Clinical Trials of Gene Editing Therapeutics Using CRISPR technology: Halachic Considerations and Guidelines
Frank Lieberman, MD, Professor of Neurology, Neurosurgery and Medical Oncology, University of Pittsburgh School of Medicine
The potential of gene editing using CRISPR technology to ameliorate or potentially cure inherited human diseases for which the relevant genetic mutation is known has led to the design of clinical trials addressing both somatic cell gene editing and germline editing. The range of disorders potentially amenable to CRISPR gene editing range from sickle cell anemia to inborn errors of metabolism to genetically inherited neurologic degenerative disorders. The ability to correct the relevant genetic mutation in the germline of the individual involved could potentially convert a carrier of a disease causing mutation into an individual at no increased of transmitting the inherited disorder than a member of the normal population.
Jewish religious law views all experimental therapies as inherently carrying risk to the subjects, and the criteria for determining if a specific experimental intervention is appropriate for human clinical trials balances the risks of the study intervention against the potential gains. Jewish religious law considers the preclinical evidence regarding safety and efficacy, the severity of the illness and length of survival expected without the experimental intervention, the risk of shortening survival or increasing morbidity, as well as the other treatment options available, in analyzing the human subject protection issues for a specific intervention. We previously discussed the halachic ramifications of CRISPR technology as compared to other therapeutic approaches to the treatment of inherited disorders as well as potential applications in cancer therapeutics which do not directly involve gene editing of the specific patient’s somatic or germline cells.
In this presentation we will discuss the guidelines for developing and applying experimental therapeutics using CRISPR as these would be used to decide whether a clinical trial design is bioethically appropriate for human subject recruitment. We will explore how the standards related to risk, potential benefit, prior human trial experience, and the severity of the illness would guide ethical decision making about human CRISPR therapeutic experimental trials.
Jewish religious law views all experimental therapies as inherently carrying risk to the subjects, and the criteria for determining if a specific experimental intervention is appropriate for human clinical trials balances the risks of the study intervention against the potential gains. Jewish religious law considers the preclinical evidence regarding safety and efficacy, the severity of the illness and length of survival expected without the experimental intervention, the risk of shortening survival or increasing morbidity, as well as the other treatment options available, in analyzing the human subject protection issues for a specific intervention. We previously discussed the halachic ramifications of CRISPR technology as compared to other therapeutic approaches to the treatment of inherited disorders as well as potential applications in cancer therapeutics which do not directly involve gene editing of the specific patient’s somatic or germline cells.
In this presentation we will discuss the guidelines for developing and applying experimental therapeutics using CRISPR as these would be used to decide whether a clinical trial design is bioethically appropriate for human subject recruitment. We will explore how the standards related to risk, potential benefit, prior human trial experience, and the severity of the illness would guide ethical decision making about human CRISPR therapeutic experimental trials.