RE:RE:RE:RE:RE:RE:RE:MY TAKE.......Although the Mtfp peptide is based upon human sequence, that doesn't guarantee that the results produced in cell culture or in mice wil translate to equivalent success in humans. It SHOULD, based upon how similar the physiologies are between mice and humans. However, no guarantee it will work as well.
I've wondered for a long time why Bioasis hasn't completed or disclosed (if completed) studies in non-human primates to validate their technology. It is extremely common to do pre-clinical non-human primate validation prior to proceeding to humans. Maybe non-human primate work has been done in some of the secretive work with the numerous companies that they are working with. Maybe, maybe not. At least with non-human primate models, one can more easily analyze the brain at the end of the experiment since one can euthanize the animal and collect the brain. It's really difficult to get those kind of protocols passed with human studies (sarcasm intended). Non-human primate work would go a long way to bolster confidence in the translatability to humans.
For example,
Armagen did a study in Rhesus macaque monkeys to learn more about their human insulin-receptor antibody vector for crossing the BBB. The last paragraph of their discussion is a good lesson: "
Our findings highlight the importance of comprehensive preclinical research in nonhuman primates to assess first-in-class therapies. While this study cannot predict whether HIR-mAb GDNF will also induce an immune response in humans, accumulated evidence using biologically-derived therapies advises caution."
Regardless stated "
what is of upmost importance now is the fact that this company and your money now depend on one shot...one phase 0 or phase 1 to demonstrate wether or not the vector will shuttle in anything past the blood brain barrier...." My concern is what will they prove with a phase 0 study? Phase 0 studies have a small number of patients, using a very small dose of the drug, and for a very short period of time. What is the goal of this Phase 0 study? Safety? Not much will be learned based on small dose and short length of time. Efficacy? Not likely because of dose and length of time? Pharmacokinetics? Yes, they should get some information about the transport, metabolism, half-life of the drug/peptide. But how will they ascertain crossing the BBB and tissue distribution? Perhaps they will attach the Mtfp to some molecule known to not cross the BBB very well, and also attach a positron emission tomography (PET)-compatible tracer that will allow them to
non-invasively determine BBB transport. That is the key.....non-invasive. These are humans, not animal models.
The financing is what it is. No use crying over spilled milk, no matter how sour that milk is. Hopefully, they use this money wisely. ""
Provided this capital is realized, it will provide the resources to obtain extremely valuable initial human data," said Rob Hutchison, chief executive officer." Clearly, the "commercial" phase of the company is not going as smoothly as they intended. Will we see the details of the Phase 0 study, or will it remain secretive? Rob told me back in June that an IND isn't necessary for a Phase 0 study and that "
we are in the process of preparing for a phase zero study, but the timing for that is predicated on material prep and patient availability." Well, it's nine months later. A voluntary corporate update from the company sure would be nice. It's been a while. Too long, in my opinion.