"Preliminary results from the first study of BIIB054 in patients with [Parkinson's] shows a favorable [pharmacokinetics], safety and tolerability profiles, providing rationale for further clinical development,"

#AAN218 Biogen's BIIB54 Posts Positive Data in Parkinson's Trial

Some more details on this, from the recent AAN conference in Los Angeles.

New Alzheimer’s and Parkinson’s Immunotherapy Data at AAN:
New Alzheimer’s and Parkinson’s Immunotherapy Data at AAN | ALZFORUM

(information on Biogen's BIIB054 is at the bottom of the page, after the heading "On to Parkinson's")

To me, the issues of most interest with respect to anti-alpha-synuclein treatments are:

Question 1: Do the treatments clear malformed alpha-synuclein from within neurons?

Why I care: If so, there is the possibility of some reversal of symptoms, since damaged (but not dead) neurons may recover instead of dying.


Question 2: Of the two main competing models about malformed alpha-synuclein propogation, which one is right. Model 1 is that malformed alpha synuclein spreads slowly from neuron to neuron, so that early in the disease, not many neurons are affected. Model 2 is that it spreads quickly but some neurons are more susceptible than others, meaning that even early on in the disease, most neurons already have malformed alpha-synuclein in them.

Why I care: If model 1 is right, clearing serum and cerebro-spinal-fluid alpha-synuclein may prevent propagation from neuron to neuron, slowing or stopping disease progression. But if model 2 is right, clearing serum and csf alpha-synuclein is useless because by the time one knows one has PD, it is already in all neurons, so propagation is not a factor in disease progression. If model 2 is right, anti alpha-synuclein therapies can only help if they work inside (rather than in-between) neurons.

Dan

PS: I'm a patient in a monoclonal antibody anti-alpha-synuclein study, but only just got started.