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StemCells Wins Second $20M Grant from CIRM for AD Work


By Catherine Shaffer
Staff Writer

An ambitious new grant for up to $20 million from the California Institute of Regenerative Medicine (CIRM) will support StemCells Inc.'s preclinical development of its HuCNS-SC neural stem cell product in Alzheimer's disease over a four-year period. The goal of the grant will be filing an investigational new drug application within that time frame.

The award is the second $20 million grant that the Newark, Calif.-based company has received from CIRM. In July, the Institute awarded it $20 million for development of HuCNS-SC cells in cervical spinal cord injury.

Those grants clearly represent significant amounts of money. StemCells CEO Martin McGlynn told BioWorld Today that the process was competitive, with around 60 applicants seeking the funds. When asked what qualities of StemCells resulted in a successful bid twice over, McGlynn said, "First and foremost, we have a proven track record in translating scientific discovery into clinical activity."

He also cited outstanding preclinical and clinical data and world-class expertise, not only within the company, but collaborators who are "the best and brightest" in the field.

McGlynn explained that the grant is actually a nonrecourse loan, meaning that it is a loan that is forgiven if the program doesn't succeed in the commercial phase. It is nondilutive funding, and no stock is issued.

StemCells appealed a previous grant and succeeded in getting CIRM to change its mind.

"This obviously is a very exciting time for StemCells Inc., because we recently announced that the cells are having a clinical effect in young boys with [Pelizaeus-Merzbacher disease (PMD)], and we also announced that the cells are having a clinical effect in the worst spinal cord injury patients," McGlynn said.

StemCells reported that six-month data from the first patient cohort in its Phase I/II trial of HuCNS-SC for chronic spinal cord injury continued to demonstrate a favorable safety profile and showed considerable gains in sensory function in two of the three patients, compared to pre-transplant baselines. The third patient remains stable.

The company presented those results at the Scientific Meeting of the International Spinal Cord Society in London.

Earlier this year, StemCells reported preliminary evidence of progressive and durable donor cell-derived myelination in all four patients who received transplantation with HuCNS-SC cells in a Phase I trial in PMD, a rare hypo-myelination disorder in children.

Clinical assessment also revealed small, but measurable gains in motor and/or cognitive function in three of the four patients. The fourth patient remained clinically stable. Those results were presented at the European Leukodystrophy Association Families/Scientists meeting in Paris.

"When you start to get confirmation in the clinic in humans of work that you saw many years ago in animals, it gets very exciting," McGlynn said.

Roth Capital Partners analyst Joseph Pantginis called the grant a "very important win" for the company and the space. "The company has a good road map for the path forward. However, a long road still awaits, in our belief, as it works to secure an IND for HuCNS-SCs in [spinal cord injury] in the U.S. and to execute on its other ongoing clinical programs," Pantginis wrote.

Pioneering embryonic stem cell (hESC) firm Geron Inc. bowed out of the field and canceled its ongoing Phase I trial of hESCs in spinal cord injury in November 2011, citing economic pressures. StemCells opened enrollment in its trial, based in Zurich, Switzerland, to patients in the U.S. and Canada, to offer an alternative to the North American spinal cord injury community cut loose from Geron's trial. (See BioWorld Today, Nov. 16, 2011, and Dec. 19, 2011.)

In spite of Geron's exit from the field, stem cell research has never been more active.

Mesoblast Ltd., of Melbourne, Australia, reported positive results in a large animal model of rheumatoid arthritis (RA) following a single intravenous injection of its allogeneic immunomodulatory adult mesenchymal precursor cells (MPCs).

The study was conducted on 30 sheep with established collagen-induced arthritis, comparing a single intravenous injection of allogeneic MPCs at one of three doses (0.3, 1 and 2 million MPCs/kg) to saline.

Thirty days later, joint synovial tissues from arthritic sheep were examined. The findings demonstrated that MPCs were immunoregulatory and concomitantly suppressed the activation and proliferation of T cells, monocytes and synoviocytes seen in active rheumatoid arthritis.

Aastrom Biosciences Inc., of Ann Arbor, Mich., reported that in a preclinical study, ixmyelocel-T protected the ischemic heart from damage in a murine model of heart failure. In the blind, vehicle-controlled study, ixmyelocel-T was tested in mice with nonacute left anterior descending coronary artery occlusion.

Four weeks after injection, those in the treated group experienced a significant decrease in infarct length compared to the vehicle control group. Animals treated with ixmyelocel-T also had reduced mortality compared to controls (22 percent vs. 44 percent), decreased infarct size and increased survival.

However, in the area of neural stem cell implants for spinal cord injury, StemCells has emerged as a clear leader, according to McGlynn. "We're the only company that has programs going on in all three regions of the central nervous system: the brain, the spinal cord and the eye," he said.

Already looking ahead, StemCells has set its sights on one more CIRM initiative designed to fund early stage clinical trials over a four-year period. StemCells has applied for that grant, worth up to $10 million, to fund a Phase II trial in PMD.