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For the first time, it targets the root cause of the onset of T1D, and clinical trials have shown that it delays the clinical onset of T1D in high-risk groups by nearly 3 years
on average.
Written by Ling Jun
On November 17, the U.
S.
Food and Drug Administration (FDA) approved a marketing application for the CD3 monoclonal antibody Teplizumab for the delay or prevention of clinical type 1 diabetes mellitus (T1D) in adults and children over 8 years of age.
It is also the world's first immunomodulatory therapy
approved for the prevention of T1D.
The industry believes that the approval of Teplizumab marks a "new era" in diabetes treatment, which targets the root cause of T1D for the first time, and clinical trials have shown that it has delayed the clinical onset of T1D in high-risk groups by nearly 3 years
on average.
"This first-of-its-kind therapy adds important new treatment options
for people at high risk of T1D.
" John Sharretts, MD, of the FDA's Center for Drug Evaluation and Research, believes that this potential to delay disease progression could free patients from the burden of
disease for months to years.
Image source: Ruijing
T1D, also known as insulin-dependent diabetes, has delayed the onset of T1D for nearly 3 years, and most of it is an autoimmune disease
.
The patient's immune system destroys the cells that make insulin, pancreatic islets β cells, resulting in insulin deficiency and elevated blood sugar, and its pathogenesis has not yet been understood, mostly thought to be related
to genetic and environmental factors.
Unfortunately, there is still no effective means to prevent and cure T1D, and patients mainly control blood glucose levels through long-term, regular insulin injections and oral hypoglycemic drugs to avoid a variety of serious complications such as diabetic nephropathy, diabetic retinopathy and neuropathy
.
Teplizumab's approval could be a game-changer
.
As an anti-CD3 monoclonal antibody, it binds to CD3 on the surface of effector T cells to inhibit the body's immune system from attacking pancreatic islet β cells from destruction
.
The key data for approval were first published in the New England Journal of Medicine in 2019, and the Phase 2 trial enrolled 76 subjects who were not diabetic but all had a family history and were diagnosed as "at high risk of clinical episodes of diabetes
.
"
Among them, the treatment group received intravenous teplizumab for 14 days, after which no other drugs were needed, and the placebo group was followed up
.
At a median follow-up of 745 days, T1D was diagnosed in 19 (43%) teplizumab-treated participants compared with 23 (72%) in the placebo group, with a relative hazard ratio of 0.
41
.
In addition, teplizumab greatly delayed the clinical onset of T1D, with a median time to diagnosis of 48.
4 months in the teplizumab group and 24.
4 months
in the placebo group.
The trial also found that the best efficacy of teplizumab occurred in the first year, when 44 percent of participants in the placebo group were diagnosed with T1D, compared with only 3 percent
in the treatment group.
Further extended trial data released in 2021 showed that the efficacy of the initial 2-week administration continued
.
At a median follow-up of 2.
5 years, 50% of patients in the teplizumab group and 78% of patients in the placebo group were diagnosed with T1D, with a mean time of onset of 59.
6 months and 27.
1 months, respectively – teplizumab delayed the onset of T1D by nearly 3 years
.
"This is a historic moment
for the T1D patient population.
" A spokesman for the pharmaceutical manufacturer said that although it cannot be done "once and for all" for the time being, the average delay of nearly 3 years of disease onset can alleviate the disease burden of the whole society, and patients have enough time to wait for the birth
of future breakthrough therapies.
The company will also conduct teplizumab clinical trials in the T1D patient population that has already developed the disease to explore whether it can further expand the indications of the drug, and the company disclosed that it completed its goal
of recruiting 300 patients for enrollment last year.
being developed simultaneously, according to the Lancet published in The Lancet According to a new model study in Diabetes and Endocrinology, an estimated 8.
4 million people worldwide had T1D in 2021 , 175,000 deaths due to T1D, by 2040 T1D The number of patients can reach 17.
4 million.
In 1922, at the age of 14, Leonard Thompson became the first T1D patient
to receive exogenous insulin therapy.
Exactly 1 century later, despite tremendous advances in the treatment of diabetes, most research has focused on optimizing insulin products and their delivery
.
In the face of such a huge burden of disease, there is also an urgent need for breakthrough treatment advances that will eventually replace daily insulin injections and free patients from continuous medication and disease surveillance
.
For Teplizumab, it took more than 20 years
from initial discovery to approval.
But teplizumab
isn't alone in exploring T1D prevention or functional cure.
At present, various cutting-edge therapies mainly focus on islet transplantation, stem cell therapy and other fields
.
In the field of stem cell therapy, at the annual scientific meeting of the American Diabetes Association (ADA) held in June this year, Vertex disclosed the clinical trial data of the stem cell product VX-880 under development, which caused a lot of sensation
.
VX-880 is an allogeneic stem cell used in T1D therapy that differentiates into islet cells and secretes insulin
.
Follow-up data showed that a man with T1D for more than 40 years had been in the normal range for more than 9 months after treatment, and no longer needed to use any exogenous insulin
.
Another woman reduced her amount of insulin needed by 30%
after 5 months of use.
However, rejection is an important pain point of VX-880 therapy, and patients receiving treatment need to take immunosuppressive drugs
for a long time.
The PEC-Encap therapy, developed by ViaCyte, attempts to solve this problem
.
PEC-Encap is the world's first embryonic stem cell-derived therapy for diabetes to enter clinical trials, which puts implanted cells into a closed "pocket" that can not only produce insulin but also "isolate" the attack of the human immune system
.
CRISPR gene editing technology is also playing a role
in T1D treatment.
The PEC-QT therapy, also developed by ViaCyte, uses CRISPR to knock out genes that trigger the expression of proteins involved in the body's rejection response, protecting the transplanted cells from attack
by the host immune system.
Currently, a Phase 2 clinical trial of PEC-Encap is underway
.
In February, ViaCyte announced that the first patient had received PEC-QT therapy, marking the first ever implantation of gene-edited, stem-derived islet β cells into the human body
.
For islet transplantation, also at this year's ADA Scientific Meeting, regenerative medicine cell therapy developer Sernova announced the results of a Phase 1/2 clinical trial of its unique cell pouch system, in which 3 T1D patients were treated with insulin for more than 2 years, 6 months and 3 months
after using innovative therapies based on the cell pouch system.
This innovative therapy consists of cell pouch pockets, islet cells and immunoprotective measures, first implanting cell pouch into the patient through minimally invasive surgery to fully integrate it with human vascularized tissue, and then injecting donor islet cells into cell pouch's microtubules to replace
the islet cells in the autologous pancreas.
It is worth mentioning that based on the cell pouch system principle, Sernova will also develop the latest cell therapy to treat hypothyroidism
caused by thyroidectomy.
Source: Medical community
Responsible editor: Tian Dongliang
Proofreader: Zang Hengjia
Editor: Zhao Jing
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