Human embryonic stem cell research has long been described with words like "potential" and "someday," words that reflect the technology's promise, but a promise to be kept in the future.
That future is now.
Stem cell therapies are happening today. Corporate giant Geron (Nasdaq: GERN) recently announced it expects to begin human clinical trials for spinal cord injuries later this year, and Advanced Cell Technologies (ACT) expects FDA approval soon to begin its own human trials for Stargardt's disease, the most common form of juvenile blindness, one that affects children as young as 10 years old.
San Diego researchers too are moving closer to real therapeutic treatments, as collaborations continue between private companies and research institutions like Scripps, UCSD and the Sanford-Burnham Medical Research Institute.
New optimism, new dollars
Last Spring President Obama fulfilled a campaign promise when he ordered the National Institutes of Health to allocate $21 million to expand its role in providing federal funds for human embryonic stem cell research. Another $5.4 million has been added from Federal Economic Stimulus Funds, effectively lifting the "ban" put in place by the Bush Administration in 2001.
For many researchers, the move was a welcome departure from the president's predecessor.
"This money is going to have a major impact," said Samuel H. Wood, M.D., CEO of La Jolla-based Stemagen. "Not only has the president reversed course on a policy that weighed scientists down for nearly a decade, he has taken a major step forward in promoting the development of stem cell-based treatments that may ease the suffering of millions of Americans who suffer from debilitating degenerative diseases for which there are currently no cures or effective treatments."
In 2008, Stemagen made worldwide headlines as the first research entity to report the successful cloning of a human embryo using adult cells. The breakthrough validated theories that it was possible to clone adult human cells. From there, scientists were just one step away from using those cloned cells to create embryonic stem cells that carried the exact DNA of the person requiring the treatment.
However, with only one state government, California, providing significant funding for stem cell research, Wood and other scientists at privately held companies had to rely on private investment dollars, since most of the money distributed through the voter-approved stem cell initiative went to public institutions and universities.
"Having additional funding sources is vital," Wood said. "Even though Californians have committed to spending $3 billion over the next 10 years, and the administration has so far only committed to spending $26 million, we feel any amount of additional money is a good thing, and we look forward to applying for it."
Not all stem cell lines created equal
Before the NIH could release the Obama money, it needed to conclude a review of existing stem cell lines and determine which were worthy of receiving federal dollars. Not all stem cell lines that have been created in the last 15 years can be used for research. In fact, most of the 13 lines provided for under the Bush Administration were unusable for research.
Embryonic stem cell lines are grown in a laboratory, and require a combination of nutrients and proteins to survive and multiply. Because most early stem cell work was done in animals, those "grower" potions all contain animal products. That made the human lines unsuitable for therapeutic use because some of the nutrients are harmful to humans. Scientists could study these lines, but they had no real therapeutic potential.
Today's stem cell lines, and indeed the ones created over the last few years, have been grown in more optimal ingredients and might have real therapeutic potential. Stemagen has created nine such embryonic stem cell lines and is maintaining them with these new, human-optimized ingredients.
So far, after careful study, the NIH has announced that 13 stem cell lines have been initially identified for funding. The NIH has said it will continue to review more in the coming months. More than 120 stem cell lines have been submitted for consideration and because all stem cell lines are different, researchers say having a variety to work with is crucial.
The lost decade
While Wood is pleased Washington has finally committed real federal dollars for new stem cell research, he can't help but wonder what might have been.
"It has been over 10 years now since human embryonic stem cells were first created," Wood points out, "and during that time, patients with devastating diseases that may be amenable only to stem cell treatments have suffered and died while politicians postured and delayed. If the promise of stem cell research is ultimately realized, and I am confident that it will be, I believe history will regard the last 10 years as 'The Lost Decade.'"
Jimenez is director of corporate communications at Stemagen.