The American Society for Neural Transplantation and Repair Considerations and guidelines for studies of human subjects. The practice committee of the society. Approved by council.

Goal: An important goal of the American Society for ably, the principal preclinical findings should be published in peer-reviewed journals. Safety issues should Neural Transplantation and Repair is to facilitate the discovery of novel cellular and molecular treatments for also be evaluated in the best and most appropriate animal model for potential toxicities including nonhuman nervous system (neural) diseases and disorders. The ASNTR recommends that investigators and members of primates if appropriate. These considerations may lead to the need to conduct the preclinical benefit studies and the Society consider the following in planning and carrying out studies of possible new treatments for nerthe safety studies separately in different models, so as to obtain the best information regarding both aspects. vous system diseases and disorders in human research subjects. A. Safety Issues for Consideration Related I. GENERAL CONSIDERATIONS to Novel Treatments The research plan should proceed according to estab1. Any treatment: Toxicity of treatments and intervenlished clinical trial principles and federal guidelines. tions, including biological products, should be deterAny special issues related to the disease or condition mined and should be within limits that are acceptable being studied should be addressed, including problems assuming that the treatments are successful. Any toxwith informed consent, the adequacy and limitations of icity should be in reasonable balance with evidence present treatments, the considerations regarding the apfor possible benefits. propriate stage of the disease for intervention, and the 2. Cell-based treatments: Fetal tissue, stem cells, other expected course of the disease left untreated or utilizing cell lines, cross-species tissue, or cell sources should the best available treatments. There should be enough be specifically tested to minimize the possibility of known about the mechanisms of the disease and the probiological contamination. For cell lines and sources posed intervention to suggest that the risks to the subwhich may have continued potential for growth, the jects are reasonable in light of a possible benefit to the tissue sources should be tested to minimize the possisubjects or society from the knowledge acquired. bility of uncontrolled growth, tumor formation, or II. PRECLINICAL STUDIES undesirable migration. Cellular products which have the potential for differentiation into varied phenoAppropriate data should be acquired in animals prior to human studies. The types of animal studies required types or undesirable migration should be assessed for those possibilities in the neural location intended might differ depending upon the target condition and the therapeutic modality that is the subject of the investigafor use. 3. Molecular and gene therapy interventions: There tion. The status of animal models for each target disorder should be considered in determining what studies are should be a justification and supporting preclinical data for the proposed choice of delivery system or most likely to offer the ability to predict any benefits. In general, the proposed treatment should be tested in the vector compared with other possibilities. Toxicity of vector constructs and any gene products on cells, tisbest animal models of the target condition, and the results should be clearly beneficial in these models and sue, and animals should be balanced against the possible benefits. The absence of effects on the germ should be peer reviewed by independent experts. Prefer-