Nikki J
Moderator
- Joined
- Mar 22, 2012
- Messages
- 15,029
- Reason
- PALS
- Diagnosis
- 04/2014
- Country
- US
- State
- MA
- City
- Boston
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.
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.
