9/11/14 Update
September 11th, 2014
1. Clinical Trials in Dogs with Type I Diabetes mellitus:
a.
The complete and very extensive paperwork (Study Protocols, Informed Consent Forms,
Review Board Applications, Budget Forms) for the initiation of the dog studies in San Diego and
at Washington State University (WSU/Pullman are submitted to the respective Institutional
Review Boards for approval of the studies (Phase I/II Clinical Trail design). In these studies the
safety, feasibility and efficacy of the SCT Hybrid Cell Therapy will be tested. The trial design is
essentially identical to the one that will be used, as presently envisaged, in human trials,
providing highly relevant and powerful large animal data in preparation for the subsequent
human studies.
b.
The Center for Veterinary Medicine (CVM) at the FDA has waived the usually required $
102,000.- fee for conducting our dog studies, indicating their interest in and support of our work.
c.
Time Line: depending on the expediency of the Institutional Review Boards at WSU and
in San Diego, we expect to carry out the first dog studies within 2-8 weeks after respective
protocol approvals have been granted and are submitted, as required, to The Center for
Veterinary Medicine (CVM) at the FDA.
d.
We are in the process of identifying the expert members of a required Data Safety and
Monitoring Board (DSMB). These are independent experts who will closely monitor, for safety
and efficacy, all data that are gathered during the entire Trial. They will report all data to the
FDA and will, at defined study points, give the “go ahead” to continue or “stop” the trials should
safety concerns arise.
e.
The needed Contracts between SCT and study site institutions are being drawn up and
reviewed by involved attorneys, liability insurance is obtained for both study sites.
f.
Master Stem Cell and Hybrid Cell Banks are finalized for release testing and shipping to
the study sites. Effective Cryopreservation protocols are in place.
2. Additional patents (IP) will be filed this month:
a.
Provisional Patents that were filed a year ago will be converted to mature patents (USA
and internationally).
b.
New Intellectual Property will be filed based on ongoing research with the continued
progress in optimizing the preparation of the therapeutic Hybrid Cells, using mouse, rat, dog and
human stem and pancreatic islet cells.
3. Studies in Diabetic Mouse Models:
a.
Spontaneously diabetic NOD (non-obese diabetic) mice (female) that develop T1DM via
autoimmune mechanisms, as patients with T1DM do, have been treated with hybrid cells and are
being followed for several weeks. Observed results resemble those previously obtained in mice
and rats that were rendered diabetic with a beta cell toxin (Streptozotocin, STZ), another
classical T1DM model.
b.
NOD/SCID (Severe Combined Immunodeficiency) Mice are being rendered diabetic
with STZ, and these are being treated with dog or human hybrid cells. Since SCID mice will
not reject dog or human hybrid cells (because of their SCID status), this model allows for the
efficacy testing of xenogeneic (foreign) cells.
SymbioCellTech Scientific Update, June 2014
June 6th, 2014
The last several months have seen significant milestones met on our path to obtaining an FDA
IND. We are working aggressively toward getting our dog trials started. To accomplish this, we
have been consulting with the Centers for Veterinary Medicine division of the FDA, developing
our protocol, and completing the studies and paperwork necessary to conduct the trial. Similarly,
we have been in negotiations with two veterinary medicine sites at which we would like to
conduct our trial in pet diabetic dogs. One site is Washington State University in Pullman, WA.
The other is Veterinary Specialty Hospital in San Diego, CA. Both sites have indicated they have
the infrastructure, experience and diabetic canine patient population necessary to conduct our
envisioned trial. We have been conducting in vitro (bench work) and in vivo (animal) studies to
characterize our cells in a manner that fulfills the FDA’s requirements. We have now isolated
islets, MSCs and ASCs from 28 dogs total, and have obtained an average of 1,072 islets per gram
of pancreas harvested, giving us an excellent cell bank with which to conduct our canine study.
Furthermore, we have obtained and studied human islets from 7 donors, and have found both
human and canine hybrids to behave in similar, beneficial ways.
