CBTP Manuscript Accepted by Oxford Academic Neuro-Oncology Advances

A new research study from the CBTP lab entitled “Combined Targeting of PI3K and MEK Effector Pathways via CED for DIPG Therapy,” was published in Neuro-Oncology Advances on May 29, 2019. The published manuscript, submitted by Dr. Uday Maachani (Dr. Souweidane Lab) on behalf of the entire research team, showed the results of a comprehensive study of midline gliomas including diffuse intrinsic pontine gliomas (DIPG), and the relationship between amplified phosphatydylinositol 3-kinase (PI3K), mitogen-activated protein kinase (MEK), and the development of these particular tumor types.

In this comprehensive study, the research team tested both PI3K inhibitor ZSTK474 and MEK inhibitor trametinib, alone and in combination in vitro  and in vivo, using various delivery methods.

The results showed that combination treatments were found to be synergistic with dosing across the  cell lines tested, and significant tumor suppression was seen when given systemically against a subcutaneous DIPG model, and when given via convection-enhanced delivery (CED) in an intracranial DIPG mouse model.  Data indicates that ZSTK474 and trametinib combination therapy inhibits malignant growth of DIPG cells in culture  and in animal models, prolonging survival. The results warrant further investigation and the team is excited by the potential for a promising new combination approach to treat DIPG via CED.

View Article Abstract Here

Download Full Article Here

Funding: This research was supported in part by Cristian Rivera Foundation, McKenna Claire Foundation, The Lyonhearted Foundation, Christian Koehler Foundation, Brooke Healey Foundation, Fly a Kite Foundation, Children’s Brain Tumor Family Foundation, Joshua’s Wish, Lily LaRue Foundation, and by the Alex Lemonade Stand Foundation’s Pediatric Oncology Student Training Grant (UT), and American Brain Tumor Association Medical Student Summer Fellowship in honor of Collegiate Charities Dropping the Puck on Cancer and Super Lucy (RC).

No Laughing Matter 2019

 

The Children’s Brain Tumor Family Foundation Hosts Second Annual “No Laughing Matter on October 22 at Carolines on Broadway

 

An All-Star Comedy Line-Up will Donate Time and Talent to the Laugh-Out-Loud Fundraiser to Help Raise Research Funding for Rare and Inoperable Brain Tumors at Weill Cornell Medicine

The room is sure to be filled with laughter infused by hope on Tuesday, October 22 at Carolines on Broadway as the Children’s Brain Tumor Family Foundation gears up tp host the second annual “No Laughing Matter,” a night of comedy to benefit the Children’s Brain Tumor Project (CBTP) at Weill Cornell Medicine. The inaugural event in 2018 raised $450,000 to benefit the groundbreaking research of Dr. Mark Souweidane and Dr. Jeffrey Greenfield, co-founders of the CBTP, and we hope to surpass that goal in 2019.

“We are so grateful to the Children’s Brain Tumor Family Foundation, the dinner committee, the performers, the honorees, the sponsors and the volunteer staff who made this event such a tremendous success.  We are so humbled to be the beneficiary of such a big event in New York City, and we were completely blown away by the support,” said Dr. Jeffrey Greenfield.

The purpose of the event is to raise much-needed research funding for rare and inoperable tumors while honoring the brave children like Walker Lipton, who have been impacted by a pediatric brain tumor diagnosis.  The incidence of brain tumor diagnoses among children has been increasing over the past 40 years, yet little progress has been made in advancing cures.  Brain tumors are the most common tumors found in children, and also the most fatal.

In addition to side-splitting comedy, the evening will include an award presentation to Emma Hill, who helped her daughter, Elizabeth, create “Elizabeth’s Hope,” before she passed away in 2012. Elizabeth’s Hope was the cornerstone for the creation of the Weill Cornell Medicine Children’s Brain Tumor Project.

IF YOU WERE UNABLE TO ATTEND, PLEASE CONSIDER MAKING A DONATION.

