Biomarker Platform That May Track Huntington’s Decades Before Symptom Onset Receives FDA Letter of Support

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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sRNA-FIND, Huntington's biomarkers

The U.S. Food and Drug Administration (FDA) has issued a letter of support encouraging sRNAlytics to further develop its sRNA-FIND platform for discovering and monitoring variants of small RNA molecules that may work as biomarkers for tracking progression of Huntington’s disease decades before symptom onset.

In addition, these biomarkers may help assess the efficacy of investigational therapies in clinical trials.

“The use of objective biomarkers to assess Huntington’s Disease in clinical trials will enable us to more accurately determine if treatments are effective. sRNAlytics’ discovery of an objective biomarker will help transform the way we think about therapeutic development and treating patients,” Blair Leavitt, professor at the University of British Columbia, said in a press release.

Currently, no biomarkers are available to detect Huntington’s disease before the onset of symptoms. Monitoring of disease progression relies on clinical measures, such as the Unified Huntington’s Disease Rating Scale and total functional capacity score. However, these measures provide limited information about disease progression in the disease’s pre-symptomatic phase.

In the body, DNA is transformed into RNA, then a process called translation begins which gives rise to proteins. Small RNAs are a type of non-coding RNA molecule that are many times involved in regulating the translation of target RNAs into proteins.

Through its sRNA-FIND platform — an artificial-intelligence computational platform that analyzes small RNA sequencing data — sRNAlytics discovered a group of small RNAs (microRNAs), called isomiRs, that are present in brain tissue samples of deceased Huntington’s patients but not in healthy controls or patients with Parkinson’s disease. These small RNAs significantly correlated with disease grade and degeneration of neurons in a brain region called the striatum — an area involved in movement initiation, response selection, and attention processes.

In collaboration with professor Richard Myers at Boston University School of Medicine and his team, sRNAlytics researchers assessed the presence of these biomarkers in the cerebrospinal fluid (CSF) — which surrounds the brain and spinal cord — of patients enrolled in the PREDICT-HD trial (NCT00051324).

They found that the small RNA biomarkers could be detected in patients who were predicted to be 20 years prior to the onset of symptoms. Additionally, the biomarkers correlated significantly with disease progression and loss of motor function.

“The small RNA profiles that sRNAlytics identified represent the most promising approach to test efficacy that I have seen to date,” Myers said. “The FDA’s recognition of these findings is an important step forward in validating their technology and accelerating their future use in clinical trials.”

sRNAlytics is currently testing their biomarkers in two additional Huntington’s studies, using CSF from 126 Huntington’s disease patients and 50 matched controls.

The company is also collaborating with Huntington’s therapy developers to identify more biomarkers using its sRNA-FIND platform.

“Receiving the Letter of Support is a major milestone in the development of our platform. In a few years, we’ll be able to look back and know that we played a role in delivering game-changing therapies for patients,” said David W. Salzman, CEO of sRNAlytics.