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

Remembering Cristian Rivera

On January 25, 2009, the world lost a smart, brave and all-around remarkable 6-year-old boy named Cristian Rivera as his two-year battle with DIPG came to an end. Three years later, we remember Cristian on the anniversary of the day he earned his wings and went to Heaven. Cristian is loved and missed dearly by his father John "Gungie" Rivera, his mother Jenelle Asencio, his big brother JC, his sister Brittnee Hudson, the rest of his friends and family and everyone at The Cristian Rivera Foundation. Throughout his illness, Cristian remained positive and never let the cancer get the best of him. Though his life was cut tragically short, Cristian made an everlasting impression with his affection, loyalty and good sense of humor. His legacy will never be forgotten as we work tirelessly in his name to find a cure for the disease that took his young life.

Monthly Newsletter January 2012

Happy New Year from the Cristian Rivera Foundation.

January is always a difficult month for us because January is the month when Cristian Rivera lost his hard-fought battle with DIPG after two years. On January 25, 2009, the world lost a smart, brave and all-around remarkable 6-year-old boy. Three years later, we remember Cristian on the anniversary of the day he earned his wings and went to Heaven. Cristian is loved and missed dearly by his father John “Gungie” Rivera, his mother Jenelle Asencio, his big brother JC, his sister Brittnee Hudson, the rest of his family and friends, and everyone at The Cristian Rivera Foundation.

Wednesday, January 4, 2012 marked two years since DIPG took the life of another amazing young boy named Javier "Buji" Villamil. During his short life, Buji was a great athlete who loved sports, George Lopez, iCarly and Scooby Snacks fruit snacks. He earned his wings a few months shy of his 12th birthday and will forever be missed by his family—including his mom Rosemary Lora, dad Gabriel Villamil and aunt Fulvia Lora, a committee member with the Cristian Rivera Foundation—as well as his friends and everyone who has had the pleasure to meet him. We at the Cristian Rivera Foundation are keeping Buji and his family in our thoughts and prayers on this difficult anniversary.

Despite the sadness we feel remembering Cristian, Buji and all the young lives DIPG has taken from us, 2012 is shaping up to be a big year for us and the entire DIPG community. Beginning this month, the Cristian Rivera Foundation officially supports the research of Dr. Oren Becher, a pediatric neuro-oncologist who has dedicated his career to improving therapies for children with brain tumors with a particular focus on identifying effective therapies for children with DIPG.

Since setting up an independent study lab at Duke University in 2010, Dr. Becher’s six-person lab has sought to solve the mysteries behind the cause, make up and cure of DIPG tumors using genetic mouse models of cancer. The lab currently generates 10-15 DIPGs per month in their mouse colony and uses the tumors to both study the biology of DIPG and to evaluate the efficacy of new cancer drugs. Through this research, Dr. Becher says he and his team are now starting to understand all of the genetic changes that DIPGs harbor, thanks in part to tumor tissue from Cristian Rivera that was donated after he passed away.

“We have analyzed all the genes in Cristian’s tumor and we learned that Cristian’s tumor had the most common genetic driver that is known to drive the formation of DIPG. Approximately 30 percent of DIPGs have this common genetic driver,” said Dr. Becher. “Coincidentally, I used the same genetic driver to develop a DIPG mouse model. So in my opinion, if we can identify drug combinations that can cure our genetic DIPG mouse model, these drug combinations will also be active in children with DIPG whose tumors harbor this common genetic driver.”

We’re proud that Cristian’s tumor tissue has been such an instrumental part of Dr. Becher’s research and we look forward to supporting and funding his work. As you know, we already support the work of Dr. Mark Souweidane and this month, he finally begins his pioneering clinical trial aimed at finding a cure for DIPG by using convection-enhanced delivery (CED) to administer radio immunotherapy to children with DIPG. Dr. Souweidane officially received FDA approval for the trial on Thursday, December 29, 2011, which is expected to begin in the last week of January. Twelve patients will be selected.

