The DIPG Collaborative "is an association of foundations unified with the mission of efficiently funding and inspiring DIPG research in hopes of finding a wider cure for cancer". From The Cure Starts now website it says that the Collaborative Council is to:
1) provide the tools and information to families whose children face the difficult diagnosis of pediatric brain cancer.
2) provide a warm and caring envirnoment to share information, discuss options and support one another.
3) make available accurate and easy to understand information for families that are newly diagnosed.
A major kick off of the DIPG Collaborative was the 2011symposium that brought 13 foundations together with researcher to find substantive ways to research DIPG jointly together. From that meeting several research infrastructure developments occurred which now really allows us to start to unravel DIPG. These included the DIPG Preclinical Consortium, the DIPG Resgistry and the DIPG Genomics Repository.
If one hasn't looked at the Preclinical Consortium, I think it is worth the time as it is impressive. It is less than 4 years from when the first cell line was promulgated in Michelle Monje's lab (from tissue donated by Dylan Jewett). Through the consoritum we now have ten institutions working together on more than 30 cell lines (as well as various animal models) trying to find the best single agents or combinations in those cell lines to be a basis for rapid translation into clinical trials. Every week there is an update on the consortiums work including DNA and RNA analysis. This is fully funded byt the Foundational Leadership Partnters of the DIPG Colaborative.
The DIPG Registry is equally impressive. There is a section for medical professionals including sections on enrollment information and consultations. There is also a section for parents where one can make contact for second opinions and also search for open clinical trials. Parents can make contact with the registry to discuss enrollment- even for children who have died. There already is some specific research in the works.
The DIPG Collaborative Symposium starts today, however, the major work of the DIPG Collaborative for foundations starts tomorrow. The first session is on objectives for the next two years. It would be really interesting to see the direction this collaborative is moving.
There are three levels of support:
Foundational Leadership Partners - The Cure Starts Now, The Cure Starts Now Australia, Jeffrey Thomas Hayden Foundation, Hope for Caroline, Soar with Grace, Reflections of Grace Foundation, The Lyla Nsouli Foundation
Foundation Partners- Smiles for Sophie Forever, Benny World, The Team Julian Foundation
Symposium Sponsors- Max Lacewell Foundation, Love Chloe Foundation, Caroline's Miracle Foundation, Ellie DIPG Research Fund, American Childhood Cancer Organization, Pray Hope Believe Foundation, National Brain Tumor Society, Lurie Children's Hospital of Chicago, Cincinnati Children's Hospital
References:
CSN DIPG Collaborative Council
http://www.thecurestartsnow.org/about/foundation/councils/DIPGcollaborative/
DIPG Collaborative
Showing posts with label DIPG registry. Show all posts
Showing posts with label DIPG registry. Show all posts
Friday, May 3, 2013
Thursday, May 2, 2013
DIPG Collaborative Symposium Begins in Cincinnati
Tomorrow the 2nd DIPG Collaborative Symposium begins in Cincinnati. This two track program brings together doctors and researchers in one track and foundations and parents in another (with a potential networking dinner Friday evening). The goal is to be able to "efficiently fund and inspire diffuse intrinsic pontine glioma cancer research in hopes of finding a wider cure for cancer". This year there is expected to be approximately 140 from 7 countries. With the diverse agenda with international speakers on the forefront of DIPG research, I suspect it will be an exciting place to be for those interested in pediatric diffuse intrinsic pontine glioma.
The 2011 DIPG Collaborative Symposium brought about some substantive advances in the infrastructure critical for DIPG research including the DIPG Registry, the DIPG Preclinical Consortium, and the DIPG Genomics Repository.
This meeting starts Friday morning with a Keynote Lecture from Canadian geneticist and researcher, Nada Jaboda MD PhD. Although the topic has not been release I am thinking- H3.3,K27M and epigenetics. To get a taste of the work she has been doing in unravelling pediatric vs adult astrocytomas (including DIPG) there is a NYU Grand Rounds titled "Rewiring the Epigenome in Pediatric and Young High Grade Astrocytomas" presented on December 18, 2012. (click here to watch the video) This work has the potential to change how we approach and treat DIPGs.
The meeting continues with a update of research funded through the collaborative and the Cure Starts Now:
Preclinica/Traslantional
* Xiao-Nan Li(Baylor)- an oncolytic picorna virus in a DIPG xenograft mouse model.
