of the brain stem, leaving the Bravin’s with a limited amount of treatment options to explore.

“It’s shocking,” Cristina said of the diagnosis. “There was a lot of anger and asking ‘Why us?” She added, “Nobody ever wants to hear anything negative about their child, let alone when they basically give her a death sentence.”

To help the Bravin’s with mounting medical and travel expenses, several fundraisers have been held in Valentina’s name including one at the Lynbrook VFW last November. Valentina’s aunt and uncle, Gina and Frank Orosz of Lynbrook, planned the event. It was attended by approximately 170 people and raised $26,000. There have also been fundraisers held in Valentina’s hometown of Farmingdale.

The Bravins didn’t waste any time to see what the possible routes of treatment were. “We basically went into search mode,” Cristina said. “We were researching every factor and treatment possible around the country and around the world.”

Their research led them to Houston, Texas where Valentina underwent 30 radiation treatments for six consecutive weeks at MB Anderson Cancer Center. The procedures started on Aug. 18 and ended Oct. 3.

Valentina, who turned five on New Year’s Eve, has been brave throughout her fight with DIPG according to her mom. “She’s a fighter,” Cristina said. “She doesn’t do things willingly, but when she does eventually comply with anything that doctors and nurses have to do she’s very tough.” Cristina said that the radiation treatment did shrink the tumor by 50 percent, but those results are usually temporary.
The Bravin’s were hoping Valentina would be accepted to a trial at Memorial Sloan-Kettering Cancer Center by Jan. 10, but according to Cristina the Food and Drug Administration (FDA) didn’t do them any favors.

“We were waiting for the FDA to approve this trial for several months,” Cristina said. “They finally approved it on Dec. 29 which didn’t leave much time for that cutoff date.” She said that Valentina missed the Jan. 10 cutoff by a few days because patients could not be admitted to the trial after 14 weeks since their last radiation treatment.

“It’s a life or death situation, but the FDA couldn’t care less,” she said.

After Valentina’s diagnosis, Cristina resigned from her job at Winthrop University Hospital and temporarily moved to Texas while her daughter underwent radiation treatment. Erik works in sales and has also taken a lot of time off in recent months.

Valentina, who likes ballet, ice skating and playing with Barbie dolls, will undergo an MRI on January 12 to check the status of her tumor. If the tumor is stable or has regressed, Cristina said Valentina may be considered for a new trial at the National Institutes of Health in Bethesda, MD.

Valentina’s grandfather started a foundation, Friends of Valentina, after the diagnosis. Cristina said the foundation is a medical trust fund to help cover expenses and to give Valentina special celebrations when she feels up to it.

If you would like to donate to or find out more information about Friends of Valentina, you can contact Gina at (516)384-9739 or Frank at (516)509-7862 or beans14orosz@netscape.net.

By Brian Croce bcroce@liherald.com

An experimental xenograft mouse model of
diffuse pontine glioma designed
for therapeutic testing

Neurooncol. 2012 Jan 10. [Epub ahead of print] An experimental xenograft mouse model of diffuse pontine glioma designed for therapeutic testing. Aoki Y Hashizume R, Ozawa T, Banerjee A, Prados M, James CD, Gupta N. Department of Neurological Surgery and Brain Tumor Research Center, University of California San Francisco, 505 Parnassus Ave., Rm M779, San Francisco, CA, 94143-0112, USA.

The prognosis for diffuse infiltrating pontine gliomas (DIPG) remains extremely poor, with the majority of patients surviving less than 2 years. Here, we have adapted standard xenograft techniques to study glioma growth in the mouse brainstem, and have utilized the mouse model for studying a relevant therapeutic for treating DIPGs. bioluminescence imaging monitoring revealed a progressive increase in signal following the injection of either of two tumor cell types into the brainstem. Mice with orthotopic GS2 tumors, and receiving a single 100 mg/kg dose of temozolomide showed a lengthy period of decreased tumor luminescence, with substantially increased survival relative to untreated mice (P < 0.001). A small molecule inhibitor that targets cdk4/6 was used to test AM-38 brainstem xenograft response to treatment. Drug treatment resulted in delayed tumor growth, and significantly extended survival. Our results demonstrate the feasibility of using an orthotopic brainstem tumor model in athymic mice, and for application to testing therapeutic agents in treating DIPG.

http://www.ncbi.nlm.nih.gov/pubmed/22231932

ALSF Announces 2011 ‘A’ Awards To Researchers In San Francisco Area

Researchers at Stanford University and the University of California at San Francisco receive prestigious awards to examine childhood cancers

Wynnewood, PA (December 2011) – Alex’s Lemonade Stand Foundation for Childhood Cancer (ALSF) today announced the recipients of the ‘A’ Award, a groundbreaking pediatric cancer research grant designed to jumpstart the careers of promising scientists in the pediatric cancer field. Michelle Monje, MD, PhD of Stanford University and William Gustafson, MD, PhD of the University of California at San Francisco were chosen from nearly 30 applicants as the 2011 recipients of the grant which totals $375,000 over the course of three years. Dr. Monje’s research will target Diffuse Intrinsic Pontine Glioma (DIPG) while Dr. Gustafson will examine neuroblastoma.