Tara

Tara is doing something…

It was a routine eye exam that led to the discovery that Tara Gordon Lipton’s six-year-old son, Walker, had a brain tumor. Life was turned upside down in an instant.

After being diagnosed with medulloblastoma, Walker’s tumor was removed and followed up with an intense series of treatments that saved his life – and left a lasting impact on the Lipton family. Today, Tara continues to help others impacted by this terrifying diagnosis through her work with the Children’s Brain Tumor Project among other philanthropic efforts.

“Walker is my youngest of four children. Upon diagnosis, I became fiercely protective of my child, stopping at absolutely nothing to ensure he was getting the best care,” said Tara.

His tumor was surgically removed by Dr. Mark Souweidane, and Dr. Jeff Greenfield, Weill Cornell Medical Center, and he followed up with an effective, albeit brutal, treatment protocol.

“It was hell. I had to resist the urge to run away every day we walked into clinic, but then when the elevator doors opened to reveal hundreds of kids on the oncology floor I realized that I can never turn a blind eye to this shocking reality,” Tara said. “I vowed that once we got through our own journey, I would continue to help others impacted by the disease.”

Today, Tara advocates for more research funding that will advance treatment options for children with rare and inoperable brain tumors. On October 16, 2018, Tara chaired “No Laughing Matter – A Night of Comedy in Support of the Children’s Brain Tumor Project” at Carolines on Broadway.  The fundraiser was a huge success with a hilarious line-up (including Ali Wentworth, Jessica Kirson and Jim Gaffigan), raising $450,000 for research. Visit nolaughingmatter2018.org for more information.

“I am forever indebted to Dr. Greenfield and Dr. Souweidane for saving my son’s life. I want to enable them to give that gift to more families impacted by this disease.”

Tara is doing something.  You can do something, too. Donate to the Children’s Brain Tumor Project today.

The Jeffers Family

The Jeffers Family is Doing Something

Sam Jeffers was in the second grade when he was diagnosed with a rare pediatric brain tumor called thalamic glioma.  His parents were told that children diagnosed with this type of tumor have a very poor prognosis.  It is a rare, inoperable tumor that is seemingly neglected by the research community.

Pediatric brain tumors are the most fatal cancer found in children.  While other childhood cancers such as leukemia have seen remarkable improvements in survival rates over the past 40 years, there has been very little research to improve outcomes for children with brain tumors because it’s so difficult to secure appropriate research funding, especially to investigate those pediatric brain tumors that are most rare.

Government grants and gifts from major foundations tend to support common cancers, where each advance benefits thousands of people. That makes sense, of course. But how can you tell a parent that not only is there no hope for their child, but that nobody is even working on it? The only way to improve the prognosis for thalamic gliomas and other rare and inoperable brain tumors in children is to study them, understand them better at the molecular level, and test innovative therapies against them.

Sam’s parents, John and Sabrina, founded the Samuel Jeffers Foundation to fund the research that children with rare and inoperable brain tumors like thalamic gliomas so desperately need.

“Our top priority is to raise enough funds to hire a full-time dedicated researcher at Weill Cornell Medicine who will study thalamic gliomas at the genomic level,” said John Jeffers.  “This isn’t just about Sam, it’s about everyone.  We have to do a better job at protecting all children.”

The Jeffers family and their extremely dedicated community of supporters have been delivering on their promise to fund a fellowship at the Weill Cornell Medicine Children’s Brain Tumor Project.  In fact, in 2018, that dream has become a reality when they made a substantial donation that will allow the lab team to acquire a dedicated researcher who will focus on this disease.  Their total contributions of almost $115,000 to the Children’s Brain Tumor Project helped the lab (1) launch the thalamic glioma registry (thalamicgliomaregistry.org), an essential tool to unite the thalamic glioma community and collect tumor tissue, (2) to hire a summer fellow who helped secure essential tumor tissue for investigation, and (3) to hire a full-time researcher in early 2019 who will take this research to the next level by sequencing the tumors, attempting to grow cell lines and avatar models, analyzing the data, conducting targeted drug screening and more.

