Community Response To Vertex Results
The media hype around Vertexs initial results is not without controversy. Those of us whove lived with this condition for years are naturally skeptical about any talk of a possible cure since weve heard it so many times before.
Regarding Vertex, many in the Diabetes Community are pointing out on social media how immunosuppression drugs are still required at this time, which is a big drawback. Others note how the Vertex data here isnt yet peer-reviewed and wont even be published in a medical journal until after the second round of clinical trials end in 2028.
Others also pointed out the critical issue of affordability, noting that Vertexs practices that are highly expensive and unaffordable for those who need them along the lines of the cystic fibrosis drug Orkambi at a list price of $272,000 per year. For those who already struggle to afford healthcare or their insulin to survive, the idea of such a high-end concierge diabetes treatment only available to some is not appealing.
Nevertheless, the JDRF praised the research findings, noting their own support in this area of research dating back to Meltons work in 2000.
Melton, by the way, is now consulting with and owns stock at Vertex, so naturally hes a big fan of this foundational work.
He says Vertexs recent announcement led his family particularly his two T1D children, Sam and Emma to have tears in their eyes.
Stop Diabetes Before It Starts
Andrea Haqq is harnessing the power of a proven therapy and a multidisciplinary team to prevent Type 2 diabetes in youth.
How it works
Childhood obesity is a precursor to insulin resistance and eventually Type 2 diabetes. Haqqs treatment uses a combination of dietary fibre and metformin a drug already used to treat Type 2 diabetes in adults to reverse obesity and insulin resistance and prevent Type 2 diabetes in children.
Yesterdays research, tomorrows treatment
Metformin has been used to treat Type 2 diabetes in adults since 1957, however it hasnt been successful in children. Haqq, a clinician-scientist at the Alberta Diabetes Institute and professor of pediatrics at the U of A, took a different approach to the drug. She and her team are using metformin alongside dietary fibre to reprogram the gut microbiome in children in a way that curbs insulin resistance. Haqq was able to pursue this line of research because of the collaborative team and clinical testing facilities at the Alberta Diabetes Institute. Their work has set the stage for a preventative therapy with the potential to reverse insulin dependence and prevent diabetes in children.
What a cure looks like
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Diabetes: Will It Ever Be Cured
While the end to diabetes is still in the distant future, strides in genetic research are showing promise.
Immunology and beta cell function have long been two core areas of research in the hunt for a cure for diabetes. But in recent years, scientists have made discoveries that could lead to genetic therapies that allow the bodys own cells to combat and even rid itself of the disease. Researchers are learning to turn gut cells into insulin-producing cells, replenish beta cells once thought decimated beyond hope, and use viral vectors to deliver genes into beta cells that may protect them from attack by the immune system.
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Nd Contender: The Bcg Vaccine From Dr Faustman
The BCG vaccine has existed for nearly 100 years. Originally designed to prevent tuberculosis in areas of the world with higher risks of developing the disease. Dr. Denise Faustman began researching the use of this vaccine in people with type 1 diabetes at the Faustman Lab at Massachusetts General Hospital.
The simple result was that the vaccine was regenerating the pancreas ability to produce beta-cells, which in-turn successfully produced insulin.
The End Of Insulin: Research Is Bringing Us Closer To A Cure Than Ever Before
A hundred years ago, diabetes became treatable. Today, five researchers are working to make insulin treatments obsolete
Lisa Szabo – 01 November 2021
Illustrations by Jordan Carson
For many of the 465 million people around the world living with diabetes, insulin is a lifeline.
Since their bodies dont produce enough of the hormone naturally or cant properly use what they do produce a strict regimen of daily injections helps people with Type 1 diabetes and around a tenth of people with Type 2 diabetes ward off life-threatening health problems.
The University of Alberta has had a hand in insulin research since the beginning, when biochemist James Collip, 24 PhD, 26 MD, 46 LLD , first purified a type that could be injected into patients with diabetes to combat excess sugar in the blood. That was a century ago. Since then, U of A researchers have vaulted off Collips life-changing discovery to develop better treatments for diabetes, including the Edmonton Protocol.
While insulin has saved countless lives, its not a treatment to take lightly. Many people with diabetes live under the fear of a slip-up an over- or under-dose that could end in a seizure, coma or death. Insulin is a lifeline, but its not a cure.
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Stem Cells May Finally Offer A Cure For Type 1 Diabetes
There are 537 million people around the world living with diabetes. And that number is growing.
When people have Type 1 diabetes, the immune system attacks and destroys the beta cells in the pancreas that make insulin. These cells regulate glucose levels in the blood which the body needs for energy. Blood sugar will continue to rise without insulin, so Type 1 diabetics must inject insulin for the rest of their lives.
But over the past 20 years, significant advancements in stem cell research and therapies have revealed promising methods of creating new insulin-making cells, which are needed to cure Type 1 diabetes.
