teplizumab

TrialNet Research Leads to FDA Approval of First Drug to Delay Type 1 Diabetes

tomd — Thu, 10 Nov 2022 20:37:07 +0000

TrialNet Research Leads to FDA Approval of First Drug to Delay Type 1 Diabetes tomd Thu, 11/10/2022 - 12:37 Media Release

Landmark findings from TrialNet research were instrumental in the Food and Drug Administration’s (FDA) November 17, 2022 approval of the first drug to delay type 1 diabetes (T1D) in people at risk.

TrialNet conducted the clinical study that discovered the immunotherapy drug teplizumab could delay T1D for 2+ years.

To develop this study, TrialNet collected data from more than 200,000 relatives of people with T1D over the past 20 years. This led to the development of the stages of type 1 diabetes, making it possible to identify who will develop T1D and move prevention research forward.

TrialNet Chair Kevan C. Herold, M.D., Yale University, served as TrialNet Principal Investigator for the study that spanned 10 years.

“We want to thank TrialNet research teams and participants in the Teplizumab Prevention Study and everyone who has participated in T1D research,” said Dr. Herold. “With your help, we are one step closer to a future without T1D.”

Study participants were relatives of people with T1D who had two or more diabetes-related autoantibodies and abnormal blood sugar levels, as identified by TrialNet screening. These individuals are thought to have a lifetime risk of clinical diagnosis nearing 100%. Of the 76 high-risk individuals who participated in the study, 55 were under age 18.

“We will build on this landmark success by continuing to study and identify new ways to slow and eventually stop T1D,” said Dr. Herold. “This accomplishment opens the door to prioritize development of new therapies for slowing and stopping T1D.”

Similar to other autoimmune diseases, such as rheumatoid arthritis, more than one drug may be needed to delay or prevent T1D in everyone. TrialNet is aiming to find other drugs and combinations that work best to offer them to all people who are at risk for T1D.

TrialNet currently has several studies at various stages, including:

The STOP-T1D Prevention Study is scheduled to open for enrollment in early 2023. This study will test a low dose of the immunotherapy drug anti-thymocyte globulin (ATG) to see if it can delay or prevent T1D in people ages 12-35 who have a 50% risk of clinical diagnosis (stage 3) within 2 years. Risk is defined by having two or more diabetes-related autoantibodies and abnormal blood sugar levels (stage 2 T1D) and at least one high-risk marker (based on test results).

TrialNet’s TOPPLE Study is currently testing the safety of a new plasmid therapy in adults diagnosed with T1D in the past 48 months. If this study results in no safety concerns, TrialNet hopes to conduct larger studies to see if this same treatment can slow down or stop T1D in people at risk.

In addition, TrialNet thanks the more than 400 people who took part in TrialNet’s Abatacept Prevention Study and Hydroxychloroquine (HCQ) Prevention Study. Results for these studies will be reported in 2023.

To learn more, visit trialnet.org/our-research.

teplizumab

TrialNet Research Continues to Close in on Type 1 Diabetes Prevention

ebatts — Wed, 03 Mar 2021 18:35:43 +0000

TrialNet Research Continues to Close in on Type 1 Diabetes Prevention ebatts Wed, 03/03/2021 - 10:35 Study Findings

New TrialNet research findings published today in Science Translational Medicine show that a 14-day course of the immunotherapy drug teplizumab provided an additional year of delay in onset of type 1 diabetes and improved insulin production, increasing the overall benefit to high-risk individuals reported in the original study findings in 2019.

TrialNet Investigator Emily K. Sims, M.D., Indiana University, reported her findings after extended follow up with participants from the Teplizumab Prevention Study, which concluded last year. That study, led by TrialNet Principal Investigator Kevan C. Herold, M.D., Yale University, was the first to show a median 2-year delay of type 1 diabetes in high risk individuals.

The latest findings show ongoing delay in diabetes, with a median time to diabetes diagnosis of approximately 60 months (5 years) in the teplizumab group vs. approximately 27 months (2.3 years) in the placebo group.

In addition to having even more pronounced disease delay, those treated with teplizumab showed improved rates of insulin production, despite exhibiting insulin loss over time prior to treatment. Participants receiving a placebo continued to show a decline in insulin production consistent with disease advancement.

Participants electing to continue being followed will be tested every 6 months, so researchers can continue to track changes. Sims and Herold are hopeful the disease delay will extend even further over time.

Study participants are relatives of people with type 1 diabetes who have two or more autoantibodies and abnormal blood sugar levels, as identified by TrialNet’s Pathway to Prevention screening. These individuals are believed to have a lifetime risk of clinical diagnosis nearing 100%. Of the original 76 individuals participating in the study, 55 were under age 18.

“These are tangible results, not only for researchers working to prevent this disease, but for family members of people with type 1 diabetes,” said TrialNet Chair Carla Greenbaum, M.D., Director of the Diabetes Research Program at Benaroya Research Institute at Virginia Mason in Seattle.

Extending the time it takes to develop type 1 diabetes is significant. It can be life changing for young children, allowing them time to reach an age where they can better cooperate and understand the rigorous treatment regimen. It also gives hope that the future will be easier for family members of people with type 1 diabetes, who are 15 times more likely to develop the disease.

