The promise of future stem cell treatments just took a significant hit. Biopharmaceutical trailblazer Geron Corporation just called it quits as far as their stem cell research program goes. Citing an uncertain economy and a need for quicker investor returns, the company will redirect their stem cell program resources to their cancer program which is seen as less risky.
It was only a year ago that Geron announced that they had enrolled their first patient in the embryonic stem cell trial. A hallmark moment for stem cell therapies, it was the first clinical trial using human embryonic stem cells (hESC) in the United States. The therapy to be tested was developed by Hans Keirstead’s team at UC Irvine. In 2005 they famously injected cells into paralyzed mice with severely damaged spinal cords and made them walk again. They’d injected the mice with oligodendrocyte progenitor cells derived from human embryonic stem cells. Oligodendrocytes occur naturally in the spinal cord. They form myelin, the layer of insulation that wraps around neurons and, like insulation around a copper wire, enable the neurons to conduct electrical signals without short-circuiting. Following spinal cord injury, neurons lose their myelin and can’t function properly. When the oligodendrocyte progenitor cells were injected into the mice, they formed mature oligodendrocytes, wrapped into a myelin sheath around the neurons, and restored normal neuronal function and enabled the mice to walk again.
Geron’s version of the progenitor cells, called GRNOPC1, would be tested to see if the same miracle could be performed on humans. What makes Geron’s decision to end the program even more unfortunate are the heroic efforts the company needed to overcome in a very long FDA approval process and a human stem cell research-unfriendly Bush administration. It took some 22 months after the original FDA approval back in January 2009 to enroll their first patient. The Phase I study was aimed at assessing the safety of GRNOPC1.
The company was advancing the frontier of a research that is still very uncertain. All too often treatments that are effective in mice or other animal models do not translate to humans. And injecting people with an unproven treatment can result in dire consequences. In the final analysis, Geron concluded that the costs outweighed the benefits. Geron’s CEO, John A. Scarlet, said in the company’s press release, “By narrowing our therapeutic area, we anticipate having sufficient financial resources to reach…value…points for shareholders…. This would not be possible if we continue to fund the stem cell programs at the current levels.” The “current levels” would be $25 million per year.
In addition to shedding itself of the stem cell program, the company is eliminating 66 full-time positions or 38 percent of its workforce. The cuts will free up about $5 million in the fourth quarter of 2011 and about $3 million in the first half of 2012. Those amounts add to a company cash flow that already made them the largest with an FDA-approved stem cell program.
Geron’s history with stem cell research goes back a ways. They helped fund research at the University of Wisconsin that led to the first isolation of human embryonic stem cells. But while Geron’s decision is a setback for taking stem cells from the lab to the clinic, the company’s budget represents only a small portion of total funding for human embryonic stem cell research in the US. While their budget was $25 million per year, the NIH funded $125 million on human embryonic stem cell research in 2010, $165 million if you count money from the American Recovery and Reinvestment Act.
There are other reasons why stem cell hopefuls can stay their despair. The company still believes in the research and is currently looking for a partner or buyer for its stem cell program. I suppose we can take that as a sign that the trial hasn’t proceeded poor at this point. In addition, the end of Geron’s program would not mean the end of stem cell trials. Another company, Advanced Cell Technology (ACT), won FDA approval in January for a human trial assessing the potential of human embryonic stem cells as a cure for Stargardt’s disease, a rare form of juvenile blindness. They are also approved for a separate trial for patients with age-related macular degeneration. Notably, two Catholic nuns are among the patients despite the fact that the Vatican opposes the research.
Phase I of another trial involving human embryonic stem cells began in January 2010. The company, Neuralstem Inc. is currently conducting trials for amyotrophic lateral sclerosis, or ALS, also known as Lou Gehrig’s disease. In ALS, the neurons that control movement die. Neuralstem hopes that neural stem cell injections will mature into functional neurons and restore movement. Twelve patients have already received injections into the lower back spinal regions. After reviewing safety data, the FDA gave Neuralstem the go ahead in October to begin injections in the upper back portions of the spinal cord.
Even in these early stages, the company has seen some promising results. Thirty-nine year old Ted Harada, a manager at a document shredding plant, was diagnosed with ALS in 2010. By the time he enrolled in the study he was having difficulty climbing stairs, raising his legs while sitting, and had to walk with a cane. Last March he received 10 stem cell injections. This past October 22, Harada had regained enough strength to finish the two-and-a-half mile Walk to Defeat ALS in Atlanta. “I still have ALS, but I’m starting to see signs of hope,” Harada told The Daily Beast.
Let’s hope Harada’s condition does continue to improve. Let’s hope ACT’s trials experience similar success. Let’s hope Geron’s stem cell program for spinal cord injury doesn’t end, that entrepreneurial entities with different financial priorities view GRNOPC1 too promising to leave dead in the water. Just as basic research is essential for long-term innovation, profit-driven interest is a powerful force in making today’s research findings into tomorrow’s treatments. Let’s hope, for the sake of spinal cord injury patients, that there’s a solution out there that will benefit us all.