Industry Voices: A pivotal time for stem cell therapeutics, as first clinical data emerge

By Martin McGlynn, President and CEO of StemCells

Stem cell therapeutics hold much promise for the treatment of serious diseases, including spinal cord injury, stroke, Alzheimer's disease and blindness. Other therapeutic approaches have been unable to provide good drugs for those diseases. Pursuing the development of innovative treatments, such as stem cells, is especially important as incidence of those diseases will only increase as the population ages, placing a heavy financial burden on the healthcare system.

Despite the promise stem cells hold for these diseases, up until now discussion surrounding the space has been restricted to the potential of the treatment, as clinical data was not yet available. Now, for the first time, we have reached the point where clinical data can be reported, and these early results have been promising, offering a first glimpse into the full potential of stem cell treatments.

Promising Results from Clinical Studies

This year, stem cell therapeutics have begun to transition from preclinical to clinical study and have made several significant clinical advances in diseases such as amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, stroke and spinal cord injury, evidenced by published and presented data. These are all serious diseases for which few or no effective treatments exist, and these studies are conducted in the worst-of-the-worst patient cases, so any clinical impacts are unexpected.

This year promising results from a study in patients with ALS were presented at the 65th Annual Meeting of the American Academy of Neurology. In this study, 6 patients treated with BrainStorm Cell Therapeutics' stem cells had significantly slower decline in overall clinical and respiratory function as measured by the ALS Functional Rating Score (ALSFRS-R) and Forced Vital Capacity (FVC) score respectively. This Phase 1/2 study involved BrainStorm's adult stem cell therapy, which utilizes bone marrow-derived mesenchymal stem cells that are differentiated into neurotrophic factor-secreting cells.

Another set of promising results involved stroke patients and were presented this year by ReNeuron. In this trial, the first 5 patients treated showed reductions in neurological impairment and spasticity, including improved speech and hand movements, which were sustained in longer-term follow up.

In addition to presented results, the body of published data involving stem cell therapeutics has been growing, with an embryonic stem cell company, Advanced Cell Technology ($ACTC), reporting early data in The Lancet last year from its studies involving patients with degenerative eye diseases. This paper reported that one patient with Stargardt's macular dystrophy and another with dry age-related macular degeneration (AMD) were able to regain vision.

StemCells ($STEM) has also been reporting early, but promising, clinical data from multiple ongoing programs in neurological disorders. Recently, one-year interim data from a Phase 1/2 study in chronic spinal cord injury were presented showing that two patients with complete spinal cord injury (no feeling below the site of injury) had multisegment gains in sensory function, first observed at 6 months and persisting at one year. One patient converted from a complete to incomplete injury. StemCells recently received approval to expand this trial from its site in Switzerland to include Canada as well.

In addition, the company has published data from a study in patients with a rare myelination disorder, Pelizaeus-Merzbacher disease, characterized by the inability to produce myelin. Successful treatment of this disease has implications for treating multiple sclerosis or cerebral palsy, and these recently reported results showed that four patients were able to create this sheathing, a world's first. StemCells is also evaluating its human neural stem cells in a Phase 1/2 clinical study in dry AMD, and IND-enabling studies for an Alzheimer's program are ongoing.

An Additional Milestone for the Stem Cell Space

In addition to these clinical advances, the stem cell space achieved another important milestone in 2012 with the approval of the first stem cell product in Canada: Osiris Therapeutics' ($OSIR) off-the-shelf stem cell product, Prochymal, for the treatment of acute graft-versus-host disease in children for whom steroids haven't worked.

This approval is an important moment for the stem cell space overall, as it sets a precedent for potential regulatory approvals in other countries. Health Canada's approval of Prochymal also brought an important treatment option to patients with a devastating disease.

Only the Beginning for Stem Cell Therapeutics

While these advances are notable, the early results that are being reported from the first stem cell trials are only indicators of the full potential for the space. More results are expected this year, including additional data from the leading stem cell study in spinal cord injury. This is a pivotal time for stem cell therapeutics, as clinical data begins to accumulate and the space advances toward midstage studies. As positive results continue to be reported, interest in the space will only grow in the future.  

Martin McGlynn is the president and chief executive officer of StemCells, a company advancing human neural stem cells with multiple ongoing programs in serious diseases such as spinal cord injury, Pelizaeus-Merzbacher disease, dry age-related macular degeneration and Alzheimer's disease. Mr. McGlynn has spent several decades in the life sciences industry in Europe, Canada and the United States. Prior to joining StemCells, Mr. McGlynn was the president of Anaquest, Inc. Before that he held positions of increasing responsibility at Abbott Labs and began his career working at Becton Dickinson.

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