Vertex's first patient's stem cell-derived therapy results in the treatment of type 1 diabetes on the 90th day

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Recent popular reports from Yimaike ★How much do you know about AAV gene therapy for hemophiliaYikemai classroom★ "Spot" G-NK cells are here! Pre-clinical data shows stronger tumor killerYimai Meng broke the news October 20, 2021 The first patient in the Phase 2 clinical trial has positive data on the 90th day
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VX-880 (STx-02) is a research-type allogeneic stem cell-derived islet cell therapy.
This cell therapy is used to treat type 1 diabetes (T1D) and achieve the stable recovery of islet cell function
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The patient received half of the target dose of VX-880 through a single infusion and immunosuppressive therapy
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After the infusion of the drug, patients showed rapid and powerful improvements, including an increase in fasting and stimulated C-peptide, an improvement in blood sugar control, a reduction in HbA1c and exogenous insulin requirements
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Dr.
Bastiano Sanna, Executive Vice President and Head of Cell and Genetic Therapy at Vertex, said: "The positive results obtained by the first patient treated with VX-880 are unprecedented
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What
is really striking is that they are at half the target dose.
under implementation
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Although too early to Shihai, these results support our clinical study of VX-880 continued to advance, and these have the potential to islet cells without the need to use immunosuppressive
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"Xander Harvard University professor, researcher Doug Melton Howard Institute The doctor said: "More than ten years ago, our laboratory had a vision to develop islet cell replacement therapy to provide functional treatment for people with T1D
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Later, our vision has great hope, namely, through stem cell-derived, fully differentiated pancreatic islet cells to provide T1D patients with new therapies that can change their lives
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"Stem cell therapy for the treatment of type 1 diabetes.
At present, diabetes is a global health problem and one of the most important causes of death.
The disease affects more than 400 million people in the world, and the prevalence rate is increasing year by year, and the global growth rate is increasing.
51%, while the existing 463 million diabetic patients, according to the growth trend to 2045 the world will have 700 million diabetes patients
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the number of people suffering from diabetes in China ranks first in the world, about 116.
4 million people
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T1D accounts for diabetics 5% to 10% of the total amount.
It is obviously characterized by early onset, usually in childhood or adolescence
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T1D is caused by the autoimmune system destroying the islet cells that secrete insulin in the pancreas, resulting in loss of insulin secretion and blood sugar control Disorders
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Lack of insulin can cause abnormalities in the body’s processing of nutrients, leading to increased blood sugar
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Hyperglycemia can lead to diabetic ketoacidosis, which over time can lead to kidney disease/failure, eye diseases (including loss of vision) ), heart disease, stroke, nerve damage and even death
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At present, the main method of diabetes treatment is insulin injection and blood glucose monitoring
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Due to the limitations and complexity of the insulin delivery system, it may be difficult for T1D patients to achieve and maintain blood glucose control balance
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in addition, in vitro complement traditional insulin therapy can not be fundamentally, and control of diabetic complications also inadequate, so the disease there is a high unmet medical need
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VX-880 is an experimental iso Gene stem cell therapy, fully differentiated, insulin-producing islet cell therapy, manufactured using proprietary technology
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VX-880 has the potential to restore the body's ability to regulate glucose levels by restoring pancreatic islet cell function (including glucose-responsive insulin production)
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VX-880 is delivered by injecting into hepatic portal vein and requires long-term immunosuppressive therapy to protect islet cells from immune rejection
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The Phase 1/2 clinical trial is a multi-center, single-arm, open-label study, mainly for T1D patients with impaired hypoglycemia awareness and severe hypoglycemia
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The test is also designed as a continuous, multi-part, to evaluate the safety and effectiveness of VX-880
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The patient who announced the 90-day trial study data was diagnosed with T1D about 40 years ago and has been dependent on exogenous insulin
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In the year before treatment, the patient experienced 5 severe and potentially life-threatening episodes of hypoglycemia
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Prior to treatment with VX-880, the patient’s insulin dose was 34 units per day, and fasting and stimulated C-peptide levels could not be detected.
The C-peptide level was a direct marker of insulin production, indicating that the patient did not make their own insulin.
Ability
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According to the Vertex study, the patient received a standard immunosuppressant regimen, which was an infusion of half of the target dose of VX-880 through the hepatic portal vein
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The fasting C-peptide, HbA1c and 7-day average daily insulin dose were measured at different time intervals 90 days after the patient received VX-880 treatment
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Through research, it was found that before receiving VX-880 treatment, fasting and stimulated C-peptide levels were not detected, indicating that there is no endogenous insulin production
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On the 90th day after receiving VX-880 treatment, the fasting peptide was 280pmol/L, which indicated that VX-880 restored glucose-responsive insulin production
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HbA1c was reduced from 8.
6% before treatment to 7.
2%, and the daily insulin dose was reduced from 34 units per day before VX-880 treatment to an average of 2.
9 units per day
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On the 90th day, the patient found that the daily use of exogenous insulin had been reduced by 91% based on the previous use
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It can be seen that VX-880 has played a great help in restoring the function of islet cells
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From a safety point of view, in the first patient, the safety of VX-880 is basically the same as the immunosuppressive regimen used in this study, and there is no serious adverse event (SAE) related to VX-880.
Most adverse events are considered mild to moderate
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The most common adverse event was severe hypoglycemia, which was not related to VX-880
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On the 90th day, the patient developed a mild rash, which was not directly related to VX-880 through research
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Based on these data, Vertex plans to continue to advance the VX-880 Phase 1/2 plan
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At present, the drug has multiple active sites in the United States, and the drug has been approved for clinical trials in Canada
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In summary, stem cells are used to treat type 1 diabetes.
As early as 2019, Time magazine included stem cells in the treatment of diabetes in the list of 12 innovations that will change medical care in the next decade
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Therefore, more and more companies/institutions are developing stem cell therapy projects for T1D
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In August 2020, Salk researchers used stem cell technology to generate the first insulin-producing human pancreatic cell population (human islet-like organoid) that can evade the immune system.
Once these "immune shielded" cell clusters are transplanted into the body, Control blood sugar without immunosuppressive drugs
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Recommended reading: Stem cell treatment of diabetes has another new trick, creating the first human islet-like organoid that evades immune surveillanceYimai Meng broke the news.
Not only that, but also many companies are committed to this, such as Evotec, Sanofi, and Nuo Nordisk (Novo Nordisk), Lilly (Eli Lilly) and Sigilon, PharmaCyte and so on
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In China, a number of projects on stem cell research have also been filed
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Reference materials: 1.
https:// -trial-dosed-with-vx-880-a-novel-investigational-stem-cell-derived-therapy-for-the-treatment-of-type-1-diabetes/