“We don’t want any other child or family to go through what Sam went through. We want to help find a cure for these rarer types of tumors that cause so much devastation in the lives of children. That’s why we strongly support the incredible work and efforts of Dr. Mark Souweidane and the Weill Cornell Medicine Children’s Brain Tumor Project.  Together, we truly believe we can change outcomes for future families,” said Sabrina Jeffers.

You can do something, too. Donate to the Children’s Brain Tumor Project today.

To learn more about what the Samuel Jeffers Foundation is doing, visit samjeffersfoundation.org.

 

Twitter Chat – September 5

HIGH GRADE PEDIATRIC BRAIN TUMORS – RESEARCH, DISCOVERY & ROADBLOCKS

Wednesday, September 5, at 11AM (Eastern).

Weill Cornell Medicine Brain and Spine Center, @WCMCBrainSpine, invites you to participate in a Twitter chat with co-hosts Dr. Mark Souweidane, co-founder of the Weill Cornell Medicine Children’s Brain Tumor Project, David Walker, Professor of Pediatric Oncology at University of Nottingham and Chair of the Children’s Brain Tumor Drug Delivery Consortium, and The Coalition Against Childhood Cancer.  The broader brain tumor research and advocacy community will be engaging in this real-time conversation about the current landscape of pediatric brain tumors.

Special thanks to our co-hosts at the Coalition Against Childhood Cancer (@CAC2) for participating in the chat and sharing among its members who support and serve the childhood cancer community.

Follow @WCMBrainSpine@DrSouweidane , @DavidWalker40, @CAC2org and @CBTDDC as we gather online by following and posting with the hashtag #KidsBrainTumors.  The team will be interacting directly with all participants, as will various members of the pediatric brain tumor research and advocacy community.

 

Add to Calendar
09/05/2018 11:00 AM
09/05/2018 11:30 AM
America/New_York
Pediatric Brain Tumor Twitter Chat (#KidsBrainTumors)
HIGH GRADE PEDIATRIC BRAIN TUMORS – RESEARCH, DISCOVERY & ROADBLOCKS
https://twitter.com/WCMCBrainSpine

 

The CBTP Declares September to be Childhood Cancer ACTION Month

In 2012, President Obama declared September to be national “Childhood Cancer Awareness Month.”  As many know, childhood cancer is represented by the gold ribbon, and as a community we have seen the “go gold” movement generate increased momentum.

Although increased awareness = increased funding = increased research = increased success in discovering new treatment options, none of this is happening fast enough for children who are in treatment today.

The Children’s Brain Tumor Project is “Powered by Families,” and among the 50+ families on our family council, the majority are bereaved.  Awareness simply isn’t enough.  This September, we are asking you to take action.

Taking action can be a grand gesture, or a simple post on social media.  Host a fundraiser, sign up for a newsletter, share the facebook page of a child in treatment, read our latest newsletter.  Any action brings us one step closer to progress.

There is a CBTP profile picture frame that can be used for the month of September by visiting www.facebook.com/profilepicframes/ and entering “Children’s Brain Tumor Project Go Gold” in the search nox.  Please consider changing yours to “go gold” for the month of September.  Follow our posts on facebook for ideas on how you can take action this month, and, most of all, we hope you are touched by the individual stories we will be sharing about the children who are part of our family council.  The Children’s Brain Tumor Project is “Powered by Families” and we look forward to sharing their touching stories with you in an effort to raise awareness and increase action.

 

Events

UPCOMING EVENTS:

June 22, 2019: Bronxville 5K with Head for the Cure

Our charity partners at Head for the Cure are bringing back the 5K previously known as the “Bronxville Road Race” to benefit the Children’s Brain Tumor Project at Weill Cornell Medicine in loving memory of Elizabeth Minter (Bronxville High School Class of 2009).  REGISTER OR DONATE HERE.

 

October 22, 2019: No Laughing Matter

Save the Date for our second annual “No Laughing Matter” comedy night fundraiser at Carolines on Broadway.

