The drugs being tested are all already approved, so safety would not be really a concern.
Not true at all. Two drugs could be very safe on their own, but in combination could cause serious adverse events or even lead to death (combining some drugs with grapefruit juice can be bad). When drugs are approved by the FDA, the obvious combinations are sometimes tested for (or theoretical interactions are called out). New combinations are basically an unknown.
After some time to collect the data, you could fairly easily compare outcomes of patients who undergo this test versus those who do not.
Unless you are running a controlled clinical trial, I'm not sure anyone would trust the data. There is too much variability among patients, that if not controlled for, could seriously skew results. Sure it might indicate a promising lead, but I doubt that's enough for insurance companies to start paying for it.
> Two drugs could be very safe on their own, but in combination could cause serious adverse events
That is true. My assumption was that only single compounds or previously tested combinations are suggested.
> Unless you are running a controlled clinical trial, I'm not sure anyone would trust the data.
I agree. I just meant it would be relatively easy to collect enough preliminary data to have a reasonable idea of how a proper clinical trial would go. It's not like going from an animal model to human trials or from a tiny cohort of late-stage patients to a larger more heterogenous group.
It's not like going from an animal model to human trials or from a tiny cohort of late-stage patients to a larger more heterogenous group.
Fair point. A retrospective analysis would provide some data. The problem is insurance companies have pretty high standards (at least for cancer drug that cost a lot)!
> The drugs being tested are all already approved, so safety would not be really a concern
I think the problem is that Notable Labs would be testing combinations of FDA-approved drugs:
Step 2: We use lab robotics to apply thousands of combinations of FDA-approved drugs...
> After some time to collect the data, you could fairly easily compare outcomes of patients who undergo this test versus those who do not
Until then, this won't be broadly available. I do think such a comparison would be interesting.
I guess you'd need to compare the expected benefit of using the Notable Labs-suggested combination to using the standard of care. You'd need to have cells from patients who did not use the Notable Labs-suggestion. This requirement could turn an 'easy comparison' into a lengthy clinical trial.
Yes we'll be testing combinations, as this brain cancer has not responded historically to single agent therapy for decades.
With combinations of existing drugs it is possible to affect multiple driver pathways in the tumor at once. Similar to the dramatic improvement of AIDS therapy and infectious disease treatment with cocktails, tumors could respond much differently to combinations instead of a "one at a time approach" that has resulted in very small survival gains in this disease.
Patients have been taking the CUSP-9 protocol without any serious adverse affects on a compassionate use basis. This has now moved into a prospective clinical trial for recurrent glioblastoma patients in Germany
http://www.anticancerfund.org/projects/cusp9-a-combination-o...
Personalized medicine services like this could allow clinicians to move beyond traditional randomized controlled trials. Commercial services such as Foundation Medicine provide test results to oncologists who then use their discretion to treat the patient outside of a clinical trial with scientific rationale based on the test itself as opposed to a standard of care.
In a severe disease like Glioblastoma where average survival is 15 months, if the majority of patients using a system like Notable Labs live substantially longer, is a control arm necessary? The HAART cocktail for AIDS treatment never went through a phase 3 trial, so there is precedent in high need diseases.
I also would like provide a link to a recent documentary that highlights the combination approach in brain cancer by telling the story of a 20 year survivor of Glioblastoma, Ben Williams. Other patients who safely used combinations of existing drugs are featured as well.
http://www.survivingterminalcancer.com/
I have not looked into your company - but what's your opinion on crowdsourcing some of this? Some of these devices are remarkably similar to 3d printers and not so tough for the hobbyist to assemble.
If you do that then the community can start banding together to test formulations and then send the promising ones to you for further analysis.
Good luck. I love the idea and I personally have a few ideas of my own in the field that I want to start in 2016/2017
Edit * of course everyday people won't necessarily have access to approved therapies - but perhaps they can test other compounds and environmental conditions (or get the FDA to somehow approve tiny little test pellets for different drugs that would be legal for hobbyists to use in testing)
> I think the problem is that Notable Labs would be testing combinations of FDA-approved drugs
I assume that only previously used combinations are suggested.
> This requirement could turn an 'easy comparison' into a lengthy clinical trial.
But at least you would have a fairly reasonable idea of how a clinical trial would go. It's not like going from an animal model to human trials. I assume they can get a large enough sample size as they can probably recruit patients easily for what can be viewed as a second opinion.
The drugs being tested are all already approved, so safety would not be really a concern.
> Insurance companies (Medicare/Medicaid included), won't pay for cancer drugs unless there is proof
After some time to collect the data, you could fairly easily compare outcomes of patients who undergo this test versus those who do not.