DRC’s Take on, “The type 1 diabetes gene TYK2 regulates βcell development and its responses to interferon-α”

Genetics in T1D

Over the years, researchers investigating type 1 diabetes have identified many genes associated with onset of the autoimmune disease. One of those genes is TYK2, which codes an enzyme (a Janus kinase) that plays a crucial role in intracellular signaling. In a study published recently in Nature Communications, a research team led by Timo Otonkoski at Helsinki University Hospital directed TYK2 knockout human iPSCs into the pancreatic endocrine lineage to decipher a dual role of the candidate gene TYK2 in pancreatic β-cells. First, depletion of TYK2 during early islet development affected the endocrine commitment, but did not affect the functionality of mature beta cells. Second, TYK2 inhibition in mature islet cells reduced vulnerability to T-cell cytotoxicity. These results identify an unsuspected role for TYK2 in β cell development and support TYK2 inhibition in adult β-cells as a potent therapeutic target to halt T1D progression.

Click HERE to read the full article.

DRC Is Excited to Share This Breaking T1D News Announced Yesterday by the FDA

The FDA has just approved Provention Bio’s Tzield™ (teplizumab-mzwv) – the first drug therapy that can delay the onset of type-1 diabetes (T1D) for those at risk of developing the disease.  This is a huge milestone for T1D research and those in the T1D community. (Read the full FDA announcement HERE.)

The average delay in the onset of T1D observed in the clinical study of Tzield was approximately 3 years, with some study participants not yet acquiring type 1 diabetes at all.  “Today’s FDA decision gives people at risk of developing type 1 diabetes the gift of time,” said Aaron Kowalski, Ph.D., JDRF CEO. “For the first time ever, we have a way to change the course and slow the development of T1D.”  (Read JDRF’s full statement on the impact of this news to the T1D community HERE.)

Tzield is the result of decades of T1D research, which began with an early scientific study.  That study led to a JDRF grant to support a trial in patients.  The success of that trial study led to further studies and support from the National Institutes of Health (NIH), eventually leading to this exciting breakthrough that will impact the future of T1D treatment.

Scientific breakthroughs such as this one, often emerge due to the inventiveness of early-career scientists.  It is DRC’s mission to connect donors with early-career scientists, enabling them to perform peer-reviewed, novel research designed to prevent and cure type 1 diabetes, minimize its complications, and improve the quality of life for those living with the disease.

Thus far, 10+ of our funded studies by early-career scientists have secured follow-on funding to continue their studies which could lead to breakthroughs like the milestone announced today.

You could help fund the next T1D breakthrough!  DONATE HERE 

November 2022 Newsletter

Please enjoy this month’s newsletter, featuring:

        • Researchers Impacting Our Mission
        • November’s Matching Gift Campaign
        • Meet Our New Executive Director
        • DRC’s Seaside Silent Auction
        • Thank You to Our Sponsors!

 

 

View the Newsletter here

For the Good of Neural Tissues and Pancreatic Islets

While organs can be transplanted from deceased donors, tissues from the nervous system rapidly lose viability. The mechanisms of neuronal death, and the potential for reversing it, remain poorly defined. Dr. Fatima Abbas, a DRC-funded investigator at the University of Utah, in collaboration with Dr. Frans Vinberg (University of Utah) and Dr. Anne Hanneken (The Scripps Research Institute, La Jolla, CA), published a paper in Nature that questions the irreversibility of neuronal cell death in the retina, an investigation that has implications for visual rehabilitation and for the future of organ transplantation. In the study, the researchers characterized neuronal death and survival and identified conditions for reviving neuronal functioning in postmortem mice and human retinas. This study is a step toward better strategies for preserving the viability and engraftment capability of tissues and cells isolated from organ donors for transplantation, including the pancreas. Given the significant overlap of genes and proteins between pancreatic islet cells and neural tissues, the findings by Abbas and colleagues may have important implications for the improvement of islet cell transplant engraftment and long-term function in type 1diabetics.

Click HERE to read the full article.

