A welcome formality

I see my patients with PSP on special clinic days when I have arranged for specialized professional help and have allotted extra time for the visits. The downside is that that it can be a dispiriting few hours, with little to offer anyone that day beyond symptomatic treatment, information and a pep talk. So I use this blog to accentuate the positive.

In that vein, I’m happy to report the drug/biotech industry’s efforts to develop a therapeutic antibody are proceeding apace. The latest tidbit is that the FDA has granted orphan drug status to the anti-tau antibody designated C2N-8E12 being developed by a joint venture of C2N Diagnostics and Abbvie. A 32-patient Phase I trial headed by Adam Boxer at UCSF will begin sometime soon. Achieving orphan status allows the company certain financial advantages and a longer patent life. Both are critically important for any new treatment for a rare disease, as the potential profits wouldn’t otherwise justify the development cost and risk.
Several other companies are working on anti-tau therapeutic antibodies, many of them aiming initially at PSP. Their ultimate Holy Grail is a treatment for Alzheimer’s, but it’s easier to conduct a clinical trial in PSP, as its progression is more readily predicted and measured. Furthermore, tau is the only protein known to aggregate in PSP, which makes that disease a simpler “model system” than AD, where both tau and beta-amyloid aggregate. The company furthest along this road is Bristol-Myers Squibb, whose tau antibody trial seeks 48 patients with PSP at 12 centers across the US and will start enrolling in a few weeks.
So I’m hoping for sunnier PSP clinic days soon!

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3 thoughts on “A welcome formality

  1. I’m happy to know that there are some parallel efforts from various pharmaceuticals in testing treatment options for PSP. I hope they will find a cure/treatment soon. My father has PSP and it is truly depressing to see how much he has deteriorated since. I would give anything to have him happy and healthy again. In your opinion – when do you think patients can start to take advantage of these treatments (pending successful trials)? 3 years from now? 5? I’m encouraging my father to hang in there and hoping it won’t be too long.

  2. desde España muchas gracias or sus informaciones y su esfuerzo hacia esta rara enfermedad. Le seguimos con mucho interés pues tenemos un familiar con este problema. Mil gracias.

  3. Best of luck with this effort. I know Adam Boxer had a lot to do with the Davunetide trial. I hope this will be more effective, though I’m slightly concerned about the potential side-effects of immunotherapies that have complicated the beta-amyloid trials.

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