Rare Disease Report

Rare Disease Treatments to Be Discovered by Machine Learning and Simulation Platform

AUGUST 09, 2017
Mathew Shanley
Earlier today, Alexion Pharmaceuticals was granted the licensing rights to operate GNS Healthcare’s Reverse Engineering and Forward Simulation (REFS) casual machine learning and simulation platform.
Alexion intends to use the platform to accelerate both the research of rare diseases and the development of novel therapies. Earlier this year, it was instrumental in finding a new target for breast cancer.
In May, GNS published data in the American Association for Cancer Research’s (AACR) journal Cancer Research in which they announced the discovery of novel targets, including TRIB1, which had positive implications for the treatment of triple-negative breast cancer. In June, it was announced that the company would be teaming up with Genentech to improve the capability to clarify disease mechanisms, recognize new targets, and diagnose patient populations more accurately.

The REFS technology is also being used by Celgene, Sema4 and other healthcare organizations to streamline their research approaches. It utilizes a never-before-seen hypothesis-free method that reverse-engineers fundamental biological and clinical models from large scale data streams, and then simulates interventions into those models to decipher the concealed impetuses of cancer development and drug response, patient by patient.
In the future, the power of REFS is expected to rapidly produce new propositions for the diagnosis and discovery of underlying pathogenic mutations linked to rare diseases, as well as innovative treatments for these more complex conditions, like multiple myeloma and Huntington’s disease (HD).
"We are delighted to partner with GNS in applying causal networks across a broad spectrum of rare diseases to decode and address their pathogenic roots," said John Reynders, PhD, Vice President of Data Sciences, Genomics, and Bioinformatics at Alexion. "I look forward to combining the GNS REFS platform with Alexion's deep expertise in data sciences to accelerate the discovery of innovative medicines for patients suffering from a rare disease."
In April, GNS Healthcare announced that nationally-regarded epidemiologist and biostatistician Julie Slezak was named Executive Vice President of Clinical Analytics, to apply her more than 2 decades of experience and expertise to the discoveries found by causal machine learning and REFS.
"We are excited to play a role in the journey to discover transformative therapies for patients with rare diseases," said Iya Khalil, PhD, Chief Commercial Officer and Co-Founder of GNS Healthcare in a press release. "As innovative companies like Alexion harness analytics and big data to answer the toughest questions about disease mechanisms and drug response, REFS unique approach of reverse engineering disease models and simulating 'what if?' scenarios unravel novel drug targets and biomarkers to help make these discoveries possible."
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