PSP treatment trials recruiting now or soon

As promised, I will now start trying to keep you updated on actively recruiting clinical trials in PSP.  Keep in mind a few things before using my list:  

  • In compiling my list, I rely heavily on  Trials in the US are required to list themselves there, but some take longer than others to comply.  Trials overseas may or not be required to have a listing on, depending on the country.  My list also includes trials I’ve heard about through the grapevine that are not (yet) listed.
  • In Phase 1 and sometimes in Phase 2, the trials do not typically allow patients completing the protocol to continue receiving the drug, no matter how well they were doing on it.  Later Phase 2 and Phase 3 trials often do allow this, but they do not commit to it in advance.  Typically, if the trial shows the drug to be ineffective (or unsafe) overall, the drug company will discontinue its program for that drug and stop producing it.  That means that even the few patients who might have been doing well on it will not continue to have access to it.
  • In theory, exceptions to that rule could exist for drugs that are being tested in both PSP and another disease such as Alzheimer’s.  If the PSP trial shows lack of efficacy but acceptable safety, the company will continue to manufacture the drug pending the outcome of the other trial and may make it available to those who completed the PSP trial.
  • For trials of neuroprotective treatment, patients may not know if they are benefitting.  (Definitions: “Neuroprotective” drugs are intended to slow the progression of the abnormal brain process in the long term, as opposed to helping the symptoms experienced by the patient in the short term.  For example, for infections, antibiotics are protective, while painkillers are symptomatic.)  To know if an individual in a neuroprotective trial has had slowing of their rate of worsening over the 12 months on the drug, their rate of worsening over at least a few months before the trial would have to have been quantitatively assessed using the same method that the trial used.  We almost never have that data, so trials work by comparing the average result from a group receiving the drug to the average from a group receiving a placebo.
  • Do not expect a neuroprotective drug to improve your symptoms.  At best, it could prevent worsening, and more likely would only slow the rate of worsening.  The trials are typically designed to detect a slowing of the rate of worsening of 30% or 40%. 
  • Bottom line: Participating in a clinical trial, especially one in Phase 1 or 2, requires some level of altruism.
  • For more information on these trials, go to and enter the drug’s name or ID number shown in the first column.

Here’s the current list to the best of my knowledge.  The ones at the bottom labeled as “may start” are awaiting funding in most cases.

Drug / ID

Transposon2aReduces tau productionBoca Raton, FL Farmington Hills, MI30 patients on drug, 10 on placebo
RT001 (di-deuterated linoleic acid ester)

Retrotope2aReduces lipid peroxidation, enhancing mitochondrial activityUniversity of Munich (Germany)Non-controlled study showed very slow PSP progression over 2 years.

Novartis1Anti-sense oligonucleotide; reduces tau productionRochester, MN; Nashville, TN; Montreal; 3 in Germany; 1 in UKRequires 4 injections into spinal space over 3 months.

AlzProtect1Reduces tau production3 sites in France~24 patients on drug, ~12 on placebo
May start recruiting in the next year:
ASN120290Asceneuron1Reduces tau misfolding and aggregation by inhibiting O-GlcNAcase?Press release info only
MP201Mitochon1Mitochondrial decoupler?Early in planning per press release
Tolfenamic acidNeuroTau2NSAID that reduces tau productionCleveland Clinic, Las Vegas + others? 
Sodium selenateGov’t of Australia2Enhances dephosphoryl-ation of tau by protein phosphatase 2Multiple, in Australia