FDA to Discuss DIPG Biopsies

On April 27, 2009, the Committee will meet to discuss the scientific and ethical issues involved in obtaining and using brain biopsy specimens to evaluate gene expression patterns in children with diffuse pontine gliomas.

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
Joint Meeting of the Pediatric Advisory Committee and the Oncologic Drugs Advisory Committee
This meeting is open to the public.

Date and Time of Meeting:
April 27, 2009, from 8 a.m. to 6 p.m.

Date and Time of the Open Public Hearing:
April 27, 2009, between approximately 1 p.m. and 2 p.m.

Deadline to Request Speaking during Open Public Hearing:
On or before April 3, 2009 (see "How to Participate in the Open Public Hearing" below)

Address of Meeting:
Washington DC North/Gaithersburg Hilton,
620 Perry Pkwy.,
Gaithersburg, MD 20877.

Contact Person:
Carlos Pena
Office of the Commissioner (HF-33)
Food and Drug Administration,
5600 Fishers Lane (for express delivery, rm. 14B-08)
Rockville, MD 20857,
301-827-3340
or
e-mail: carlos.pena@fda.hhs.gov

Agenda:
On April 27, 2009, the Committee will meet to discuss the scientific and ethical issues involved in obtaining and using brain biopsy specimens to evaluate gene expression patterns in children with diffuse pontine gliomas.

FDA intends to make background material available to the public no later than 2 business days before the meeting. If FDA is unable to post the background material on its Web site prior to the meeting, the background material will be made publicly available at the location of the advisory committee meeting, and the background material will be posted on FDA's Web site after the meeting. Background material is available at
http://www.fda.gov/ohrms/dockets/ac/acmenu.htm, click on the year 2009 and scroll down to the appropriate advisory committee link.

How to Participate in the Open Public Hearing:
Oral presentations from the public will be scheduled between approximately 1 p.m. and 2 p.m. on April 27, 2009.

Interested persons may present data, information, or views, orally or in writing, on issues pending before the committee. Notify the contact person (listed above) on or before April 3, 2009, if you would like to make a formal oral presentations the day of the meeting. Submit a brief statement of the general nature of the evidence or arguments they wish to present, the names and addresses of proposed participants, and an indication of the approximate time requested to make their presentation. The contact person (listed above) will notify interested persons regarding their request to speak by April 6, 2009.

Time allotted for each presentation may be limited. If the number of registrants requesting to speak is greater than can be reasonably accommodated during the scheduled open public hearing session, FDA may conduct a lottery to determine the speakers for the scheduled open public hearing session.

Written submissions may be made to the contact person (listed above) on or before April 13, 2009.

Persons attending FDA's advisory committee meetings are advised that the agency is not responsible for providing access to electrical outlets. FDA welcomes the attendance of the public at its advisory committee meetings and will make every effort to accommodate persons with physical disabilities or special needs. If you require special accommodations due to a disability, please notify the contact person (listed above) at least 7 days in advance of the meeting.

FDA is committed to the orderly conduct of its advisory committee meetings. Please visit our Web site at http://www.fda.gov/oc/advisory/default.htm for procedures on public conduct during advisory committee meetings.

Avastin

On March 31, 2009 -- this Tuesday -- the FDA will hold an open public hearing to discuss approval of Avastin as a single agent in previously treated glioblastoma multiforme. The hearing will take place from 8:30 a.m. to 4:30 p.m. at the Hilton Washington DC/Silver Spring. Although this hearing is for an adult indication, we believe this matter deserves the attention of the brain tumor community at large. Moreover, we are seeing Aavastin being used with our DIPG kids more and more frequently.

There is some concern in the adult community that a negative review from the FDA will result in insurance companies refusing to pay for Avastin for any brain tumor patient.

