1. How do you plan to deal with mutations/regrowths? Would the tumor have to be repeatedly resected for subsequent in vitro analyses?
2. What is your plan for dealing with impending LDT regulation?
The thing I'm most interested in is how you're tying the in vitro results to in vivo efficacy, as well as your reimbursement strategy, but refurb already asked those 2 questions.
1. Great question, this is why we'll be focused on newly diagnosed glioblastoma patients. If they do recur and have a surgery to remove the recurrent (and hypothetically mutated) tumor, we would test that tissue as well and compare its drug response profile as well as any biomarker changes.
We'll also be using in vivo xenografts to try to model mutations/regrowths, by continuously observing treated mice to see if we get a regrowth. The mice have faster metabolisms than humans so tumors can sometimes regrow faster in them than in patients.
If we have a regrowth in a mouse, we could then culture that tumor in vitro for a screen to develop a plan to treat a patient's recurrence before it occurs. This has been proven out already as a strategy by another company using xenografts, Champions Oncology.
2. Since this LDT regulation change is still in motion we are observing it very closely. In discussions we've had with experts on the topic they have highlighted that any changes will be implemented gradually over time.
Sure, just shoot me an email at one of the addresses in my profile.
It'll be very interesting if you can robustly translate murine xenograft data into successful clinical outcomes. Two potential hurdles I can see are (1) high cost per patient despite relying on generics, from all the animal models (though maybe you can get an adventuresome ACO on board: http://www.hhs.gov/news/press/2015pres/03/20150310b.html) and (2) insufficient characterization of the mutations/drug interactions/interpatient variability state space, due to the relatively low GBM incidence rate (compared to other cancers).