Personalized medicine may be in our future

Approach is hindered by higher cost of development

August 25, 2006|By Jon Van. | Jon Van.,Chicago Tribune

It's a dream called personalized medicine: Genetic and technological advances offer the tantalizing prospect that doctors can identify maladies with such precision that their therapies nearly always work.

An example of personalized medicine is Herceptin, a breast cancer treatment that blocks cancer-promoting proteins. Herceptin can be effective when given to the 20 percent to 30 percent of women whose tumors produce high levels of targeted proteins, and those women can be identified by genetic testing before treatment.

But most breast cancer patients would not benefit from Herceptin therapy, so it is not routinely prescribed.

This is the medical and business quandary facing personalized medicine. It is opposite to the drug industry's approach of generating profits from blockbuster, one-size-fits-all prescriptions.

The smaller the potential number of beneficiaries, the more expensive it is to develop drugs and procedures for them, which can mean that there are therapies out there ready to help patients that cannot reach them.

Medical researchers believe that many, perhaps most, treatments of ailments from cancer to heart disease could be similarly enhanced through greater knowledge of an individual's genetic profile. In some cases there is a recognition of which types of drugs might work with subsets of patients, and in many other cases even those elementary issues are not understood.

Experts say the mainstream medical and pharmaceutical industries are aware of personalized medicine and in many cases support the approach, but that it is so early in the process of discovery that they are not necessarily ready to delve in.

"It's not easy to bring diagnostics and therapeutics - the hallmark of personalized medicine - together," said Edward Abrahams, executive director of the Personalized Medicine Coalition, a nonprofit group whose members include biotech and big pharmaceutical firms. "Typically, they're developed by different companies. Combining them means you have to figure out how to share value. But we're optimistic the rate of progress will accelerate in the near future."

Because of economic considerations, observers are deeply pessimistic that the approach will take hold.

"There are lots of technologies that work, but they aren't available because they're not profitable," said Burton Weisbrod, a Northwestern University economics professor.

In the meantime, early in the process of developing personalized medicine, it is the researchers who are taking a lead, looking beyond their laboratories to figure out ways to get treatments to patients.

Dr. Edwin Stone, for example, is a widely respected scientist who studies genetic connections to blindness. For more than a decade Stone and his colleagues at the University of Iowa have characterized genetic abnormalities associated with eye diseases.

"We'd do our research, write the results and publish in medical journals," said Stone, sitting in his ophthalmology lab on the Iowa City campus. "Once we published, we figured our job was done. Someone else would develop tests and market them to physicians."

But that never happened, and it took a while for economic reality to dawn on the academic scientist.

"The diseases we study are too rare," Stone said. "There's no money to be made because the market is just too small."

Rare diseases are formally defined as those that afflict fewer than 200,000 people. But while each single malady may be rare, taken together they affect 25 million Americans.

Stone and his colleague, Dr. Val Sheffield, decided the only way their research findings could help patients would be for them to create and offer genetic tests to primary-care physicians

Their goal is to develop a clinically useful test for every eye disease that has a known genetic connection.

However, getting into the testing business, even on a nonprofit basis, presents challenges that can be daunting.

Stone's university-based testing laboratory, operating with a $5 million grant from the Carver family of Muscatine, Iowa, charges patients only for materials and technician time needed to perform tests. Stone tries to get those costs below $500 per test rather than the thousands it typically costs to do such work in a research setting.

Thomas Casavant, a professor of bioinformatics and computational biology in Iowa's engineering college, has a team devoted to automating processes for Stone's lab in order to reduce costs and increase research output.

Another example of personalized medicine, according to a recent report in the Proceedings of the National Academy of Sciences, is bucindolol, a drug once thought to be useful in treating heart failure that initially failed but now may find new life.

While bucindolol flunked clinical trials with heart failure patients, two academic researchers found that it helps a subset of patients who share a particular genetic variation.

Baltimore Sun Articles
Please note the green-lined linked article text has been applied commercially without any involvement from our newsroom editors, reporters or any other editorial staff.