Position Statements
Meeting with Celtic Pharma to discuss Xerecept
All, or nearly all, children diagnosed with diffuse intrinsic pontine glioma (DIPG) are given high doses of dexamethasone to combat brain edema. The side effects of dexamethasone are often horrific and range from immobilizing weight gain, to insatiable hunger (during a time when the disease causes paralysis of the esophagus), to thinning and splitting of the skin. (Dexamethasone also inhibits the body’s ability to heal itself, exacerbating this problem.) These and other side effects severely limit the ability of children to fight the battle against this devastating disease and to enjoy their young lives. It is the position of Just One More Day for Love, Hope & a Cure that Xerecept should be made available to children with DIPG, both via clinical trials and compassionate use. It is inhumane to deny this resource to these suffering children and their families.
FDA Discussion of DIPG Biopsies
On April 27, 2009, the Committee met 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.
It is the position of Just One More Day for Love, Hope & a Cure that every parent of a child who is diagnosed with a diffuse pontine glioma should be provided with full and complete information regarding biopsy of the tumor, including both the pros and cons of such a procedure. Further, every parent should be advised that there are respected neuro-surgeons who are experienced and willing to perform biopsies when the parents and the surgeon agree that doing so is advisable.
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.
Last update July 30, 2009