Gene therapy for hearts Competition: Scientists and biotechnology companies are in a high-stakes race to treat heart disease with gene therapies that stimulate blood vessel growth.

November 01, 1998|By Mark Guidera | Mark Guidera,SUN STAFF

It's been almost a year since Dr. Ronald G. Crystal and Dr. Todd Rosengart of New York Hospital-Cornell Medical Center opened the chest of a 60-year-old man and did what no surgeon had done before: They injected a gene-loaded solution into the man's heart in the hope of stimulating blood vessel growth.

The goal of the pioneering treatment is to alleviate a recurrence of the pain and heart damage that can result from blocked coronary arteries.

Aside from making medical history, the doctors touched off a crowded and intense race to develop gene therapies for heart and vascular diseases.

Almost a dozen biotechnology companies have entered this high-stakes race, and gene therapy experts think even more will begin cardiovascular-related gene or protein therapy programs in the next several years.

The field includes two Maryland companies, privately held GenVec Inc. of Rockville and Vascular Genetics Inc., which is partly owned and bankrolled by publicly held Human Genome Sciences Inc., also of Rockville.

Unheard of a few years ago, gene-based drugs have emerged as one of the most promising the therapies for treating heart and vascular diseases. Some experts believe gene therapies for heart disease are most likely to be the first to be mass commercialized.

The aim of the treatments is to use normally occurring genes to stimulate the body to produce the proteins responsible for blood vessel growth, known as angiogenesis.

The body produces the proteins in response to artery blockages and heart damage, said Crystal, but in quantities too small to benefit those suffering from heart disease.

By getting the body to grow new vessels, it is hoped that the heart will be spared further damage from blocked arteries and other conditions that rob it of oxygen and other nutrients.

Some experts are referring to this experimental treatment as a "biological bypass."

If human experiments such as those conducted by Crystal, Rosengart and others continue to show promise, gene therapy for heart disease could revolutionize cardiology, experts say.

"I believe this could potentially provide a quantum leap in how people with cardiovascular diseases are treated in the future," said Paul Fischer, president and chief executive officer of GenVec.

At stake is a huge potential market. Heart disease remains the leading cause of death annually in the United States.

More than 58 million Americans have cardiovascular disease, according to the American Heart Association.

Almost a third of them, 14 million, have coronary heart disease, a debilitating condition that can lead to heart attack and death. Heart disease is considered epidemic among Westernized nations, so the worldwide potential market is much larger.

The heart association estimates that more than $171 billion is spent annually in the United States to treat people with heart and vascular troubles.

Biotechnology company executives say it's too early to calculate how much gene therapies for the heart might cost if they are approved for commercialization by government regulators, including the Food and Drug Administration.

Also, it will be several years before company researchers have a clear idea of what types of heart and circulatory conditions the treatments might best treat and hence the size of the potential market.

But there are indications of how valuable the pioneering technology is:

Drug giant Warner-Lambert Co. struck a deal with GenVec in September 1997 to license rights to its BIOBYPASS cardiovascular gene therapy. The deal is worth up to $100 million if the treatment is approved for marketing. Warner-Lambert has paid GenVec $13.5 million and purchased $2 million of the company's stock.

Human Genome Sciences entered a joint venture with St. Elizabeth's Medical Center in Boston and Cato Holding, a Research Triangle, N.C.-based company in November 1997 to launch Vascular Genetics Inc. Human Genome agreed to supply $600,000 in start-up funds. Under the venture, Human Genome will assist Dr. Jeffrey Isner, a cardiologist and gene treatment pioneer at St. Elizabeth's Medical Center, in developing a gene treatment for vascular disease.

German pharmaceutical giant Schering AG bought a 20 percent equity stake in Collateral Therapeutics Inc. of San Diego in May 1996 for $5.7 million and agreed to underwrite $5 million annually in research and development costs in exchange for rights to market GENERX, Collateral's leading gene treatment for coronary heart disease. Collateral also gets $20.5 million for regulatory approval to market angiogenic gene treatments and sales royalty payments.

Surgical instrument maker Boston Scientific Inc. of Natick, Mass., bought privately held CardioGene Therapeutics in July for an undisclosed amount.

At the time of the purchase, Art Rosenthal, chief development officer for Boston Scientific, said the company believes "gene therapy is the next frontier for treating patients suffering from blocked or completely occluded arteries, as well as a host of cardiac problems."

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