We are so excited that Dr. Souweidane’s path to a cure has finally gotten the green light, so imagine our reaction when we also heard that Dr. Souweidane performed his first DIPG biopsy on a young girl named Olivia Boccuzzi on Friday, January 6. Olivia was only 22 months old when she was diagnosed with DIPG on September 21 of last year. After six weeks of radiation and medication, an MRI showed that Olivia’s tumor exhibited a significant amount of necrosis, explained Olivia’s mother Enza. Olivia’s parents consulted with Dr. Souweidane, who agreed to perform the biopsy and, after one night in the ICU, we’re proud to report that Olivia returned home and the operation was a success.

All these huge steps toward a cure empower is to continue working tirelessly in Cristian’s name to find a cure for the disease that took his young life. Please help us get even closer to our goal of curing DIPG once and for all by visiting http://cristianriverafoundation.org/donation.html and making a tax-deductible donation via Paypal. You can also mail a check to the Cristian Rivera Foundation at 40 West 37th Street, Suite 402, New York, NY 10018. Every donation big or small brings us closer to curing DIPG and we appreciate all of the amazing support we have received so far.

One Cristian Rivera Foundation committee member in particular has shown amazing strength and courage in the fight against DIPG and Cristian Rivera Foundation founder John “Gungie” Rivera wanted to take a moment to recognize her for her dedication to our cause.

As founder of the Cristian Rivera Foundation, I would like to take a moment to express my sincerest gratitude to one of our most active and dedicated committee members, Solange Osorio. Solange has been a committee member basically since the foundation began and even though she has never had or lost a child to DIPG, she has shown more devotion to finding a cure and comforting and helping DIPG families than anyone I know. She selflessly dedicates time from her busy schedule every week and has even put her education second to the needs of the DIPG community. She volunteers at every Cristian Rivera Foundation event, attends many other DIPG events, and has provided the décor for every Cristian Rivera Foundation Gala, even going so far as to skip shopping for her outfit to the Third Gala just to make sure that everything was running smoothly. Solange has shown amazing compassion towards DIPG families and has been the communicator between the foundation and the DIPG community. I communicate with the doctors and certain foundations, and she deals with everyone else. She always remembers to send a Christmas card to a DIPG family, an email to find out how a sick child is doing, a message of encouragement in tough times, an acknowledgment of DIPG Angel anniversaries and everything in between. She is well-known within the DIPG community for the kindheartedness and care she puts into every interaction. Thank you for all of your hard work and dedication. Thank you for being my friend and standing by me in my quest to rid the world of DIPG. For this and so much more, I Love Her.

Everyone at the Cristian Rivera Foundation is grateful for Solange’s contribution, along with the donations and support we receive from every member of the Cristian Rivera Foundation family. Remember to visit www.cristianriverafoundation.org, which we’ve recently revamped with new tabs with informative DIPG content such as ongoing clinical trials, helpful information for understanding DIPG biopsies, and the latest news from the Cristian Rivera Foundation and the entire DIPG community. You can also make a tax-deductible donation on our website, or purchase Cristian Rivera Foundation merchandise, like official foundation wristbands, t-shirts, wood beaded bracelets and necklaces with your choice of bronze or silver charm. The official Cristian Rivera Foundation Merchandise Catalog is available for you to download and show your friends, family and colleagues all of the exciting products we offer in support of our cause. Also make sure to follow us on Twitter @tweetcrf and on Facebook at www.cristianriverafoundation.org to get the latest updates on all our upcoming events.

Next month, the Cristian Rivera Foundation will host a special charity performance of DC-7, The Roberto Clemente Story at the Puerto Rican Traveling Theatre in Times Square. DC-7 is a bilingual musical that tells the amazing story of Hall of Famer Roberto Clemente, one of the most beloved figures in the history of Major League Baseball. The Society of the Educational Arts (SEA), under the direction of Manuel Moran, has partnered with Cristian Rivera Foundation founder John “Gungie” Rivera and Cristian Rivera Foundation Committee Members Ralph Mercado III and Miriam Colon to bring this amazing production to New York for a limited run.

DC-7 was recently nominated for 7 ATI Awards (Independent Theater Awards), including Best Musical. The show has already won 6 ACE Awards from the Latin Critics Association, including Best Musical, Best Director and Best Book for writer-director Luis Caballero, Best Actor for Modesto Lacen, Best Actress for Lorraine Velez and Best Supporting Actress for Xiomara Rodríguez.

