A new therapy for treating Type 1 diabetes using stem cell therapy!

Promising early results show that longstanding Harvard Stem Cell Institute (HSCI) research may have paved the way for a breakthrough treatment of Type 1 diabetes. Utilizing research from the Melton Lab, Vertex Pharmaceuticals has developed VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with type 1 diabetes (T1D). In conjunction with immunosuppressive therapy, VX-880 produced robust restoration of islet cell function on Day 90 in the first patient in its Phase 1/2 clinical trial.

The patient was treated with a single infusion of VX-880 at half the target dose in conjunction with immunosuppressive therapy. The patient, who was diagnosed with T1D 40 years ago and has been dependent on exogenous (injected) insulin, achieved successful engraftment and demonstrated rapid and robust improvements in multiple measures. These included increases in fasting and stimulated C-peptide, improvements in glycemic control, including HbA1c, and decreases in exogenous insulin requirement, signifying the restoration of insulin-producing islet cells.

VX-880 is not only a potential breakthrough in the treatment of T1D, it is also one of the very first demonstrations of the practical application of embryonic stem cells, using stem cells that have been differentiated into functional islets to treat a patient, explained Doug Melton, Ph.D., co-director of HSCI, is the Xander University Professor at Harvard and an Investigator of the Howard Hughes Medical Institute. Unlike prior treatments, this innovative therapy gives the patient functional hormone producing cells that control glucose metabolism. This potentially obviates the lifelong need for patients with diabetes to self-inject insulin as the replacement cells “provide the patient with the natural factory to make their own insulin,” explained Melton.

These results from the first patient treated with VX-880 are unprecedented. What makes these results truly remarkable is that they were achieved with only half the target dose,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. “While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression.”

“As a surgeon who has worked in the field of islet cell transplantation for decades, this approach, which obviates the need for an organ donor, could be a game changer,” said James Markmann, M.D., Ph.D., Professor of Surgery and Chief of the Division of Transplant Surgery at Massachusetts General Hospital. “We are excited to progress this unique and potentially transformative medicine through clinical trials and to patients.”

“More than a decade ago our lab had a vision for developing an islet cell replacement therapy to provide a functional cure to people suffering from T1D,” said Melton, a founder and one of the first co-chairs of the Harvard Stem Cell and Regenerative Biology Department. “These promising results bring great hope that stem cell-derived, fully differentiated islet cells could deliver a life-changing therapy for people who suffer from the relentless life-long burden of T1D. I’m so grateful that Harvard and the Harvard Stem Cell Institute have supported this work.”

For more information on this clinical trial, please click on the link below:

https://hsci.harvard.edu/news/new-therapy-treating-type-1-diabetes

 

 

 

Diabetes Can Lead to Amputations, But Stem Cell Treatment Offers Hope!

 

 

One of the most dangerous complications of diabetes is a foot ulcer that won’t heal, but now a preliminary study finds that a type of stem cell found in body fat may be a powerful remedy for these severe foot wounds. Diabetes Can Lead to Amputations, But Stem Cell Treatment Offers Hope!

The study included 63 patients with non-healing diabetic foot ulcers who were given injections of cells from their own body fat. Over the next year, the treatment healed the ulcers in most patients. Researchers said the study — conducted in Nicaragua — lays the groundwork for a similar trial in the United States, to replicate the findings.

Diabetic foot ulcers are open sores or wounds that affect roughly 15% of people with diabetes, according to the American Podiatric Medical Association (APMA). For some, the wounds refuse to heal and can become infected — sometimes leading to amputation.

In the United States alone, studies show that more than half of all amputations are diabetes-related — and non-healing foot ulcers are usually the reason why. People with diabetes are vulnerable to foot ulcers for several reasons, explained study author Dr. Michael Carstens.

To read the entire article, please click on the link below:

https://www.usnews.com/news/health-news/articles/2021-04-14/diabetes-can-lead-to-amputations-but-stem-cell-treatment-offers-hope

Stem Cell transplants can benefit some Type 2 diabetes patients.

 

Durham, NC — Type 2 diabetes patients who are not overweight and who have had the disorder for less than a decade can benefit from stromal stem cells transplanted from their own bone marrow, according to a study published today in STEM CELLS Translational Medicine.

