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Presidential Election Result Good News for Stem Cell Sector

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By Peter Winter
BioWorld Insight Editor

At the beginning of his first term in office U.S. President Barack Obama overturned President George W. Bush's existing executive order that banned federal funding of human embryonic stem cells (hESC) research with the exception of a small number of existing stem cell lines.

The easing of restrictions on stem cell research was expected to pave the way for massive investments into the space and lead to pharmaceutical and biotech companies developing robust product pipelines based on stem cell therapies. However, there have been some bumps in the road that has served to keep both big pharma companies and venture capitalists on the sidelines to date.

This may change following a series of important recently announced early advances in the clinic, increases in federal funding for all forms of stem cell research and a regulatory approval of a manufactured stem cell product. This momentum, which has been building in 2012, is likely to continue. Now that President Obama has been re-elected for a further term the stem cell research community can look forward to a favorable political climate to support their work going forward.

Steady Advances Being Made

Although the sector hasn't grown as fast as many people would like, the field is advancing on a much broader and deeper front compared to a decade ago, Martin McGlynn, president and CEO of StemCells Inc., told BioWorld Insight.

His company has been working on the therapeutic uses of human neural stem cells (HuCNS-SC) since 1999 and this research is just beginning to bear fruit.

"While we are still at the very early stage in uncontrolled trials, the data we are seeing from these studies are confirming what we have seen in animal models to date," McGlynn said.

Their research, published in the Oct. 10, 2012, issue of Science Translational Medicine, describes parallel preclinical and Phase I successes in stem cell transplantations for demyelinating disorders. In the clinical trial, four babies with Pelizaeus-Merzbacher disease, a rare genetic myelination disorder, were implanted with HuCNS-SCs.

All patients subsequently showed preliminary evidence of progressive and durable donor cell-derived myelination and three of the four of them showed modest gains in their neurological function, which suggests a departure from the natural history of the disease, McGlynn noted.

Although clinical benefit cannot be confirmed in a trial without control patients, the small but measureable gains in function at one year may represent signals of a clinical effect to be further investigated in a controlled trial with more patients.

In the animal studies, cells that had been frozen and preserved were still able to form functional myelin when they were thawed and transplanted into mice with a genetic defect that leads to a lack of myelin. The cells in the animals studied formed functional myelin, though with a significant delay after transplantation. (See BioWorld Today, Oct 15, 2012.)

StemCells also recently 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.

Equally excited about the recent progress in stem cell research is Gary Rabin, chairman and CEO of Advanced Cell Technology (ACT). He told BioWorld Insight that the company has treated 13 patients to date in three ongoing trials for dry age-related macular degeneration (dry AMD) and Stargardt's Disease (SMD) using human embryonic stem cell (hESC)-derived retinal pigment epithelial cells.

The plan for each trial is to enroll a total of 12 patients, with cohorts of three patients each in an ascending dosage format, from 50,000 to 200,000 hESC-derived RPE cells. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with dry AMD or SMD at 12 months, the study's primary endpoint.

ReNeuron plc, of Guildford, UK, is also on track to begin a Phase II study next year for its neural stem cell therapy. It recently presented data from a study in stroke patients that indicated five out of six patients showed reductions in neurological impairment and spasticity, including speech that was less slurred and improved hand movements. (See BioWorld International, Aug. 22,2012.)

The hundreds of ongoing clinical trials are pointing us toward the future of medicine, Bernie Siegal, executive director of the nonprofit Genetics Policy Institute (GPI), told BioWorld Insight. The GPI organizes the World Stem Cell Summit and its eighth meeting to be held in Florida early next month is reflective of just how far the stem cell field has evolved, Siegal noted.

"At this year's Summit event there will be a greater emphasis on the presentation of clinical data in sharp contrast to the agenda of our first meeting. While we are still at the foundation stage of cell therapy, the explosion of activity is certainly very encouraging."

Public Markets

It also has been a good year for stem cell companies on the public markets, boosted by a regulatory approval and encouraging clinical trials results.

For example, Osiris Therapeutics Inc.'s stock value has risen 82 percent year-to-date. The company's allogeneic stem cell treatment for graft-vs.-host disease (GvHD) in children received the world's first regulatory approval in Canada of a manufactured stem cell product and the first therapy approved for GvHD – a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected, many within just weeks of diagnosis. (See BioWorld Today, June 7, 2012.)

Newark, Calif.-based StemCells Inc. has seen its shares rise 113 percent YTD and the shares of San Diego-based Cytori Therapeutics Inc. are up almost 50 percent in the same period. The company recently began a Phase I/II trial of its cell therapy for refractory heart failure resulting from myocardial ischemia. The randomized, double-blind trial of safety and feasibility will enroll 45 patients at six centers in the U.S. who will be randomized to cell therapy or inactive placebo injection. All patients will have adipose tissue removed. The tissue is used for processing into adipose-derived stem and regenerative cells. (See BioWorld Today, Oct. 12, 2012.)

The year also has seen Verastem Inc., of Cambridge, Mass., raise $55 million through an initial public offering to help support its pipeline of small-molecule compounds that target cancer stem cells (CSCs). (See BioWorld Today, Jan 30, 2012.)

The excitement surrounding CSCs lies in the fact that these comparatively rare cells (making up about 1 percent to 3 percent of all cells in a tumor) are implicated in the recurrence of tumors and have been identified in a wide range of cancers, highlighting their importance as therapeutic targets.

Toronto, Canada-based Stem Cell Therapeutics Corp. (SCT) has signed an agreement with University Health Network (UHN), through its commercialization agent MaRS Innovation, which provides the company with an option to an exclusive worldwide license to a cancer stem cell program. (See BioWorld Today, Nov.9, 2009.)

With the promising clinical advances in the past year and an assurance that, at least for the next four years, the climate for research will be supportive, the sector looks as though it has a bright future ahead.