Visit www.teatrosea.org to find out more about the show and keep checking our website, Facebook and Twitter for the latest details on dates, times and ticket prices for our special night at the theater. We look forward to seeing you there and we look forward to all of the great things ahead for us in 2012. Full Steam Ahead!

Sincerely,
“Cristian’s Mommy” Jenelle Asencio, John “Gungie” Rivera “Forever Cristian’s Daddy,” and all of the members of the Cristian Rivera Foundation Committee: Aldo Marin, Alex Garcia, Andy Epstein, Anthony Mason, Camilo, Carlos Colon, Carlos Keyes, Dr. Bear Walker, Dr. Ben Velazquez, Carlos R. Colon, Dolores Catania, Doug E. Fresh, Edward Caban, Elis Pacheco, Fernando Ferrer, Fulvia Lora, Funkmaster Flex, Hamlet Peralta, Jaison Newring, Jay Hernandez, Jeff Lavino, Jerry Chaparro, Jerry Salerno, J.I. Starr, Jim Jones, John “Jelly Bean” Benitez, Johnny Nuñez, Jose Alberto “El Canario,” Joseph Mbeh, Kamar De Los Reyes, Karen Cerna, Katie DeFlorio, Kenan Thompson, Khaliah Ali, Little Louie Vega, Luis Guzman, Manuel Alejandro Ruiz (Boy Wonder), Mark Rosner, Matthew “Mateo” Rajkumar, Melissa Chung, Miriam Colon, Murray Richman, Odalys Molina, Pablo Diaz, Ralph Mercado III, Ramon E. Javier, Ray Wong, Ricardo Cardona, Rickie Ricardo, Robert Vazquez, Ruben Sierra, Solange Osorio, Steve Cox, Tedsmooth, Tony Ferrara, Victor Martinez, Vito Bruno, William Padilla and William Pla.

Dr. Mark Souweidane Performs Successful DIPG Biopsy

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.

Celebrity Chef and Cristian Rivera Foundation Committee Member Ricardo Cardona Caters Delicious Home-Cooked Meal for "Moms for Mikey" Auction Winners

The Cristian Rivera Foundation, along with Committee Member and Celebrity Chef Ricardo Cardona, donated an event package that was auctioned at the Mikey Czech Foundation's annual fundraiser gala, "Moms for Mikey," on Saturday November 19, 2011 at The Woodway Country Club, Darien, CT.


Robert and Dana MacNaughton bid a winning $7,500 for Chef Cardona to personally cater a meal for 8 people, including food, liquor and servers. They invited three couples over a delicious meal of miso sea bass, churrasco and traditional steak accompanied by lobster salad. For dessert, Chef Cardona impressed the guests with chocolate cake, apple empanadas and flan. "The dinner was phenomenal and unbelievably delicious," said Mrs. MacNaughton, who noted that while she and her husband have visited some of the best restaurants in the world, they were absolutely stunned by Chef Ricardo Cardona's amazing recipes. "Everybody called back next day and complemented the food and expressed their gratitude for such a delicious dinner."

This was Mr. MacNaughton's second year attending the event and the first for his wife. Though they don't personally know a child with DIPG, the MacNaughton's 4 ½-year-old son Stewart was diagnosed with Medullo Blastoma two years ago. They feel that children with cancer don't receive enough funding for treatment and wanted to help with their generous $7,500 bid.

"It was a very unique, almost one in a lifetime opportunity," said Mrs. MacNaughton. "If the same prize is offered again next year, I would do it again and definitely recommend it to everybody."

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

By Katie Sullivan ksullivan@timesshamrock.com
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

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

By Brian Croce bcroce@liherald.com

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.

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

J 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.

The Cristian Rivera Foundation mourns the loss of Samuel Tyler Kidd

Samuel Tyler Kidd

Monday January, 9 2012 was a sad day for the entire DIPG community as Heaven gained another DIPG Angel. Eleven-year-old Samuel Tyler Kidd lost his battle with DIPG at 9:30 that morning. His parents, Mark and Mindy, will always remember him as vibrant, fun-loving, caring little boy. He had a huge heart of pure gold and always smiled no matter what. Tyler put up a brave fight against DIPG and was an example of incredible courage and strength. The Cristian Rivera Foundation extends our sincerest sympathy and condolences at what is surely an unimaginably difficult time for Tyler's family and friends. Samuel Tyler Kidd will always stay in our thoughts and prayers.

