this treatment The type 1 diabetes approach hasn’t been clinically validated on a large scale, but it suggests that Vertex Pharmaceuticals is at least on the right track.
The Boston-based biotechnology company announced on the morning of October 18, 2021, that a diabetic patient Test results for:
He was at Massachusetts General Hospital After three months of treatment, an injection of Vertex’s experimental stem cells, the patient’s daily insulin requirements dropped by more than 90%, and no serious side effects were found. To be on the safe side, he only took half the target dose.
This patient with type 1 diabetes has diabetes age For 40 years, it had previously been dependent on exogenous insulin. In the year prior to treatment, he experienced 5 severe, life-threatening symptoms of hypoglycemia.
Before treatment, the patient’s insulin dose was 34 doses per day units, and fasting and stimulated C-peptide levels were undetectable, suggesting that the patient was unable to produce insulin on his own.
Day 90 after treatment, fasting C-peptide was 280 pmol/L, reflecting restoration of basal insulin production; daily insulin doses were reduced from 34 units per day before treatment to an average of 2.9 units per day over the 7-day period, resulting in a 91% reduction in daily exogenous insulin use.
Vertex Executive Vice President and Bastiano Sanna, PhD, Head of Cell and Genetic Therapies, said: “These results are unprecedented. What’s really striking is that they were achieved at half the target dose. Although it’s early days, these results support our clinical research. continued progress.”
< span>Professor James Markmann, Chief of Transplant Surgery at Massachusetts General Hospital, said: “As a surgeon who has worked in islet cell transplantation for decades, I am delighted to have a clinical trial that will allow patients to use This potentially transformative, organ transplant-free approach could be an industry game changer.”
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This patient is just the first in a small clinical trial that will enroll up to 16 people, and Vertex is encouraged by the results , published the results of the study.
The treatment basically turns stem cells into insulin-producing Islet cells, similar to the beta cells naturally found in the pancreas.
Type 1 diabetics’ pancreatic beta cells are destroyed, lead to an absolute lack of insulin. These new cells developed by Vertex can enter and take their place, a form of regenerative medicine.
Vertex CEO Jeff Leiden said: “This It’s a milestone in medicine, and suddenly we have a different kind of treatment. It’s not just about treating the symptoms, it’s getting to the root of the disease.”
This therapy, known as VX-880, can be traced back to the Harvard lab run by Doug Melton, who worked with his son and daughter After being diagnosed with type 1 diabetes, he changed the direction of his research to focus on the replacement of pancreatic beta cells with stem cells.
Melton founded the company Semma Therapeutics to advance research, which was not tested in animals until this year.
Vertex acquired Semma in 2019 for $950 million.
In the United States alone, there are approximately 1.6 million. Control their blood sugar by monitoring their blood sugar levels and injecting insulin multiple times a day.
Leiden said insulin injections still cannot rule out side effects, and sometimes even Serious, such as blood clots and strokes.
Leiden said: “Insulin It’s a lifesaver, but it won’t cure the disease at all. Our goal is to make these patients insulin dependent.”
Leiden hopes that the VX-880 will fundamentally solve this problem, although it may take years before it hits the market.
Vertex claims that this For the first time, stem cell therapy in patients with type 1 diabetes has demonstrated the recovery of islet cell function.
Currently, Vertex plans to The treatment is being tested on people with diabetes because the process requires a dose of the drug to suppress the patient’s immune system so that it doesn’t attack new cells.
Vertex is also working on a way to avoid using these An alternative to the drug: A capsule that converts stem cells into islet cells and implants in the abdomen. The capsule wall acts as a barrier against the body’s defense system, allowing islet cells to regularly produce insulin and deliver it into the bloodstream.
Competitor ViaCyte, also begins stem cell therapy Type 1 Clinical trials for diabetes, but a Vertex spokesman said the results in patients treated with VX-880 were the best of any trial reported so far.
Sources| The Boston Globe, Business Wire span>