DRC’s Take: Vertex to Acquire Chief T1D Stem Cell Competitor in All-Cash Deal

Vertex, whose VX-880 stem cell therapy for type 1 diabetes has cleared clinical proof of concept, is acquiring ViaCyte, a private biotech that has also reached clinical trials with its own stem cell therapy. In the $320 million all-cash deal, Vertex will acquire ViaCyte’s human stem cell lines, manufacturing facilities, and other relevant intellectual property.

While both companies are pursuing stem cell-based approaches to treating type 1 diabetes, their methods differ. The Vertex therapy involves injecting synthetic islet cells into patients. By comparison, the ViaCyte therapy uses gene-edited, immune-evasive stem cells encapsulated in implantable devices.

Both companies have reported data from clinical trials.

Data released by Vertex in October 2021 showed that the first patient who received the treatment had a lower average HbA1c (8.6% to 7.2%) and a significantly reduced reliance on insulin injections. Results from a second patient have also been reported and data from additional trial participants are expected later this year or early next year. In June 2021, ViaCyte revealed that a single patient had also experienced a drop in HbA1c (7.4% to 6.6%) but still required insulin injections.

“VX-880 has successfully demonstrated clinical proof of concept in T1D, and the acquisition of ViaCyte will accelerate our goal of transforming, if not curing T1D by expanding our capabilities and bringing additional tools,” Vertex CEO Reshma Kewalramani said in a statement.

Click HERE to view the full article.

DRC in the Community: President and Chair, C.C. King’s Presentation on Insights into Medical Research

On Monday, May 23rd, DRC’s President and Chair, C.C. King, Ph.D., spoke at Del Mar Foundation’s speaker series, DMFTalks. Over 30 members of the community came to listen to C.C. talk about the importance of medical research. His talk addressed the importance of medical research. He began by sharing the process of approving medication through the FDA and how in-depth that procedure can take. This transitioned into the significance of model systems in three categories; Cancer, the self, and Type 1 Diabetes (T1D). In researching human disease, model organisms allow for a better understanding of the disease process without the added risk of harming an actual human. By the end of the presentation, he illustrated how Diabetes Research Connection (DRC) is a vital organization that funds early-career scientists who have bold and out-of-the-box ideas without the years of experience that other researchers have, therefore often don’t receive the funding necessary to make headway with their research. DRC is an organization that proudly acts as the seed funding for many of these projects, allowing them to gain momentum and credibility to help them receive larger grants to bolster their research. Many of these projects use model systems to help validate their hypotheses and have often received follow-on funding from the results they have garnered and have made headway in T1D research.

Click HERE to watch C.C.’s presentation!

CONNECTING FOR A CURE: June 2022 Newsletter

DRC has distributed over $400,000 to research projects like Dr. Dwyer’s and Dr. Zhu’s in Request for Application (RFA) 2 2021 alone. We received an unprecedented number of Letters of Interest (LOIs) in our last RFA and are funding even more innovative projects than ever. View our “Support a Project” page to see what other research projects we are currently committed to funding by clicking here. Take a look at our latest newsletter, where we feature some quotes from our newest grant recipients, show DRC in the community, and highlight our newest volunteers.

Click this link to view our June newsletter that we mailed out previously this month about what we’ve been up to and the impact we are making together. It takes a community to connect for a cure!

Diabetes Research Connection Awards $400,000 to Eight Promising T1D Studies

After rigorous peer review by DRC’s Scientific Review Committee, eight early-career researchers were awarded $50,000, totaling $400,000 in seed funding for their work to find the cause, treatment, and cure for T1D. 

Click HERE to read the Press Release

Meet a DRC Volunteer: Kaya Keutler

Kaya was diagnosed with type 1 diabetes when she was 13. That was also the point she knew she wanted to become a scientist and work in the research field of diabetes. Fast forward 15 years, Kaya is working on getting her doctoral degree at the Oregon Health & Science University. However, Kaya has realized that she wants to spend less time doing science and more time communicating current scientific knowledge to relevant audiences and the public. Her work for the DRC brings her one step closer to that goal. 