A Special Thanks to Cristian Rivera Foundation Committee Member Solange Osorio

Cristian Rivera Foundation
Committee Member
Solange Osorio

As founder of the Cristian Rivera Foundation, I would like to take a moment to express my sincerest gratitude to one of our most active and dedicated committee members, Solange Osorio. Solange has been a committee member basically since the foundation began and even though she has never had or lost a child to DIPG, she has shown more devotion to finding a cure and comforting and helping DIPG families than anyone I know. She selflessly dedicates time from her busy schedule every week and has even put her education second to the needs of the DIPG community. She volunteers at every Cristian Rivera Foundation event, attends many other DIPG events, and has provided the décor for every Cristian Rivera Foundation Gala, even going so far as to skip shopping for her outfit to the Third Gala just to make sure that everything was running smoothly. Solange has shown amazing compassion towards DIPG families and has been the communicator between the foundation and the DIPG community. I communicate with the doctors and certain foundations, and she deals with everyone else. She always remembers to send a Christmas card to a DIPG family, an email to find out how a sick child is doing, a message of encouragement in tough times, an acknowledgment of DIPG Angel anniversaries and everything in between. She is well-known within the DIPG community for the kindheartedness and care she puts into every interaction. Thank you for all of your hard work and dedication. Thank you for being my friend and standing by me in my quest to rid the world of DIPG. For this and so much more. I Love Her.

Remembering Javier "Buji" Villamil

Javier "Buji" Villamil

Wednesday, January 4, 2012 marked two years since DIPG took the life of an amazing young boy named Javier "Buji" Villamil. During his short life, Buji was a great athlete who loved sports, George Lopez, iCarly and Scooby Snacks fruit snacks. He earned his wings a few months shy of his 12th birthday and will forever be missed by his family—including his mom Rosemary Lora, dad Gabriel Villamil and aunt Fulvia Lora, a committee member with the Cristian Rivera Foundation—as well as his friends and everyone who has had the pleasure to meet him. We at the Cristian Rivera Foundation are keeping Buji and his family in our thoughts and prayers on this difficult anniversary.

DIFFUSE INTRINSIC PONTINE GLIOMA: COLLABORATION IS LEADING TO CHANGE

Written by: Dr. Loice Swisher

Dr Loice Swisher whose daughter Tori is a ten year medulloblastoma survivor.  Loice is an FDA Patient Representative and emergency medicine physician. She is pictured here on a family vacation in Utah.

“It's been almost two years since Sam was diagnosed in December 2006. The only improvement that I've seen during this time is that we have this wonderful site!” [the DIPG Internet Yahoo support list and discussion group]

So said “Sheila” (in December 2008), whose young grandson had died in February 2008 from a diffuse intrinsic pontine glioma.

A diffuse intrinsic pontine glioma, known as DIPG, is perhaps the most feared pediatric brain tumor because of the dismal survival statistics and devastating clinical course. This tumor tends to strike four to ten year olds with approximately half of these young children dying in the first year and 80-90% by the end of the second.

Despite more than 200 trials, no treatment has been found to be effective for long term survival in DIPG. For some children, steroids and radiation allow for a ‘honeymoon’ with relief of symptoms. But this is often followed by a relentless advancing of the disease and tragically, death months later.



Basic science research into this tumor has been frustratingly difficult. A significant hurdle has been the lack of tumor tissue on which to carry out tests.

In 1993, the standard of care for DIPG in the United States changed, as biopsy provided no improvement in survival over neuro-imaging in typical pediatric diffuse pontine tumors. Since that time, biopsies of pediatric DIPG have been uncommon resulting in the scarcity of tumor material for research. At the time “Sheila” wrote, there were no published reports on cell lines, no animal models and no molecular/genetic studies.

The changes in the medical community’s approach to a disease are often evident much before the patient community is aware of them because the time from concept to study to publication of a research paper can take years. In 2008, change was beginning in DIPG research. The heart-wrenching post from grandmother “Sheila” launched an effort towards earlier awareness of research endeavors as well as international advocacy collaboration.

