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Abstract Amyotrophic Lateral Sclerosis (ALS) disease severity is usually measured using the subjective, questionnaire-based revised ALS Functional Rating Scale (ALSFRS-R). Objective measures of disease severity would be powerful tools for evaluating real-world drug effectiveness, efficacy in clinical trials, and for identifying participants for cohort studies. We developed a machine learning (ML) based objective measure for ALS disease severity based on voice samples and accelerometer measurements from a four-year longitudinal dataset. 584 people living with ALS consented and carried out prescribed speaking and limb-based tasks. 542 participants contributed 5814 voice recordings, and 350 contributed 13,009 accelerometer samples, while simultaneously measuring ALSFRS-R scores. Using these data, we trained ML models to predict bulbar-related and limb-related ALSFRS-R scores. On the test set (n = 109 participants) the voice models achieved a multiclass AUC of 0.86 (95% CI, 0.85–0.88) on speech ALSFRS-R prediction, whereas the accelerometer models achieved a median multiclass AUC of 0.73 on 6 limb-related functions. The correlations across functions observed in self-reported ALSFRS-R scores were preserved in ML-derived scores. We used these models and self-reported ALSFRS-R scores to evaluate the real-world effects of edaravone, a drug approved for use in ALS. In the cohort of 54 test participants who received edaravone as part of their usual care, the ML-derived scores were consistent with the self-reported ALSFRS-R scores. At the individual level, the continuous ML-derived score can capture gradual changes that are absent in the integer ALSFRS-R scores. This demonstrates the value of these tools for assessing disease severity and, potentially, drug effects.

Passive acoustic monitoring is a well-established tool for researching the occurrence, movements, and ecology of a wide variety of marine mammal species. Advances in hardware and data collection have exponentially increased the volumes of passive acoustic data collected, such that discoveries are now limited by the time required to analyze rather than collect the data. In order to address this limitation, we trained a deep convolutional neural network (CNN) to identify humpback whale song in over 187,000 h of acoustic data collected at 13 different monitoring sites in the North Pacific over a 14-year period. The model successfully detected 75 s audio segments containing humpback song with an average precision of 0.97 and average area under the receiver operating characteristic curve (AUC-ROC) of 0.992. The model output was used to analyze spatial and temporal patterns of humpback song, corroborating known seasonal patterns in the Hawaiian and Mariana Islands, including occurrence at remote monitoring sites beyond well-studied aggregations, as well as novel discovery of humpback whale song at Kingman Reef, at 5∘ North latitude. This study demonstrates the ability of a CNN trained on a small dataset to generalize well to a highly variable signal type across a diverse range of recording and noise conditions. We demonstrate the utility of active learning approaches for creating high-quality models in specialized domains where annotations are rare. These results validate the feasibility of applying deep learning models to identify highly variable signals across broad spatial and temporal scales, enabling new discoveries through combining large datasets with cutting edge tools.