Because this is an issue that may very well directly impact the DIPG community specifically, and most certainly will impact the entire brain tumor community, we delivered the following letter in support of Avastin for the Committee's consideration:


ODAC Committee Members:

We, the Board members of Just One More Day, are writing to urge the Committee to recommend approval of Avastin as a single agent in recurrent glioblastoma multiforme.
Just One More Day is a non-profit foundation formed by parents to help other parents whose children have been diagnosed with pediatric diffuse intrinsic pontine gliomas (“DIPG”), a cancerous tumor which invades the brainstem. DIPGs are almost always terminal, with most children dying within one year of diagnosis. In thirty years, and despite multiple different trials, no chemotherapy has been found to be effective against DIPG. With Avastin, however, there is hope.

Although DIPGs are generally considered inoperable, DIPG lesions were biopsied as part of a recent French study. The study revealed that the vast majority of DIPGs are high-grade gliomas, with many being glioblastoma multiforme.

In our network of parents we have seen many of our children with DIPGs stabilize with Avastin. In fact, we have recently been overjoyed by dramatic regression of the DIPG in Andrew, son of one of our Board members, after he received a single cycle of Avastin. Avastin has provided a flicker of hope in the dismal world of diffuse intrinsic pontine gliomas.

We realize that the question before the committee is whether to recommend approval of Avastin as a single agent in recurrent glioblastoma. Unfortunately, the call for written testimony did not indicate whether the question addresses both adults and children or only adults. Regardless, we believe it is important that our voice be heard on the matter. We are directly impacted by what is being done for adults with brain tumors, as treatments are usually not approved for children until they pass through the approval process for adults. Articles such as Antiangiogenic Therapy Using Bevacizumab in Recurrent High-Grade Glioma: Impact on Local Control and Patient Survival (J. Neurosurg. 2009 Jan; l 10(1): 173-80)) are encouraging and reflect what we are seeing with our children.

We ask the committee pause and reflect on the impact of this recommendation to the entire brain tumor community -- including our children. Previously there has been so little hope for children with DIPGs. Even a little more time with our children – even, as our name reflects, just one more day -- is incredibly precious to us. Please help the flame of this hope continue to burn and, we hope, to grow even brighter.

None of our board members have financial conflicts regarding the approval of Avastin.

Thank you for your time and consideration.



Background information is available at-
Meeting background http://www.fda.gov/ohrms/dockets/ac/cder09.html#OncologicDrugs
27 page document from Genetech http://www.fda.gov/ohrms/dockets/ac/09/briefing/2009-4427b1-01-FDA.pdf

May is Brain Tumor Awareness Month

Please join us for UmbrELLA of HOPE a family fun event in remembrance of Ella Hope Hauschildt and families fighting Diffused Intrinsic Pontine Glioma (DIPG).

On December 7, 2007 our lives changed forever, our daughter Ella Hope was diagnosed with a DIPG a rare and inoperable brain tumor. During Ella’s fight we tried conventional and alternative treatments, but to this day there is still no known treatment that is able to destroy this tumor.

Ella was born into heaven on November 21, 2008 after an 11 ½ month battle. On that day our focus changed from fighting for our little girl’s life, to fighting for brighter days for all children and families dealing with this dreadful tumor.

I ask that you join us in this fight on Saturday May 16, 2009 at the UmbrELLA of HOPE family fun event http://www.umbrellaofhope.com/ to remember Ella and all children fighting this disease and to support Just One More Day.org.

Just One More Day.org is committed to providing information and support for families affected by Diffused Intrinsic Pontine Glioma, promoting awareness and funding research for a cure. The event will include a family walk on the trails of Lake Front Park in Prior Lake, Minnesota. Other events include:

* Kids bounce houses
* Face painting
* Clowns
* Music entertainment
* Story telling
* Much more.

To register for the Family Fun Event please go to http://www.umbrellaofhope.com/ . We look forward to seeing you and we appreciate and applaud your willingness to walk with us in this battle!!!

Donations of coloring books, crayons, markers, colored pencils and craft items will also be collected for Riah’s Rainbow http://www.riahsrainbow.org/. Riah’s Rainbow was created by the Klien family in memory of little Mariah Klien who passed away from DIPG on August 31, 2008. Their goal is to bring smiles to the faces of children who have to endure a day, a week or even months in the hospital. All donations will be delivered to Fairview University Hospital. Please consider bringing a donation to bring smiles to kids who are battling cancer or chronic illnesses.

