October 15, 2012

Stem Cells: the Promise, the Cost, the Future

I like seeing the latest news about stem cell research. The amazing technology certainly won’t extend my own 83-year-old life beyond its estimated 90 years, but who knows what impact the science may hold for my kids, grandchildren, and great grandkids.

There’s lots to love about the idea of curing, not just treating, heart disease, Alzheimer’s, HIV/AIDS, diabetes, cancer, Parkinson’s, ALS, macular degeneration – and other conditions, like spinal injuries – without drugs or complicated surgeries, using only our own genetic material. It strikes me as the most elemental – and at the same time the most extraordinarily complex – medicine ever.

The great promise for stem cell therapy hit the headlines again last month when ABC’s morning TV personality Robin Roberts received a bone marrow transplant to treat MDS, a condition that affects production of blood cells. In a quick, five-minute procedure, Robin received millions of stem cells from her sister through a syringe. We should know soon if these new cells – guided by their own genetic coding -- properly found their way into Robin’s bone marrow and have begun to function normally.

Even with the successes and awesome promise of stem cell therapies, there are reasons why we aren’t reading about regular breakthroughs in the New England Journal of Medicine. Even the Michael J. Fox Foundation – our country’s most public proponent of stem cell research -- announced earlier this year that it planned to diversify its funds into other areas of medical exploration… areas it thought might yield more immediate results.

Follow the Money
So, why isn’t progress on stem cell therapy zipping full speed ahead? Jeffrey M. O’Brien, author of a fascinating article in the October 8, 2012 issue of Fortune, suggests a simple answer:
The obstacles along the road from lab rat to human patients are many, of course, but the biggest by far is money. With the dramatic events in the lab, you might think that a gold rush would be under way. That's far from true. Long time horizons, regulatory hurdles, huge R&D costs, public sentiment, and political headwinds have all scared financiers. Wall Street isn't interested in financing this particular dream. Most stem cell companies that have dared go public are trading down 90% or more from their IPOs. 
We shouldn’t expect Big Pharma to lead the way, O’Brien suggests: “A few drug companies have kicked the tires on stem cells over the years, but waiting for them to undo the current model is akin to banking on Big Oil to rethink energy."

Just last year, Geron – a Silicon Valley biotech company – injected four people who had spinal injuries with some genetic compound that had allowed previously paraplegic rats to walk again. There were no adverse reactions and initial indications were positive, but, according to O’Brien, “…Geron discontinued the program and exited the stem cell field, citing economic conditions and an uncertain path to market.”

Waiting for the Pipe Bomb Explosion
The article tells the story of Robert Lanza, chief scientist at Advanced Cell Technology (ACT). Earlier this year, in The Lancet, Lanza wrote about a clinical trial involving two women with age-related macular degeneration (AMD) who were injected with retinal cells engineered from human embryonic stem cells. The results have been good; both women report important vision improvement. Since then, dozens of AMD patients have undergone the same procedure with similar good results. O’Brien reports what happened next:
ACT became embroiled in a patent-infringement lawsuit that drained the coffers and forced the sale of key assets. "We were okay for a year or two, and then we ran out of that money," Lanza recalls glumly. "We were living hand to mouth." The phones were turned off; the water cooler and coffeemaker were hauled away. Meanwhile, a nearby stem cell company was targeted by a pipe bomb, putting the entire industry on edge. "I was always listening for noises," he says. "There were busloads of picketers outside our office." 
Still, the news isn't all bad. Three years ago, pharmaceutical giant Novartis increased programs for regenerative medicine, “focusing on stem cells both as therapies and as tools for drug discovery.” GlaxoSmithKline has a $25 million, five-year partnership with the Harvard Stem Cell Institute. Emphasizing how young the science still is, Jason Gardner, R&D chief for GSK’s regenerative medicine unit, said: "It's been a renaissance for stem cell science in the last two to three years. Of course lots of the science is risky and early. So we are very interested in risk-sharing partnerships that also share in the upside."

Californians Give the Research a Boost
According to O’Brien:
The biggest patrons by far of stem cell research are the citizens of California -- who have, over the years, led the country on health care issues from pollution standards to anti-tobacco legislation to bicycle helmets. Eight years ago voters passed Proposition 71, allocating $3 billion to establish the California Institute for Regenerative Medicine (CIRM) and create a nexus for biotech research that would be analogous to Silicon Valley.
As a result, $1.7 million of that money has already been awarded, and there are 12 new research centers in the state. The CIRM has also gotten wealthy patrons to pony up. Insurance magnate Eli Broad and Business Wire founder Lorry I. Lokey each gave $75 million to fund research centers there. In total, the CIRM raised more than $560 million from donors to build a whopping 823,000 square feet of brand new research space in California.

