Geron: Betting the farm on embryonic stem cells

WASHINGTON (Reuters) - Academic researchers working with human embryonic stem cells may be in doubt about their future after a U.S. court ruling on federal funding this week, but for California-based Geron Crop the field is wide-open.

Geron is preparing to test the powerful cells in people with newly injured spinal cords for the very first time after getting a go-ahead from the U.S. Food and Drug Administration at the end of July.

The California-based company is arguably the closest to demonstrating that human embryonic stem cells might regenerate damaged tissue.

"This is the next wave -- cells as therapeutics," Chief Executive Officer Dr. Thomas Okarma said in a telephone interview. "Living cells are going to be tomorrow's pills."

Researchers have hoped all along that the cells might regrow tissue missing from the pancreas of a child with type-1 diabetes, or replace the brain cells destroyed by Parkinson's. Geron plans to use the cells directly to treat damaged spinal cords.

While Monday's federal court ruling does not affect Geron, which has used private funds for its stem cell work for more than a decade, Okarma is dismayed and hopes it will be overturned.

"Certainly there is an indirect impact. We would love to have more academic collaborators," Okarma said in a telephone interview. "This puts the kibosh on those temporarily."

Okarma hopes the ruling will encourage Congress to revive a bill that explicitly permits human embryonic stem cell research -- a bill passed with bipartisan support twice and vetoed twice by former president George W. Bush.

REVIVING LEGISLATION

"This absolutely needs to be pushed through Congress before the election and stop this silliness," Okarma said.

Human embryonic stem cells were discovered by an academic researcher -- James Thomson at the University of Wisconsin, in 1998.

But because of U.S. legislation called the Dickey-Wicker amendment forbidding the use of federal funds to destroy human embryos, Geron early on set up a private agreement with the University of Wisconsin and holds patents on many of the early lines, or batches, of embryonic stem cells.

Embryonic stem cells have many seeming magical properties. They can develop into all of the tissue types in the body.

They can also proliferate indefinitely in the lab and Okarma hopes Geron can exploit this property to make profits and transform medicine.

Most cells die after awhile when you try to grow them in the lab, but embryonic stem cells do not and scientists can share them, almost like sourdough starter for bread.

Okarma says this makes them superior to cells offered as "alternatives" to embryonic stem cells, such as induced pluripotent stem cells or adult stem cells.

Eventually, Okarma hopes Geron can grow huge batches of human embryonic stem cells in bioreactors, like many other biotechnology products such as monoclonal antibodies.

"We see value far beyond today's pharmaceuticals and we cannot fall into the trap that the new cancer therapies have, where a course of treatment costing $100,000 buys you a few weeks of life," Okarma said.

SINGLE INJECTION

Geron hopes a single treatment will help most patients.

The company is now setting up the first clinical trial using a type of nerve cell called a glial cell to fix injured spines. Geron's product is a primitive glial cell derived from human embryonic stem cells.

Glial cells are what Okarma calls the nursemaids of the nervous system, helping form the protective myelin sheath around nerve strands and producing important chemical signals.

Geron will inject these cells directly onto injured spinal cords to see if they can help regrow the damaged connections and help paralyzed patients regain at least some movement.

If they work in people like they do in animals, the cells will make their way to the damaged areas and nurture the bruised neurons, helping them reconnect and start sending signals again.

And because they come from embryos, there should be no issues with immune rejection, Okarma says, although the first volunteers will get low doses of immune suppressing drugs, just in case.

The doctors who will perform the tests are being trained on how to use the cells and examine the patients in a consistent way. Then they will wait for a patient who has just been paralyzed, perhaps by diving into the shallow end of a swimming pool, or in a car accident.

"This is the high season, if there is such a word that is appropriate, for the frequency of these injuries," Okarma added.

(Reporting by Maggie Fox; editing by Andre Grenon)