BRAIN RePAIR & ORGANØID 1NTEL by Minee Choi

The KAIST BRAIN RePAIR & ORGANOID INTELLIGENCE Lab is …

… a team of researchers at KAIST studying diseases of the nervous system, focusing on Parkinson’s diseaes and Dementia. These diseases are challenging to treat due to the difficulty of accessing the diseased brain and their individual heterogeneity. To overcome the inherent limitations of studying brain disorders, we use human stem cells generated from real patients (hiPSC). This approach allows us to profile the diseased state of a patient’s individual brain cells and identifying mechanistic subtypes using a ‘disease-in-a-dish’ paradigm. By utilizing patient-derived iPSCs, our overarching goal is to develop a preclinical testbed for brain disorders. We also employ AI to predict the most suitable drug combinations for individual patients’ pathological and cellular subtypes, thereby overcoming the limitations of biological experiments in accurately capturing the heterogeneity of diseases.

… 파킨스, 치매 등 난치성 인간 뇌질환의 병리 기전을 연구하고, 뇌질환 정복에 반드시 필요한 뇌질환 치료제 전임상 테스트베드를 개발하는 연구실입니다. 이를 위해 우리는 뇌질환 환자 역분화 줄기 세포 (hiPSC)를 표적 뇌세포 또는 뇌-오가노이드로 분화시켜, 환자 개인의 뇌 세포 질병상태를 프로파일링하고, 그에 맞는 기전적 하위 유형을 찾는 ‘접시 속의 질병’ 패러다임을 사용합니다. 또한 질병 특이성(이질성) 판별에 있어 오류 가능성이 높은 생물학적 실험 관측의 한계를 극복하기 위해 인공지능을 활용함으로써 개별 환자의 발병 인자에 가장 적합한 약물 조합을 예측합니다.

주요 연구분야

  1. 환자 유래 줄기 세포 뇌 오가노이드 기반 뇌질환 치료제 전임상 테스트베드 개발
  2. 퇴행성 뇌질환 핵심 병리 기전 연구: 뇌세포 사멸, 단백질 응집, 미토콘드리아 기능 장애
  3. 뇌 오가노이드 지능 연구: 오가노이드 임베디드 컴퓨팅

Finding New Keys to the Deadlock of Human Brain Disorders

One Goal

We are dedicated to making brain diseases more curable than yesterday. The primary aspiration of this endeavor is to foster a more profound understanding of the molecular and cellular origins of Neuro-Degenerative, -Psychological & -Developmental diseases & disorders, thereby enabling a more strategic approach to the innovation of new therapeutic intervention.

Two Faces of 'One-Size-Fits-All' Approach

The most common therapeutics in neurodegenerative diseases, such as Dopamine Agonists and Levodopa, follow a ‘one-size-fits-all’ approach. This approach applies the same treatment regimen to all patients with a particular condition, regardless of individual differences. While this method simplifies the treatment process, making it easier to standardise care and ensure consistency across different healthcare providers, it often fails to account for the unique characteristics and needs of individual patients. As a result, this can lead to less effective disease management and an increased risk of adverse effects, highlighting the need for personalised medicine.

Three Challenges

Our mid-term objective is to develop a bespoke therapeutic platform that addresses three major challenges associated with human brain diseases.

  1. The distinctive complexity inherent in the human brain
  2. The clinical variability presented on an individual level
  3. The predominantly unknown etiological factors of the human brain diseases
[BBC] Animal brains vs. human brains - let the Battle of the Brains commence!
[BBC] Animal brains vs. human brains - let the Battle of the Brains commence!
[The Lancet] Report finds no common cause for mystery brain disease.
[The Lancet] Report finds no common cause for mystery brain disease.
[WHO] There is no cure for Parkinson's disease, but therapies including medicines, surgery and rehabilitation can reduce symptoms.
[WHO] There is no cure for Parkinson’s disease, but therapies including medicines, surgery and rehabilitation can reduce symptoms.

Four Powerful Keys

Our therapeutic platform is underpinned by four pivotal technological advances.

Patient-induced pluripotent stem cell (iPSC) , facilitating the implementation of the ‘Disease in a dish’ model. This approach is instrumental in profiling an individual’s brain cells and elucidating the specific mechanisms driving their disease.
Brain organoid , often described as “Mini-brain”, a powerful tool in neuroscience, offering a unique window into the human brain’s complexities, diseases, and potential treatments.
High-resolution live cell imaging , a technique used in cell biology to study living cells using time-lapse microscopy. This technology allows scientists to observe the dynamic processes and behavior of cells in real-time, providing valuable insights into cellular function, morphology, and interactions.
Artificial intelligence (AI) , harnessed to discern and comprehend the heterogeneity of these diseases, thereby contributing to the development of a predictive model individualized for each patient.

Five-fold Enhancement in Human Brain Repairability

A Recent Breakthrough : AI-based Prediction of Disease State and Cellular Subtypes in Parkinson’s Disease (PD)

The workflow of our classifier predicting cellular subtypes in Parkinson's Disease (PD)
The workflow of our classifier predicting cellular subtypes in Parkinson’s Disease (PD)
Our classifier, trained on cell profiles of key organelles, predicts disease states with an accuracy of 82%.
Our classifier, trained on cell profiles of key organelles, predicts disease states with an accuracy of 82%.

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