The ‘A’ Award joined a prestigious line of medical and nursing grants from Alex’s Lemonade Stand Foundation in 2009. The award was created in an effort to find the best and brightest young researchers and encourage them to build life-long careers in the pediatric cancer field. The Foundation believes that young researchers are integral components of finding new treatments and cures, and by providing support for their research these investigators will utilize their talents toward pediatric oncology.

Dr. Monje, an Assistant Professor of Neurology and Neuro-Oncology at Stanford University, will focus her research on DIPG, a highly aggressive and difficult to treat brain tumor. DIPG is the second most common malignant brain tumor in children and the leading cause of pediatric cancer death with a survival time after diagnosis of less than a year. Monje and her team have developed the first published experimental model system to study DIPG and have thus discovered molecular factors that drive tumor growth. Now, they will use this experimental model system to test therapeutic strategies combining two drugs to target both the cells responsible for tumor initiation and those responsible for tumor expansion. If successful, Monje would seek to translate her findings into a clinical trial for children battling DIPG.

Dr. Gustafson will examine high-risk MYCN amplified neuroblastoma, a deadly form of childhood cancer which frequently becomes resistant to chemotherapy and radiation. In his research, Dr. Gustafson will develop models of this high-risk disease for pre-clinical testing, and he will utilize cutting-edge chemistry to direct targeted therapy against the Aurora A protein, known to be critically important both in driving cell growth and in maintaining high MYCN levels. Through these studies, Dr. Gustafson will ultimately work toward novel and clinically tolerable therapies which target drug-resistant neuroblastoma.

Along with the funds provided to ‘A’ Award recipients, the award will also include access to ALSF’s Scientific Advisory Board for periodic consultation and a choice of reference books to enhance the researcher’s personal pediatric oncology library. New in 2011, the Foundation will also give recipients the choice of equipment to aid in their research (up to $10,000 value), funding to attend one medical conference of their choice and the opportunity to meet other ‘A’ Award recipients to collaborate and share ideas.

The ‘A’ Award will mark the final grants allocated from Alex’s Lemonade Stand Foundation for 2011. However, the Foundation will begin accepting applications for its newly announced Bridge Grant program for NIH childhood cancer research applications that received excellent scores but did not receive funding on December 15, 2011. In an effort to keep the projects of these researchers on track while they reapply for funding, ALSF’s Bridge Grant will provide $100,000 over a 12 month period. Applications will be taken on a rolling review basis with a deadline of January 9, 2012 or until capacity is reached. A total of three grants will be funded in this cycle, with a second cycle planned for the summer of 2012.

For more information on the ‘A’ Award, Bridge Grants or Alex’s Lemonade Stand Foundation’s grant program, visit: www.ALSFGrants.org.

Alex’s Lemonade Stand Foundation 2011 ‘A’ Award Recipients

Michelle Monje, MD, PhD, Stanford University, Palo Alto, CA

A Combinatorial Approach to Target the Tumor-Initiating and Transit Amplifying Cellular Subpopulations in Diffuse Intrinsic Pontine Glioma (DIPG)

Diffuse Intrinsic Pontine Glioma (DIPG) is the second most common malignant brain tumor in children and the leading cause of pediatric cancer death. We have developed the first published experimental model system to study DIPG, and using this have discovered some of the molecular factors that drive DIPG tumor growth. We now propose to use our mouse model system to test a therapeutic strategy combining two drugs. The purpose of this proposal is to develop a strategy that will target two types of cells in the tumor – those responsible for tumor initiation, and a distinct population responsible for tumor expansion. If efficacious, we would seek to translate the findings quickly to a clinical trial for children suffering from DIPG.

William Gustafson, MD, PhD, University of California San Francisco, San Francisco, CA

Blockade of p53 and Aurora A in Therapy Resistant Neuroblastoma

High-risk MYCN-amplified neuroblastoma is typically responsive to therapy at diagnosis, becoming resistant to chemotherapy/radiation at relapse. Mirroring human disease, the well established TH-MYCN model of neuroblastoma, developed in our lab, is responsive to conventional chemotherapy. To cure MYCN-amplified high-risk neuroblastoma, we propose to: 1) Develop models which accurately reflect the genetics and behavior of relapsed, chemotherapy refractory disease. I have developed a drug-resistant version of the TH-MYCN model by crossing it with models defective in p53, leading to a defective response to chemotherapy, and mirroring a similar defect in resistant childhood disease. I plan to characterize the response of this model to conventional chemotherapy as well as novel, targeted therapies including those subsequently described. 2) Development of novel and clinically tolerable therapies which target drug-resistant neuroblastoma. I propose to use cutting-edge chemistry to direct targeted therapy against the Aurora A protein, known to be critically important both in driving cell growth, and in maintaining high MYCN levels. I have already generated a new class of Aurora A inhibitors which block both Aurora functions (current drugs only block cell growth) which we predict will have improved activity against MYCN amplified neuroblastoma.