In this third in a series of podcasts focused on rare disease patient advocates jointly produced by The Burrill Report, the California Healthcare Institute, and the Children’s Rare Disease Network, we speak to Susan Cornelius, business advisor to The Nicholas Conor Institute for Pediatric Cancer Research. When her granddaughter struggled against cancer, she was frustrated that new technology was unavailable to her and that all doctors could offer were surgery, radiation or chemotherapy – what she characterized as cut, burn, or poison the child for a disease, in her granddaughters case, that was genetically-based. Cornelius lost her granddaughter to cancer in 2008, but now works to bring about changes to the way research is conducted.

 

In this second in a series of podcasts focused on rare disease patient advocates jointly produced by The Burrill Report, the California Healthcare Institute, and the Children’s Rare Disease Network, we speak to Beth Anne Baber, CEO of The Nicholas Conor Institute for Pediatric Cancer Research. When Baber’s son Nicholas was diagnosed with stage 3 neuroblastoma she and her husband, both cancer researchers, fully understood the seriousness of the diagnosis. Because of the location of the tumor, radiation and surgery were not options. But as they explored treatments they discovered a startling fact. Most cancer therapies used to treat children were about 25 years old and drug companies weren’t generally pursuing new therapies for children with various cancers because the markets simply weren’t large enough. Her institute offers a new model. It seeks out industry partners to help develop specific technologies it wants to bring into the clinic while helping to fund that work.

In this first in a series of podcasts focused on rare disease patient advocates jointly produced by The Burrill Report, the California Healthcare Institute, and the Children’s Rare Disease Network, we speak to Debra Miller, president and founder of Cure Duchenne. When Miller’s five-year-old son was diagnosed with Duchenne Muscular Dystrophy, she faced grim news. The progressive muscle loss caused by the disease typically leaves these children wheelchair-bound by age 10 and robs them of their lives by 20. As she and her husband explored treatment options and ways they could become to raise money for research, they found that existing organizations were often focused on palliative care and focused on academic research rather than working with industry. Miller and her husband launched Cure Duchenne to help fund promising research at biotech companies in the hopes of seeing these therapies developed and commercialized. We spoke to Miller about her son’s story, new approaches to treating Duchenne, and the problems of funding cutting-edge research

 

The second quarter saw the markets come roaring back as the Dow Jones Industrial Average rose 12 percent and the Nasdaq Composite Index soared 20 percent.  The biotech sector didn’t fare as well, but managed to end the quarter in positive territory as the Burrill Biotech Select Index gained 3 percent. Nevertheless, some biotech stocks, such as Dendreon, showed they could be explosive, while others just seemed to blow up. We spoke to Peter Winter, editor-in-chief of The Burrill Report about the sector’s performance in the second quarter, the financing and dealmaking activity, and what to expect in the second half of 2009.

For four decades, an informal network of public-private partnerships has collaborated to support the advancement of the life sciences. The result has been jobs, wealth creation, improved healthcare, and prospects for new sources of energy. But those partnerships are cracking under pressure from a range of forces including constrained public budgets, the turmoil in capital markets, and the prospects of healthcare reform. Corey Goodman, former president of Pfizer's Biotherapeutics and Bioinnovation Center and an adjunct professor at the University of California, San Francisco, will be featured as a panelist at The first BioEconomy Summit, which will be held June 25 in Silicon Valley. We spoke to Goodman about the pressures on the bioeconomy, what needs to be done to make sure it continues to grow, and why policymakers should care about its future health. 

 

More information on The BioEconomy Summit can be found on the event’s website.

 

Since the mapping of the human genome in 2003, the pace of new discoveries about the genetic underpinning of diseases and the ability to use this information to better segment patients for targeted therapies has advanced at a dizzying pace. The emergence of personalized medicine as a powerful force reshaping healthcare is starting to change everything from the way drug companies develop therapies to the way doctors treat patients. But barriers remain from realizing the benefits of this new approach. We spoke to Ed Abrahams, executive director of the Personalized Medicine Coalition about “The Case for Personalized Medicine,” a new report from the organization. Burrill & Company, publisher of The Burrill Report, is a member of the Personalized Medicine Coalition. We talked to Abrahams about the promise of personalized medicine, hurdles that need to be overcome, and the critical role it could play in healthcare reform.

 

In just a matter of days, the biotechnology industry’s attention will shift to Atlanta. No, we’re not expecting a major pronouncement from the Centers for Disease Control and Prevention. Instead, the 2009 BIO International Convention will kick off in The Big Peach May 18. It is the largest annual gathering for the industry drawing people from 60 countries. The event this year comes at a time of great change for the industry, so we thought we’d check in with Peter Winter, editor in chief of The Burrill Report, about the mood going into the convention, the hot topics this year, and what to look for at the conference.  

