DIPG/DIPT Discussion

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A searchable blog on DIPG research, DIPG news, recent publications, DIPG Foundations, DIPG researchers, clinical trials as well as other issues relating to Diffuse Intrinsic Pontine Tumors- both Diffuse Intrinsic Pontine Gliomas (DIPGs) and Atypical Pontine Lesions (APLs).

For parents, family and friends of children with DIPG looking for information and connection to others dealing with DIPG please check the buttons on the right hand side for resources.

Tuesday, July 13, 2010

French Present DIPG Biopsy Data at ISPNO

Back in the summer of 2005 the Institut Gustave Roussy started a study with upfront stereotactic biopsy in children with diffuse intrinsic pontine gliomas.


In the July 2007 edition of The Journal of Neurosurgery, the results neurosurgical results of 24 children who underwent biopsy were published. In this there was no mortality and transient morbidity in two patients.

At the 2010 International Society of Pediatric Neuro-Oncology in Vienna, the French group presented pathologic data regarding the upfront biopsy results with pediatric DIPG. There were 60 children who underwent biopsy and which there were 35 frozen sample in which they could undertake array comparative genomic hybridization or gene expression profiles. The median age of the children was 7.3 years and the overall survival was 10.5 months. Histopathologically, there were 10 grade II, 14 grade III, 10 grade IV and one that could not be graded.

Neither the MRI characteristics or the tumor grade were correlated with survival.

Two patterns were found by genetic analysis. One pattern showed enrichment for neural patterning genes (particularly regarding PDGFRa). The other had angiogenic and adhesion genes. The patterns did not relate to tumor grade. The first group had a worse prognosis with a majority of children in this group having overall survival less than the median survival.

The authors concluded that they have found two DIPG molecular subgroups ‘characterized by chromosomal regions and potentially therapeutic targets’.
These results seem to dovetail well with the previously reported post-mortem study in March in JCO from Sick Kids in Toronto.

Whole-genome profiling of pediatric diffuse intrinsic pontine gliomas highlights platelet-derived growth factor receptor alpha and poly (ADP-ribose) polymerase as potential therapeutic targets
J Clin Oncol. 2010 Mar 10;28(8):1337-44. Epub 2010 Feb 8.

Sunday, July 11, 2010

From "Brain Tomour" World Edition 2010. International Brain Tomour Alliance

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 24 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.

Tuesday, June 8, 2010

Compassionate Access Act Capital House Briefing

Abigail was 21 when she ran out of options for her cancer. Her oncologist urged the family to try to get access to an experimental drug targeted at EGFR called C225 (now called Erbitux). Contacting the pharmaceutical companies, congressional representatives, the media and other influential people was to no avail. Abigail died on June 9, 2001. Ten hours after the greatest tragedy in his life, Frank Burroughs, decided to dedicate his life to furthering the cause of expanded access. In November 2001, a foundation was started bearing his daughter’s name- Abigail’s Alliance for the Better Access to Developmental Drugs.

Now there is a House Bill (H.R. 4732) called The Compassionate Access Act of 2010 which seeks expanded access to drugs in development for patients with serious disease. A summary of the bill is at-

On Thursday June 10th there will be a Capital House Briefing on the Compassionate Access Act held in room 122 of the Cannon House Building. Abigail Alliance cofounder, Steven Walker, will be giving a Power Point presentation. Interested persons are invited to attend.

Those who can’t attend can still support this bill. Contact the Rep's office in DC. Ask for the health care aide. Request the aide to go to the Congressional Briefing on June 10 at 9 (122 House Cannon Building- Rep Diane Watson is sponsor and her office may know more details if they need) Ask for the Rep to consponsor the bill. To find your representative’s office

Wednesday, June 2, 2010

Journal Watch (continued)

“The lack of tumour tissue has led to stagnation in biological and genetic studies.”

The paper’s main focus was in understanding problems in overcoming the lack of specimens for analysis of DIPGs. The authors did identify that recent reports indicate “that biopsies in DIPG are safe and performed with acceptable low and only transient morbities while accomplishing meaningful analysis.” However, they also felt that using living tissue is ‘most defensible’ once there are therapeutic options in which the patient will benefit from in obtaining such samples.
The authors report on post-mortem tumor examination to try to get out of this catch 22 situation (one can only do a biopsy if there is benefit to the individual however one will not uncover those things that might be of benefit without tissue).
In regards to the 9 donations-
  • Eight of the children died at home.
  • Two children (age 10 and 11) expressed their own wish to donate.
  • No parent expressed regret in donation.
  • All families were offered disclosure of the autopsy result.
Although the numbers are very small, the paper is of significance in highlighting that such a program can be untaken— even with such logistical difficulties as serving an area of 1 million square kilometers with the children dying at home. Not only can such research happen but it can also uncover important biologic/genetic findings. For example, 2 of the 9 tumors were PNETs instead of gliomas.
It is interesting that the authors concluded with a statement indication for such a program to work there has to be cooperation of the primary neuro-ocnologists with the palliative care team, the pathology department and hospital management. This cooperation is critical in overcoming barriers and logistical issues.