In 2005 the biopsy debate had heated up again. St Jude Children’s Research Hospital in Memphis, Tennessee (USA) responded with a concerted effort to approach families for post-mortem tumor donation for research resulting in more molecular information on DIPG. Since many children die at home, far from St Jude, the emotional and logistical challenges were numerous.

A family responded to the financial issues raised by these challenges by establishing a foundation called Tyler’s Treehouse (established in 2006), specifically started to fund the logistical aspects of this study.

Over the ensuing years, many families with DIPG children have provided the ultimate gift to the research community involved with these studies of their child’s tumour tissue. Some families as far away as Australia and South America have donated their child’s tissue. The St. Jude efforts haven’t lead to publication yet, however, The Hospital for Sick Children (“Sick Kids”) in Toronto, Canada published the first whole genomic analysis of DIPG tumors in February 2010.

Their French colleagues took a different approach, with a clinical trial including upfront stereotactic biopsy of pediatric DIPG. In the July 2007 issue of the Journal of Neurosurgery the surgical results were published. With 33 children there was no mortality and only two children had transient morbidity.

The combined effect of the French stereotactic biopsy results and the molecular analysis studies from “Sick Kids” in Toronto has lead to renewed efforts for future clinical trials to include molecular analysis from stereotactic biopsy samples.

The development of animal models is also emerging.



At the 2008 ISPNO (International Symposium on Pediatric Neuro-Oncology) conference in Chicago (USA), Dr. Oren Becher won the best basic science presentation award for his genetically engineered mouse model of brainstem glioma. The excitement of potentially being able to study this tumor in a mouse model has resulted in requests for Dr Becher’s mice from several others interested in studying brainstem glioma.

For some time, the non-availability of resected tumor tissue for the development of cell lines has met with failure, even to the point of new researchers being discouraged from pursing this direction.

In the summer of 2009, Stanford University in California revealed that Dr. Michelle Monje had been able to culture neurospheres from post-mortem pediatric DIPG tissue using a stem cell technique. This breakthrough in DIPG research at Stanford has lead to an EGFRviii vaccine being introduced to the pediatric brain tumor community for the first time as well as other research. Some of this has been funded through the Kyle O’Connell Foundation.

Truly exciting events have been two international meetings of researchers and clinicians to discuss DIPG. The Fondo Alicia Pueyo hosted the first conference in Barcelona, Spain in February 2009. The second event was hosted by The Hospital for Sick Children in Toronto with funding support by Just One More Day and B.R.A.I.N.child.

We are now seeing a change in DIPG research - and the international collaboration of parents, advocates, clinicians and researchers that is making this happen.

Mikey Czech's father Steve Czech interviewed on Today

The Mikey Czech Foundation funds new hope for cancer research

Written by: Rachel Kirkpatrick, Editor

When Mikey Czech was diagnosed in January 2008 with a diffuse intrinsic pontine glioma (DIPG), a rare pediatric brain tumor, his parents Steve and Jennifer were told there was little that could be done. 

Up until that point, doctors in the United States were hesitant, if not unwilling, to biopsy these brain tumors due to their locations in the brainstem and the perceived risks. Children with DIPGs were often diagnosed, went through intense, experimental chemotherapy or radiation, and they died, as Mikey did exactly nine months later at age 11. 

At the foundation's fourth annual "Soaring Towards a Cure" gala on November 19, the Czechs announced that a major clinical trial is being launched in the U.S. that could provide more information than ever on the molecular makeup of these DIPG tumors and, in turn, the best way to treat them. 

"This just goes to show you that when people get together, when you focus on something and are relentless in its pursuit, you can change anything," Steve told the Advertiser. 

On a mission 


DIPG is one of the most aggressive and deadliest forms of brain tumors diagnosed in children between the ages of three and 16, according to the foundation. DIPGs are inoperable, incurable and — until recently — unable to be biopsied. Approximately 80% of patients diagnosed with DIPGs die within one year of diagnosis; nearly 100% die within five years of diagnosis. 

"Conventional treatment consists of general radiation and chemotherapy," according to the foundation. "Alternative treatments include various clinical trials (approximately 250 over the past 30 years) which, to date, have proven no more effective than conventional treatment." 

