A caregiver has asked me, as CurePSP’s Chief Clinical Officer, to list the most important clinical research advances in PSP of 2023. Happy to oblige. Here are my top five in no particular order. 

• The FDA approved a combination of two drugs called taurursodiol and sodium phenylbutyrate with the brand name “Relyvrio” for use in amyotrophic lateral sclerosis (ALS; Lou Gehrig disease).  A trial in PSP has already started to recruit patients.  The drugs address an issue in the mitochondria shared by the two diseases in different sets of neurons.
• Tau PET ligand APN-1607 received go-ahead from the FDA to proceed to a pivotal Phase 3 trial.  Such a trial began recruitment in December in the US and will involve multiple other countries as well.  The compound would allow a diagnosis of PSP in early or equivocal cases by being taken up by the abnormal tau protein in the brain and imaged.
• A drug called TPN-101 was found to be safe and well-tolerated in a Phase 1 trial of 30 patients with PSP.  The drug counters inflammation in the brain by reducing the transcription of ancient viral DNA in our genome.  Next is a small trial for efficacy.
• A simple, remote, gait-monitoring system with only three sensors proved able to distinguish the gaits of PSP and PD.  Further testing for its ability to document progression or improvement will follow.
• PET imaging of frontal lobe synapses showed good correlation with the PSP Rating Scale and with the results of cognitive testing.  This is different from typical PET in neurodegenerative disease, which images glucose utilization or protein aggregates.  The work suggests that synaptic imaging could be a good diagnostic marker in the earliest, pre-symptomatic stages of PSP.

But the most important piece of news is that several drug companies are planning to start clinical treatment trials in the next year or two. I’ll report on all that as it happens.