Madison researchers making major breakthroughs in stem cell work
Apr 03, 2012 (The Wisconsin State Journal - McClatchy-Tribune Information Services via COMTEX) --
This story first appeared in the Sunday edition of the Wisconsin State Journal newspaper.
Stem cells derived from the skin and blood of blind people are morphing into retina-like balls in Dr. David Gamm's lab at UW-Madison.
WiCell Research Institute and the Waisman Center, both connected to the university, are growing stem cells to help researchers around the country prepare for clinical trials.
Cellular Dynamics International, a company at University Research Park, is producing more than 2 billion stem cells a day, selling most to pharmaceutical companies for drug testing.
Nearly five years after the most recent major stem cell discovery in induced pluripotent stem cells, mature cells reprogrammed to their embryonic state -- progress continues on many fronts.
"We're at the beginning of having some revolutionary approaches to medicine enabled by this technology," said Dr. Tim Kamp, director of UW-Madison's Stem Cell and Regenerative Medicine Center and an organizer of the seventh annual Wisconsin Stem Cell Symposium this month.
Madison is well known for stem cell achievements: UW-Madison's James Thomson was the first scientist, in 1998, to grow human embryonic stem cells in a lab.
In 2007, Thomson and colleague Junying Yu of UW-Madison discovered human iPS cells, the stem cells made by reprogramming adult cells, at the same time as Shinya Yamanaka of Japan. The cells, made from skin or blood, provide an alternative source of stem cells without destroying embryos.
Both types of stem cells, believed capable of becoming each of the more than 200 cell types in the body, could someday offer cell therapies for patients with Parkinson's disease, heart disease, diabetes, cancer and other conditions. For now, the cells are mostly being used to better understand diseases and to screen and test drugs.
Nationally, the stem cell picture today is mixed.
A judge last year threw out a lawsuit that threatened to end the Obama administration's expanded funding of human embryonic stem cell research. But the plaintiffs appealed, and the case remains in court.
Geron, a California company that in 2010 launched the first clinical trial using human embryonic stem cells -- in patients with spinal-cord injuries -- stopped the study in November, citing financial difficulties.
But Advanced Cell Technology, of Massachusetts, said in January its study of human embryonic stem cells on patients with macular degeneration, a cause of blindness, might have improved the vision of two patients.
In Madison, the stem cell landscape has been marked by gradual and sometimes quiet shifts in recent years.
A once-fiery squabble over three of UW-Madison's stem cell patents appears to have been resolved in November. An official from the U.S. Patent and Trademark Office upheld a patent challenged by two groups, California-based Consumer Watchdog and New York-based Public Patent Foundation. An appeals board still must rule, however.
The patent office previously upheld two other challenged stem cell patents, with minor changes, held by the Wisconsin Alumni Research Foundation, the university's tech-transfer organization.
The National Stem Cell Bank, a repository of 21 lines, or colonies, of stem cells initially approved for use in research paid for with federal money, ended two years ago with little notice. It was housed at WiCell, an affiliate of WARF, at University Research Park.
The federal contract for the bank expired after the Obama administration expanded federal funding for stem cell lines created with private money, to 152 lines today.
WiCell continues to house and distribute the initially approved lines, including Thomson's original five lines, through its own stem cell bank. The bank also has about three dozen other lines, including embryonic stem cells grown without animal products and reprogrammed stem cells.
Since Geron announced in November that it will license or sell its stem cell properties to other companies, inquiries at WiCell from potential customers have picked up, said Erik Forsberg, executive director of WiCell.
"It was very quiet for a while, but now there's an interest in using embryonic stem cells for clinical applications," Forsberg said.
At Waisman Biomanufacturing, a lab inside the Waisman Center near UW Hospital, embryonic and adult stem cells are grown in super-clean conditions required for clinical trials, said Derek Hei, the facility's technical director.
Through an $8.8 million federal contract awarded in 2010, the lab is helping researchers at UW-Madison and other institutions prepare for clinical trials, mostly by using the cells in animal studies.
'Eye is leading the way'
Gamm, a pediatric ophthalmologist, said blind patients are among those with the most potential to benefit from stem cell technologies.
"The eye is leading the way," said Gamm, who will speak at the stem cell symposium April 11 at Promega Corp.'s BioPharmaceutical Technology Center in Fitchburg.
He's one of more than 80 researchers on campus whose work involves stem cells.
In his lab at the Waisman Center, Gamm is studying stem cells made from the skin and blood of patients with retinitis pigmentosa, a group of genetic defects that causes blindness.
His research last year illustrated how stem cells could help doctors tailor treatments to patients better than methods available today. Gamm grew stem cells from a blind woman who had a rare form of retinitis pigmentosa into retinal cells and showed that vitamin B6 could overcome her mutation. A commonly used test had said the vitamin wouldn't help her.
In a study published last month, Gamm grew stem cells derived from a healthy person's blood into a more organized, layered, retina-like structure in a dish.
Gamm plans to use such mini-retinas to screen a variety of drugs that might help patients, such as the vitamin B6. Stem cell therapies are likely years away, but the treatments could be particularly well suited to the eyes, which are relatively easy to access and monitor compared with other parts of the body, he said.
At Cellular Dynamics
Cellular Dynamics sells tiny vials of heart, blood and brain cells, made from induced pluripotent stem cells, to all of the top 25 pharmaceutical companies, along with biotech firms and academic researchers, said Chris Parker, chief commercial officer.
The pharmaceutical companies use the cells to test drugs for side effects and to screen drug candidates, Parker said.
Cellular Dynamics, which plans to start selling liver cells this year, has created some 200 cell lines from a variety of donors and patients, Parker said -- "everything from a neonatal foreskin to a 92-year-old deceased woman."
Using a $6.3 million federal grant awarded last year to the Medical College of Wisconsin in Milwaukee, the company is making heart cells from stem cells derived from 250 patients in Alabama and Utah. Researchers will use the cells to study left ventricular hypertrophy, which can cause heart failure and stroke.
Cellular Dynamics, which has more than 100 employees, plans to open satellite offices in other states this year and move to a larger building in the Madison area next year, Parker said.
Stemina Biomarker Discovery, which has a dozen employees in Madison, uses stem cells to create tests for developmental disorders such as autism. It also uses the cells to evaluate the safety and effectiveness of drugs.
Other companies in Madison that work with stem cells or make products used in the work include CellCura, Cell Line Genetics, InvivoSciences and Primorigen Biosciences, which collectively have about 30 employees.
Madison's stem cell enterprise may not be as big as those in Boston, San Diego, San Francisco or other big cities on the coasts. But Madison likely has more people per capita working in the field -- and a drive to stay on top, Parker said.
"We want to prove to the world that this can be done here," he said.
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