New drug selected for platform trial

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Nikki J

Mar 22, 2012
The HEALEY ALS Platform Trial is adding a new investigational drug, DNL343, to continue to research new treatments for ALS (Amyotrophic Lateral Sclerosis or Lou Gehrig’s disease).

The trial,is a trial in which multiple investigational drugs are tested and evaluated simultaneously to accelerate the development of potential new ALS therapies. Drug candidates that enter the platform trial are chosen by a group of expert ALS scientists and members of the Healey & AMG Center Science Advisory Committee.

DNL343 targets eIF2B, a key regulator of the integrated stress response (ISR). In neurons exposed to cellular stressors, inhibition of the ISR by DNL343 restores protein synthesis and dissolves pre-formed TDP-43 containing stress granules. This effect of DNL343 is of clinical interest because TDP-43 containing stress granules are thought to lead to TDP-43 inclusions, a hallmark of ALS pathology. DNL343 is being developed by Denali Therapeutics, a publicly owned biotechnology company based in South San Francisco, CA, and is not yet approved for use in any country. The Healey & AMG Center for ALS at Mass General and Denali Therapeutics recently signed an agreement to work together to design a trial of DNL343 in the HEALEY ALS Platform Trial.
This trial sounds like it will be quite limited due to the candidacy be qualified to the ALS experts from MGH. Is that inaccurate?
The platform trial which has been going on for a while with other drugs already finished and trehalose currently has 40 something sites in the US. It is probably one of the more inclusive ones IIRC correctly it is within 3 years of onset and breathing over 50 mostly. sometimes a particular regimen has extra rules based on the safety profile of the study drug. You don’t have to travel to mgh. Screening would be done at whatever site someone went to by their doctors and staff.

MGH started the trial and coordinates it. Much of the funding was from Sean Healey and his friends and company to start this

I am already participating in the Regimen E, trehalose, trial. And looking forward to the next opportunity.
My local clinic is starting their efforts for activation of the Regimen F.
Maybe I can do that trial?
You can only do one trial at a time. If you are finished with trehalose and haven’t received any for whatever washout period ( usually 30 days) when the new arm starts you could screen for it. They won’t let you do ole trehalose or any other experimental medication when in another trial
This is interesting thanks Nikki. My husband’s dr mentioned the Platform trial and said he could do it, but we wanted to get him started on the medications first. Now that we have that sorted out we should re-visit the trial.
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