As the swine flu spreads across more and more countries, the number of people infected with the disease for now remains low. But public health officials are worried that a deadly pandemic may be unfolding before our eyes. The World Health Organization has pushed its influenza pandemic alert to the second highest level and instructed all countries to immediately activate their pandemic preparedness plan. Alexandra Stern, associate director of the Center for the History of Medicine at the University of Michigan, has conducted extensive research into the response of U.S. cities to the 1918-1919 influenza pandemic. We talked to Stern about the challenges of containing a pandemic in our interconnected world today, about why efforts to contain the 1918 pandemic failed in some major cities, and what lessons public health officials can learn from the past.

The Dow Jones Industrial Average posted its worst January performance ever and worst February since the Great Depression, and then in March it did an about face and generated one of its biggest one-month gains in years. The IPO market still remained closed to biotechs, but on the financing side the numbers actually looked pretty good in the first quarter as the industry posted a 47 percent increase over the same period a year ago. With Roche’s purchase of Genentech, the collective market cap for the biotech sector sunk below the $300 billion mark, but the deal also fuels the discussion about whether it even has meaning anymore to talk about a pharmaceutical industry and a biotechnology industry that are separate and distinct from each other. We spoke to Peter Winter, editor of The Burrill Report about the first quarter numbers, where the money went, and what it all means.

There are signs that the economy may be stabilizing. After six consecutive months of decline, factory orders rose for the first time in February. The stock market, after a dismal start to the year, soared in March and has now moved into positive territory for the year. And there are some signs of the housing market leveling off. Nevertheless jobless numbers continue to reach records and the pharmaceutical and biotech industries have not been immune to the job cuts. A tally by The Burrill Report found in the first quarter alone, 90 companies announced nearly 30,000 job cuts as the number of new U.S. claims for state unemployment benefits jumped to a 26 year high last week. John Challenger, CEO of the outplacement firm Challenger, Gray, & Christmas, offers some perspective on the job losses in the life sciences, how companies should go about their downsizing, and what newly displaced workers should do to get new jobs. 

President Barack Obama named Margaret Hamburg, former New York City Health Commissioner, to head the FDA. At the same time, he also named Baltimore City Health Commissioner Joshua Sharfstein as Hamburg’s principal deputy. Sharfstein previously worked for Democratic Congressman Henry Waxman, a harsh critic of the pharmaceutical industry. When Obama announced his choices, much of his comments focused on food safety issues, an area of public embarrassment for the agency during the past year. Little, however, was said about the agency’s mission to regulate drugs and medical devices. We spoke to Peter Pitts, former FDA associate commissioner and president of the Center for Medicine in the Public Interest about Obama’s selections, what the President’s picks will mean for industry, and the challenges ahead for Hamburg and Sharfstein in their new jobs.

President Barack Obama’s executive order removing barriers to scientific research involving human embryonic stem cells overturned Bush era restriction that significantly limited federal funding of this work. At the same time, Obama issued a memorandum on scientific integrity – part of an effort, he said, to “restore scientific integrity in government decision making.” We spoke to Stacie Propst, vice president of scientific policy and outreach for the advocacy group Research!America about the Obama executive order, the changes he is bringing to the nation’s science policy, and whether there’s any hope for science to become delinked from politics in the future. 

This week, leaders in the House and Senate joined to introduce legislation to reform the United State patent system. This follows failed efforts at patent reform in the previous two sessions of Congress. The high-technology industry has long pushed for patent reform, but their efforts have been at odds with the biotechnology and pharmaceutical industries, which are concerned that the legislation would weaken patent protections and consequently cripple their ability to raise capital. We spoke to the Biotechnology Industry Organization’s CEO Jim Greenwood about the new legislation, how it differs from previous versions, and the concerns that remain for the biotech companies.

Stock prices have tanked, financial markets have seized and companies are laying off staff and putting promising projects on ice.  In the midst of all of this grim news it’s easy to overlook an important milestone for the biotechnology industry.  Peter Winter, editor of The Burrill Report, though, has been crunching the numbers and he says some 40 years after the industry began, it actually hit a major milestone in 2008 by turning profitable for the first time in its history. It’s not all good news by any means, but we talk to Winter about this major milestone for the industry, what we should make of it, and the emerging world of the biotechnology haves and the biotechnology have-nots.

