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The main goal of the Precision Medicine Initiative for brain cancer is to use precision science – and the genomic information from each patient’s brain tumor – to develop new personalized cancer treatments. Glioblastoma Multiforme or GBM is the most common type of primary brain cancer and has a very poor prognosis.

While no two tumors are alike, all GBMs are characterized by aggressive growth, invasiveness and resistance to standard therapy.  The current treatment for GBM includes surgical removal, radiation therapy and chemotherapy. However, the vast majority of patients fail the initial therapy within 18 months of diagnosis and second line treatment options are limited. Today all patients with GBM are treated with the same anti-cancer drug. This does not make sense because each patient’s tumor is unique.

A tumor from one patient can have one set of genetic abnormalities that are very different from another patient’s tumor. The personalization of cancer treatments means that one size does not fit all. Treatment personalization in brain cancer has been limited to a “one mutation: one drug” strategy. This simplistic approach ignores the complexity of the inner machinery of a cancer cell. Precision Medicine has been showcased front and center as the future of cancer care by President Obama and other politicians. However, there are not many examples out there of how precision science is being developed to address a specific challenging medical condition.

The Precision Medicine initiative for brain cancer does exactly that. The goal of the initiative is to find personalized cancer treatments powered by a precision science methodology. Although we are talking about brain cancer, it is important to note that we are developing a Precision Medicine, scientific methodology that can be used to tackle ANY cancer. This is what we are doing:

 

Let’s say a person develops severe headaches and an MRI shows a brain tumor. We typically then perform a biopsy and if it’s safe, we surgically remove the tumor. Here is an example of a precision neurosurgery being performed through a minimally invasive NICO brain path tube. Part of the surgical specimen is then sent to the pathologist to confirm the diagnosis of glioblastoma and the rest of the specimen is sent to our research laboratory.

Under the auspices of an Institutional Research Board approved protocol, and after informed consent, we take the research tumor sample and split it into 2 pieces.

One piece goes to our lab where we grow the patient’s tumor in culture. The other piece of the brain tumor undergoes next-generation genomic sequencing to establish a comprehensive mutational profile of that patient’s specific cancer. Then in collaboration with our industry partner, Cellworks, we use this mutational profile to build a virtual, mathematical In-SILICO tumor model.

The simulation predictions are then compared to the real patient tumor cells that have been growing in our laboratory. We take the “real data” from experiments in our lab and optimize and validate the virtual tumor model.

We then run an experiment where all FDA approved targeted drug combinations are tried on the virtual tumor cell to identify the optimal drug combination that maximizes the cell kill for that specific brain tumor. This is made possible by leveraging a database of all approved targeted drugs, including non-cancer drugs.

In the last step we use the patient’s cancer cells and expose them to this unique and personalized drug combination to ensure that it effectively kills the patient’s cancer cells.

We believe that this innovative precision science methodology holds the key for getting us a step closer to finding effective, novel and personalized therapies for Glioblastoma (and potentially for other cancers?).