Disease Sufferers Lobby at Biotech Conference

Posted on: Friday, 11 May 2007, 21:00 CDT
http://www.redorbit.com:80/news/heal...ource=r_health

BOSTON _ Patients offer poignant reminders of the high stakes involved in connecting the biotechnology dots.

After all, for them it's a matter of life and death.

Carrie Smith was a country singer in Nashville, Tenn., with a new baby and a promising career when doctors told her in 2002 that multiple sclerosis was causing her excruciating leg pains, severe dizzy spells and other symptoms.

"I wasn't able to sing. I wasn't able to be a good wife. And I wasn't able to care for my daughter," Smith said.

Smith eventually began treatment with Rebif, a medication marketed by EMD Serono Inc. and Pfizer Inc. The results were dramatic, she said, adding that she felt confident enough to carry her child again and resume playing music.

"I feel like my life was given back to me," Smith said.

Celebrating breakthroughs in basic research and chasing big business deals are the usual focus of the nation's largest biotechnology conference. But people suffering devastating diseases sought to use BIO 2007 this week to lobby for quicker access to cures. Indeed, they were invited.

Actor Michael J. Fox, who started a foundation that funds research after he was stricken with Parkinson's disease, challenged the industry to remove obstacles slowing or even blocking new cures. He argued that more progress should be expected from an enterprise driven by brilliant minds and backed with billions of dollars.

"American taxpayers are funding the greatest discovery engine in the world, yet we fail to provide incentives for our scientists to convert their relevant findings into improvements in human health," said Fox, who lives with recurring tremors, in a keynote address at BIO 2007.

Lori Lober, a Kansas City, Mo., cancer survivor, echoed Fox.

Seven years ago doctors told Lober she had an advanced case of breast cancer that had spread. Chances of survival, they said, were slim.

Lober overcame the odds thanks to treatment that included a drug produced by biotechnology industry pioneer Genentech Inc.

"The traditional therapy offered me no hope," said Lober, who later founded the Touched by Cancer Foundation.

It remains to be seen how well the words of Smith, Fox and Lober will be heeded.

The BIO conference caters to companies touting biotech breakthroughs with hopes of attracting investor cash and state officials pitching incentives with hopes of landing economy-boosting prospects.

This year was no exception. BIO 2007 drew more than 22,000 officials from around the world. Missouri Gov. Matt Blunt and U.S. Sen. Pat Roberts of Kansas were among those representing 48 states and numerous foreign countries that jostled for attention on a sprawling convention floor.

Making the biggest economic-development splash was Gov. Deval Patrick of Massachusetts, a state already rich with top universities, a bustling base of venture-capital firms and a powerful hub of biotech companies.

Patrick announced a plan to channel $1 billion over 10 years to build new life sciences centers, provide research grants and distribute new batches of stem cells to scientists.

Kansas officials, meanwhile, drew attention to their $588 million initiative.

Blunt met with company officials and talked up his plans to channel more money toward higher education and research.

Such efforts are expected to help the economy. But it is also important to remember the ultimate aim of many biotechnology endeavors, Blunt said in an interview at Missouri's pavilion playing up a "Built for Biotechnology" theme.

"The benefits for mankind with this technology are so tremendous," he said. "That is something we should never lose sight of. To help patients, that is what this is really about."

Cracking the genetic code with the Human Genome Project and other biotech advances have spawned an era of previously unimaginable possibilities, said Greg Simon, president of advocacy organization FasterCures.

Cancer, amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease), Parkinson's and cystic fibrosis are among the maladies that innovations emerging from medical research labs are likely to fell, Simon said.

"In the lifetime of my generation, most of the diseases we are dealing with, if not cured, should be turned into treatable chronic diseases," Simon said.

But while the science is promising, Simon said, other factors were worrisome:

_Researchers must do a better job of collaborating.

_More money must be directed to translating discoveries into therapies.

_Greater attention must be paid to filling gaps in a system that can require 17 years to take an idea for a treatment to a new drug.

"The system we have won't get there," Simon said. "We have to have a new system."

Leaders at the Ewing Marion Kauffman Foundation in Kansas City also have identified the same roadblocks. They are joining Simon and others in campaigns to remove them.

The federal government has been the dominant source of funding for medical research. But new organizations are emerging with the power to force changes, said Lesa Mitchell, a Kauffman Foundation vice president who helped organize and moderate a panel discussion at the conference.

The Bill & Melinda Gates Foundation, The Michael J. Fox Foundation, the Stowers Institute for Medical Research in Kansas City and various initiatives of former Wall Street financier Michael Milken are among the philanthropic ventures pumping major money into medical research. Some projections, Mitchell said, indicate these private sources could soon surpass federal funding.

Put simply, those who write the checks can write new rules.

With federal money more challenging to obtain, scientists will respond to foundation requirements such as discoveries being announced more quickly and widely. Foundations also are demanding focus on cutting-edge areas rather than well-trod paths likely to produce successful experiments but few major advances.

"We are seeing the philanthropists pushing the envelope," Mitchell said.

Both patients and philanthropists are getting more attention from the industry.

Jim Greenwood, chief executive of the Biotechnology Industry Organization, made sure foundations and patients had prominent slots at this year's conference. He also included them in forums aimed at matching companies with investors and peers who might work on new projects.

"We are giving people the opportunity to custom-make deals in a way I don't think is happening right now," Greenwood said in a recent interview.

Foundations want to pay for cures, disease groups need help and biotech companies must pursue the most lucrative projects if they hope to survive. Mixing the three groups can provide new solutions to the problems they all face.

"Put all those things together and you get some really interesting combinations," Greenwood said.

Darren Baker suddenly saw the significance of his life's work in a new light when he became a patient.

A researcher at Biogen Idec, a Cambridge, Mass., biotechnology company, Baker was diagnosed with non-Hodgkin's lymphoma after seeing his doctor in 2003 about a pain in his chest.

Baker received treatment with Rituxan, a drug developed by his employer.

He described his time as a patient as a "quite eye-opening" experience and the source of a newfound motivation for his own scientific work.

"Nothing drives a researcher more than to see a drug in the clinic helping people," Baker said. "I hope biotechnology continues to deliver on the promise."