 

 

 

PREVIOUS EVENTS:

Nov 14, 2018: Cristian Rivera Foundation 10th Annual Celebrity Gala

Cristian always said, “I Love You Ten.” This year marks the 10th anniversary of the Cristian Rivera Foundation Celebrity Gala to benefit the CBTP in his memory.  Save the Date and be sure to follow the Cristian Rivera Foundation on Facebook for event updates.

 

Nov 4, 2018: TCS New York City Marathon

Dr. Jeff Greenfield and Dr. Mark Souweidane will be joining the Children’s Brain Tumor Family Foundation on November 4 as they run 26.2 miles through NYC to benefit pediatric brain tumor research.  Sponsor a mile at greenfieldmarathon.org or souweidanemarathon.org.

 

October 19, 2018: Play it Forward Golf Tournament

The McKenna Claire Foundation will be hosting a golf tournament including a putting contest, longest drive, closest to the pin, hole in one and a ball drop after the tournament.  Read more.

 

October 16, 2018:  No Laughing Matter, a Night of Comedy to benefit the Children’s Brain Tumor Project

The Children’s Brain Tumor Family Foundation is proud to present this laugh-out-loud fundraising event at the world famous Carolines on Broadway.  Visit nolaughingmatter.org for sponsorship information and to purchase tickets.

 

 

October 14, 2018: Head for the Cure 5K to benefit CBTP

Join us for the Head for the Cure Metro NYC 5K Run/Walk as we raise funds, awareness and hope for the Children’s Brain Tumor Project! Read more.

 

Elizabeth's Hope

Sept 29, 2018: 6th Annual Round Robin for Cancer Research

Members of the Bronxville Field Club are invited to sign up for a morning of tennis and bridge to benefit Elizabeth’s Hope.  Members can register here.

 

Sept 5, 2018: Twitter Chat with @DrSouweidane – #KidsBrainTumors 

Join us in an engaging discussion on “High Grade Pediatric Brain Tumors – Research, Discovery & Roadblocks.  Visit CBTP.org for more information and to add to your calendar.

 

September, 2018: Childhood Cancer ACTION Month

September is childhood cancer awareness month, but raising awareness isn’t enough, we’re asking our supporters to take action.  Read more.

 

September, 2018: “Go Gold” with Team Campbell

Every September, Team Campbell sells lawn signs to help raise awareness.  Please contact us at info@teamcampbellfoundation.org for availability.

 

 

August 4, 2018: Muddy Puddles Mess Fest to Benefit Children’s Brain Tumor Project

A fun and “messy” celebration of kids being kids in honor of those who can’t.  For more information, visit their website for more information and to get tickets.

 

 

CBTP Published Research

OXFORD ACADEMIC NEURO-ONCOLOGY ADVANCES – MAY 2019

Combined Targeting of PI3K and MEK Effector Pathways via CED for DIPG Therapy

Raymond Chang, Umberto Tosi, Julia Voronina, Oluwaseyi Adeuyan, Y Linda Wu, Melanie E Schweitzer, David J Pisapia, Oren J Becher, Mark M Souweidane, Uday B Maachani.  Download full manuscript here.

 

ACS CHEMICAL NEUROSCIENCE – MAY 2019

Real-Time, in Vivo Correlation of Molecular Structure with Drug Distribution in the Brain Striatum Following Convection Enhanced Delivery.

Tosi U, Kommidi H, Bellat V, Marnell CS, Guo H, Adeuyan O, Schweitzer ME, Chen N, Su T, Zhang G, Maachani UB, Pisapia DJ, Law B, Souweidane MM, Ting R.ACS Chem Neurosci. 2019 May 15;10(5):2287-2298. doi: 10.1021/acschemneuro.8b00607. Epub 2019 Mar 6.
PMID:30838861

 

NATURE – JANUARY 2019

Tracking tumour evolution in glioma through liquid biopsies of cerebrospinal fluid.