I was diagnosed with type 1 diabetes when I was 13. One of my closest cousins had lived with diabetes for about 10 years back then, so it wasn’t an unknown disease to me. Still, it felt like a bad diagnosis when an indifferent doctor delivered it to me in front of his note-taking students at a university clinic. Today I recognize it as a life-changing moment and am grateful for it, as it has made me the person I am. It also has significantly impacted my career decisions. I knew I wanted to become a scientist and work on a cure for diabetes back then. Although actively working in the field of diabetes research today, I now know it’s not that simple. There is so much we still need to learn about the disease and its treatment, and that includes both basic science as well as coming up with smart solutions for the everyday life of diabetic patients. “Let’s see what the science says” is a phrase I often use both at work and while engaging in managing my diabetes. I’ve found that what I know as a scientist does influence my treatment decisions quite a bit. I’ve reached out to volunteer for the DRC as I want to give others the power to make science-based treatment decisions and to better understand their disease, the research around it, and their options.

Kaya is now helping this organization by translating complex scientific language from DRC-funded researchers so that the community can understand the project better.

National Tell a Story Day: A Co-Founder Shares Her Experience

Amy Adam’s son was diagnosed with T1D when he was five years old. She served on the Board of Directors and various committees for the Juvenile Diabetes Research Foundation (JDRF) Chicago chapter for 10 years and was a contributing writer to Insulin Free Times. This is her second term on the DRC Board, and she has served as the Lay Person Review Committee Chair since DRC’s inception.

It’s hard to believe that 30 years, hundreds of thousands of blood tests and shots, innumerable doctors’ visits and procedures, and countless renewed vows to find that elusive combination of food, activity, and insulin have passed since my own child was diagnosed with Type 1 Diabetes. Time has propelled us forward like tumbleweeds in a dust storm, yet the rigorous demands and challenges of diabetes have clung stubbornly to him every step of the way. One of the most significant personal challenges I have encountered along this path is having to stand by and watch my child’s indomitable spirit rise and fall as this disease continues to roll along with his organs, tissues, and psyche firmly in its grasp.

You can’t outgrow Type 1 Diabetes, and it is progressive. Most Type 1 diabetics develop at least one complication, and close to 50% of Type 1 diabetics will develop disabling or even life-threatening complications over their life despite their best efforts to control the disease. Perhaps due to multiple autoimmune diseases, my child struggles more than many. 

Fortunately, early on our path with this disease, we met the most extraordinary doctor who medically guided us through some of his darkest hours and gave us the only trustworthy source of hope we have ever felt through his research for a cure for this insidious disease. Years later, when Dr. Hayek introduced me to an equally impressive man named David Winkler and asked me to join them in their endeavor to create a diabetes research organization with a different paradigm, I was all ears.

Our founding vision for The DRC (Diabetes Research Connection) was to empower the community of young, innovative scientists, people affected by diabetes, and their supporters to propel unique and promising Type 1 Diabetes research ideas that weren’t receiving adequate attention by other organizations. Each selected project would have to be endorsed by a group of world-renowned diabetes experts who had volunteered their time to evaluate the merit of these projects to qualify for funding. Then the projects would be written in lay-friendly terms and posted on our website, where potential supporters could choose to support the projects that “spoke” to them based on their own experiences and knowledge of the disease. The scientists would provide regular updates on the progress of their project, good, bad, or inconclusive. Outcomes would be published on our website, adding to the body of diabetes knowledge in a united effort to eliminate diabetes.

I support the Diabetes Research Connection because despite our best efforts to control what we were told was a manageable disease, the only source of control we have ever felt is where we put our money towards curing it. Join us and influence the future of diabetes research; by selecting the research that is meaningful to you and your experience, you may help influence the work that leads to a cure for Type 1 Diabetes.

Amy’s initial vision for co-founding DRC has come to fruition. In this organization’s 10th year, they have funded 48 projects, invested $2.4 M in innovative T1D research, and 12+  researchers received approximately $12M in follow on funding.