Making the Promise a Reality
Authors: Inder M. Verma
Journal: Molecular Therapy (2002)

Last week I attended the 54th Annual National Hemophilia Foundation Meeting in Orlando, Florida, joining hundreds of parents of children with hemophilia, adults severely debilitated by bleeding disorders, nurses, caretakers, healthcare providers, research scientists, and, of course, little boys with the disease. The mission of the scientists in attendance was to educate the concerned groups and offer them hope for a future free of the consequences of the diseases. Patients who are now grandfathers spoke about the tremendous progress in the treatment of bleeding disorders from the days of blood transfusions, fear of devastating viral infections, and the use of purified recombinant proteins. Both the longevity and the quality of life of affected individuals have steadily improved in the past few decades. The prospect of gene therapy as a new modality in the armamentarium of treatments for bleeding disorders was enormously exciting for those in attendance. “The thought that my son could get a single injection of the gene to make him normal is just unimaginable,” said one mother. My reaction to her response was to ask myself whether we were making unrealistic promises. The preclinical data in rodent and canine models is spectacular—a single injection of a viral vector and in some cases naked plasmid DNA leads to normal bleeding times in otherwise diseased animals. Clearly, these are exciting results on which to base clinical trials. But the public does not always distinguish the promising findings generated by scientists in experimental model systems from the reality of clinical trials in humans, which are fraught with unexpected adverse events as we have seen over the past couple of years. Are we giving false hope to the patients? I believe not—it is essential that we convey to the patients and their families the excitement we feel for the experiments we do. Despite the fact that most speakers emphasized the preliminary nature of their data from animal experimentation, many in the audience wanted to believe that it was only a matter of time before similar treatments worked in people. This is simply human nature, and no degree of caution or careful caveats will overcome that. It is easy to see how hope can beget hype! The gene therapy community, despite what its critics may say, must not shy away from enthusiastically communicating their research findings to the public and patients. But, the path from the test tube to the bedside is not a straight one and we must make every effort to emphasize the uncertainty of the process of discovery to cures for human disease. At the same time, we need to keep the patient population informed of new discoveries—not to give them false hope, but to make them aware of new therapeutic possibilities. The patients can engage their physicians and primary care givers to keep them informed of new developments from these discoveries. Gene therapy offers an entirely new modality of medicine to cure diseases without compromising the quality of life. It is a young field, but one that has the potential to influence the vast spectrum of human disease ranging from genetic and degenerative diseases to cancer. I think of those many boys with hemophilia at Disneyworld in Orlando, unabashedly wishing each other “a magical day,” and who one day may have a quality of life like any other normal child. That is the promise of gene therapy that we must work to make a reality in the coming years.