Miller AM, Shah RH, Pentsova EI, Pourmaleki M, Briggs S, Distefano N, Zheng Y, Skakodub A, Mehta SA, Campos C, Hsieh WY, Selcuklu SD, Ling L, Meng F, Jing X, Samoila A, Bale TA, Tsui DWY, Grommes C, Viale A, Souweidane MM, Tabar V, Brennan CW, Reiner AS, Rosenblum M, Panageas KS, DeAngelis LM, Young RJ, Berger MF, Mellinghoff IK.
Nature. 2019 Jan;565(7741):654-658. doi: 10.1038/s41586-019-0882-3. Epub 2019 Jan 23.
PMID: 30675060

 

NATURE – JANUARY 2019

18F-Radiolabeled Panobinostat Allows for Positron Emission Tomography Guided Delivery of a Histone Deacetylase Inhibitor.

Kommidi H, Tosi U, Maachani UB, Guo H, Marnell CS, Law B, Souweidane MM, Ting R.ACS Med Chem Lett. 2018 Jan 17;9(2):114-119. doi: 10.1021/acsmedchemlett.7b00471. eCollection 2018 Feb 8.

Nature. 2019 Jan;565(7741):654-658. doi: 10.1038/s41586-019-0882-3. Epub 2019 Jan 23.

PMID:30675060 FREE DOWNLOAD

 

LANCET ONCOLOGY – AUGUST 2018

Convection-enhanced delivery for diffuse intrinsic pontine glioma: a single-centre, dose-escalation, phase 1 trial.

Souweidane MM, Kramer K, Pandit-Taskar N, Zhou Z, Haque S, Zanzonico P, Carrasquillo JA, Lyashchenko SK, Thakur SB, Donzelli M, Turner RS, Lewis JS, Cheung NV, Larson SM, Dunkel IJ.Lancet Oncol. 2018 Aug;19(8):1040-1050. doi: 10.1016/S1470-2045(18)30322-X. Epub 2018 Jun 18. Erratum in: Lancet Oncol. 2018 Aug;19(8):e382.
PMID: 29914796

 

JOURNAL OF NEUROSURGERY – JULY 2018

Volume of distribution and clearance of peptide-based nanofiber after convection-enhanced delivery.

Singh R, Bellat V, Wang M, Schweitzer ME, Wu YL, Tung CH, Souweidane MM, Law B.J Neurosurg. 2018 Jul;129(1):10-18. doi: 10.3171/2017.2.JNS162273. Epub 2017 Sep 8.
PMID:28885119

COLD SPRING HARBOR MOLECULAR CASE STUDIES – APRIL 2018

Precision oncogenomics in pediatrics: a personal reflection.

Campbell C, Greenfield JP.Cold Spring Harb Mol Case Stud. 2018 Apr 2;4(2). pii: a002865. doi: 10.1101/mcs.a002865. Print 2018 Apr. Review.
PMID: 29610396. Free PMC Article

 

JOURNAL OF NUCLEAR MEDICINE – MARCH 2018

PARP-1-Targeted Radiotherapy in Mouse Models of Glioblastoma.

Jannetti SA, Carlucci G, Carney B, Kossatz S, Shenker L, Carter LM, Salinas B, Brand C, Sadique A, Donabedian PL, Cunanan KM, Gönen M, Ponomarev V, Zeglis BM, Souweidane MM, Lewis JS, Weber WA, Humm JL, Reiner T.J Nucl Med. 2018 Aug;59(8):1225-1233. doi: 10.2967/jnumed.117.205054. Epub 2018 Mar 23.

 

JOURNAL OF NEUROONCOLOGY – MARCH 2018

Patterns of relapse for children with localized intracranial ependymoma.

De B, Khakoo Y, Souweidane MM, Dunkel IJ, Patel SH, Gilheeney SW, De Braganca KC, Karajannis MA, Wolden SL.J Neurooncol. 2018 Jun;138(2):435-445. doi: 10.1007/s11060-018-2815-7. Epub 2018 Mar 6.