Riding the wave, CIRM president Alan Trounson sounds psyched, and eager for a few key wins: “We need to demonstrate that cell therapies will work. There is an urgency to do that now. The science stands up that we can cure HIV. My feeling is, let's get that done.”

O’Brien uses several analogies to describe how we may be arriving at some critical juncture:
There's a pattern to how scientists describe the potential impact of stem cells on the economy and society at large. Think about the Interstate Highway System or the Internet. They weren't built overnight, but once they got rolling, they manifested themselves in ways no one dared imagine. And increasingly, despite the economic hardship, prominent scientists suggest we have arrived at an inflection point. "The potential to make advances in this field has never been better. In part that's because of the wonderful tools at our disposal -- genetic sequencing, imaging techniques, cell sorting. We can do things in a day that used to take half a year. And the cost is going down," says Arnold Kriegstein, director of the UCSF center. "The last 10 years have been turbulent times, almost like the Wild West, but the landscape is now better defined. The field is stabilizing. It's maturing. I'd be surprised in 10 to 15 years if there aren't some cell-based therapies that work." 
When O'Brien says "think about the Interstate Highway System or the Internet" I think about President Eisenhower who advocated spending billions of federal tax dollars on building the Interstate Highway System and which would be Dead on Arrival with today's Tea Party Republicans and I think of the billions of dollars that the Defense Department spent on developing the internet but which would never be spent on stem cell research. \

Philanthropist Eli Broad – the guy who gave CIRM $75 million – is bullish, at least on the promise of stem cell therapy: ”The greatest promise of improving the human condition is in stem cell research and genomics. I cannot predict all the economic benefits any more than I could have predicted the economic benefits of NASA. I cannot predict how it goes from here to bedside, but it's going to happen."

Others Say “Not so Fast”
Andy Grove is considered one of the godfathers of the semi-conductor industry, and therefore understands how “game-changing” industries are born. He also has a personal stake in stem cell development: a PWP, he’s participating in a clinical trial for a Parkinson's drug; he has invested in four biotech funds; and has pledged about $40 million to the Michael J. Fox Foundation. Nonetheless, O’Brien finds him full of “doom and gloom.” When O’Brien asks if stem cell therapy is poised to revolutionize medicine the way the transistor transformed communications decades ago, Grove rejects the comparison: "The sun shone on that industry. The government had a stake, the consumer had a stake, and the telecommunications industry had a stake. This industry is just as important, but after that the similarities are gone."

But some scientists are thinking creatively. Hans Keirstead founded UC Irvine’s stem cell research program and chairs the advisory board for California Stem Cell (CSC). That organization now makes money by selling “high-purity cell platforms” to Big Pharma for drug screenings. In turn, CSC uses that revenue to develop stem cell therapies for cancer, spinal muscular dystrophy, and ALS.

Like the entrepreneur he is, Keirstead also seeks to lower development costs, a regular obstacle for stem cell progress. O’Brien explains Keirstead’s plan this way:
He wants to offshore clinical trials in the same way corporate America has outsourced business processes. Many stem cell scientists worry about the U.S. losing a stem cell arms race to China. But Keirstead wants to drag the industry there on his terms. He recently founded China Stem Cell in Shanghai, and expects to start conducting trials there before long. "If you look at the old companies, with all their ups and downs and tiring out investors, they just have so much internal baggage," he says. "Running trials in China would be cheaper. I'm not advocating that people skirt FDA quality. Take the standards with you. You can do 100 or 1,000 patients there and be 20% of the cost of an American trial." 
Is Stem Cell Therapy Inevitable?
Whatever the obstacles -- regardless of political or moral opposition, and in spite of the gigantic challenges to turn the science into some kind of workable and profitable enterprise – O’Brien seems to think we’re moving inexorably toward a future that includes revolutionary stem cell therapies:
According to a recent Gallup poll, 62% of Americans now consider embryonic stem cell research to be morally acceptable, and that attitude is pretty consistent across all age groups. It's a healthy sign that public sentiment is strengthening in the way it often does with scientific breakthroughs. First we fear the different or unknown. Then we realize how much it may help us or the ones we love. It's hard to imagine that there was once moral outrage over in vitro fertilization now that it's become fodder for reality television.

O’Brien’s article was very interesting. You can find it here.

The National Institutes of Health (NIH) posts excellent information on its site. Here’s their info about stem cells
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