One of the huge problems with chemotherapy is that it beats up normal cells- especially bone marrow. The toxicity limits how far the doctors can increase dosages before it is just too much for the body. One novel idea is to try to make the normal bone marrow cells more resistant to chemotherapy. This is exactly what researchers are trying to do with a gene therapy which effects MGMT. This will allow for increasing doses of temozolomide.
Dr Geoffrey McCowage at The Children's Hospital of Westmead in Sydney Australia has spent the last 15 years developing a trial which removes bone marrow stem cells, genetically modifies the NA repair protein and then reinserts them into the patient. The hope is that this modification will allow a patient to withstand escalating chemotherapy while better targeting the tumor. In fact, this is more than theory. This first of kind therapy for brain tumor kids is open in trial.
This Phase 1 intervention is open to several different types of brain tumors including brainstem glioma of diffuse pontine type and all the following recurrent tumors- medulloblastoma, ependymoma, atypical teratoid rhaboid, high and low grade glioma.
Erin is the first child enrolled. You can read at-
Last month she had reached a year from diagnosis- and in the picture she looks great.
The trial is being funded by the Kid's Cancer Project (formerly Oncology Children's Foundation), Sporting Chance Cancer Foundation and the Australian governmental funding agency, The Department of Innovation, Industry Science and Research.
Erin is hoping to raise $50,000 for The Cure Starts Now for DIPG research.
Note- A similar trial seems to be open in the US at the NIH for adult glioblastoma patients.
Brave Erin's Inspirational Battle Against Cancer- http://www.news.com.au/national-news/south-australia/brave-erins-inspirational-battle-against-cancer/story-fndo4dzn-122658419720
MGMT Cancer Gene Therapy Project- http://www.thekidscancerproject.org.au/Research/Gene-Therapy-Trial.aspx
Cancer Gene Therapy Project (Westmead Research site)-
Australian New Zealand Clinical Trial Registry (ANZCTR)-
NIH Adult Trial with Newly Diagnosed GBM- http://www.clinicaltrials.gov/ct2/show/NCT01269424