Stem cells in action

The use of stem cells in clinical trials
Stem Cells and the treatment of genetic diseases
Therapeutic use of embryonic stem cells

To date only one clinical trial using human embryonic stem cells has received FDA approval for a phase I study. The study is being run by Geron, based in the US. The study group are people with thoracic spinal cord injuries with complete loss of locomoter and sensory activity below the site of injury. Interestingly the trial is limited to patients with recent injuries (within 14 days), as animal models have shown that stem cell treatment is ineffective at later time points, most likely due to scar tissue formation. The trial has been recently halted as the FDA scrutinise new pre-clinical data. Although Geron are using human embryonic stem cells, the actual treatment is with in vitro differentiated cells, that is, the stem cells that have already been switched into cells of the neural lineage by scientists in the laboratory. This trial has been recently halted as the FDA scrutinise new pre-clinical data.  

Stem cell tourism

Many countries with lower regulatory or ethical approval standards already have clinics advertising stem cell therapies for a range of conditions. Many (if not all) of these are offering unproven therapies (there may not even be any prior animal studies testing safety or efficacy). It seems that the promise of improvement, even if small, is enough to tempt those in desperate need. The dangers associated with these types of unproven treatments are very real and have already been shown in at least one well publicised case. A young boy received an experimental stem cell transplant in Russia to treat a rare inherited disease (Ataxia Telangiectasia). He was later found to have developed tumours in his brain and spinal cord which had originated from the transplanted donor cells.

 

The future

www.clinicaltrials.gov and the Australian clinical trials website provide regularly updated information about federally and privately supported clinical research in human volunteers. In Australia we have the National Stem Cell Foundation and Stem Cells Australia.

 

Information about clinical trials in Australia and New Zealand can be searched 
using the Australian and New Zealand Clinical Trials registry. An international website with clinical trial information is Clinical Trials Portal.

The International Society for Stem Cell Research has published guidelines for patients and families interested in stem cell research and treatments. This can be downloaded here

If members of the public are concerned or have questions about any of the issues raised here, they can contact the Australasian Society for Stem Cell Research at info(at)asscr.org.  

Useful websites:

National Stem Cell Foundation

US government clinical trial database 

International Society for Stem Cell Research

Osiris therapeutics

Mesoblast 

Written by Gary Brooke, PhD
Mater Medical Research Institute
Raymond Terrace
South Brisbane
Queensland 4101