 

PEDIATRIC BLOOD CANCER – JANUARY 2018

A phase II study of radioimmunotherapy with intraventricular 131 I-3F8 for medulloblastoma.

Kramer K, Pandit-Taskar N, Humm JL, Zanzonico PB, Haque S, Dunkel IJ, Wolden SL, Donzelli M, Goldman DA, Lewis JS, Lyashchenko SK, Khakoo Y, Carrasquillo JA, Souweidane MM, Greenfield JP, Lyden D, De Braganca KD, Gilheeney SW, Larson SM, Cheung NV.Pediatr Blood Cancer. 2018 Jan;65(1). doi: 10.1002/pbc.26754. Epub 2017 Sep 22.
PMID:28940863

 

PEDIATRIC BLOOD CANCER – SEPTEMBER 2017

A phase II study of radioimmunotherapy with intraventricular 131 I-3F8 for medulloblastoma.

Kramer K, Pandit-Taskar N, Humm JL, Zanzonico PB, Haque S, Dunkel IJ, Wolden SL, Donzelli M, Goldman DA, Lewis JS, Lyashchenko SK, Khakoo Y, Carrasquillo JA, Souweidane MM, Greenfield JP, Lyden D, De Braganca KD, Gilheeney SW, Larson SM, Cheung NV.Pediatr Blood Cancer. 2018 Jan;65(1). doi: 10.1002/pbc.26754. Epub 2017 Sep 22.
PMID: 28940863

 

PALLIATIVE SUPPORT CARE – SEPTEMBER 2017

Rapid autopsy of a patient with recurrent anaplastic ependymoma.

Lange KR, Fischer C, Rajappa P, Connors S, Pisapia D, Greenfield JP, Beltran H, Rubin M, Mosquera JM, Khakoo Y.Palliat Support Care. 2018 Apr;16(2):238-242. doi: 10.1017/S1478951517000864. Epub 2017 Sep 25.
PMID:  28942746 Free PMC Article

JCO PRECISION ONCOLOGY – JUNE 2017

Next-Generation Rapid Autopsies Enable Tumor Evolution Tracking and Generation of Preclinical Models.

Pisapia DJ, Salvatore S, Pauli C, Hissong E, Eng K, Prandi D, Sailer VW, Robinson BD, Park K, Cyrta J, Tagawa ST, Kossai M, Fontugne J, Kim R, Sigaras A, Rao R, Pancirer D, Faltas B, Bareja R, Molina AM, Nanus DM, Rajappa P, Souweidane MM, Greenfield J, Emde AK, Robine N, Elemento O, Sboner A, Demichelis F, Beltran H, Rubin MA, Mosquera JM.JCO Precis Oncol. 2017;2017. doi: 10.1200/PO.16.00038. Epub 2017 Jun 14.
PMID: 29333526 . Free PMC Article

 

Dr. Souweidane’s Trial Findings Published in Lancet Oncology

Dr. Souweidane’s Trial Findings Published in Lancet Oncology

On behalf of Dr. Mark Souweidane and the Children’s Brain Tumor Project, we are proud to announce that the Lancet Oncology has published the highly anticipated results of Dr. Mark Souweidane’s first-ever dose escalation study using convection-enhanced delivery (CED) in patients with diffuse intrinsic pontine glioma (DIPG). The milestone Phase I clinical trial, which investigated the safety of using CED to bypass the blood-brain barrier and administer a drug directly to a brain stem tumor site, resulted in no serious adverse effects and no dose-limiting toxicity; researchers conclude that the technique is safe to use in children.   

This publication validates a career-long hypothesis that Dr. Souweidane put forth more than 20 years ago, and was only made possible by the families who entrusted their children in his care by enrolling in the trial, and the donors who have provided the financial support to enable this research (read his inspiring blog here).

The results are so promising, they are expected to eventually change the landscape of future pediatric brain tumor protocols.   

View the article abstract here.

Read more here…

Dr. Mark Souweidane and patient, Brooke, who participated in the breakthrough Phase 1 clinical trial.