F.D.A. Approves a Stem Cell Trial

WARNING: Long post with no pictures. But it's important.
Thanks to Rayne for passing it along!

By Rob Waters
Jan. 23 (Bloomberg) -- Geron Corp. won approval from U.S.regulators to begin the first human test of embryonic stem cells,treating people who have spinal cord injuries.The test will attempt to restore movement to paralyzed people by injecting neurons made from embryonic cells to the siteof injury. The U.S. Food and Drug Administration today lifted a hold placed on the research last May after Geron persuaded regulators the procedure is likely to be safe, said Thomas Okarma, chief executive officer of the Menlo Park, California-based company.

While thousands of patients around the world have been treated with adult stem cells and have shown mixed results, no humans have been given cells derived from embryos in an approved trial. Geron’s test will provide the first look at whether embryonic stem cells can regenerate.

“This goes beyond pills and scalpels,” Okarma said in a telephone interview yesterday. “This is a different kind of innovation. This is a single injection of living cells that may restore function to a tissue damaged by injury.” Susan Cruzan, a spokeswoman for the FDA, confirmed in a telephone interview yesterday that the agency will let Geronconduct preliminary safety trials.

Geron rose by $1.09, or 21 percent, to $6.31 at 9:33 a.m.New York time in Nasdaq Stock Market composite trading. An earlier gain of 26 percent today was the biggest in 3 ½ years. The shares have risen 9.9 percent in the 12 months through yesterday.

Other Trials Possible
The FDA’s action may help pave the way for other companies to get permission to start their own trials, said Christopher Thomas Scott, director of Stanford University’s Stem Cells in Society program.

“Geron is a path-breaking company in getting the first stem-cell trial,” Scott said in a telephone interview yesterday.“The message is that FDA now feels comfortable with the measure of risk the first trial will contain for the first few patients.”

Two other U.S. companies, Advanced Cell Technology Inc. of Los Angeles, and closely held Novocell Inc., based in San Diego, are using embryonic stem cells to develop therapies and are working to begin clinical trials.

The chances that the cell treatment will harm patients are much smaller than the chance the technique won’t help them, said Joseph DiMasi, an economist with the Tufts University Center forthe Study of Drug Development in Boston. Less than one biotechnology drug out of three that enter clinical trials is approved, DiMasi said. That risk of failure also applies to Geron, which has spent $45 million preparing an FDA submission on the stem-cell treatment.

Stem Cells Powerful
Embryonic stem cells, derived from embryos just days after conception, are able to form any of the roughly 210 cell types in the human body. The treatment has been politically and ethically charged since its discovery 11 years ago. Because the extraction of the cells leads to the destruction of human embryos, former President George W. Bush barred federal funding for stem-cell colonies created after 2001.

President Barack Obama, like many Democrats and some Republicans in Congress, backs such research, as does his pick for science adviser, Thomas P. Holdren. Bush’s limitation led to reduced support for academic and corporate research on embryonic stem cells. Obama said during his campaign that he favored overturning the restriction.

No Academic Data
Bush’s policy discouraged research and made it harder for Geron to persuade the FDA that the stem cells were safe and had the potential to help patients, Okarma said.“Normally when the FDA gets an application for something brand new, they consult the published literature to find confirmation of the claims in the application,” Okarma said. In this case, the lack of research funding meant that academic scientists had published nothing on the particular cell types that Geron is trying.

“The only publications on these cells and spinal cord injury come from Geron,” he said. The company’s early research showed the cells have enabled rats with damaged spines to walk. Most spinal cord injuries bruise, rather than sever, thecord, damaging the long fibers of nerve cells known as axons. This damage disrupts the flow of electrical signals from thebrain to other parts of the body needed to control movement, resulting in paralysis. Geron has shown in 24 studies involving1,977 rats and mice that stem cells help reconstitute a layer of insulating material known as myelin that protects the axons,Okarma said.

Looking for Safety, Benefit
In the first phase of the research, Geron will treat eight to 10 patients with spinal cord injuries that left no function below the site of the injury. Researchers will inject the patients’ spines with a type of nerve cell that makes another known as an oligodendrocyte. Geron has learned to make oligodendrocytes in large quantities from embryonic stem cells.

While the main focus of the initial study will be to ensure that the cells don’t cause harm, the researchers will also look for signs of improvement in the patients. If the cells prove safe in the first group of patients, Geron will seek FDA clearance to increase the dose and later to treat less severely injured patients, Okarma said.

The company will now seek approval from the ethics boards of seven medical centers around the U.S. to begin conducting the study with the goal of beginning to treat patients this summer, Okarma said. While he declined to estimate how long it might take to complete all the studies and seek FDA clearance to market the therapy, he predicted that early success would speed the process.“We are treating a desperate patient population that has no hope of any recovery whatsoever,” he said. “If we see efficacyin these early studies, the excitement will be hard to overestimate.” If the treatment works, “this will be fast-tracked and I don’t think we’ll be needing thousands of patients.”

The NY Times has more about the political timing of the approval.

The Christopher and Dana Reeve Foundation responds here. Watch Peter Kiernan, Chairman of the Board on Good Morning America here.