In a randomized clinical trial at Vinmec Research Institute of Stem Cell and Gene Technology in Hanoi, Vietnam, researchers investigated the safety and potential therapeutic value of administering bone marrow stromal stem cells to patients with Type 2 diabetes. In each case, the cells were autologous, or taken from the patients’ own bodies.

A total of 30 adult patients with different body mass indexes whose Type 2 diabetes histories varied from one to 25 years were recruited for the study. Each received two infusions of the cells intravenously or by injection into an artery that supplies blood to the pancreas.

To read more, please click on the link below:

Bone marrow stem cell transplants can benefit some Type 2 diabetes patients, study shows

 

New hope for stem cell approach to treating diabetes – Washington University Study

Researchers at Washington University School of Medicine in St. Louis have tweaked the recipe for coaxing human stem cells into insulin-secreting beta cells and shown that the resulting cells are more responsive to fluctuating glucose levels in the blood. Here, the new beta cells appear red as they secrete insulin in response to glucose.
Credit: Millman lab, Washington Universityientists working to develop more effective treatments for diabetes are turning to stem cells. Such cells can be transformed into cells that produce insulin, the hormone that controls blood sugar.

But there’s a major challenge: the amount of insulin produced by theses cells is difficult to control.

Now, by tweaking the recipe for coaxing human stem cells into insulin-secreting beta cells, a team of researchers at Washington University School of Medicine in St. Louis has shown that the resulting cells are more responsive to fluctuating glucose levels in the blood.

 

When they transplanted the beta cells into mice that could not make insulin, the new cells began secreting insulin within a few days, and they continued to control blood sugar in the animals for months.

The new study is published Jan. 17 in the journal Stem Cell Reports.

“We’ve been able to overcome a major weakness in the way these cells previously had been developed. The new insulin-producing cells react more quickly and appropriately when they encounter glucose,” said principal investigator Jeffrey R. Millman, PhD, an assistant professor of medicine and of biomedical engineering. “The cells behave much more like beta cells in people who don’t have diabetes.”

The researchers now believe it may be time to evaluate whether the same stem-cell approach could produce insulin and effectively control blood sugar in people.

Millman was a part of a research team at Harvard that, in 2014, converted skin cells into stem cells and, in 2016, did the same thing with skin cells from a patient with diabetes. Each time, the stem cells were then treated with various growth factors to coax them into insulin-secreting beta cells. The beta cells, however, didn’t work as well as the researchers had hoped.

“Previously, the beta cells we manufactured could secrete insulin in response to glucose, but they were more like fire hydrants, either making a lot of insulin or none at all,” he said. “The new cells are more sensitive and secrete insulin that better corresponds to the glucose levels.”

For this study, Millman’s laboratory still grew beta cells from human stem cells, but they made numerous changes to the “recipe” for producing insulin-producing beta cells, treating the cells with different factors at different times as they grew and developed to help the cells mature and function more effectively.

After that process was complete, the researchers transplanted the beta cells into diabetic mice with suppressed immune systems so that they wouldn’t reject the human cells. Those transplanted cells produced insulin at levels that effectively controlled blood sugar in the mice, functionally curing their diabetes for several months, which, for most of the mice in the study, was about the length of their lives.

As laboratory researcher rather than a clinician, Millman said he can’t predict exactly when such cells may be ready for human trials but believes there are at least two ways that stem cell-derived beta cells could be tested in human patients.

“The first would be to encapsulate the cells in something like a gel — with pores small enough to prevent immune cells from getting in but large enough to allow insulin to get out,” he said. “Another idea would be to use gene-editing tools to alter the genes of beta cells in ways that would allow them to ‘hide’ from the immune system after implantation.”

Millman said that if stem cell-derived beta cells are proven safe and effective for people with diabetes, his method of manufacturing the cells quickly could be ramped up to an industrial scale. In his laboratory alone, his team is able to grow and develop more than a billion beta cells in just a few weeks.

Story Source:

Materials provided by Washington University School of MedicineNote: Content may be edited for style and length.


Journal Reference:

  1. Leonardo Velazco-Cruz, Jiwon Song, Kristina G. Maxwell, Madeleine M. Goedegebuure, Punn Augsornworawat, Nathaniel J. Hogrebe, Jeffrey R. Millman. Acquisition of Dynamic Function in Human Stem Cell-Derived β CellsStem Cell Reports, 2019; DOI: 10.1016/j.stemcr.2018.12.012

 

 

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