Back in July, the Swiss pharmaceutical company Roche offered $43.7 billion, or $89 a share, for the 44 percent of Genentech it doesn’t already own.  Since then, talks have not progressed well. Roche last week said it would launch a hostile bid for the company at $86.50 – less than it originally offered. Genentech, which has told Roche it’s worth $112 a share, asked its shareholder not to act on the bid. But with the latest maneuvering by Roche came filings with the U.S. Securities and Exchange Commission that shed new light on the behind the scenes wrangling between the two companies. We spoke to TheStreet.com’s senior columnist Adam Feuerstein, who is also responsible The Street’s investment newsletter Biotech Select, about the emerging drama between the two companies and where it will all end.

The Obama Administration’s drive to reform healthcare suffered a serious setback when the president’s pick to lead the effort, Capitol Hill veteran Tom Daschle, suddenly bowed after embarrassing tax revelations. Daschle withdrew his nomination to head the U.S. Department of Health and Human Services. In addition, he said he will not serve as director of the newly created White House Office of Health Reform. The end of Daschle’s cabinet bid was a blow since many believed the Democrat from South Dakota, who spent 26 years in Congress, was uniquely qualified to lead the reform effort. Daschle was respected on both sides of the aisle and was viewed as someone who could shepherd complex and controversial healthcare reform through the rough-and-tumble world of Washington politics. What’s more, he had penned the book Critical: What We Can Do About the Health-Care Crisis, which provides a blueprint for overhauling the U.S. healthcare system. We spoke to Henry Aaron, a senior fellow at the Brookings Institution, about what Daschle’s withdrawal means to healthcare reform and where the Obama administration goes from here.

After a long and often frustrating back-and-forth with regulators, Geron said it had won clearance from the U.S. Food and Drug Administration to begin human clinical trials of its experimental therapy derived from human embryonic stem cells to treat patients with acute spinal cord injury. It is the first human embryonic stem cell-derived therapy to enter the clinic. We spoke to Bernard Siegel a leading expert on stem cell policy and the founder and executive director of the Genetics Policy Institute, about the significance of Geron finally getting a green light from the FDA to begin clinical trials and what might be ahead for the field under the new Obama administration.

As the Obama administration takes over in Washington, the biotechnology industry is readying for a busy year on the legislative front. Ongoing fights over issues such as patent reform and biosimilars will continue, but new ones surrounding healthcare reform, the regulatory environment, and comparative effectiveness could dramatically alter the landscape for the industry. And, with the seizing of capital markets, the biotechnology industry is turning to Washington to help with a more immediate crisis as 300 publicly traded biotechnology companies— 75 percent of all public biotech companies – have less than one year of cash remaining, and 120 of those have less than six months cash on hand. We spoke to BIO CEO Jim Greenwood and BIO chairman Joshua Boger about what’s in store for the industry under the new administration.

It’s that time of year when thousands of executives, bankers and investors involved in the life sciences industry emerge for the JPMorgan Annual Healthcare Conference and check if they see their own shadows. The conference, which brings together more than 7,000 people for four days of presentations and meetings at the Westin St. Francis hotel in San Francisco, runs January 12 through 15.  Traditionally, the industry has looked to the meeting to gauge sentiments on the outlook for the new year, appetite for deals, and how eager investors will be to buy shares in IPOs. Though not many IPOs are expected to be filed in the first half of the new year, raising money will likely be among the top concerns of many of the companies gathered for the event. Even companies not invited to attend will fill the lobby of the St. Francis and meet in surrounding hotels and restaurants. We spoke to G. Steven Burrill, CEO of Burrill & Company and the Publisher of The Burrill Report about this year’s conference and what he’ll be looking for as he makes his way through meeting rooms.

Many of us will be happy to see 2008 over. It was a year where the economy tanked, stocks sank, and capital became a lot harder and more expensive to obtain. We spoke to Burrill & Company CEO G. Steven Burrill, publisher of The Burrill Report, about the year past and the outlook for 2009 as he completes his latest book on the outlook for the life sciences industry. This is the first in a regular series of conversations we’ll be conducting with Mr. Burrill. Here we begin with a look at the capital markets.

About a year and a half ago Ed Holmes along with his wife Judy Swain, both distinguished researchers in the United States, found themselves lured to Singapore where the government was backing its great ambitions to become a leading biotech center with billions in investment. Holmes, the executive deputy chairman of A*STAR's Biomedical Research Council, is helping lead the second phase of Singapore's biomedical sciences initiative with a focus on translational efforts. He is working to build capabilities and attract the talent needed to translate basic research into potential new drugs, devices and diagnostic tools. We spoke to Holmes, a member of the TJOLS advisory board, about both the successes Singapore has enjoyed and the challenges it continues to face.

