Pluristem Therapeutics (NASDAQ: PSTI) is opening the doors to the future of cell therapy being available to the masses as standardized, off-the-shelf personalized medicine.
As what I believe is the most forward-thinking and practical player in cell therapy, Pluristem has leaped ahead with standardization in its manufacturing and its delivery methods, independent of location and independent of the experience of the healthcare provider.
Pluristem just announced that its full-scale, clinical-to-commercial grade manufacturing facility currently being built in Israel reached a major milestone with quality control and process validation now underway, the last step toward making the facility ready to produce FDA-quality stem cells. Capacity is huge; product made there will be worth $1 billion to the company.
While standardizing manufacturing is critical, perhaps equally important is the ability to consistently freeze, store, ship and then thaw the cells at the point of care. That's why Pluristem announced it created an as of yet unnamed thawing device.
Accepting new clinical methods is difficult, but one sure way to smooth the path to quicker physician adoption and widespread use is through standardization. The reason is because science is based on replication. Laboratory testing of a scientific hypothesis must use the exact recipe described by the inventor. If results can be repeated, then the hypothesis is proven. After repeated many times in the lab, validated through clinical trials, and approved by regulatory agencies, the hypothesis becomes medical fact.
The thawing device news did not get much attention, but look closer. One of the biggest problems facing stem cell therapy companies that have circumvented the political controversy of embryonic tissue usage is replicating clinical results. The science is new, the techniques are rustic, and the responsibility falls to the laboratory technicians and nurses that help prepare injections. Pluristem will be offering what sounds like an easy to use system where a frozen sample of its PLX cells can be popped into a small device and thawed quickly and - here's the key - uniformly to provide a dose just like the one before it and the one after it. Uniform doses and predictable results. Potential differences in handling stem cells prior to administration will no longer have to be factored into differing results between patients, and clinical observations will be more credible.
A comprehensive search for marketed thawing devices for frozen stem cell samples led to one product, the Brooks RapidThaw, a somewhat clunky and unattractive lab bench instrument with what looks like limited specifications. The most boastful feature is fully automated thawing of up to four racks of samples, which doesn't speak much to its uniformity nor is that a claim with the device. The biggest advantage with the RapidThaw is reducing thaw time down from two hours. Pluristem's device claims to be rapid, which in lab time means under ten minutes.
Introducing a thawing device is a terrific competitive gain for Pluristem and should do a lot to further their progress in clinical trials, and beyond. Literature shows that other stem cell therapy hopefuls depend on kitchen-variety methods to thaw their samples. With the first stem cell treatment ever approved in Canada last May, Osiris Therapeutics (NASDAQ: OSIR) still has yet to launch Prochymal for graft-versus-host-disease, so no clinical standardized procedures are recorded yet, although from its scientific publications its success could have been facilitated by easy, reproducible stem cell processing that gave them the allure of an off-the-shelf product ready to treat disease in an acute setting. Mesoblast Limited (OTC:MBLTY) appears a bit more behind in lab methods, where stem cell samples are simply washed with saline prior to treatment.
A good example of lab standardization furthering medical progress was the push to use glycated hemoglobin, or HbA1c, as a measure of someone's blood glucose levels in diabetes. Originally proposed as a simple, verifiable test for the disease, repeated clinical trials finally resulted in evidence that the chronic complications of diabetes are highly correlated with controlling blood sugar as measured by HbA1c. Since then, diabetes has been recognized as the epidemic that it is.
Pluristem's sleek overall design of its medical technology and implementation is impressive - from harvesting and processing cells, shipping them to clinical trial sites around the world through Fisher BioServices, a top-shelf biologic material currier and part of Thermo Fisher Scientific (NYSE: TMO), to now making them dependable to use in an off-the-shelf, easy and efficient manner, ending in the simple administration of an intramuscular injection. And their timing is good. Earlier this month, two researchers in stem cell work were recognized by the Nobel Prize for medicine, a first for this kind of therapy and sure to boost its credibility. In another area, the President's Council of Advisors on Science and Technology has been complaining that drug companies are spending $31 billion a year on clinical trials but drugs are not getting to patients faster because of insufficient clinical protocols, the under-utilization of technology, and a lack of clinical trial standards. The NIH and FDA are working to fix this.
Anyone selling new medical ideas or products knows it takes the equivalent of a gun to the head to get a doctor to change procedure. While this may seem counterintuitive given the innovation of our healthcare system, it makes sense in a world of time-stressed physicians reluctant to surrender familiar techniques that don't scare patients or give referring colleagues pause.
I believe Pluristem will be recognized for the game changing work it's doing, not only within its own world of pushing forward in clinical studies, building out manufacturing and choosing the right partners, but also in the larger scientific and clinical landscape, by facilitating the progress of innovative medicine through simple standardization.