The "Considerations and Guidelines for Studies of Human Subjects" will be published Dec. 24 in the journal Cell Transplantation. Lead authors were Eugene Redmond Jr., MD, chair of the ASNTR Practice Committee and director of Neural Transplantation and Repair at Yale University, and Thomas Freeman, MD, co-chair of the Practice Committee and director of the Neural Reconstruction Program at the University of South Florida and Tampa General Hospital.
The guidelines were endorsed by the society's full membership, which includes most of the world's researchers working on applications of fetal tissue, stem cells and gene therapy for brain repair.
"Inadequate preliminary data or study design flaws have sometimes set the field back -- we hope these guidelines help investigators 'get it right' the first time," said Dr. Redmond.
"Clinicians are beginning to have the capability to modify the nervous system through cell therapy, transplants, gene therapy and the advancing developments of stem cell therapies," Dr. Freeman said. "Our goal is to make sure these new treatments are developed in a scientifically rigorous fashion and in a manner as safe as possible for patients."
The explosion of neuroscience research in recent years has led to the growth of novel, investigational cell and gene replacement therapies for Alzheimer's disease, Parkinson's disease, stroke and other brain disorders. At the same time, researchers have been criticized by other scientists and the public who argue that some therapies put patients at disproportionate risk because they have been rushed to clinical trials without sufficient preclinical findings of safety and effectiveness.
The ASNTR recommendations support existing federal regulations while addressing new concerns associated with neural transplantation and repair, including:
- Duration and type of preliminary studies (laboratory and/or animal studies) required before it is ethical and appropriate to study a treatment in human beings
- Design of clinical studies for cellular treatments and for gene therapy. For example, the authors write, a gene therapy study for a fatal disease with no treatments might be ethically acceptable with more unknowns and possible toxicity than a similar study for a nonfatal condition with treatments available.
- Use of control groups, which might include "sham" surgery or "imitation operations" to control bias.
- Medication management that balances patient safety with optimizing study of a new treatment's outcome.
- Conflicts of interest. The authors recommend investigators fully disclose ownership of patent interests, large stock or stock options, paid consultant positions, or membership in scientific advisory boards of companies that might benefit from a study.
- Appropriate publication of results. The authors hope the recommendations will carry weight with local Institutional Review Boards that oversee clinical studies, Dr. Redmond said, making it more difficult for unethical or hurried researchers or companies to potentially harm patients enrolled in trials