A couple of years ago shares of Geron Corporation (GERN) spiked to a near billion dollar market cap - an impressive feat for a company with nothing past Phase II trials at the time - on hype created by its embryonic stem cell treatment for spinal cord injuries (SCI) circulated the investing circuits. The trial was the first-ever approved by the FDA utilizing embryonic stem cells, and its initiation ushered in a new wave of hope for the millions around the globe suffering from the effects of severe spinal cord injuries, including hundreds of thousands in the United States alone.
Another wave of hope for SCI patients hit the wires in early October, when Doctors announced a new technology that could have just as easily been born in a scene in a Bruce Willis Sci-Fi movie. This technology allowed monkeys to use their brains to move the virtual hands of an avatar, a breakthrough that was hyped to become a huge benefit for those suffering with paralysis resulting from severe SCI. This technology also allowed subjects to 'virtually' feel the texture of the objects they were touching through an outside robotic exoskeleton.
While the above events created significant buzz, Geron's treatments are still many years away from market and the sci-fi adventures described with the robotic exoskeleton left lingering questions as to whether medicine will ever advance enough to allow the paralyzed to walk, touch and fell on their own again, never mind an avatar that does it for them.
A solution might not be as far away as we thought, judging by results from preclinical studies in monkeys by a small, emerging company with answers.
Preclinical studies of a technology developed by InVivo Therapeutics (OTCBB: NVIV.OB) have allowed paralyzed rats and monkeys to walk again, within weeks of initial treatment, and the company is just awaiting the green light from the FDA to begin its first trial in humans.
The preclinical results, already published in the Journal of Neuroscience Methods in 2010, marked the first time that paralyzed monkeys were able to walk again following treatment for paralysis and were convincing enough to allow InVivo to land a high profile defection from Geron in early October when Edward Wirth, M.D., Ph.D. was named NVIV's new Chief Science Officer (CSO).
Dr. Wirth, a renowned leader in the field of regenerative medicine, led Geron's spinal treatment initiative and will join InVivo in early December and will lead the human trials that will be necessary for an eventual FDA approval. With the technology and data from the preclinical trials already before the FDA, and with the manufacturing, science and materials already in place, the company is just awaiting the regulatory green light to move forward with the human phase.
So what is this novel technology that has has invigorated hope in the SCI-treating community landed the company's co-founder, Robert Langer, ScD, on the short list of candidates for the Nobel Prize in medicine for 2011?
It all starts with InVivo's biopolymer scaffold device (BSD) that has, in early studies, demonstrated a therapeutic effect in healing the spinal cord. While most other companies relevant to the the field are developing treatments and methods with a pharmaceutical basis, InVivo's BSD is being developed as a medical device and addresses the serious threat of secondary injuries to the spinal cord that generally lead to the most severe cases of SCI and paralysis.
The scaffold, as described by company founder and former paralysis patient himself Frank Reynolds, can be customized to fit each spinal injury and can be administered right there in the operating room when an SCI patient first arrives into the ER. Ninety percent of SCI patients are not initially paralyzed, rather they only suffer from spinal shock, only to become paralyzed by secondary injuries caused by inflammation, bleeding and scarring. Again, InVivo's BSD looks to heal the spinal cord in a timely manner before those secondary injuries take effect. The BSD is convenient enough to be administered in conjunction with the current standard of care, which consists of installing rods and screws in and around the damaged area of the spine, and wards off those devastating secondary injuries by allowing the spine to heal itself quickly.
If the secondary injuries can be warded off, the theory holds, then so can paralysis.
In support of that theory, 100% of the monkeys treated with InVivo's scaffold in preclinical studies were up and running within 12 weeks. The mood is encouraging that move to human trials from non-human primates will prove successful, given the similarities in anatomy and bodily structure between the monkeys used in the preclinical studies and humans; it's the shift from rats to monkey that was the larger technological leap.
With that said, should the human trials turn out positive, then the path to approval could be fairly quick since the scaffold is being developed as a medical device and not as a new drug. Medical devices are generally approved in a more expedient manner than new drugs by the FDA, so even with the human trials still yet to commence for InVivo, it's possible that the BSD can be on the market literally years before Geron's regenerative embryonic cell treatment.
This, however, brings up an interesting transitional point; the potential is there that treatments such as Geron's will be best administered utilizing InVivo's scaffolding technology. That makes InVivo's growing patent portfolio that much more valuable.
In addition to treatment with the scaffold alone, InVivo has already tested its device in combination with drugs and/or cells, although not yet in human trials. The company plans to continue that testing line and eventually file for approval of its scaffold in combination with the combination therapies. As outlined in this video clip, the company has thus far been very successful with the combination applications as well as with the scaffold alone, and an injectable, gel-based scaffold is also being pushed through the developmental phases. In fact, it's treatment with the said hydrogel that Mr. Reynolds says produced the best results in the completed monkey studies, according to the above-linked clip.
Once InVivo has secured FDA approval for the scaffold as a medical device, additional approvals for the combination therapies will also be sought after. Assuming the products or treatments that will be combined with the BSD are already FDA approved, then the path for the combination approvals should also come relatively quickly.
The vast treasure chest of worldwide patents compiled by InVivo is still growing, protecting its technology well into the future and opening the door for licensing and partnership revenue, as I alluded to previously in discussion with Geron's experimental treatment.
Additionally, the BSD is also being discussed to treat indications other than just SCI, opening the door to other highly-lucrative markets.
The possibilities presented by combining InVivo's scaffold with other drug and cell therapies has already led to a strategic partnership with the Miami Project, an organization that has to date raised nearly half a billion dollars in search of a cure for SCI-induced paralysis. This collaboration will study InVivo’s tissue engineering technology in conjunction with the Miami Project’s Schwann cells and was featured last month at the Buoniconti Fund to Cure Paralysis 26th Annual Great Sports Legends Dinner fundraising event.
In commenting on the Miami Project collaboration, CEO Frank Reynolds noted, “We look forward to the results of our preclinical work with the Miami Project, and are hopeful that the combination of the scaffold and Schwann cells will improve the therapeutic effect of both technologies. Our scaffold has shown excellent results in non-human primates and in a rodent contusion model treating paralysis following acute spinal cord injury. We remain on target to begin a 10-patient pilot study in acute spinal cord injury in 2012.”
With human trials on slate, and with stellar preclinical results, the makings of a paradigm shift in the treatment of SCI could be underway.
Disclosure: Long NVIV.