In Vitro: In vitro work has focused on complete characterization of our Hybrid cells, including
extracellular markers and physiologic release of insulin in response to glucose. As shown in the
figure below, we have demonstrated that our canine hybrid cells that are intended for
implantation secrete insulin in response to glucose at levels comparable to those of freshly
isolated canine islets.
We have also demonstrated that these canine cells do not express the antigens necessary for them
to be recognized by the immune system. We have been streamlining our fusion process, and
comparing different fusion methods in order to make it as efficient as possible in order to
generate the greatest number of insulin-producing hybrids.
In Vivo: We have begun studies in Non-obese Diabetic (NOD) mice to test our protocol in an
autoimmune rodent model of T1DM (as opposed to our STZ rat and mice models previously
studied). At the same time, we are starting FDA required studies of our canine and human hybrid
cells in an immune compromised mouse strain: Severe Combined Immuno-deficient (NODSCID) mice.
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Financial and Administrative Update, June 2014
June 5th, 2014
Funding
Two of our Finance Advisory Committee members, Dr. Joel Katz and James Horton, along with
Allen Gardner, another investor, have helped SCT to raise an additional $2,555,000 during the
2nd round of financing, (which is due to close on July 31, 2014). All funds generated were from
angel investors and all membership shares were priced at exactly $50.00 each.
Through the enormous efforts of Jim Horton, Managing Partner of ICT Holdings, SCT received
$2,000,000 from a single source, H2M Capital, LLC.
These additional funds will sustain the company until the third or fourth quarter of 2015.
Our thanks to Allen, Joel and Jim for the countless hours of work they dedicated to help us reach
our goal. Their efforts are greatly appreciated by all of us and the funds will enable SCT to
concentrate on the research going forward.
Financial
The current burn rate is approximately $120,000 per month for the first quarter. The lab made
two significant acquisitions to help in its research. A large autoclave was purchased and is used
for sterilization of liquids and solids, as well as cages for the immune compromised mice. These
animals are being used to test SCT’s human and canine cell lines. A new hood was also
purchased which is needed for sterile handling of the immune compromised mice. These items
totaled approximately $45,000 and will last for an extensive period of time. SCT does not
estimate any further material purchases of equipment for the lab within the next year.
We are still evaluating our budget additions in the areas of the dog studies and the additional
facilities that may be necessary going forward in relation to the dog trials.
Administration
Finance Advisory Committee, Science Advisory Committee and Board meetings are being
planned for the near future. SCT is currently waiting for information from outside sources that
would be relevant to the upcoming meetings.
We would like to thank everyone for their support. Research has its own pace, and our investors
are a key element in this venture. SCT will post the minutes from the upcoming meetings for
your review.
Thank you,
The Management of SCT
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Scientific Update, Oct. through Dec. 2013
December 18th, 2013
We have been focusing on reproducing our fusion and characterization of islet cells with stem
cells derived from canine and human tissues. We have now generated and characterized fusion
lines from 12 dogs and 3 human donors. We have been very pleased to find that both human and
canine cell lines and fusion products grow and passage well in our hands, and that they make
insulin, glucagon, and somatostatin. In this fashion, the hybrid cells represent a mini version of
the islet.
Figure 1, below, shows fusion of human islet cells (red) to human MSCs (green), and
demonstrates that we can fuse human cells from primary tissues with high efficiency, as we will
need to do for our clinical trial and off the shelf application.
Figure 1 : Fused human cells appear orange or bicolored.
Figure 2, below, is an image of a culture of fused canine β-MSCs stained for the three primary
islet hormones, insulin (green), glucagon (red) and somatostatin (purple), and nuclear content
(blue). These data demonstrate that the generated hybrid cells reproduce the cell biology of an
intact islet in the physiological ratios. Figure 2a is a split image; Figure 2b shows the composite.
Figure 2a: Fused, redifferentiated canine β-MSCs stained for insulin (green), glucagon (red),
somatostatin (purple), nuclei (blue).
Figure 2b:: enlarged composite image from figure 2a, bottom, right panel.
Click on any figures to view an enlarged version.