As Senior Vice President, Research and Development at Amgen, Joe Miletich leads the translational science efforts for the biotechnology giant. As such, Miletich serves as both a gateway and guardian over the company’s future. This critical part of Amgen’s R&D effort unlocks the promise of early discoveries by demonstrating their potential to alter disease in a beneficial way. We caught up to Miletich at the BIO 2008 Confernece and talked to him about the changing process of translational research and the sometime challenge of following where science leads.

“Given the volatility and the pace of scientific discovery in recent years, there is a need for a fact-based analysis to better understand which innovations are emerging as the most promising and, conversely, which technologies are losing ground,” said Matthew Hudes, U.S. managing principal, Biotechnology, for Deloitte Consulting's Life Sciences & Health Care practice. Using data collected over the past six years, Deloitte researchers created a three-stage measurement model covering discovery, development and realization to measure the inputs and outputs of innovation during the process of bringing new products to market. We caught up with Hudes, a member of the TJOLS business advisory board, at the BIO 2008 Conference and talked about the emerging technologies reshaping the biotech sector and how exactly you go about measuring innovation.

For nearly 40 years, the Harvard-MIT division of Health Sciences and Technology has worked to bring innovations in non-medical fields such as information technology, engineering and materials science to the patient bedside and to clinical research. The catchword for the program is convergence with faculty focusing on fostering collaboration among researchers from disparate fields. Now, HST through a unique program aimed at training a new class of highly versatile health professional, is taking its model to India. The move could lead to new treatments and technologies.

For Reg Kelly, the director of the California Institute for Quantitative Biosciences, or QB3, research breakthroughs are not enough. Kelly notes that Q2’s mandate is not only to improve public health, but California’s economic well being as well. To that end, QB3 has aggressively pursued relationships with industry, sought to find sources of funding for faculty created start-ups, and even created incubator space for new companies within its headquarters on the Mission Bay campus of the University of California, San Francisco to see that researchers’ discoveries get translated into products that can ultimately benefit public health.

No one would ever confuse William Boyd with Steve Austin, the iconic and bionic hero of the 1970’s TV series the six million dollar man. Boyd, who spent his career at a Nabisco bakery in Houston mixing dough to make Ritz Crackers, though, may become something of a bionic man himself. In 1979, Boyd developed retinitis pigmentosa, a genetic condition that causes a degeneration of the natural photoreceptors that line the retina in the back of the eye. Thirty years after his diagnosis, Boyd may be one of the first patients implanted with an artificial retina in the hopes of restoring limited sight to him.

 

In a major victory for the medical device industry, the U.S. Supreme Court ruled that medical device makers could not be sued over harm done by devices that had gone through the U.S. Food and Drug Administration’s premarket approval process. The 8-1 decision in the case of Riegel v. Medtronic decision reaffirms two previous lower court rulings. The medical device industry said the decision makes clear that the FDA, and not a patchwork of state regulation and jury verdicts, is the determiner of the safety and efficacy of medical devices. Jaxon White, chairman of MedMark Insurance Group, discusses the significance of the decision with Tjols' editor-in-chief William Patrick.

As the emerging science of nanotechnology converges with biopharmaceutical sciences, the blurring boundary lines threaten to create a nightmare for regulators. As Lori Andrews reports in the February issue of the Journal of Life Sciences, both the U.S. Food and Drug Administration and the U.S. Patent and Trademark Office are bring criticized for their approaches as they attempt both to encourage and to impose order on the field.

Evidence-based medicine could help physicians navigate the complexities of patient care by collecting, interpreting and disseminating data on outcomes. At first, it would seem to be nothing more than common sense. After all, who wants medicine based on mere opinion? If outcomes data confirm a treatments benefits, doctors should use it: if it’s harmful they shouldn’t, but as David Gollaher writes in the February 2008 issue of The Journal of Life Sciences, while evidenced-based medicine can improve quality and cut costs in the U.S. Healthcare system there are obstacles, not the least of which are doctors.

Though in 2007 concerns over patent reform and efforts to create a pathway for so-called follow-on or generic biologics dominated the policy front for the industry, no resolution on these issues were reached. As we head into an election year, healthcare reform will likely take center stage, but these unresolved issues will continue to remain high on the agenda for the industry. David Gollaher joins Bill to discuss the big policy concerns of 2007 that continue to be front and center for the industry as we move forward.

eHarmony specializes in getting singles to connect. Now the matchmaking site hopes landmark research will show that good health, not just marriage, is a possible outcome of its pairings. If a dating service seems an unlikely venue for serious research that could influence both health psychology and personalized medicine, it helps to remember that eHarmony’s business proposition is not to find you a few dates, but a partner for life. Gian Gonzaga, senior research scientists with eHarmony talks to Bill about the research being conducted by the company.

