ALS actively protective biology, novel causal targets and mechanistic biomarkers
Transformational ALS research also predicts responders to existing & candidate ALS therapies
Causative combinatorial analysis
Key findings
- Genetic basis of 32 disease targets, 19 of which we believe to be new to ALS
- First genetic support for existing medications that are used in the treatment of ALS and its symptoms
- Mechanistic patient stratification biomarkers to select patients most likely to benefit from existing therapies
- Actively protective biology in genes which may be acting to counter disease risk factors and prevent disease
- Genetic evidence to support the development of new precision medicines and rapid, early diagnostic tools
AI-led research discovers actively protective biology, novel causal targets, drug repurposing candidates and mechanistic patient stratification biomarkers in ALS
New research by PrecisionLife, presented at the Northeast ALS Consortium Annual Meeting 2024, describes a series of important advances for our understanding of ALS disease biology.
Causative combinatorial analysis has uncovered the genetic basis of 32 disease targets, 19 of which we believe to be new to ALS, and five which are involved in the mechanisms of action of existing therapeutics (riluzole, retigabine, baclofen).
A unique new approach to finding actively protective biology has identified additional targets that appear to have the ability to be disease resistance genes, functioning in a way to counter disease risk factors and prevent disease onset.
One example of the protective genes is MTRR, which codes for an enzyme involved in vitamin B12 biology (an ultra-high dose was recently approved as in Japan for slowing progression of functional impairment in ALS), and another is GRIP1 which has previously been linked to rare reversal cases.
Mechanistic patient stratification biomarkers tied to these groups could be used to select patients most likely to respond to each of these therapies.
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See how we're driving precision medicine in CNS disorders and view more precision neuroscience research from PrecisionLife.
