This was on the other forum i am on - quite exciting if it works!🙂Bev
The Faustman Lab at Massachusetts General Hospital, run by Denise L.
Faustman, MD, PhD, is moving rapidly through the clinical trial
challenge to test and possibly establish a vaccine using a generic drug,
Bacillus Calmette-Gu?rin (BCG) to reverse Type 1 diabetes.
The experiments have moved from mice to a clinical trial in humans, and
the testing has passed every research milestone ahead of schedule -- an
astonishing feat for any clinical research project. The Phase I safety
trial in people with Type 1 diabetes is near completion. The next step
is to find, in a Phase II study, the possible dose and frequency of
administration of BCG vaccinations that will benefit patients with Type
1 diabetes.
What makes these trials unique? First, unlike other immunosuppressive
therapies for autoimmune diseases that harm both healthy and
disease-causing T-cells, this treatment appears to provide a way to
achieve "targeted removal" of only autoimmune disease-causing cells. BCG
works by causing the release of a natural protein in the body called
tumor necrosis factor, or TNF. In mice, temporarily elevating TNF levels
destroys the autoreactive T-cells, allowing the insulin-producing cells
of the pancreas to regenerate and produce insulin. TNF in mice also
elevates a good population of T-cells, thus restoring the immune system
to near normal. In other words, the treatment literally reverses Type 1
diabetes, at least in a mouse model of diabetes. In laboratory
experiments, TNF also destroys the autoreactive cells in human blood
samples. TNF does not harm the normal, healthy T-cells that help fight
infection, leaving the immune system intact.
The human clinical trials seek to expand these findings in human
subjects. The BCG trial is also unique since BCG is a generic drug that
has been used widely as a vaccine for more than 80 years in humans.
Currently, BCG is used in small doses as a vaccine against tuberculosis,
and it is used in larger doses as a bladder cancer therapy. Because it
has been used for so long and in so many humans, the safety of this
vaccine is well-established. This fact allows the trials to move more
quickly through safety studies compared to clinical trials that test new
drugs.
With a generic drug, it can move BCG to the public rapidly if the drug
is indeed found to be safe and effective in patients with Type 1
diabetes. The phase I safety study is following the enrolled patients
over months looking for changes in the T-cells and also testing the
reliability of the blood tests for tracking disease-causing T-cells.
The BCG trial is one of the few translational studies in human testing
where the animal data showed disease reversal in diabetic mice that had
hyperglycemia, not just in mice with normal blood sugars and with a
predisposition to diabetes. Therefore, this human trial at Mass General
is designed to treat people with established Type 1 diabetes unlike most
human testing that focuses only on not yet diabetic patients at risk for
the disease or newly diagnosed diabetics with a short time period of
high blood sugars.
The Faustman Lab at Massachusetts General Hospital, run by Denise L.
Faustman, MD, PhD, is moving rapidly through the clinical trial
challenge to test and possibly establish a vaccine using a generic drug,
Bacillus Calmette-Gu?rin (BCG) to reverse Type 1 diabetes.
The experiments have moved from mice to a clinical trial in humans, and
the testing has passed every research milestone ahead of schedule -- an
astonishing feat for any clinical research project. The Phase I safety
trial in people with Type 1 diabetes is near completion. The next step
is to find, in a Phase II study, the possible dose and frequency of
administration of BCG vaccinations that will benefit patients with Type
1 diabetes.
What makes these trials unique? First, unlike other immunosuppressive
therapies for autoimmune diseases that harm both healthy and
disease-causing T-cells, this treatment appears to provide a way to
achieve "targeted removal" of only autoimmune disease-causing cells. BCG
works by causing the release of a natural protein in the body called
tumor necrosis factor, or TNF. In mice, temporarily elevating TNF levels
destroys the autoreactive T-cells, allowing the insulin-producing cells
of the pancreas to regenerate and produce insulin. TNF in mice also
elevates a good population of T-cells, thus restoring the immune system
to near normal. In other words, the treatment literally reverses Type 1
diabetes, at least in a mouse model of diabetes. In laboratory
experiments, TNF also destroys the autoreactive cells in human blood
samples. TNF does not harm the normal, healthy T-cells that help fight
infection, leaving the immune system intact.
The human clinical trials seek to expand these findings in human
subjects. The BCG trial is also unique since BCG is a generic drug that
has been used widely as a vaccine for more than 80 years in humans.
Currently, BCG is used in small doses as a vaccine against tuberculosis,
and it is used in larger doses as a bladder cancer therapy. Because it
has been used for so long and in so many humans, the safety of this
vaccine is well-established. This fact allows the trials to move more
quickly through safety studies compared to clinical trials that test new
drugs.
With a generic drug, it can move BCG to the public rapidly if the drug
is indeed found to be safe and effective in patients with Type 1
diabetes. The phase I safety study is following the enrolled patients
over months looking for changes in the T-cells and also testing the
reliability of the blood tests for tracking disease-causing T-cells.
The BCG trial is one of the few translational studies in human testing
where the animal data showed disease reversal in diabetic mice that had
hyperglycemia, not just in mice with normal blood sugars and with a
predisposition to diabetes. Therefore, this human trial at Mass General
is designed to treat people with established Type 1 diabetes unlike most
human testing that focuses only on not yet diabetic patients at risk for
the disease or newly diagnosed diabetics with a short time period of
high blood sugars.
