April 17, 2019

SOURCE: AANS 219: SB623 Demonstrated Statistically Significant Improvement in Motor Function Among Patients with Chronic Motor Deficit from Traumatic Brain Injury (TBI) | DNA RNA and Cells | News Channels

Positive results from the STEMTRA Phase 2 trial were presented today at the American Association of Neurological Surgeons (AANS) annual scientific meeting

Data demonstrates the possibility of a stem cell treatment for chronic motor deficit from TBI

MOUNTAIN VIEW, CA, USA I April 16, 2019 I The SanBio Group (SanBio Co., Ltd. and SanBio, Inc.), a scientific leader in regenerative medicine for neurological disorders, today announced that SB623 met its primary endpoint, with patients treated with SB623 achieving an average 8.7 point improvement from baseline in the FMMS, versus 2.4 in the control group, at 24 weeks, in a Phase 2 trial of patients with chronic motor deficit from traumatic brain injury (TBI). Detailed results from the STEMTRA Phase 2 trial evaluating the efficacy and safety of SB623 were presented today at the American Association of Neurological Surgeons (AANS) annual scientific meeting in San Diego.

SB623 is an investigational product made from modified and cultured adult bone marrow-derived mesenchymal stem cells that undergo temporary genetic modification. Implantation of SB623 cells into injured nerve tissue in the brain is expected to trigger the brain’s natural regenerative ability to recover lost motor functions.

According to the Centers for Disease Control (CDC), approximately 5.3 million people in the United States are living with a TBI-related disability and more than 280,000 people annually suffer permanent disability.i The effects of TBI are often long-lasting, with more than one-third of severe TBI patients displaying a neuromotor abnormality two years following an injury.ii

“Traumatic brain injury is among the most common health conditions faced worldwide, yet its devastating and long-lasting effects are often overlooked and underestimated,” said Dr. Okonkwo, M.D., Ph.D., professor of neurological surgery and director of the Neurotrauma Clinical Trials Center (NCTC) at the University of Pittsburgh. “The results of this study are truly groundbreaking, demonstrating the possibility of regenerating the brain following injury—a finding that could have significant implications for research in traumatic brain injury and other brain diseases.”

In this clinical study involving a total of 61 patients, 46 were treated with SB623 and 15 underwent sham surgery as a control group. Improvement was measured by the change from baseline in the Fugl-Meyer Motor Scale (FMMS) score. This scale measures changes in motor impairment and a 10 or more point improvement has been considered a clinically meaningful threshold in the context of acquired brain injury.3 Of patients treated with SB623, 18 (39.1%) reached this threshold compared to one control patient (6.7%). This difference was statistically significant (p=0.044).

No new safety signals were identified. The most commonly reported adverse event were headaches and 34.4% of patients treated with SB623 had headache up to 7 days after surgery. There were no significant differences in the rate of adverse events between patients treated with SB623 and placebo (p=0.25).

“These data are the first in the world to demonstrate the possibility of a stem cell treatment that may regenerate brain cells following traumatic brain injury, which affects thousands of people each year and has no underlying cure,” said Keita Mori, chief executive officer at SanBio. “These results represent a significant step forward in the development of regenerative medicines for neurological disorders, and we are thrilled to be able to share these findings at the largest gathering of brain surgeons in the world.”

SanBio plans to initiate a Phase 3 trial for SB623 for the treatment of chronic motor deficit from TBI by the end of fiscal year ending January 31, 2020. The company is also aiming to submit an application for manufacturing and marketing approval for its TBI program in Japan during the fiscal year ending January 31, 2020 (February 2019–January 2020), using the conditional and term-limited authorization system for regenerative medicine products under the Revised Pharmaceutical Affairs Act of Japan.

About the STEMTRA Trial

STEMTRA is a 12-month, Phase 2, randomized, double-blind, surgical sham-controlled, global trial evaluating the efficacy and safety of SB623 compared to sham surgery in patients with stable chronic motor deficits secondary to traumatic brain injury. In this study, SB623 cells were implanted directly around the site of brain injury.

To be eligible for this trial, patients (ages 18-75) must have been at least 12 months post-TBI and had a Glasgow Outcome Scale extended (GOS-E) score of 3-6 (e.g., moderate or severe disability). Patients must also have been able to undergo all planned neurological assessments and had no seizures in prior three months. The primary endpoint was mean change from baseline in Fugl-Meyer Motor Scale (FMMS) score at six months. The STEMTRA trial enrolled 61 patients from 13 surgical and 18 assessment sites in the U.S., Japan and Ukraine.

About SanBio, Inc.