J Neurooncol. 2010 May 16
Post mortem Examination in diffuse intrinsic pontine glioma: challenges and chances.
Angelini P, Hawkins, C, Laperriere N, Bouffet E, Bartels U.
Division of Paediatric Haematology and Oncology, The Hospital for Sick Children, Toronto, Canada

Thursday, May 27, 2010

Journal Watch

Sick Kids is becoming a powerhouse for diffuse intrinsic pontine glioma research.
This past February, Sick Kids hosted an International Think Tank with approximately 45 researchers, clinicians and advocates to specifically address issues regarding this tumor. In February, this same team published the first whole genomic profiling of DIPG tumors highlighting platelet derived growth factor receptor alpha as a potential target for therapy. In the May issue of the Journal of Neuro-oncology, the same team published their six year experience with post-mortem examination of diffuse intrinsic pontine gliomas.

Between July 2002 and June 2008, 27 children were diagnosed by a combination of clinical symptoms and radiographic appearance to have a diffuse intrinsic pontine glioma. The clinical symptoms included cranial nerve palsies, ataxia and long tract signs lasting less 6 months. The radiographic criteria included that the mass was at least 2/3 of the pons without an exophytic component.
Here are some of the statistics that were presented regarding these 27 children
  • There was an even gender split- 13 male and 14 female.
  • The median age at diagnosis was 7.1 years (range - 0 days to 16.9 years) .
  • Twenty- four children had radiation. The three that did not have radiation were of a very young age.
  • Sixty three percent of the children had a partial or complete clinical response to radiation.
  • The media time to progression at the end of radiation was 3.9 months (range – 0.8 to 13. 6 months).
  • The median time from diagnosis to death was 10.1 months (range – 0.3- 26.3 months).
Autopsy tumor donation was discussed with 21 of the families. Ten consented. Regarding the six families where this was not discussed- 4 children had transferred care to another provider and with two families the physicians felt that the family was too overwhelmed.

Due to logistical issues only nine of the ten consented donations were obtained. All specimens were adequate for nucleic acid. Interestingly, two of the nine samples were found to be primitive neurectodermal tumors (PNETs). The other seven were glioblastomas.


J Neurooncol. 2010 May 16
Post mortem Examination in diffuse intrinsic pontine glioma: challaneges and chances.
Angelini P, Hawkins, C, Laperriere N, Bouffet E, Bartels U.
Division of Paediatric Haematology and Oncology, The Hospital for Sick Children, Toronto, Canada

Saturday, March 20, 2010

Treatment Upon Progression

Trials for Children with Recurrent DIPG

The following information was gathered by Sue Rooney, mother of Quinn Rooney and JOMD Board member. Sue attempted to contact at least one institution for each open clinical trial to determine both whether a child with DIPG is eligible to participate in the study and to ensure the study is still open. If you find that any of this information is incorrect, please email info@JustOneMoreDay.org so that the list may be corrected. Further, if you are aware of an open trial that is not listed, please email us that information as well.

This list is provided for the sole purpose of helping individuals research. No assessment of the efficacy or advisability of any of the treatments listed has been made or is implied by inclusion in this list. Please note that the order listed was purely random! Please consult your own medical practitioners to determine appropriate medical treatment. Thank you!

1) A Phase I Study of MK-0752 in Pediatric Patients With Recurrent or Refractory CNS Malignancies

Please refer to this study by its ClinicalTrials.gov identifier: NCT00572182

2) ABT-888 and Temozolomide in Treating Young Patients With Recurrent or Refractory CNS Tumors

3) Study of Peptide Vaccination With Tumor Associated Antigens Mixed With Montanide in Patients With CNS Tumors
ClinicalTrials.gov Identifier: NCT00935545

4) An Open Label Dose Escalation Safety Study of Convection-Enhanced Delivery of IL13-PE38QQR in Patients With Progressive Pediatric Diffuse Infiltrating Brainstem Glioma and Supratentorial High-grade Glioma

5) AZD2171 in Treating Young Patients With Recurrent, Progressive, or Refractory Primary CNS Tumors
Mark Kieran, MD, PhD, Protocol chair Ph: 617-632-4907;Â 866-790-4500

(close to being full per Dr. Kieran)

6) Enzastaurin in Treating Young Patients With Refractory Primary CNS Tumors

(this will be closing soon per Dr. Blaney (2/15/10))

7) Antineoplaston Therapy in Treating Children With Brain Tumors

8) Topotecan in Treating Young Patients With Neoplastic Meningitis Due to Leukemia, Lymphoma, or Solid Tumors
(DIPG not technically excluded but may not qualify per Dr. Susan Blaney)

9) Karenitecin in Pediatric Patients With Refractory or Recurrent Solid Tumors N10010) (KTN10010)

Susan Blaney, MD 832-822-4586 smblaney@txccc.edu

10) Phase 2 Study of Nimotuzumab in Pediatric Recurrent Diffuse Intrinsic Pontine Glioma
ClinicalTrials.gov Identifier: NCT00600054

11) Ixabepilone in Treating Young Patients With Solid Tumors or Leukemia That Haven't Responded to Therapy

12) Valproate and Etoposide for Patients with Progressive, Relapsed or Refractory Neuronal tumors and Brain Metastases, Phase I clinical trial

Dr. Johannes Wolff 800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)


13) Phase I Study Of Single Agent Perifosine For Recurrent Pediatric Solid Tumors
For more information and to inquire about eligibility for this study, please contact

Dr. Oren Becher at 212-639-2152 or Dr. Ira Dunkel at 212-639-2153.