* Oren Becher (Duke)- systemic and direct delivery of a PDFGR-alpha antibody.
* Suzanne Baker (St Jude)- establishment and characterization of DIPG renewable tissue resources
* Patricia Baxter (Texas)- Novel BMI-1 inhibitors in brainstem xenograft mouse models.
* Michele Monje (Stanford)- combined targeted therapy for cellular subpopulations in DIPG.
Clinical
Mark Souweidane (Cornell)- CED trial update
multiple- DIPG Registry update
This will be followed by various other sessions especially revolving around biopsy, biologic understanding of DIPG and current trends in clinical trials. One of the most anticipated things for me is the Xerecept session on Friday afternoon. It has been years of waiting for this drug to see if it can decease the suffering caused by steroids in so many DIPG children (click here for video). Hopefully something substantive will come from this session.
The other track- the parents and foundation track- will meet primarily on Saturday to discuss the Collaborative objectives, structure, prior efforts and success.
The event will culminate with a Once in a Lifetime Gala which looks to be spectacular (sold out- 1200 seat). This year's three ring circus them features the Cincinnati Circus and Nik Walenda.
Reference:
DIPG Collaborative Symposium Meeting Agenda
https://csn.donordrive.com/assets/csn/files/$cms$/100/2097.pdf
DIPG Preclinical Consortium-
http://pptiohsu.blogspot.com/2011/12/open-science-forum-dipg-preclinical.html
DIPG Registry-
http://www.dipgregistry.org/
The 2011 DIPG Collaborative Symposium brought about some substantive advances in the infrastructure critical for DIPG research including the DIPG Registry, the DIPG Preclinical Consortium, and the DIPG Genomics Repository.
This meeting starts Friday morning with a Keynote Lecture from Canadian geneticist and researcher, Nada Jaboda MD PhD. Although the topic has not been release I am thinking- H3.3,K27M and epigenetics. To get a taste of the work she has been doing in unravelling pediatric vs adult astrocytomas (including DIPG) there is a NYU Grand Rounds titled "Rewiring the Epigenome in Pediatric and Young High Grade Astrocytomas" presented on December 18, 2012. (click here to watch the video) This work has the potential to change how we approach and treat DIPGs.
The meeting continues with a update of research funded through the collaborative and the Cure Starts Now:
Preclinica/Traslantional
* Xiao-Nan Li(Baylor)- an oncolytic picorna virus in a DIPG xenograft mouse model.
* Oren Becher (Duke)- systemic and direct delivery of a PDFGR-alpha antibody.
* Suzanne Baker (St Jude)- establishment and characterization of DIPG renewable tissue resources
* Patricia Baxter (Texas)- Novel BMI-1 inhibitors in brainstem xenograft mouse models.
* Michele Monje (Stanford)- combined targeted therapy for cellular subpopulations in DIPG.
Clinical
Mark Souweidane (Cornell)- CED trial update
multiple- DIPG Registry update
This will be followed by various other sessions especially revolving around biopsy, biologic understanding of DIPG and current trends in clinical trials. One of the most anticipated things for me is the Xerecept session on Friday afternoon. It has been years of waiting for this drug to see if it can decease the suffering caused by steroids in so many DIPG children (click here for video). Hopefully something substantive will come from this session.
The other track- the parents and foundation track- will meet primarily on Saturday to discuss the Collaborative objectives, structure, prior efforts and success.
The event will culminate with a Once in a Lifetime Gala which looks to be spectacular (sold out- 1200 seat). This year's three ring circus them features the Cincinnati Circus and Nik Walenda.
Reference:
DIPG Collaborative Symposium Meeting Agenda
https://csn.donordrive.com/assets/csn/files/$cms$/100/2097.pdf
DIPG Preclinical Consortium-
http://pptiohsu.blogspot.com/2011/12/open-science-forum-dipg-preclinical.html
DIPG Registry-
http://www.dipgregistry.org/
Friday, April 12, 2013
St Jude Looks at Long Term DIPG Survivors
At a poster session for the 3rd biennial Pediatric Neuro-Oncology Basic and Translational Research Conference next month, physicians from St Jude Research Hospital will be presenting a retrospective review of their patients who had prolonged survival despite being given a diagnosis of diffuse intrinsic pontine glioma.
