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Testimony of Michael J. Werner, Esq.

On behalf of the Biotechnology Industry Organization (BIO), regarding the Proposal That Calls For A Moratorium on Some Human Gene Therapy Protocols before the NIH Recombinant DNA Advisory Committee.</p>

Good afternoon. My name is Michael Werner. I am bioethics counsel for the Biotechnology Industry Organization (BIO). BIO represents more than 900 biotechnology companies, academic institutions, and state biotechnology centers engaged in biotechnology research on medicines, diagnostics, agriculture, pollution control and industrial applications.

The proposal before you today would impose a moratorium on gene transfer technology protocols using viral vectors, unless the protocol is a treatment of last resort for a life threatening illness.

This proposal is ill advised and shortsighted. It will delay vital research that holds great promise to treat many deadly and disabling diseases. Adoption of the proposal will simply mean more suffering. We urge you to vote against it.

Gene Therapy's Promise

Gene therapy is focused on finding ways to introduce genes into cells in order to correct a cell malfunction, to add a new function to a cell, or, in the case of cancer, to add a gene to a cancer cell that causes that cell to die. A variety of different types of gene delivery systems are used to deliver genes into target cells. Some systems are made from modified viruses, which appear to be very efficient at getting genetic information into cells. There are many different types of viral based systems. Each has its own characteristics and safety profile. The goal is to match the appropriate delivery system with the gene, the target cell and the disease, in order to develop effective therapeutics.

There are many potential medical benefits of finally knowing all the genes that make up the human being. Gene therapy allows us to actually put that knowledge to work, by creating products based on these genes. There are about 5000 genetic diseases including hemophilia, Huntington's disease and cystic fibrosis. For patients with these diseases and others, gene therapy offers hope that finally their diseases can be efficiently controlled or cured, not just diagnosed.

Hundreds of clinical trials have been initiated over the years as government and private industry have worked together to research numerous ways for this technology to reach its potential.

Many of the gene therapy clinical trials that are active today are in an early stage of development. These early phase studies (Phase I/II) are specifically designed to evaluate the safety of the vector under investigation. These trials continue to be in early development because researchers are exploring their options with indications, routes of administration, dosing regimes, patient populations, combination therapies, and novel vectors. Many studies also are designed to evaluate the maximum tolerated dose, with the expectation that adverse events will occur at some dose level. This approach is standard in any drug development process.

Recent reports indicate that this hope for gene therapy is well placed. For example, several gene therapy products have demonstrated safety, and have begun to demonstrate efficacy in cancer clinical trials. In addition, recent studies in France have shown that infants suffering from Severe Combined Immunodeficiency Disease (SCID) have had their immune systems completely restored by gene therapy.

Moreover, we all saw the front-page stories in many newspapers last week that described preliminary data about how gene therapy could help to the thousands of Americans who suffer from hemophilia. Ironically, since the pending proposal only allows gene therapy using viral vectors if it is a treatment of last resort for a life threatening illness, some people with hemophilia might not be able to receive this treatment if the proposal is approved.

Parallels To Other Technology

History is filled with examples of scientific and medical discoveries that took years, if not decades, to perfect. For example, in the 1970s and 1980s interferons and monoclonal antibodies were touted as wonder drugs against a range of diseases, particularly cancer, because of their stunning success in laboratory experiments. Both were tagged "magic bullets." These bullets, however, were blanks initially because of difficulties in getting them to reach their targets in humans.

After years of research, interferons were first approved in 1985 for hairy cell leukemia. During the next 15 years, as research progressed, these natural human proteins emerged as effective treatments against a whole range of viral diseases and cancers, including melanoma, lymphoma, hepatitis and multiple sclerosis. The discovery of therapeutic applications for interferons is far from over.

Monoclonal antibodies also were a disappointment after being touted for their extraordinary cancer fighting abilities. It took almost 20 years to figure out how to get them to work in humans. The first antibody drug for cancer finally was approved for patients in 1997 for non-Hodgkin's lymphoma. Since then antibodies have proved effective for breast cancer, inflammatory bowel disease and respiratory viruses in children. Antibodies in development target an even broader range of diseases, from asthma to leukemia to prevention of organ transplant rejection.

The years of persistence, dedication and investment of biotechnology companies are paying off for the millions of patients who benefit from interferons and monoclonal antibodies. Biotechnology companies are committed to spending the time and money to bring gene therapy to patients. We believe gene therapies will work. It is not surprising that it is taking time. It is a challenging undertaking.

Oversight of Gene Therapy

Biomedical research has already brought us 100 new drugs and vaccines and helped millions of people who have serious and debilitating illnesses.

The biotech industry is committed to reaching future scientific advances cautiously and safely. Anything less would be a gross disservice to the patients we are trying so hard to help, an abandonment of our own principles, and a clear violation of the established regulatory framework.

The tragic death of Jesse Gelsinger has raised questions about the oversight of gene therapy. It is important to remember however that gene therapy is subject to greater oversight than virtually any other technology. The FDA, through its statutory role as the regulator of drug development, the NIH in its role as overseer of federal research dollars and the RAC as the forum for public discussion, have served to protect patients while ensuring that important research moves ahead.

BIO Recommendations for Gene Therapy Oversight

BIO looks forward to working with the FDA, NIH, and the RAC as they develop initiatives to ensure that clinical trials are performed responsibly and public confidence is justified.

As you know, this was the subject of discussion here yesterday. BIO has proposed a responsible oversight system for gene therapy that would provide adverse event reports to the NIH and FDA through a harmonization of FDA and NIH reporting rules. Under our plan the respective and complementary roles of the agencies would remain the same. FDA would remain the agency with regulatory authority and the ability to approve a trial or put a trial on hold. The RAC would maintain its important role as an educational and advisory body.

As you know, the industry's willingness to provide adverse event data to the RAC is contingent upon an agreement between NIH and industry that would memorialize how the data will be used. We urge OBA to work with FDA and industry to develop a process that will provide the RAC with adverse event data it needs to do its job effectively and ensure that this information will be used appropriately. BIO reiterates its offer to work with OBA and FDA on this matter. We look forward to this collaboration.

A useful first step would be an analysis and report of data from gene transfer protocols to determine the nature and extent of adverse events that have occurred and how they have been addressed. Data Dr. Patterson presented yesterday should be included.


The promise of gene therapy technology has not yet been realized. However, despite the recent tragedy at UPenn, data obtained in gene therapy trials thus far are encouraging. Biotechnology companies involved in gene therapy development will continue to present to the patients enrolling in clinical trials and the public a practical and balanced assessment of the benefits and risks of the technology.

The biotechnology industry is committed to providing the resources necessary to fully realize the promise of gene therapy for the treatment of serious medical conditions such as cancer, cardiovascular disease, and genetic and metabolic diseases. It is vital that patients with these conditions have access to novel and innovative therapies.

Although improvements can and will be made in the oversight system for gene therapy, a moratorium is not appropriate. It will not make gene therapy safer. It will simply delay scientific progress, deny treatment to patients and halt a promising area of research. The proposal is unnecessary and dangerous. Stopping gene therapy will hurt patients, not help them.

Thank you.