DIPG Digest

DIPG News brought to you by:
Just One More Day for Love, Hope & a Cure
http://www.justonemoreday.org/


Medical News
p16 promoter methylation in the serum as a basis for the molecular diagnosis of gliomas.: http://www.ncbi.nlm.nih.gov/pubmed/19240607?ordinalpos=5&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_DefaultReportPanel.Pubmed_RVDocSum

Interferon-beta, MCNU, and conventional radiotherapy for pediatric patients with brainstem glioma.: http://www.ncbi.nlm.nih.gov/pubmed/19260101?ordinalpos=3&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_DefaultReportPanel.Pubmed_RVDocSum

Targeting multiple kinases in glioblastoma multiforme.: http://www.ncbi.nlm.nih.gov/pubmed/19243279?ordinalpos=29&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_DefaultReportPanel.Pubmed_RVDocSum

National Childhood Brain Tumor Prevention Network Act of 2009

Advocacy Resource Center-
http://www.justonemoreday.org/Advocacy/Advocacy.html
http://tinyurl.com/b7zsku

Yahoo Group-
CBTPN · Childhood Brain Tumor Prevention Network: http://groups.yahoo.com/group/CBTPN/

State Resolutions to Support S. 305 and H.R. 653: http://www.justonemoreday.org/Advocacy/StateResolutions.html


FDA Open Public Hearing on Avastin

March 31, 2009
from 8:30 a.m. to 4:30 p.m. Hilton Washington DC/Silver SpringThe Ballrooms8727 Colesville Rd., Silver Spring, MD301-589-5200.

Information from the FDA: http://www.fda.gov/OHRMS/DOCKETS/98fr/oc20098-nm.pdf
Background information available March 29, 2009: http://www.fda.gov/ohrms/dockets/ac/09acdocs.html


Foundations News

Moss Report Newsletter provided by MJ's Children's Glioma Cancer Foundation: http://www.marcjr.org/FoundationMembers/MossReport/tabid/461/Default.aspx

The Cure Starts Now http://www.thecurestartsnow.org/

The Cure Starts Now is happy to welcome Jon and Anne Cinnelli and Marc and Emily Quayle to our strategic council. Both families know all to well the struggles we face in the fight against brain cancer and have made a major financial contribution to The Cure Starts Now and we look forward to their leadership contributions now. Their place on the strategic council will help guide us to the first 10-13 branches of CSN opening by years end. They will also help the existing strategic council decide on funding more research grants (our second funding cycle in 6 months). We are excited about the new partnerships and being able to broaden our operations. If you would like more information in joining these families in their combine fight against brain cancer, please contact me at brooke@thecurestartsnow.org.
Please join us on April 4th in Macon Georgia for the Imagine a Cure Music and Food Festival benefitting The Cure Starts Now. Organized by Patsy Mercer whose relative, Brayden Harrell, lost his battle with brain cancer. The event will be a wonderful tribute to him and other children who have and will battle brain cancer. Visit http://2009imagineacure.com/ for more details and we hope to see you there.


For past issues of DIPG Digest please visit http://www.justonemoreday.org/ParentPointers/DIPGDigest.html

Please submit information you feel will be helpful to DIPG families to JustOneMoreDay@cfl.rr.com subject: DIPG Digest

Copyright 2008 - 2009 Just One More Day for Love, Hope & a Cure, Inc. All rights reserved. The materials and links provided on this site have been prepared for information purposes only and should not be construed as advice or opinions on any specific facts or circumstances. Medical research concerning disease and treatments is an ongoing process. Readers should not act upon this information, but should obtain advice from physicians, medical institutions or other professionals, as appropriate.

