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AI-Aided Collaboration to Discover Neuro Disease Drugs

Mature neuron

(National Institute of General Medical Sciences, NIH)

9 May 2022. Two biotechnology and health technology companies are discovering precision treatments for neurological disorders with simulation models for individual patients. The collaboration brings together Arvinas Inc. in New Haven, Connecticut, a developer of therapies that degrade disease-causing proteins, and GNS Healthcare in Somerville, Massachusetts that uses artificial intelligence algorithms to simulate disease activity in individual patients.

Arvinas and GNS Healthcare seek to discover new therapies for chronic, debilitating neurological disorders such as Parkinson’s, Alzheimer’s, and Huntington’s diseases. Not only do these conditions affect a large number of people, estimated by GNS Healthcare at six million in the U.S., but they also present a range of symptoms and varying progression rates, which suggests a need for more precise and personalized treatments rather than a single therapy.

Arvinas discovers and develops treatments for cancer and neurological diseases that aim to degrade proteins in the body responsible for these diseases. The company’s technology, based on research by its founder Craig Crews at Yale University, and called proteolysis targeting chimeras or Protac, harnesses E3 ligases, a group of proteins in the body that acts as a natural defense against disease-causing mutated or misfolded proteins. E3 ligases, says Arvinas, add a targeting molecule called ubiquitin, sending disease-causing proteins to the proteasome, a cellular complex that breaks down the proteins into smaller peptides.

The company says this mechanism allows for weak binding to target proteins, and in preclinical studies, is shown to cross the blood-brain barrier. Arvinas has several therapy candidates for neurodegenerative diseases still in early-stage research, to go along with a number of cancer therapies in preclinical and clinical development.

Causal factors, not just associations

GNS Healthcare helps biotech and pharmaceutical companies discover new therapies with a technology called “virtual patients” using simulation algorithms to represent individual patients. The company draws on data for its algorithms from a host of large-scale databases representing genomics, electronic health records, demographics, pharmacy and medical claims records, imaging, and mobile devices. Aggregating these individual patient models, says GNS Healthcare, reveals causal factors and not just associations in the data.

The company says its work already uncovered 10 new and unique targets for Alzheimer’s disease treatments, by reverse-engineering networks linking gene expressions, metabolites, and clinical data. In Sept. 2017, Science & Enterprise reported on an analysis of data from two clinical trials by GNS Healthcare, where its machine-learning algorithms identified factors that predict the decline of motor functions in individuals with Parkinson’s disease.

In their collaboration, Arvinas and GNS Healthcare are applying their respective processes to discover new drugs using the Protac technology of protein degradation for treating neurodegenerative diseases. The collaboration aims to better understand the workings of protein degradation therapies in individual patients, as well as model those findings in simulated clinical trials. Data from simulated trials are expected to highlight possible clinical outcomes, better identify patients for trials, and connect treatment options to different types of patients.

“We believe this collaboration,” says Arvinas vice president for neuroscience and platform biology Angela Cacace in a GNS Healthcare statement, “will provide novel insights and will help us understand the underlying mechanisms of complex neurodegenerative diseases to inform our discovery and development efforts with the aim of improving patient outcomes.”

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