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.



Reference:

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

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
sharon.gardner@nyumc.org

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
info@burzynskiclinic.com

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.)

jwolff@mdanderson.org

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

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".
http://dipg.blogspot.com/2013/04/are-there-survivors.html
http://dipg.blogspot.com/2013/04/10-and-15-year-survivors.html

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

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.