These physicians reviewed the records of DIPG patients at St Jude from October 1, 192 to May 31, 2011. Of the 191 patients there were 5 long time survivors with a median time from diagnosis being 9.3 years! It seems 4 out of 5 of these patients had some atypical clinical or imaging features that put them on the side of potentially better outcomes than the average.
All these survivors underwent neurocognitive testing. Interestingly, and unfortunately like so many other children who undergo brain radiation, cognitive function was impacted in 4 of 5 patients- two falling in the range of borderline/mild mental retardation. These findings will become increasingly important when there is better survival statistics for DIPG.
The researcher point out that prolonged survival does not equal cure. Two of the patients had progression years after their initial treatment with radiation.
It will be interesting to see what comes of this. Certainly the DIPG Registy could become very important to bring the information on all long term survivors together at one place.
Reference:
Clinico-radiologic characteristics and neurocognitive assessment of long-term survivors of diffuse intrinsic pontine glioma
https://soc-neuro-onc.conference-services.net/reports/template/onetextabstract.xml?xsl=template/onetextabstract.xsl&conferenceID=3467&abstractID=738281
These physicians reviewed the records of DIPG patients at St Jude from October 1, 192 to May 31, 2011. Of the 191 patients there were 5 long time survivors with a median time from diagnosis being 9.3 years! It seems 4 out of 5 of these patients had some atypical clinical or imaging features that put them on the side of potentially better outcomes than the average.
All these survivors underwent neurocognitive testing. Interestingly, and unfortunately like so many other children who undergo brain radiation, cognitive function was impacted in 4 of 5 patients- two falling in the range of borderline/mild mental retardation. These findings will become increasingly important when there is better survival statistics for DIPG.
The researcher point out that prolonged survival does not equal cure. Two of the patients had progression years after their initial treatment with radiation.
It will be interesting to see what comes of this. Certainly the DIPG Registy could become very important to bring the information on all long term survivors together at one place.
Reference:
Clinico-radiologic characteristics and neurocognitive assessment of long-term survivors of diffuse intrinsic pontine glioma
https://soc-neuro-onc.conference-services.net/reports/template/onetextabstract.xml?xsl=template/onetextabstract.xsl&conferenceID=3467&abstractID=738281
Friday, April 5, 2013
10 and 15 year Survivors
Meet Connor and Tori.
Their names have been around the internet for some time. I absolutely believe that their parents were told the awful prognosis of DIPG at diagnosis and although I have never seen the MRIs I don't doubt that the lesion in these children's brains were diffuse and intrinsic and pontine.
Connor was just 5 weeks old when he was diagnosed with a DIBSG (diffuse intrinsic branstem glioma). The parents knew something wasn't right about a week earlier when he wasn't latching on and seemed exhausted. A seizure sent him to Cincinnati Children's ER. During the admission for failure to thrive a golfball size tumor was found in his brainstem. At that young age there were no therapeutic options. He was sent home with a feeding tube to die. In those first two years he had spectroscopy scans which showed high levels of choline and other cancerous indications but it didn't change. As far as I can tell his last MRI still had residual tumor at age 2. In September, this happy healthy boy will have his 10 year anniversary from diagnosis. His mom has a caringbridge site to give hope to other DIPG parents.
Tori seems like a regular teenage girl. However, not long after she was born that thought that we would ever know seemed beyond impossible. Tori was born in June 1998 and a few weeks later was found to have some typical DIPG symptoms. Her eyes had trouble tracking and the side of her face seemed stiff. AN MRI showed a brainstem glioma (that is what they all were called at that time). The doctors told her parents she would die. With no conventional options, they searched for alternatives. This year she wrote her own update. This fall will be 15 years since diagnosis for her!
One can ask a million times but no one knows why these children have survived. What we do know is that there have been other cases of spontaneous regression of diffuse pontine lesions in neonates. It has been reported in the literature before.
A case from Germany is remarkable similar to the two above. A 7 week old infant was found to have an MRI "strongly suggestive of diffuse pontine glioma". By 27 months the lesion was gone.
A slightly different situation (also from Germany) is the first reported case of spontaneous remission that I am aware of. A 2-year-old boy was found to have a large, diffuse pontine tumor extending into the midbrain. A biopsy showed a fibrillary astrocytoma. He received a shunt but no other conventional therapy. By the age of 6.6 years the tumor was no longer visible on imaging.
