2. Academic Validation
  3. Development of iPSC-based clinical trial selection platform for patients with ultrarare diseases

Development of iPSC-based clinical trial selection platform for patients with ultrarare diseases

  • Sci Adv. 2022 Apr 8;8(14):eabl4370. doi: 10.1126/sciadv.abl4370.
Glen Lester Sequiera 1 2,  Abhay Srivastava 1 2,  Niketa Sareen 1 2,  Weiang Yan 1 2,  Keshav Narayan Alagarsamy 1 2,  Elika Verma 1 2,  Mohamad Reza Aghanoori 3 4,  Michel Aliani 3,  Ashok Kumar 5,  Paul Fernyhough 3 4,  Cheryl Rockman-Greenberg 6,  Sanjiv Dhingra 1 2
Affiliations

Affiliations

  • 1 Institute of Cardiovascular Sciences, St. Boniface Hospital Albrechtsen Research Centre, University of Manitoba, Winnipeg, Canada.
  • 2 Regenerative Medicine Program, Department of Physiology and Pathophysiology, Max Rady College of Medicine, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, Canada.
  • 3 Division of Neurodegenerative Disorders, St. Boniface General Hospital Albrechtsen Research Centre, University of Manitoba, Winnipeg, Canada.
  • 4 Department of Pharmacology and Therapeutics, Max Rady College of Medicine, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, Canada.
  • 5 Centre for Systems Biology and Bioinformatics, Panjab University, Chandigarh 160014, India.
  • 6 Department of Pediatrics and Child Health, Max Rady College of Medicine, Rady Faculty of Health Sciences, University of Manitoba, Winnipeg, Canada.
PMID: 35394834 DOI: 10.1126/sciadv.abl4370
Abstract

A "Leap-of-Faith" approach is used to treat patients with previously unknown ultrarare pathogenic mutations, often based on evidence from patients having dissimilar but more prevalent mutations. This uncertainty reflects the need to develop personalized prescreening platforms for these patients to assess drug efficacy before considering clinical trial enrollment. In this study, we report an 18-year-old patient with ultrarare Leigh-like syndrome. This patient had previously participated in two clinical trials with unfavorable responses. We established an induced pluripotent stem cell (iPSC)-based platform for this patient, and assessed the efficacy of a panel of drugs. The iPSC platform validated the safety and efficacy of the screened drugs. The efficacy of three of the screened drugs was also investigated in the patient. After 3 years of treatment, the drugs were effective in shifting the metabolic profile of this patient toward healthy control. Therefore, this personalized iPSC-based platform can act as a prescreening tool to help in decision-making with respect to patient's participation in future clinical trials.

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