Doctor: We can change the world with human embryonic stem cells

Editor’s note: This week, The Chart is taking a closer look at the most important health stories of 2010. One was stem cell research, a topic with which Dr. John McDonald  is very familiar. McDonald is director of the International Center for Spinal Cord Injury at Kennedy Krieger Institute in Baltimore. The longtime stem cell researcher, who was one of Christopher Reeve’s physicians, provides his perspective on the first human clinical trial of embryonic stem cell research.

By Dr. John McDonald
Special to CNN

We are living in an amazing time.

This year marked what just a decade ago many believed would be an impossible feat - the first human has been injected with cells from human embryonic stem cells (hES). hES cells, and embryonic stem cells in general, are one of the greatest scientific tools for discovery of the 21st century.

The clinical trial brings together the best we have to offer in central nervous system research to address the difficult problem of spinal cord injury.

It is a phase I open label safety trial. To be included, individuals have to have suffered a complete thoracic spinal cord injury, which means no movement or sensation below the injury level. The injury to the spinal cord must have occurred between the third and 10th thoracic neurological levels, and the individual has to be injected with the stem cell therapy, called GRNOPC1, within seven to 14 days after the injury.

Many of my fellow researchers expect that the trial will be safe because previous trials have demonstrated the safety of cell transplantation in the injured central nervous system.

Following this initial safety trial, it will take three to five years to complete a trial that evaluates the effectiveness of this approach. If successful, a FDA-approved cellular treatment for spinal cord injury could be developed and on the market within five to seven years.

This trial is tremendous progress forward given the discovery of the hES cell in 1998. Researchers advanced at an incredible pace while facing political barriers that severely limited federal funding for research. This success is based on a quarter century of progress across many fields. It represents over a billion dollars of investment, largely provided by the National Institutes of Health, and the hard work of millions of American workers.

As a physician working with patients at the International Center for Spinal Cord Injury at Kennedy Krieger Institute, I witness their unbelievable dedication and perseverance throughout treatment. This trial means much more to my patients, and to all those living with spinal cord injury, than one single treatment option. It represents hope and the indication of near-term treatments. It is the tip of the iceberg. During the next five years, we can expect that many strategies for repairing the central nervous system will come to a similar stage of human testing.

Although this trial is evaluating transplantation of hES cells, the greatest effect of these cells will not be as a direct treatment. It will be from the use of hES cells as a scientific tool of discovery, accelerating progress across multiple fields and leading to effective repair and recovery. The human genome sequencing project is similar to the tools offered by hES cells, and like the human genome project, hES cell tools will change our world.

What we collectively decide today will affect our children and generations to come. This is an awesome responsibility, and one that requires risk and investment. The primary risk is of the unknown, and this is not new or unique to hES cell technology. Remember the similar fears surrounding the idea of sequencing the human genome? Imagine where we would be today if we made the wrong decision out of fear or from personal ethics?

We must not fear knowledge. We must be decisive and clear in purpose and allow science to benefit from all of its tools.

Learn more about Dr. John McDonald's work at www.spinalcordrecovery.org.