In 2005, two US neurosurgeons reported two cases of spontaneous regression of diffuse brainstem lesions in neonates. These two newborns were found to have limb weakness and cranial nerve abnormalies. MRIs in the first week after birth revealed a large pontomedullary tumor. Neither child received conventional treatment. The authors related that the children were normal at age 4 and 10.
A followup letter to the editor to the above case report came from Texas Children's relating a similar story although the child presented at 11 weeks of age. At the time of publication she was 12.
These authors stressed that nearly all patients with diffuse brainstem lesions do poorly- including neonates. In 2008 Sick Kids published a case report on two infants with "large, expansive, non-enhancing, intrinsic pontine masses diagnosed as DIPGs. They were treated only with supportive care and died by 2 weeks of age. One of the children underwent autopsy and was found to have a brainstem PNET.
We have no idea how often this happens. This is why the DIPG Registry is critical in understanding this disease. Both families and physicians can enroll patients including children who have died.
The bottom line is: 1) we really don't understand DIPGs, and 2) there are children who have been diagnosed with DIPGs-based on the incomplete knowledge that we currently have- who truly have had spontaneous regression of their tumors and 3) most of the children with report spontaneous regression have been very young.
Their names have been around the internet for some time. I absolutely believe that their parents were told the awful prognosis of DIPG at diagnosis and although I have never seen the MRIs I don't doubt that the lesion in these children's brains were diffuse and intrinsic and pontine.
Connor was just 5 weeks old when he was diagnosed with a DIBSG (diffuse intrinsic branstem glioma). The parents knew something wasn't right about a week earlier when he wasn't latching on and seemed exhausted. A seizure sent him to Cincinnati Children's ER. During the admission for failure to thrive a golfball size tumor was found in his brainstem. At that young age there were no therapeutic options. He was sent home with a feeding tube to die. In those first two years he had spectroscopy scans which showed high levels of choline and other cancerous indications but it didn't change. As far as I can tell his last MRI still had residual tumor at age 2. In September, this happy healthy boy will have his 10 year anniversary from diagnosis. His mom has a caringbridge site to give hope to other DIPG parents.
Tori seems like a regular teenage girl. However, not long after she was born that thought that we would ever know seemed beyond impossible. Tori was born in June 1998 and a few weeks later was found to have some typical DIPG symptoms. Her eyes had trouble tracking and the side of her face seemed stiff. AN MRI showed a brainstem glioma (that is what they all were called at that time). The doctors told her parents she would die. With no conventional options, they searched for alternatives. This year she wrote her own update. This fall will be 15 years since diagnosis for her!
One can ask a million times but no one knows why these children have survived. What we do know is that there have been other cases of spontaneous regression of diffuse pontine lesions in neonates. It has been reported in the literature before.
A case from Germany is remarkable similar to the two above. A 7 week old infant was found to have an MRI "strongly suggestive of diffuse pontine glioma". By 27 months the lesion was gone.
A slightly different situation (also from Germany) is the first reported case of spontaneous remission that I am aware of. A 2-year-old boy was found to have a large, diffuse pontine tumor extending into the midbrain. A biopsy showed a fibrillary astrocytoma. He received a shunt but no other conventional therapy. By the age of 6.6 years the tumor was no longer visible on imaging.
In 2005, two US neurosurgeons reported two cases of spontaneous regression of diffuse brainstem lesions in neonates. These two newborns were found to have limb weakness and cranial nerve abnormalies. MRIs in the first week after birth revealed a large pontomedullary tumor. Neither child received conventional treatment. The authors related that the children were normal at age 4 and 10.
A followup letter to the editor to the above case report came from Texas Children's relating a similar story although the child presented at 11 weeks of age. At the time of publication she was 12.
These authors stressed that nearly all patients with diffuse brainstem lesions do poorly- including neonates. In 2008 Sick Kids published a case report on two infants with "large, expansive, non-enhancing, intrinsic pontine masses diagnosed as DIPGs. They were treated only with supportive care and died by 2 weeks of age. One of the children underwent autopsy and was found to have a brainstem PNET.
We have no idea how often this happens. This is why the DIPG Registry is critical in understanding this disease. Both families and physicians can enroll patients including children who have died.
The bottom line is: 1) we really don't understand DIPGs, and 2) there are children who have been diagnosed with DIPGs-based on the incomplete knowledge that we currently have- who truly have had spontaneous regression of their tumors and 3) most of the children with report spontaneous regression have been very young.
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