DIPG Awareness Wristbands

DIPG Awareness Wristbands
100% pure silicone wristbands
They glow in the dark
$3.00 (including shipping)

To purchase please visit
http://www.justonemoreday.org/giftStore/JOMDMerchandise.html

http://justonemoreday.org/

The Georgian Senate Resolution

On March 5th, the State Senate of Georgia passed a resolution calling the President and the Congress of the United States to support and enact the “National Childhood Brain Tumor Prevention Act of 2009”. One of the key people in getting this passed was Marsi Thrash, one of our DIPG Foundations Alliance Members. Marsi urges others to become involved- even reworking the wording of the Georgia resolution for different home states (full text of the resolution below). Our DIPG families can be a leader for all pediatric brain tumors by advocating to pass the National Childhood Brain Tumor Prevention Act of 2009 (S. 305 and HR. 653). You can find out more about how to be involved from –
http://www.justonemoreday.org/Advocacy/Advocacy.html

A RESOLUTION

Urging the President and Congress of the United States to support legislative efforts to enact the "National Childhood Brain Tumor Prevention Network Act of 2009"; and for other purposes.

WHEREAS, tumors kill more children than any other disease, and brain tumors are the second most common type of cancer in children; and

WHEREAS, childhood brain tumors are the leading cause of death from solid tumors in children; and

WHEREAS, the World Health Organization has recognized and defined new types of brain tumors, many of which occur in children; and

WHEREAS, the causes of most childhood brain tumors are unknown, but it is clear that these tumors have substantial costs for the affected children, their families, and society as a whole and can result in death and the loss of many years of potential life; and

WHEREAS, the prognosis for most children suffering with brain tumors is dismal and survivors face lasting adverse health effects; and

WHEREAS, because of the relatively low overall incidence of childhood brain tumors, such tumors frequently do not receive sufficient attention and research funding; and

WHEREAS, no single institution has a sufficient number of patients to independently conduct research that will adequately address the causes of childhood brain tumors; and

WHEREAS, there has been no comprehensive study analyzing all relevant clinical, biological, and epidemiological aspects of childhood brain tumors to identify potential risk factors and determine the cause of such tumors; and WHEREAS, existing national cooperative clinical oncology groups primarily investigate treatment options and prognosis and do not typically examine the origins of childhood brain tumors or the risk factors associated with such tumors; and

WHEREAS, a significant majority of children with brain tumors are first treated by neurosurgeons and not by the oncologists typically involved in such groups.

NOW, THEREFORE, BE IT RESOLVED BY THE GENERAL ASSEMBLY OF GEORGIA that this body does hereby urge the Congress of the United States to enact and the President to sign H.R. 653 and S. 305, creating the National Childhood Brain Tumor Prevention Network.

BE IT FURTHER RESOLVED that the Secretary of the Senate is authorized and directed to transmit an appropriate copy of this resolution to the Honorable Barack Obama, President of the United States, and each member of the Georgia congressional delegation.

A simple way to change the future

I personally know of 135 children who have passed away because of a DIPG since my daughter Alicia was diagnosed with this terrible cancer in 2005. After their children were diagnosed, these 135 families had about 12 months to accomplish a lifetime of dreams with their children. They will never see their child go on their first date, drive a car, graduate from high school, get married or have their own child.

Diffuse intrinsic pontine glioma is one of the worst brain cancers, with no real effective treatment and no cure. Some doctors believe finding a cure for DIPGs may very well lead to a cure for all brain tumors. We desperately need to fund research. We need to change the future for our children and grandchildren.

Thank you for supporting Just One More Day and helping us raise awareness for DIPGs. Now I'm asking you to take it a step further; I'm asking you to visit http://www.firstgiving.com/21687 and develop your personal page to benefit Just One More Day for Love, Hope and a Cure.

If 100 of our wonderful supporters donate just $5.00, we would raise $500.00. But, if each of us sets up FirstGiving page and 50 of our friends donate $5.00, the amount we raise increases to $25,000.00. With this money we could begin funding a research protocol - the first step to finding a cure.

Starting a page at FirstGiving.com is quick and simple. After your page is ready, you can e-mail it to friends, family and colleagues. You will continue to raise awareness about this rare disease and will also allow those who wish to help to donate by credit or debit card in an easy, secure, online transaction. Please take a moment and look at my daughter's FirstGiving page at http://www.firstgiving.com/jomdashley and consider her plea for an answer to the question that changed her life.

Together we can make a difference. Together we can find a cure.

Thank you for your time and support.
With Hope,
Christine Martin
Angel Alicia's Mom
http://www.firstgiving.com/jomdchristine