Susan Solomon is an advocate for more research into a promising treatment for many conditions: human stem cells. “They are our bodies’ own repair kits, because they are pluripotent — they can morph into any cells in our bodies.” We can use them to model and study disease, but she hopes for much more. She hopes, and believes, that our children will look at Alzheimers, and many other diseases, the way we now look at polio.
But this field is under siege, and it became clear early on that researchers would need safe-haven laboratories to study stem cells. So Solomon and her friends founded the New York Stem Cell Foundation (NYSCF). That had an amazing side effect. Medical research is dominated by large organizations and, says Solomon, “Sometimes they can’t get out of their own way or ask the right questions.” There is a huge gap between academic laboratories and pharmaceutical companies, one that they hope to bridge.
Her group is trying to develop a new technology: a genetically diverse array of thousands of stem cell lines, which could allow us to harness the promise of the human genome project, and produce safer, cheaper cures, and produce them faster.
Stem cells were first identified in 1998. By 2008, a scientist at the NYSCF was able to take skin cells, turn them into stem cells, then into motor neurons — the kinds that are damaged in ALS, or motor-neuron disease. Using that, he could watch the progress of the disease in a dish in living human cells. He found they were dying in very different ways than thought — they were being attacked by other cells. This was not possible to see before the model provided by the stem cells — it would have been like looking at a plane crash before the black box. Says Solomon, “Stem cells are the black box for diseases.”
The most amazing part is that they can study the progress of diseases long before symptoms are apparent. Right now, drug discovery takes 13 years, costs $4 billion, and has a 99% failure rate. “That’s a terrible business model, but also is a horrible social model.” Currently we test on animals, then artificial cells. But “we’re not mice, and you can’t go into a living person with an illness and just pull out a few brain cells or cardiac cells and then go and fool around in a lab to see if you have cancer.”
But with stem cells you can.
That’s an incredible advance, but Solomon thinks it’s not yet enough — because we can’t just look at cells from a few people. We’re not all the same! People are different and respond to diseases differently. That, she says, “seems totally obvious, but it is not the way the pharmaceutical industry has been developing drugs, because until now it hasn’t had the tools.”
There are other problems. We have the human genome, but the map is like having a computer program without a computer. To make a stand-in for the human population, we need to collect stem cells from the entire human population. But making a stem cell line previously has been difficult, inexact and time-consuming. So they’ve built a robotic system. That system can generate thousands of identical stem cell lines that will be avatars for all of us. To start, they could re-screen all the drugs that are out there to see how safe they are in different populations. But there’s much more to come, here at the threshold of personalized medicine.
Photos: James Duncan Davidson