Preliminary results from Biogen's Phase 1 study of alpha synuclein & PD (BIIB054)
 

"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

Update
 

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.