Bad News about Both PSP/CBD Experimental Trials in 2012

I wanted to be sure everyone saw the very bad news that came out in 2012:  the two experimental drug trials in PSP — Davunetide and Nypta.

Hopefully the search will continue for a medication that can slow the progression of PSP and CBD — both tauopathies.  And hopefully cures can eventually be found for these diseases.

Adam Boxer, MD, the neurologist at UCSF’s Memory & Aging Clinic and the principal investigator of the Davunetide trial, has spoken forcefully in the past about pharmaceutical companies using PSP — rather than Alzheimer’s Disease — as the testing ground for tau-modifying agents.  I hope the failure of Davunetide doesn’t mean that Dr. Boxer’s theory has been disproven.

A small number of people in our local support group participated in the Davunetide trial; UCSF was the lead institution.  (A total of 313 people participated worldwide.)  And a couple of our group members participated in the pilot trial of Davunetide done at UCSF in PSP, CBD, and FTD.  I don’t believe anyone in our group participated in the Nypta trial.  (UCLA was the nearest institution participating.)  I would like to thank those who participated.  Without their sacrifice, we would not have known whether this medication was effective or not.

Anecdotal evidence in at least one local participant suggested
Davunetide was effective.  I guess that was placebo effect?

The manufacturer of Davunetide, Allon Therapeutics, had a phone call with investors the day the results were announced in mid-December 2012. Janet Edmunson, the chair of CurePSP, listened to a recording of the call.  One point that she passed on — that was not in the press release — was that perhaps not enough of the medication was given. Would a higher dose work?  Allon said the results are being further analyzed to “determine if there is any evidence of an effect or explanation for the absence of an effect.”

Apparently, the bad news about the worldwide Nypta trial came out in early 2012.  UCLA was the nearest participating medical center.  I don’t think Dr. Boxer mentioned this failed trial at the October symposium.  Noscira, the manufacturer of Nypta (tideglusib), said that the experimental medication failed in PSP but a trial in Alzheimer’s Disease would continue in 2012.  No results of that have been announced yet.

This seems to be the path of most medical research — some steps
forward and lots of steps back (or sideways).  We’ll just have to keep hoping for a breakthrough as the researchers continue to plod away.

If you’d like to read the details on the bad news, check out:

Allon Therapeutics press release regarding Davunetide
Excerpt:  “The study had co-primary outcome measures: the Progressive Supranuclear Palsy Rating Scale (PSPRS), and the Schwab and England Activities of Daily Living (SEADL).  Data analysis failed to detect an effect on either the PSPRS or the SEADL.”

Noscira CEO letter regarding Nypta (tideglusib)