14) Sunitinib in Treating Young Patients With Refractory Solid Tumors

15) PF-02341066 in Treating Young Patients With Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma

16) Intracerebral Clysis in Treating Patients With Recurrent Primary Brain Tumors
Jeffrey Bruce, MD jnb2@columbia.edu (reference NCT 00452959)

17) A Trial of RAD001 and Avastin in Children With Recurrent Solid Tumors

18) PF-02341066 in Treating Young Patients With Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma

19) Perifosine With Temsirolimus for Recurrent Pediatric Solid Tumors
Please refer to this study by its ClinicalTrials.gov identifier: NCT01049841
Contact Dr. Oren Becher at 212-639-2152 or Dr. Ira Dunkel at 212-639-2153

20) Pazopanib Hydrochloride in Treating Young Patients With Solid Tumors That Have Not Responded to Treatment
(This trial is temporarily closed with an expected date of reopening in mid March 2010)

21) Trabectedin in Treating Young Patients With Solid Tumors That Have Relapsed or Not Responded to Treatment

22) Cixutumumab and Temsirolimus in Treating Young Patients With Solid Tumors That Have Recurred or Not Responded to Treatment

23) Doxercalciferol in Recurrent Pediatric Solid Tumors

24) Immunotherapy for Patients With Brain Stem Glioma and Glioblastoma
Email: Marites.Francisco@cshs.org

Wednesday, February 17, 2010

Finding Hope--Are There Survivors?

Update- since 2010 when this was published there have been several children who have passed away; however several children are still doing quite well.   Starting April 2013 there will be a series of posts on survivors.   You can also search the blog with the keyword "survivor".

Parents new to DIPG often ask, "Are there survivors?"

The children below--though treated very differently--have one thing in common; they have beaten the odds. Their stories provide hope for families who come after them.

Annabelle-dx 9/2005

Benny-dx 1/2008

Bridget-dx 8/2006

Clay-dx 12/2007

Connor-dx 9/2003

Cooper-dx 8/2007

Dasia-dx 3/2005

Dylan-dx 1/2004

Emily-dx 1/2007

Erica-dx 4/2005

JohnRobert-dx 4/2007

Kayla-dx 8/2006

Kayla-dx 8/2005

Laurel-dx 9/1999
Well 1/2010

Nikki-dx 1998

Nina Rose-dx 3/2006

Quinn-dx 5/2007
Well 2/2010

Ross-dx 10/2001
Well 1/2010

Sam-dx 9/2000
Well 1/2010

Tori-dx 8/1998

Sunday, February 7, 2010

Survey of DIPG Parents

We are reaching out to all parents of children who have been diagnosed with DIPG to seek information regarding each family's experience after the DIPG diagnosis.

We have been asked to gather information from families for consideration by clinicians and researchers who are focused on DIPG. To that end, we've prepared a survey which seeks information regarding individual experiences, thoughts and feelings, as well as suggestions for changes and improvements. The input of parents who have traveled the path will be invaluable help to those who, unfortunately, will certainly come behind us. (Both parents of a child who has been diagnosed with DIPG are encouraged to complete the survey -- each parent is certain to have a different perspective, even with regard to experiences with the same child.)

The survey results will be compiled in a report which will be shared at the end of February. The hope is that this will assist the medical community in better meeting the needs of children and families. (We also plan to make the information available to the DIPG community as a whole.)

We know the DIPG journey is difficult, no matter where you are on the path. Please accept our apologies if you are reading this at a particularly painful or difficult time. We would never want to hurt you or cause you additional pain, and you should feel no pressure or obligation to participate in this project.

Because we are on a short time frame, there is also a short time for completing this survey. Please note that the survey will close at midnight on February 14, 2010. We sincerely hope you will be able to find time during the next week to share your thoughts and experiences and we thank you for contemplating participating in this effort.

Please contact us at info@JustOneMoreDay.org if there is anything at all we can do to support you and your family.

Click here to begin the survey.

Again, thank you.

Saturday, January 9, 2010

Remembering 2009

We seize hope as we enter 2010 in the world of DIPG. There are new clinical trials on the horizon and exciting research is on the way. There seem to be more researchers and clinicians focused on DIPG and our children

In the meantime, we shall not forget the brave children who were taken from their families by DIPG in 2009. There are 66 names on this list, and we certainly are not aware of every child who died because of diffuse intrinsic pontine glioma during the last year.

With much hope that this number will be significantly lower in 2010, we honor and remember: