Stem Cells: A Promise Deferred?

Oct 01, 2012

Tucked away in a tiny room at last summer's BIO convention, adjacent to a big top vendor circus that would make even P.T. Barnum jealous, a handful of stem cell company executives convened for a panel titled "Stem Cell Therapies...Fact or Fiction?" The meeting was sparsely attended. After trotting out some compelling statistics in support of their chosen field—that stem cell research spending in 2011 was $1.2 billion; that, as of June 2012, 4,067 clinical trials involving stem cells were ongoing worldwide; and that stem cell products will earn upwards of $21 billion in sales by 2020—the executives moved from a discussion of facts to address the fictions.

Eduardo Bravo, CEO at TiGenix, a Belgium-based cell therapy company, said the most problematic fiction was the initial hype around stem cells. "Pharma jumped in and burned their fingers," he said.

Pharma's fingers are still smarting. It's true that the vast majority of stem cell companies are in the earliest stages of product development, and the sector has not been immune to the typical setbacks common in bringing forward a new class of drugs. Tumors in patients receiving human embryonic stem cells (hESC), or immune rejection in allogeneic transplant—a term that refers to patients receiving stem cells originating from another person, embryo or fetus—helped to scare off potential investors, especially those that weren't already frightened by ethical concerns and political or regulatory uncertainty. But as stem cell companies have developed work-arounds and solutions to these problems, they still haven't been able to fully assuage would-be funder's fears.

Time for the pitch

"Seven years ago, partnering discussions with Big Pharma lasted 15 seconds," said Bravo. "Now they last 15 minutes." Even as mid-sized organizations with stem cell programs like Celgene and Shire have "increased the pressure on Big Pharma" to take notice or be left behind, Bravo said the future primary players in stem cell therapeutics won't likely emerge from the Big Pharma set. "I think it will be someone new, like Samsung, General Electric, or Fuji."

Maybe that's fitting. After all, stem cell therapies are fundamentally different than small molecules in that small molecules were designed to address symptomatology, whereas "the promise of cell-based therapies is that they offer the prospect of restoring lost function and preserving against degeneration," says Martin McGlynn, CEO at Stem Cells Inc. "These are areas where the pharmaceutical industry's efforts in the past have not been stellar." The problem is that small molecules aren't the game-changers for patients or blockbuster sources of revenue they once were. As pharma moves increasingly toward specialty products and biologics that block the pathways leading to loss of function, perhaps it's time to reconsider stem cell therapies as an additional area for future growth. Stem cell technologies "could be partnered much earlier if there was vision and support from the [Big Pharma] C-suite, if they said to their executives, 'Go out and put some place-holder bets on the table. You don't have to go for winners, but put us in a favorable position if any of these technologies do pan out in controlled trials,'" says McGlynn.

What the numbers say

While stem cell companies continue searching for the funding and partnerships that will pay for the next round of clinical trials, early-stage data continues to accumulate. With the help of stem cell therapies, embryonic and otherwise, recent animal model tests have shown subjects to regain hearing, memory, and motor function. Human subjects with macular degeneration have demonstrated improvements in vision; paraplegics have regained sensation; and patients with aplastic bone marrow—a condition in which the body fails to produce the critical hemtaopoetic cells that in turn produce red blood cells, white cells, and platelets—have been essentially cured, their lives saved by stem cells. There are other examples of early successes with stem cells, and other programs targeting many of the biggest unmet needs in disease areas like Parkinson's, Huntington's, ALS or Lou Gehrig's disease, traumatic brain injury, stroke, Alzheimer's, multiple sclerosis, cerebral palsy, lysosomal storage disorders, diabetes, hematologic cancers, peripheral artery disease, and critical limb ischemia. Stem cells have been shown to provide protection even from radioactive bombs.

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