HMO Kaiser Permanente is helping set up farmers markets at its medical centers across the nation on the principle that good food leads to good health. Since the first weekly markets opened at the Kaiser facility in Oakland in May 2003, farmers markets have sprouted at 32 Kaiser centers in California, Oregon, Washington, Colorado, Georgia and Hawaii. As journalist Theresa Moore reports in the December issue of The Journal of Life Sciences, it’s one thing to tell patients to eat more fruits and vegetables and its another thing to make high quality produce available at its medical centers.

 

In 2008, Menlo Park, California-based Geron expects to begin a much-anticipated clinical trial in which doctors will transplant a type of neural stem cell into humans. The trial is significant because, if successful, it would represents the first time that a product derived from embryonic stem cells could be shown to be safe and effective. But as author Ann Parson reports in the December issue of The Journal of Life Sciences, even though the company’s expectations are modest, success in treating patients with spinal cord injuries would validate a lengthy and costly effort.

The notion of personalized medicine is seductive. Proponents say it will increase the efficiency of drug development, cut wasteful spending on therapies that don’t work for certain patients, and deliver more effective treatments. But as Daniel S. Levine reports in the November issue of The Journal of Life Sciences, the adoption of personalized medicine on a large scale faces significant barriers. The science of identifying appropriate biomarkers for diagnostics can be evasive. The business models to align pharmaceutical and diagnostic companies’ interests remain undefined. And the regulatory and reimbursement models are not yet in place to allow companies to recoup their sizable R&D investment in products designed to reach smaller and smaller populations within a subset of a disease.

Empowerment through genetics is now at everyone’s finger tips – or so we’re told. A quick trip online will direct you to tests that claim to identify your genetic propensity for diabetes, breast cancer, cystic fibrosis, or iron overload disease. You can secretly check your baby’s paternity and learn its gender in as few as six weeks from conception. You can go to Target.com and buy a genetic home collection kit for the whole family, with results that tell you how your DNA makes you unique. But as Sally Lehrman reports in the November issue of The Journal of Life Sciences, some question whether the trend toward making genetics testing as easy as fixing a TV dinner is putting the market ahead of the science.

Reserved, introspective, uncomfortable with head-to-head competition, Elizabeth Blackburn might seem an unlikely role model and mentor for young scientists. But her predilection for exploring unconventional explanations and her aversion to jostling for position in a crowded race led her to a seemingly obscure byway that became a thriving research field with significant implications for human health. The functions of telomeres and telomerase in the aging of cells influences human aging, and telomerase also plays a role in the growth and metastasis of cancer. Catherine Brady, author of the newly released Elizabeth Blackburn and the Story of Telomeres: Deciphering the Ends of DNA joins Bill to discuss the career of this pioneering scientist.

The Journal's Editor-in-Chief offers some thoughts on the biomolecular economy and how biological principles are informing organizational structures.

For all the size and scope of its medical infrastructure, Houston lags behind in translating biomedical research into biotech commerce. A 2005 Ernst & Young study showed that California was leading the nation with more than 400 biotechnology companies, but Texas was toward the end of the list with fewer than 50. In the current issue of the Journal of Life Sciences, Ellen Durckel takes a look how the biggest city in Texas is hoping institutional partnerships will change that.

A Glioblastoma is a relatively rare illness, but a nasty one. About 10,000 patients get diagnosed with it in the United States annually and only half of them will live a full year after their diagnosis. Even when the tumors have been surgically removed from the brain, half the time they will return within six months. But a vaccine in tests for this aggressive cancer has patients living more than twice as long as those treated with radiation and chemotherapy and, as author Bruce Goldman reports in the October issue of the Journal of Life Sciences, has some wondering if it could serve as a model for other types of cancer.

Maxygen CEO Russell Howard has said that “Life is characterized not only by death, but by reproduction, passing on the DNA, the thread that produce progeny and is the real essence of life. Most businesses are the genesis of another business. They may spin off another company, and the parent may die, but the spin-offs survive and succeed. I use the analogy not only of inevitable death, but of life’s continuous process of recreation.” In 2002, Monitor’s Chris Meyer took a close look at Maxygen, itself has been an extraordinarily adaptive enterprise, following an evolutionary biological model when it comes to its organization, leadership, and product development. In this month’s Journal, Meyer revisits the company five year’s later.