A re-post of my
short writeup on the TDI forum (sign-in required).
Some quick notes on the gathering:
Steve Perrin did a keynote of sorts. He reported good results in mice from the new molecule TDI200 - a mouse antibody for SOD1. The Gilenya small-group trial also did well in the biomarker department - measuring lymphocytes and other signs of immune activation. They cooperated with MGH's James Berry. The FDA-approved dose was too high, though, as it was based on the MS dosing. Steve noted that he does not recommend running out and getting Gilenya yet. He noted that a PMP goal ideally would be to predict the slope of decline for clinical trials beforehand.
Fernando Vieira did a talk on 15 years of pharmacological experiments at TDI and particularly on two recent candidates:
- TDI200 targeting misfolded SOD1: an antibody sticking to misfolded but not wild-type SOD1. This is a collaborative effort with Neurimmune. TDI200 has a rather amazing 21 day plasma half-life. Good results with mice! Survival extension in females by 13 days. Males are SOL.
- Guanabenz - blood pressure drug, approved; target - Eif2alpha. reduces ER stress. A bit of a concern with mouse body weight and aggression. Only 1.8 hour half life. CSF levels much higher than plasma. Reduced Chop protein and apoptosis. But hastened progression! TDI will try similar drugs now to target the same path but hopefully without the nasty progression-hastening side effects.
Matvey Lukashev: another PMP-oriented talk on isogenic cellular models of FALS and such.
Ajay Verma - Biogen. An interesting talk on imaging approaches, CSF measurement, CNS delivery, means to speed up spread of drug in CNS (vest to vibrate the subject). Shame they haven't got anything good to deliver to CNS for now.
Panel:
My question on cocktails drew a blank -- need at least one drug known to work on biomarkers before cocktailing. There was a consensus that once biomarkers are available, drugs will appear.
Grabbing people off-camera yielded a couple of interesting tidbits: TUDCA got a recommendation, if used carefully enough to avoid horrid diarrhoea experienced by some. I asked how the stuff works and got a shrug. Edaravone data is said to look very solid.
There was a strong presence and much energy from PALS/CALS. Glad I dragged my carcass over there. Peace to all.
