Our Bodies, Our Cells?

By Robert Oldham. Dr. Oldham is chairman of the South Carolina Biotechnology Association and CEO of Cancer Therapeutics Inc.

It is now possible to insert one person's gene into another person's cells. It is also possible to grow an individual's cells outside the body in large numbers -- to be therapeutically readministered to the same patient.

Soon, we will be able to create tissues and organs from one's own cells to be used for the therapeutic benefit of that individual. Just as a suit can be tailor-made for an individual, so too will medicines be "customized" for the individual patient. Such medicines will be much more effective than today's broadly targeted, mass-market drugs -- and will have fewer or no side effects, because most will use single-function proteins or cells that already exist in the body.

How will these new therapies be regulated by the Food and Drug Administration? Will they have to run the gauntlet of today's hugely expensive and time-consuming clinical trials? Curiously, the answer may be found in good measure by answering the question: Who owns an individual's genes, cells and proteins?

Under the system designed for mass-marketed drugs, clinical trials are required to show that an agent is both safe and effective -- that it works in a statistically significant number of people better than a placebo or the accepted standard treatment. In contrast, the new biological therapies will be more like chemical surgery. Every patient is unique; nobody considers it appropriate or workable to impose a single regulated standard on surgeons. Indeed, they aren't doing their job if they don't tailor each procedure to the individual patient.

The regulatory question intersects with a growing body of law that holds that cells, tissues and a person's unique proteins are, to some as yet uncertain degree, private property. One of the most spectacular cases arose in the 1980s after Dr. David Golde, then of the UCLA Medical Center, developed a specialized cell line from the spleen of a patient, John Moore, who was found to produce a large volume of the proteins that speed the growth of bone marrow. Both doctor and patient eventually came to envision substantial profits from using the cell line to produce various proteins that might become important medicines.

Mr. Moore, the patient, sued, alleging that the cells, property rights and intellectual property actually belonged to him and requested that a licensing fee or royalty fee come to him from any profits that might ensue from the use of his cells. In 1990, the California Supreme Court ruled against him on the profit issue but upheld a patient's general right to withhold his or her tissues from commercialization.

Extended to the treatment realm, such decisions may have the happy effect of drawing a circle of autonomy around the budding new doctor-patient relationship in the era of genetically based medicine. Trying to insert a new layer of regulation between a patient and the use of his or her own cells would run smack into a fundamental problem of property rights. Preserving these property rights will be a good thing for the advancement of medicine.

Biotechnology companies are now highly focused on the individual characteristics of the genome, including deciphering single nucleotide polymorphisms (SNPs) as a way to determine how to make drugs work better in smaller and smaller subsets of patients down to the individual human being. These SNPs are the key to understanding individual susceptibility to disease and the custom tailoring of therapy.

Furthermore, the recent demonstration that stem cells can be developed both from the bone marrow and from fat cells indicates that ultimately we will learn how to develop a whole series of cellular therapies from an individual's own cells. It may be possible, in the very near future, to grow lifesaving stem cells from an individual to be used by an individual or closely related family member.

In the past, such arcane considerations of property rights over biological materials were passed over rather lightly by physicians, scientists and the companies that used these materials. Often, it was assumed that simple informed consent gave adequate rights to commercial development of tissues.

In addition, many of the individuals who "donated" such tissues were no longer living. They were in no position to express an ongoing interest in such property rights.

Nowadays huge banks of tissues, cells and genes exist and biotechnology companies are racing against time to develop genetic profiles and databases utilizing these materials. Much remains to be decided with regard to property rights for genetic materials and perhaps we will have to wait for the courts to properly adjudicate in this area.

But it is readily within the purview of the FDA to remove one large area of uncertainty that could stymie the emergence of these lifesaving therapies. A good model is the way blood banks are regulated today. It has traditionally been deemed acceptable to receive one's own blood, the blood of one's family, and appropriately typed blood from other individuals without efficacy studies. Simply stated, it is sufficient to assure that blood is safe and that the procedures for extracting, storing, handling and distributing it are safe. This is the realistic standard that should apply to individualized cellular therapy.

What's needed now is some formal recognition from the FDA that its role is limited to assuring the safety of the procedures when an individual requests that his own cells be readministered. Our cells belong to each of us. The FDA should put itself on record supporting that fundamental proposition.