The public’s hope for stem cell cures remain high, but scientists warn that many basic problems with this approach remain to be solved – and that investors demand profits far faster than research can be finished.
“We’ve created a system that is unrealistic and short sighted,” said Dr. Darrell Kotton, director of Center for Regenerative Medicine at Boston University’s Boston Medical Center.
Few of the 38 publicly traded stem cell companies will survive, said Philip Reilly, a physician, attorney and venture partner with Third Rock Ventures.
It is very difficult to produce returns in “venture time,” defined as less than five years, said Reilly. “There is tremendous disconnect,” between investors and biologists’ timelines, he said.
The best strategy, he believes, is for a small company to show data with enough promise – what he called “an inflection point” – that it wins the interest of a larger company, with the resources to carry the research across the finish line.
“Few will make it,” he said, of existing companies. At almost all, the cost per share has fallen below the opening price. “They were launched too soon.”
Greater government funding would help, the panelists agreed, because it would build a stronger foundation of basic research.
Because of limited federal funding, federal support for hESC research has historically been small, the panelists agreed. Private companies, states and philanthropists now spend more money than the federal government to support stem cell research.
“The NIH has a budget of $30 billion,” said Reilly. “Given the amazing work in stem cells, $30 billion is a drop of bucket that could be used for innovative products.”
Embryonic stem cells are undifferentiated “blank” cells that do not have a specific physiological function, but which can be turned into more specialized cells that perform desired functions, Kotton said.
Less controversial are so-called induced pluripotent stem cells (iPS), which are generated by reprogramming adult cells that have already differentiated into a specific cell, such as a skin cell.
The hope is that these specialized cells can be developed into tissues that replace those damaged or destroyed by disease.
Because of the pressure for quick progress, scientists go after the so-called low-hanging fruit of stem cell treatments – those projects that appear to have the least amount of risk and the maximum benefit, such as treatments for skin or blood disease as opposed to organ regeneration.
Kotton predicted that new lungs, for instance, won’t be built in his lifetime, but in future generations.
Rather than replacing full organs, it is far more feasible to use stem cells to develop a large bank of cell cultures to test drugs for patients suffering from a wide variety of diseases.
There’s already at least one successful example of this strategy. Kotton’s lab used stem cells to recreate a heart ailment called “Long Q Syndrome.” Then these sick cells were subjected to many combinations of drugs. When the ideal regimen was identified, it was tested in a sick child in New York City. The child’s irregular heart beat stabilized, he said.
This provides proof, he said, that stem cells “can already be applied to a real patient to understand disease and predict drug therapies.”
However, not much thought has been given to how those products – once they reach the market – might be regulated, said George Annas, a bioethicist at Boston University.
It is important that issues of oversight be addressed in a way that encourages scientific innovation but also protects the patients for whom these treatments might provide great relief, he said.