Antisense trial for SALS on horizon

[Nikki J]

As many of you know antisense genetic therapy is hopeful for FALS. The SOD1 trial is in phase 3. C9 is in a phase1-2. FUS is coming to trial in the next year

however Ionis has an antisense targeting ATXN-2 TDP-43 Is involved which is found in SALS ( and many FALS). They SAY they expect a trial to start in the latter part of this year. My experience is that it may well take longer but this is incredibly hopeful news for SALS

 

[Anjum nazir]

N what about people like me who are fals but do not know the exact gene involved ...what should we people do even without hope for future therapies

 

[Nikki J]

You might have the TDp-43 issue. Most PALS do including a lot of FALS. However this is a proposed phase 1 trial that has not yet started not a treatment that is being given.

 

[Nikki J]

Not recruiting yet but listed on clinicaltrials.gov

ClinicalTrials.gov

www.clinicaltrials.gov

there are arms for people with the specific mutation and without. Without needs to be at least El escorial probable With can be possible. The only genetic categories excluded are sod1 and fus presumably because TDP-43 isn’t an issue for them.

 

[Nikki J]

This is now enrolling -4 sites California, Georgia, Maryland and Missouri