Biotech company Vertex Pharmaceuticals recently began a clinical trial where it plans to treat 17 participants who have Type 1 diabetes with new insulin-making cells derived from stem cells. The first patient in the trial, Brian Shelton, has had positive results. After 150 days, Shelton was able to reduce the amount of insulin he injects by 92%.
Other global companies are also working to cure diabetes, such as ViaCyte, CRISPR, and , one of the biggest insulin manufacturers in the world.
Watch the video to hear how stem cell therapy has changed Shelton’s life and what other diabetes cures are being developed.
Impact Trial: A Unique Opportunity For Adult Patients Run By Leading Eu Experts
Among the four interventional trials currently being conducted under the INNODIA umbrella, the ImotopeTM approach, developed by Belgian company Imcyse, is one such immunotherapeutic being investigated in a Phase 2 study . ImotopesTM are based on modified peptides that induce cytolytic CD4 T cells to kill other immune cells involved in the destruction of a specific target, in this case, insulin-producing cells in the pancreas. This action works without affecting other functions of the immune system. Prof Mathieu explains the approach: The aim of this approach is to interfere early enough in the disease progression, so the aberrant immune response is halted. The immune system is recalibrated to stop the destruction of beta-cells and block the autoimmune response. Through this intervention, the pancreas maintains its natural ability to produce insulin and the rest of the immune system continues to function fighting infections. It is a very interesting, different approach and the safety profile we have seen so far is very impressive. A more detailed description of Imcyses technology is available in a previous article.
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Moving Research Forward Solving The Mysteries Of T1d Together
To advance their immunotherapy as a safe and effective therapy for patients with early T1D, Imcyse has joined forces with INNODIA, gaining access to a broad supportive research network. To really make a difference, everyone needs to work together, says Prof Mathieu. Newly diagnosed patients need to get the opportunity to participate in these studies, clinicians need to inform their patients about ongoing intervention trials, companies need the patient perspective, there is this aspect of public funding and the industry side. This is why networks such as INNODIA are so important.
Nanotherapy Offers New Hope For The Treatment Of Type 1 Diabetes
- Date:
- Northwestern University
- Summary:
- Individuals living with Type 1 diabetes must carefully follow prescribed insulin regimens every day, receiving injections of the hormone via syringe, insulin pump or some other device. And without viable long-term treatments, this course of treatment is a lifelong sentence. Now a team of researchers has discovered a better way.
Individuals living with Type 1 diabetes must carefully follow prescribed insulin regimens every day, receiving injections of the hormone via syringe, insulin pump or some other device. And without viable long-term treatments, this course of treatment is a lifelong sentence.
Pancreatic islets control insulin production when blood sugar levels change, and in Type 1 diabetes, the body’s immune system attacks and destroys such insulin-producing cells. Islet transplantation has emerged over the past few decades as a potential cure for Type 1 diabetes. With healthy transplanted islets, Type 1 diabetes patients may no longer need insulin injections, but transplantation efforts have faced setbacks as the immune system continues to eventually reject new islets. Current immunosuppressive drugs offer inadequate protection for transplanted cells and tissues and are plagued by undesirable side effects.
Specifying the body’s attack
Scott, also a member of CARE, said he wanted to see how the drug could be enhanced by putting it in a nanoparticle and “controlling where it goes within the body.”
Research hits close to home
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The Main Contender For A Type 1 Diabetes Cure
While you may see a variety of news articles each year with researchers claiming theyve found a successful cure for type 1 diabetes, the majority fail in animal trial and are never heard from again because they dont pass the early stages of long-term efficacy and safety.
The majority of it is really lined with hope, explainsKristina Figueroa, MSPH, who is an expert in type 1 diabetes clinical research and public health, and a passionate patient advocate.
Like most of us, Figueroa says that after her own type 1 diabetes diagnosis at age 6 in 1996, her healthcare team told her a cure was just around the corner. Due to the half dozen cures of type 1 diabetes in mice each year, it can seem that way.
Weve made huge advances, adds Figueroa, but were still nowhere close.
The researched treatment efforts closest to a successful cure come down to one, maybe two, that have the potential to progress through each critical trial phase.
And hopefully, one day becoming available to all patients with type 1 diabetes.
Hope For A Type 1 Diabetes Cure
Citations:
- MLA style: “Hope for a type 1 diabetes cure..” The Free Library. 2006 International Medical News Group 29 Jun. 2022
- Chicago style:The Free Library. S.v. Hope for a type 1 diabetes cure..” Retrieved Jun 29 2022 from
- APA style: Hope for a type 1 diabetes cure.. > The Free Library. . Retrieved Jun 29 2022 from
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Origins Of Vertex Diabetes Research
DiabetesMine readers may recall that Vertex moved into the T1D space back in 2019, acquiring stem cell startup Semma Therapeutics that had been founded by high-profile researcher Dr. Doug Melton of Harvard Stem Cell Institute in Cambridge, Massachusetts.
