BSN PSP/CBD Conference – Adam Boxer

Advances in Therapeutic Development for CBD & PSP

Adam Boxer, MD, UCSF MAC

Clinical Research Updates

    • There are some treatments being tested in patients that really have a chance to make a difference
    • Can ultimately present us with a standard treatment in the long run
  • Advancements in PSP publishing’s
    • Multidisciplinary intensive rehab therapy (MIRT)
      • Rehab in PSP is extremely helpful
      • Improvements that people saw over a month of intensive rehab was greater than any drug anyone has ever tried
    • Tau Therapeutic Targets
      • We don’t know exactly why, but in PSP & CBD, the tau protein has a large part in these diseases
      • Chemistry of tau protein is being researched more
      • Most advance testing are therapies that block the spread of abnormal forms of the tau protein, from one nerve cell to another.
        • Think that antibodies may be of help in blocking this spread
  • Current clinical PSP Therapies
    • Microtubule stabilizing agents (Epothilone, Davuntide, Abeotaxane)
    • Post-translational modification/aggregation (Methylene blue, Tideglusib, O-glc-NACase inhibitors, Salsalate)
    • Anti-sense oligonucleotides
      • Immunotherapies to tau or phosphor-tau (mAbs, active vaccines)
    • Other approaches
      • Young plasma infusions
      • Hypnotics (Suvorexant and Zolpidem) (sleep medicines)

Challenges for PSP therapeutic development

    • Late diagnosis
    • Clinical heterogeneity (need a tool to measure the effects of the drug for people with different types of symptoms)
    • Rare disease (are there enough patients?)
    • Narrow pipeline (we need more drugs to test)
      • Richardson’s (PSP-RS/NINDS-SPSP) Most clinical trials formulate around this form of PSP.
      • Other variants of this are PSP, PSP-P, PSP-CBS, PSP-SL, and PSP-F
      • If we can diagnose people reliably during psychiatric and earlier phases, we can be more certain on how to treat
      • Treat people that can benefit from early treatment, and detect people very early. First symptom might be a single fall, (suggestive of PSP), but what we want to do is get to test clinical trials at this stage, or even before.  (Prevention trials)
    • Looking at different features of the disease within different people, looking at eye movement, falls, movement capabilities, behavior, language, and cognitive function. By rating people on each different domains, we think we can detect PSP better and diagnose people easier
    • ARTFL research, LEFFTDM, 4RTNI (Clinical research networks) are recruiting patients all over the country. (4 in California).  Recruiting about 900 people, goal is about 1500 people. Studying people to develop new tools to measure disease progression.