SanBio is a regenerative medicine company headquartered in Tokyo and Mountain View, California, with cell-based products in various stages of research, development and clinical trials. Its proprietary cell-based investigational product, SB623, is currently in a Phase 2b clinical trial for treatment of chronic motor deficit resulting from ischemic stroke, and in a Phase 2 clinical trial for treatment of chronic motor deficit resulting from traumatic brain injury. More information about SanBio, Inc. is available at SanBio - Official Site.

i Centers for Disease Control and Prevention. Report to Congress on Traumatic Brain Injury in the United States: Epidemiology and Rehabilitation. National Center for Injury Prevention and Control; Division of Unintentional Injury Prevention. Atlanta, GA. 2014.
ii Walker WC, Pickett TC. Motor impairment after severe traumatic brain injury: a longitudinal multicenter study. Journal of Rehabilitation Research & Development. 2007;44(7):975-982.

1 Feys HM et al., 1998; van der Lee JH, et al., 2001

SOURCE: SanBio

Did you notice the SB263 is implanted during brain surgery? The patients are severely injured with severe motor dysfunction.

The ischemic stroke trials were a failure.

Yes I did notice that. It is not known whether or not IV or IT applications will work yet. But the trials for TBI panned out apparently. It surprised everyone that it didn't continue to work for stroke patients as there were some miracle cases broadcast earlier on. I imagine that with TBI you have more of the underlying tissue infrastructure remaining and thus a larger chance of meaningful recovery, whereas with stroke you are missing a huge percentage of the brain.

Meaningful recovery means the patient regained 10% more motor control than without the SB623 treatment. None of the trials suggest anything that would help with PCS.

They're just looking at motor deficits right now. It is likely that after it is approved the uses will be expanded to other affected functions. No reason to believe it can't work for PCS.

There are plenty of reasons to believe it will not work for PCS. PCS relates to a very complex high level of neurological function. They inject it into the targeted damaged area. PCS is a global problem for most.

Motor control is a very primitive neurlogical function.

The difference is like learning to say your ABCs vs learning how to do the Friday New York Times crossword puzzle in ink.

Hate to break it to you but it's already been done. The Dr. I saw has helped several people improve noticeably. This was with autologous stem cells without genetic modification. I think he is a little underqualified to be doing this but it's been done.

The various claims thus far have included improvements in vision, sleep, memory, emotional stability, memory etc. I have spoken to a number of his patients, several of whom were in car crashes. The results vary, but are all these primitive brain functions too?

It is likely that it will help, and again, IV and IT use have not been tried yet. These methods will allow the cells to cover more of the brain, as they are known to migrate on their own, and so you may get a more global improvement in function.

Placebo.......

These are not FDA approved uses because there are no FDA approved uses. They get FDA exemptions under a Generally Regarded As Safe, GRAS, exemption.

I've seen these lower level recoveries many times.

Go ahead and spend $10,000 for a try of stem cells.

Read the SB623 study results carefully.

So first we only get improvements in motor skills because motor skills are primitive functions within the brain. Stem cells can't help with PCS. Then I cite examples of improvements, but oh, they are only "lower level" so they don't count. Or they are "placebo," a highly dubious claim.

So people sleeping longer are experiencing a placebo effect? People who no longer require glasses are experiencing a placebo effect? People who are no longer alienating their friends through emotional outbursts are experiencing a placebo effect?

These claims are purely ridiculous. Without some physiological repair taking place, these things aren't going to improve. Placebo effects require a healthy brain to begin with for many things. However you engage the machinery, it needs to actually exist and function. This should be fairly obvious. Some of these people are also taking the brain gauge test before and after the treatment, which is scientifically based. You can't just say it's all in their heads. These claims are untenable. I also find it amusing that these "placebo effects" are lasting indefinitely.

Either you are asleep for longer or you're not. The fact is that SB623 is modified in a way that is specifically regenerative. The fact that you can get some recovery without doing this suggests that more targeted therapies may work better.

They are also investigating whether or not this therapy can help with various forms of dementia as well as other neurological problems, which can affect language function. But I suppose language function is too primitive to count too should we see any improvements.

Are these anecdotal claims or clinically verified claims? There have been many of these anecdotal false starts over the years. Progesterone was thought to be a miracle cure until true clinical trials were done.

Anxious persons are highly influenced by suggestion. There are many symptoms that are magnified by anxiety and seriously reduced by reductions in anxiety or by beliefs that a treatment is the big cure.

What is the cost of these stem cell treatments? Where I live, they are offered for many different conditions but cost $8000 to $10,000.

The SB623 is not an IV or IT delivery. It is a surgical delivery.

Studies show that the more expensive a treatment is, the better the patient response to the treatment. This holds true with sham treatments, therapeutic treatments, generic drugs, and branded drugs. Up to 60% of many clinical trial positive responses can be placebo. A good trial will need to calculate the placebo factor.

btw, We have had half a dozen do off shore stem cell treatments on NT. No sustained benefit.