A D-Dad himself, Melton made big news in 2013 when his research was hailed as a breakthrough. Though a few years later, his original work was disappointingly retracted. Still, the essence of his approach was solid, and his 2014-founded startup Semma a mashup of the names of his two T1D kids, Sam and Emma went on to become one of the first funding projects of JDRFs venture philanthropy T1D Fund in 2017.
Meltons work has been a fascinating example of research aimed at using stem cells to create new insulin-producing cells in the body.
Vertex had been focused largely on cystic fibrosis up until then, but by buying Semma, the company entered the diabetes cure research game. At a price tag of $950 million, it was considered the largest T1D cure-focused transaction on record to date.
Specifically, Vertex bought and has pushed forward on Semmas two-pronged approach to this research:
That first prong is what Vertex is focusing on first, with the implantable device coming later down the road.
Stop The Immune Attack
In type 1 diabetes, the immune system is unable to tell the difference between harmful germs and the body’s cells and tissues, so the immune system attacks the bodys own insulin producing beta cells.
One way to cure diabetes could be to persuade a badly behaved immune system to accept the beta cells as part of the body.
Some of the immune system culprits attacking the beta cells are T cells. T cells develop in the thymus gland and, from a naïve or undifferentiated state, they are gradually educated to become helpers, warriors, or regulators.
In type 1 diabetes, the helper and warrior T cells work together to kill the beta cells, while the regulators appear unable to stop this from happening.
A/Prof Grey’s team worked up a vaccine, called BCMA-Fc, that re-dressed this imbalance. When given to mice with a disease similar to type 1 diabetes, the vaccine prevented diabetes by increasing the number of regulator T cells and reducing the number of warriors.
This work shows the potential of stopping the immune attack as a way to prevent type 1 diabetes. It has supported the testing of a related compound, called rituximab, in people with promising results. In a USA trial, people kept the ability to produce their own insulin, after diabetes had started, for up to 18 months. Further study will test whether taking this drug repeatedly will continue to maintain insulin production.
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A Cure For Type 1 Diabetes For One Man It Seems To Have Worked
A new treatment using stem cells that produce insulin has surprised experts and given them hope for the 1.5 million Americans living with the disease.
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Brian Sheltons life was ruled by Type 1 diabetes.
When his blood sugar plummeted, he would lose consciousness without warning. He crashed his motorcycle into a wall. He passed out in a customers yard while delivering mail. Following that episode, his supervisor told him to retire, after a quarter century in the Postal Service. He was 57.
His ex-wife, Cindy Shelton, took him into her home in Elyria, Ohio. I was afraid to leave him alone all day, she said.
Early this year, she spotted a call for people with Type 1 diabetes to participate in a clinical trial by Vertex Pharmaceuticals. The company was testing a treatment developed over decades by a scientist who vowed to find a cure after his baby son and then his teenage daughter got the devastating disease.
Mr. Shelton was the first patient. On June 29, he got an infusion of cells, grown from stem cells but just like the insulin-producing pancreas cells his body lacked.
Now his body automatically controls its insulin and blood sugar levels.
Its a whole new life, Mr. Shelton said. Its like a miracle.
But, he said, bottom line, it is an amazing result.
How Stem Cells Could Fix Type 1 Diabetes
Encapsulated stem cell-derived islets could shield cells from the immune system.Credit: Ref. 8
Insulin has been one of the most transformative discoveries in medicine. The isolation of this hormone in 1921 made type 1 diabetes a treatable, rather than a terminal, illness. However, there is growing hope that 100 years later, insulin therapy for T1D might be on the brink of obsolescence.
Insulin is crucial to maintaining safe levels of glucose in the blood. It is produced in the pancreas by cells, which continuously detect circulating glucose concentrations and secrete insulin accordingly the higher sugar levels go, the more hormone is released to counteract the increase. In T1D, however, the cells are destroyed by a persons own immune system.
The cause of this autoimmunity, which typically manifests in childhood, is incompletely understood, but the effect is clear: with neither cells nor insulin, circulating sugar levels remain constantly, toxically elevated. This chronic hyperglycaemia damages blood vessels and nerves, leading to an accumulation of ill-health effects and, if untreated, death.
Now, though, a growing number of scientists and physicians are talking about curing T1D. Their focus is not supplying the body with insulin, but rather replacing the cells that make it.
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Targeting A Cure: A Resource For Type 1 Diabetes Cure Research
On May 1, the American Diabetes Association is publishing an updated edition of our book, Targeting a Cure For Type 1 Diabetes: How Long Will We Have to Wait? The eBook is now available for purchase and download in the ADA store at ShopDiabetes.org. The book can be found at this address: . We hope this will give Targeting A Cure the opportunity to reach a larger audience, and so we have decided to update our original preview of the book from diaTribe #34 in anticipation of the rerelease. We have revised the original article where appropriate to reflect areas in which the research has evolved since the books original 2011 publication. Below, we present short excerpts from Dr. Robert Ratners foreword and Dr. Aaron Kowalskis introduction. They are both experts in the field of diabetes research, and we are honored to begin our book with their words of wisdom.