StemCells, Inc. Exits SCI Study

Posted by Sam Maddox in Research News on May 31, 2016

This is a sad story to report. A major clinical trial to treat chronic spinal cord injury has been halted after 17 patients were dosed with neural stem cells. The reason, according to Stem Cells, Inc.: The effect wasn’t what they hoped for, and indeed seemed to decline after time.

What’s more, StemCells, Inc. is running out of money and is going full terminal -- winding down,” as they say.

The news is shocking. As regular readers know, we have followed StemCells Inc. for many years. The preclinical basis (the animal studies) for the study, came from Aileen Anderson and Brian Cummings at the University of California, Irvine. Anderson’s lab is part of the Reeve Foundation's International Consortium on Spinal Cord Injury.

You might recall the upbeat post here just last November: StemCells Inc. released early data from the so-called Pathway trial, which targeted chronic cervical spinal cord injury:

Five of six motor complete quads treated long after their injuries with stem cells recovered strength in their hands. Four got better dexterity and fine motor control in their hands and fingers.

Three treated patients were able to pick up a key, put it in the lock and turn it 90 degrees.

Said StemCells Inc. chief medical officer Stephen Huhn, "... we have three patients who pre-transplant were unable to pick up a key and insert it into a slot — after transplant they were able to pick up the key and insert it into the lock and turn it at the full 90 degrees ..."

“These findings,” said Huhn, “demonstrate the ability of our HuCNS-SC® cells to improve both muscle strength and motor function, thereby changing the trajectory of recovery following a spinal cord injury.”

So what happened? You can read the full, depressing press release here. The gist of it is that yes, there was a measured effect of the stem cell injections but the “magnitude” of effect over time did not trend well enough to spend more money running the trial. The company concluded “that achieving the primary endpoint objective of the Pathway Study would be unlikely.”

Here’s Huhn from today’s news release, demonstrating a major pivot on the trajectory of his outlook:

.... we believe we see a biological signal in many of the patients. Equally important, the first cohort of the study also confirmed the safety of cell administration into the cervical cord. The collective human data we have generated across all of our studies reinforce our belief that our cells have an excellent safety profile and that there are neurological and retinal disorders with unmet need that may be helped by cell transplant. Unfortunately, the company does not have the resources to implement changes in our development program to permit further investigation.

Readers who follow SCI research know the story of Geron, the first stem cell trial targeting spinal cord injury. That company bailed after just five patients but the rights to the study were acquired by BioTime, dba Asterias, and the study has since rebooted. StemCells, Inc. appears to be looking for a savior, too. Here’s founder and Stanford scientist Irv Weissman, from the release:

That we did not see significant recovery of motor functions in the Pathway Study is disappointing given the company’s nearly complete restoration of motor and sensory functions with HuCNS-SC cells in spinal cord injured immunodeficient mice, the recovery of sensory responses in patients with thoracic spinal cord injury, and the many other encouraging clinical and preclinical studies with these cells. Given the collective strength of past data with these cells, we sincerely hope others will pick up the many questions we have about the variability of results seen in the Pathway Study. Naturally, over the next few weeks, we will endeavor to find a party able to continue the development of this very promising technology, which is so important not only for current and future patients with these devastating diseases, but also for the field of brain stem and progenitor cell therapies.”