Actually, there has been a recent trial that was successful in humans. Genervon has announced a successful Phase IIa trial for it's neurotrophic master regulator, Motoneuronotrophic Factor (MNTF), also known as GM608.

MNTF is a neurotrophic molecule that binds on very specific receptors of the nervous system. It is an integral component of human fetal development. It is able to correct inadvertent errors in that development and thus be life sustaining and potentially therapeutic.Throughout the embryonic development process, there are crucial regulatory pathways that oversee cell proliferation/apoptosis, differentiation, repair, inflammation/anti-inflammation, and overall developmental balance. MNTF is a specific master regulator that participates in regulating embryonic nervous system development.

The trial was very small, 6 participants, 4 with the drug and 2 placebo. As one of the trial participants, I have some reservations about how it was conducted and some of the results. Nevertheless, there were enough promising results to get excited about the potential of MNTF and hope that an expanded Phase II/III trial gets started soon. The drug was also successful in treating ALS patients and has received Orphan Drug and Fast Track status from the FDA for that disease.

The results that I question somewhat are the ones involving PD clinical data. There were no results showing drug vs. placebo group. The company reported on the drug group pre-test and post test. So, although the results showed a highly significant improvement on UPDRS and other clinical measures, they don't account for a very likely placebo effect. I'm not sure why there was no results comparing the two groups, other than a very small sample size.

On the other hand, the results involving biomarker data were very exciting.
1.) MNTF significantly increased the neuroprotective biomarker BDNF at week 2 when compared between GM608 treated and placebo groups, p value =0.035. BDNF has potent effects on survival and morphology of dopaminergic neurons; its loss could contribute to death of these cells in PD.
2.) MNTF dramatically lowered the levels of the inflammatory biomarker MMP9 at week 2 to -14.88% (lower is better) while the biomarker in the placebo group increased to +1.31%, an indication of disease progression. MMP9 gene is associated with the risk of PD.
Genervon is now in discussions with the FDA to determine their next step. As I said, for ALS they have been fast tracked, given orphan drug status and applying for compassionate use status. They are now trying to get the same for PD. We'll have to wait and see. Likely, they will be approved for a larger and longer term clinical trial. In any case, we finally had some positive human results from a neurotrophic drug study.

I'm going to also post this as a separate thread.

http://finance.yahoo.com/news/generv...011900395.html


Gary

Gary, that is good news. Thanks for participating in the trial. I hope more studies are done.

Yet more info on the successful study using intranasal delivery of stem cells!

"The research demonstrated that many of the stem cells delivered intranasally survived for at least six months in the brain; that the stem cells rapidly migrated preferentially to the damaged areas of the brain; and that motor control showed significant improvement."

http://offandonakpdrag.blogspot.com/...good-news.html

http://usatoday30.usatoday.com/news/...ose/55787500/1

you realize this research was in rats? as a courtesy, i'd appreciate you'd put that in the title line in future posts. you could spend 24/7 posting here links to snippets of pd research in mice and rats that scientists feel compelled to put out to get funding/attention but what are you trying to accomplish here?
just my opinion, i'll just stop reading your posts.

Yes rats and mice are often used before human trials. As a Parkinson's patient, a successful rat trial brings some hope that perhaps the success can be possible in humans too! The apparently successful AMGEN GDNF trial that was terminated early was a human trial. We are all able to post information that interests us and readers can decide whether to read it or not. I will take your suggestion though and place the rat/mice info in the title line.