The Comments section just received a trial participant’s personal report and some good questions about the anti-sense oligonucleotide (AS0) trial sponsored by Novartis.  As explained in my last post, all the patients in that trial have completed their participation and the data are being analyzed.  First, here’s the comment unedited (except for correcting the location of the PSP conference and my bracketed clarification in the fourth line), followed by my response.

IT WORKS! My husband was in the trial. No cure but a great year – his condition was improved in so many ways. I have been waiting for someone at Cure PSP to put out the word. I assumed they were going to present at the Toronto conference but they did not.

We are so sad they [Novartis] refuse to continue to give it to us. Novartis has a published policy to keep giving people in a trial drugs that provide help but denied us with the excuse it was phase 1. Even if it is too expensive when administered through the spine it provides evidence that this horrible disease can be managed and greatly improves life. We would love to go to Novartis and show them how it works. Hopefully they are not just proceeding on using it for Alzheimers. We have been watching and waiting to see if they release results. They need to publish them. HOPE MATTERS! Kathy and Steve from San Diego, California.

Kathy and Steve:

I’m so glad that you, Steve, had symptomatic benefit from NIO752.  You raise several good points deserving separate responses:

• The known molecular mechanism of the drug would merely slow down, or hopefully halt, the future worsening of the disease, not improve it in absolute terms.  People entering clinical trials may start paying new attention to their health and changing their habits regarding diet, hydration, exercise, physical and other types of therapy, compliance with concomitant medication, and discontinuation of unnecessary concomitant medication that might have been producing side effects.  There’s also the possibility of a placebo effect.  All this, of course, could make someone entering the trial feel better and is why establishing true drug efficacy requires a control group. 
• The reason not to provide the drug post-trial to Phase 1 trial completers is that the purpose of Phase 1 is to establish safety, and until that trial is over and the data analyzed, the drug’s safety remains unknown in people with PSP.  Even if the trial is over and the drug found to be safe, that applies only to the duration of the trial.  So, allowing a trial completer to continue to receive the drug for longer would be taking a safety risk, and there would no longer be a placebo group to use in assessing the magnitude of that risk.
• That said, there’s a slight theoretical possibility that the reduction in tau production caused by NIO752 could actually allow recovery of normal function by some brain cells that were malfunctioning but still salvageable, producing an immediate symptomatic improvement.  But that might have no relationship to any long-term neuroprotective benefit.
• I do have some good news in response to your hope that Novartis is still pursuing PSP rather than just Alzheimer’s.  I’ve heard that the company has been seeking information useful for the design of an efficacy PSP trial.  That suggests that such a trial is being at least provisionally planned. 
• The results of Phase 1 trials are not always be published in journals, but they are often presented as posters at conferences and are announced by the company in the form of press releases.
• Yes, HOPE DEFINITELY MATTERS!