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FDA Lifts Hold on Geron Stem Cell Trial, Shares Leap

BioWorld Today Washington Editor

WASHINGTON – The FDA's blessing Friday of the world's first clinical trial of a human embryonic stem cell (hESC) therapy has not only launched what may be "the dawn of a new era" for medical treatment, but if successful, the therapies could alleviate much of the burdensome costs plaguing the nation's health care system, insisted Geron Corp. CEO Thomas Okarma.

"We see this as going well beyond the power of pills and scalpels, which really only temporize the endless progression of chronic disease, to an era where a single injection of cells is actually curative," Okarma told BioWorld Today.

Regulators Friday lifted the clinical hold on Menlo Park, Calif.-based Geron's Phase I trial of its experimental hESC therapy GRNOPC1 in patients with subacute thoracic spinal cord injuries, which was imposed last year after preclinical results showed a higher frequency of small cysts in animals than had earlier been observed. (See BioWorld Today, Aug. 28, 2009.)

But Geron developed new markers and assays as additional release specifications for GRNOPC1 , which contains hESC-derived oligodendrocyte progenitor cells, and completed an additional confirmatory preclinical animal study, whose results were apparently enough to convince the FDA to permit the company to move forward with the therapy in humans.

Geron's shares (NASDAQ:GERN) rose 17.3 percent Friday, or 83 cents, to close at $5.63.

"We are obviously very pleased," Okarma said.

Geron expects to study 10 patients at seven U.S. medical centers, he explained.

The multicenter Phase I trial is designed to establish the safety of GRNOPC1 in patients with "complete" American Spinal Injury Association (ASIA) grade A subacute thoracic spinal cord injuries.

Once safety in that patient population has been established, Geron plans to seek FDA approval to extend the study to increase the dose of GRNOPC1, enroll patients with complete cervical injuries and expand the trial to include patients with severe "incomplete" injuries, or those on the ASIA scale of grade B or C.

Okarma said the protocol of the trial has remained "essentially the same" as it was before the FDA initiated the clinical hold, with the only change being the dose escalation in cervical patients after completing the safety study in thoracic injuries.

Although the primary endpoint of the trial is safety, the protocol includes secondary endpoints to assess efficacy, such as improved neuromuscular control or sensation in the trunk or lower extremities, he noted.

The objective of the trial, Okarma said, is to shift the outcome of the complete spinal-cord injured patient "from one of no hope for any recovery to an outcome much like the incomplete patients who can respond to physical therapy.

"If we were to achieve that, it would be a very dramatic demonstration of utility," he said.

During a conference call with investors and analysts Friday, Okarma said Geron and its study sites plan to "erect as many walls as we possibly can" to ensure the security and integrity of the Phase I trial's data.

Because the company was concerned that media would be "camping out in the waiting room to follow the patient home after the injection," Geron has kept the identity of the sites "pretty quiet," he said.

Okarma said that the inclusion-exclusion criteria for the study are "so rigid as to be so absolutely certain that every patient who is admitted to this trial is absolutely an ASIA-A patient, so that if there are any improvements in function or sensation we can be sure to ascribe them to the therapy and not to some spontaneous recovery."

He insisted that the "scalability" of GRNOPC1 "can't be overemphasized," noting that Geron aims to eventually manufacture the therapy for "hundreds and ultimately tens of thousands of patients," which Okarma said not only would ensure product uniformity, but also would lead to low cost of goods.

"That would be critical to enable the widespread adoption of this technology, which obviously we hope goes well beyond acute spinal cord injury," he told BioWorld Today.

"Because we know that we can manufacture literally at scale any cell in the human body, there are no diseases or injuries that are beyond the reach of this approach. The value to society if this is successful and widely adopted can't be overestimated," Okarma added.

Although the FDA has lifted the hold on Geron's trial, the firm and others involved in hESC and other regenerative medicine technologies still face the battle of convincing Congress to spend more funds on such innovations, Okarma said.

He blamed the high costs of cancer therapies, with some costing as much as $100,000 per course, as being the source of policymakers' fear of innovation.

But for that price or less, the potential for hESCs "is permanent cure, and that is the value proposition inherent in what we are developing here," Okarma argued.

"We are at the vanguard of a real revolution, not only in therapeutic impact, but in the ability to deliver this broadly in a cost-containment environment," he contended.

Okarma noted that his firm has been working with Reps. Diana DeGette (D-Colo.) and Michael Castle (R-Del.) on legislation aimed at creating grants and tax credits, similar to the qualifying discovery therapeutic tax credit, and other incentives and resources for the regenerative medicine field. (See BioWorld Today, July 6, 2010.)

He said the lawmakers plan to introduce the bill this fall.

The company also has been closely watching House and Senate bills that are seeking to codify the executive order signed last year by President Obama reversing the restriction on federal funding for hESC research. (See BioWorld Today, March 10, 2009, March 11, 2009, Dec. 3, 2009, and April 29, 2010.)

Advanced Cell Technology Inc. CEO William Caldwell, whose firm is awaiting the go-ahead from the FDA on its own Phase I/II hESC clinical trial, was in Washington last week also promoting that legislation.

A win like Geron's last week, Caldwell told BioWorld Today, could be the trigger to move that legislation forward.

Published: August 2, 2010