Following Mikey's death, the Czechs identified the foremost expert in the world on DIPG, Dr. Mark W. Kieran of Harvard Medical School/Dana-Farber Cancer Institute ­— one of the five doctors they consulted following Mikey's diagnosis. 

"We were very impressed with him," Czech said. "We went back to him and said, 'Look, everyone but you said this tumor was not biopsiable, why?'" 

The Czechs learned that while neurosurgeons in the U.S. were not willing to do these biopsies, Parisian neurosurgeons are ­— and have, successfully. In fact, at that time the Czechs met with Kieran, the doctor told them there had been 20 biopsies taken of these tumors by French neurosurgeons and not one of them had a single complication. 

With backing from the foundation, Kieran set out to convince 20 U.S. hospitals that this procedure was not as dangerous as had been believed. 

"He convinced the neurosurgeons from 20 U.S. hospitals here with the Parisian neurosurgeons and said, look, here's the technique," Czech said. "And once they saw that technique, they said, 'You're right — we can do this.'" (A list of the participating U.S. hospitals is available at NCAdvertiser.com.) 

Now, The Mikey Czech Foundation, in conjunction with six other foundations, is funding a new clinical trial which was approved on November 11, allowing these tumors to be biopsied in the United States for the first time. Participating foundations include: The Zach Carson DIPG Fund, The Ellie Kavalieros DIPG Fund, The Prayers From Maria Foundation, Children's Hospital of Los Angeles Imaging Center, The Pediatric Brain Tumor Research & Clinical Fund at Dana-Farber Cancer Institute, and Stop & Shop Pediatric Brain Tumor Research Fund. 

A new laboratory 

A second goal of the foundation is to establish a dedicated translational research laboratory where these tumors can be studied and remedies can be developed. 

With the support of the aforementioned foundations, Kieran obtained the live tissue samples of the 20 biopsies from children in Paris and took them to the Broad Institute of Boston (a joint venture between Harvard Medical School and the Massachusetts Institute of Technology), where the tumor cells can be deciphered to identify their molecular make-up using the "latest and greatest" in nanotechnology and biomedical engineering, Czech said. 

The Broad Institute has already identified 2,000-plus different mutations that cause cancers in general. 

"We're going to launch this next phase, the U.S. phase of this," Czech said. "What's going to happen at these 20 centers where the kids get diagnosed is, the day they get diagnosed, they are going to get biopsied. Those live tissues will be flown to Broad and they're going to identify what the molecular markers are, what the mutations are, and compare them to the 'encyclopedia' of mutations that are already known in an effort to identify existing remedies that can be used to treat these DIPG tumors. 

"The idea being that each one of these kids now, for the time, is going to have his or her own custom-made treatment, as opposed to just throwing everything at the wall and seeing what sticks," Czech said.

Just $700,000 

In the two and a half years The Mikey Czech Foundation has been in existence it has raised more than $1.5 million in funding. As Czech points out, this has been accomplished at the grassroots level, with no corporate or government involvement. 

"The extent to which people have reached out has been incredible," Czech said. "I always refer to New Canaan as my 18,000-person family, because of the way the community has embraced us. We'll always be grateful." 

It has taken 10 years for researchers to get to this point and it was done, notably, with $700,000. Czech knows that with consistent funding and support, the possibilities are endless. 

There are no pharmaceutical companies willing to write a big check for research — nor is the federal government — at least for now, because this cancer is so rare. So when Czech, a hedge-fund manager, raised his second fund, the $1.1 billion Czech Asset Management, LP, last year, he made a contractual commitment to donate a fixed percentage of his personal profit from the hedge-fund to The Mikey Czech Foundation. 

"For the first time, there is a meaningful, sustainable source of funding for efforts solely directed toward pediatric brain tumors," Czech said. "And that's what it is going to take — there has to be a sustainable source to keep these people interested in researching it, but also to attract other researchers into the field." 

Nearly four years since the loss of his only son, Czech is finding a comfortable balance in life. 

"It's very simple: it's family, faith, foundation and the fund, and the fact of the matter is, the fund and foundation's success are very much aligned," Czech said.