One $five-million-project is titled "Outliers in Glioblastoma Outcome: Moving the curve forward." This five-year investigation seeks to discover why approximately two percent of glioblastoma multiforme (GBM) patients the outliers live far beyond the average survival time of 18 months. GBM is the most common and aggressive form of malignant primary brain tumor; 98 percent of people diagnosed with GBM live less than 18 months.
"A major challenge with brain cancer is that people survive such a short time," said Catherine (Bracken) Ivy, founder and president of The Ben & Catherine Ivy Foundation. "If this research enables patients to live longer, clinicians and researchers will gain a better understanding of how this disease works, which will bring us time to move closer to a cure."
"The tireless and dedicated support of programs like the Ivy Foundation is helping transform ideas into medical reality," said TGen President and Research Director Dr. Jeffrey Trent.
By precisely identifying the billions of molecular building blocks in each patient's DNA through whole genome sequencing, TGen researchers hope to discover the genetic differences between those patients who survive only a few months, and those who survive longer because their brain cancer develops more slowly.
Using these genetic targets, TGen researchers will identify those patients most likely to benefit from the current standard of care, and those who might best benefit from alternative or new experimental treatments.
"If we can identify patients who will likely only survive a few months on current standard of care regimens we can then prioritize those patients for personalized clinical trials," said Dr. David Craig, TGen's deputy director of bioinformatics and one of the project's principal investigators.
First-in-Patient Clinical Trial Studies
In the second $five million project, "Genomics Enabled Medicine in Glioblastoma Trial," TGen and its clinical partners will lead first-in-patient clinical trial studies that will test promising new drugs that might extend the survival of GBM patients.
This multi-part study will take place in clinics across the country and TGen laboratories.
This project begins with a pilot study of 15 patients, using whole genome sequencing to study their tumor samples to help physicians determine what drugs might be most beneficial.
To support molecularly informed clinical decisions, TGen labs also will examine genomic data from at least 536 past cases of glioblastoma, as well as tumor samples from new cases, developing tools that will produce more insight into how glioblastoma tumors grow and survive. TGen also will conduct a series of pioneering lab tests to measure cell-by-cell responses to various drugs.
"We expect to identify genes that play a crucial role in this cancer's survival and that may be crucial to the survival of other types of cancer as well," said Dr. Michael Bittner, co-director of TGen's Computational Biology Division.
To get new treatments to patients as quickly as possible, this five-year study will include a feasibility study involving up to 30 patients, followed by Phase II clinical trials with as many as 70 patients. TGen intends to team with the Ivy Early Phase Clinical Trials Consortium that includes: University of California, San Francisco; University of California, Los Angeles; the MD Anderson Cancer Center; Memorial Sloan Kettering Cancer Center; University of Utah; and the Dana-Farber/Harvard Cancer Center.
The results of these clinical trials should not only help the patients who join them, but also provide the data needed for FDA approval and availability of new drugs that could benefit tens of thousands of brain cancer patients in the future.
"Working with physicians, the project will aim to get new drugs to patients faster, deliver combinations of drugs that might be more effective than using a single drug, quickly identify which therapies don't work, and accelerate discovery of ones that might prove promising for future development," said Dr. John Carpten, TGen's deputy director of basic science, director of TGen's Integrated Cancer Genomics Division, and another of the project's principal investigators.
In addition to helping patients as quickly as possible, the projects should significantly expand Arizona's network of brain cancer experts.
"It's a tremendous opportunity to find more solutions for the patient diagnosed with brain cancer," said Ivy, who also is working to establish additional clinical trials in the Phoenix area, giving local patients more treatment options. "The clinical trials are very exciting because they can impact the patient today."
Ivy's Commitment to Brain Cancer Research
Ivy has investigated research institutions nationally and internationally, learning everything she can about how to cure brain cancer. She is determined to help find better treatment options and improve the quality of life for patients with brain tumors.
Because brain cancer is rare compared to many other cancers, it does not receive as much media attention, government funding or pharmaceutical industry support. Standard treatment involves removal of the tumor, though surgery fails to get all of the cancer. Surgery is followed by radiation and chemotherapy using a drug with limited effect for most patients. There currently is little else that can be done to extend life expectancy.
The status quo is not acceptable to Ivy. More than anything, she said, the Ivy Foundation wants to instill hope and solutions for people diagnosed with brain cancer.
The Ivy Foundation's overarching goal over the next seven years is to double the life expectancy of brain cancer patients from 18 to 36 months. And in working with TGen, Ivy said she has found three key values that align both organizations:
- Patient-focused research.
- Conducting the best research possible in a cost-effective manner.
- Making progress immediately.
"I hope that people with brain cancer and brain tumors know that there is a community of people working very hard to try and help them," Ivy said. "We're not saying we're going to cure it tomorrow, but at least we're moving the needle. We will never give up until we find a cure.''
Provided by The Translational Genomics Research Institute
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