Relatives of people with type 1 diabetes are urged to get free Pathway to Prevention screening. TrialNet screening can detect risk years before symptoms appear, opening the door for early intervention with monitoring and clinical trials testing ways to prevent and delay type 1 diabetes. In-home test kits make it possible to get screened without leaving home.

teplizumab

TrialNet highlights research advances during Diabetes Awareness Month and World Diabetes Day

ebatts — Sun, 01 Nov 2020 21:39:21 +0000

TrialNet highlights research advances during Diabetes Awareness Month and World Diabetes Day ebatts Sun, 11/01/2020 - 13:39 Research Spotlight

Each November, we join the diabetes community to recognize Diabetes Awareness Month and World Diabetes Day on November 14th. This year, as TrialNet begins to celebrate 20 years, we share major research advances made possible by our incredible study participants and research teams around the world.
Identifying early stages of T1D

More than 200,000 relatives of people with T1D have taken part in TrialNet risk screening. Besides learning their personal risk, they played an important role in helping identify the early stages of T1D. Because it promotes earlier disease identification and provides opportunity for intervention, staging classification was recommended for adoption by the JDRF, American Diabetes Association (ADA), and Endocrine Society in 2016.

Early T1D identification—prior to symptoms—is extremely useful. The earlier in the disease process someone is diagnosed, the more beta cells likely remain. And this gives us the opportunity to slow or stop further destruction, preventing type 1 diabetes. In addition, for people participating in T1D research including close monitoring, risk of diabetic ketoacidosis (DKA), a serious and sometimes life-threatening condition, decreases from 30% to 3%.

Advances in T1D prevention

In 2019, TrialNet’s Teplizumab Prevention Study made headlines as the first to delay T1D onset for 2 or more years in high risk individuals.

Teplizumab is an immunotherapy drug that interferes with the body’s destruction of its own beta cells. While previous studies showed teplizumab could prolong insulin production in people recently diagnosed, this was the first study to prolong insulin production in people at high risk.

According to findings, median time for people in the control group to develop clinical diabetes was just over 24 months while the treatment group averaged 48 months before progressing to diagnosis. See more details here.

Participants electing to continue being followed are being tested every 6 months, so researchers can continue to track changes.

Also in 2019, findings from TrialNet’s ATG/GCSF New Onset Study reported low-dose thymoglobulin (ATG) preserved insulin production and improved long-term blood sugar control for 2 years in people newly diagnosed with type 1 diabetes. Hemoglobin A1c levels were significantly lower in those treated with low-dose ATG, as compared to placebo. See more details here.

In 2017, TrialNet reported findings from the Oral Insulin Prevention Study, our largest study ever with more than 500 participants. TrialNet tested oral insulin in a daily pill to see if it could delay or prevent T1D from progressing from stage 1 to stage 2, ultimately preventing clinical diagnosis in those at risk (stage 3).

While oral insulin did not prevent or delay T1D in the majority of participants, it did delay onset an average of 31 months in a sub-group of 55 people, supporting our understanding that not everyone develops T1D in the same way. See more details here.

Current prevention studies

TrialNet is currently conducting two prevention studies testing immune therapies:

Hydroxychloroquine (HCQ) Prevention Study - now enrolling
TrialNet is testing the drug hydroxychloroquine (HCQ) to see if it can delay or prevent early stage T1D (stage 1) from progressing to abnormal glucose tolerance (stage 2), and clinical diagnosis (stage 3). HCQ is already used to reduce symptoms and progression of other autoimmune diseases, such as rheumatoid arthritis and lupus. This is the first study to see if it can prevent or delay T1D. Learn more here.

Abatacept Prevention Study – no longer enrolling
TrialNet is testing the drug abatacept to see if it can delay or prevent progression of early stage T1D (stage 1 or stage 2), to clinical diagnosis (stage 3). In earlier studies of people newly diagnosed (stage 3), abatacept helped slow down disease progression. We should know in the next year if this drug is also effective in delaying T1D progression in pre-diagnosed people. Learn more here.

Impact for people living with T1D

The work TrialNet is doing to try to control the immune system in early stages of T1D has the potential to directly benefit people who have been living with T1D for a long time.

“Even if we could give someone with T1D new insulin producing cells, we would still need a way to keep the immune system from attacking them,” explains Dr. Greenbaum. “All the drugs shown to be effective in slowing T1D progression are immune therapy drugs. This clearly tells us it’s the immune system we need to keep under control.”

What’s next?

Prevention
With a several studies in the pipeline, we aim to answer questions including these:

Now that we have evidence that immunotherapies can slow disease progression, which drugs or combination of drugs work best at what stage?
If we treat at an even earlier stage, can we delay the disease even longer?

People living with T1D
Later this year, we’ll be launching a phase 1 study for people diagnosed with T1D within the past 4 years. This brand new therapy is being tested for safety and tolerability. If this study results in no safety concerns, we plan to conduct a larger study to see if this same treatment can slow down or stop T1D in people at an earlier stage of disease, before clinical diagnosis.

“Every time we do a study, we learn something about the benefits and risks of the therapy, but we also learn more about the disease,” explains TrialNet Chair Carla Greenbaum, M.D. “We know that not everyone responds to the same treatment in the same way. Our goal is to eventually be able to match the exact therapy to the needs of individual participants.”

A few drops can make a big difference!

TrialNet T1D risk screening looks for type 1 diabetes years before it appears. Know you or your child’s risk for T1D with a free blood test delivered to your door.

Sign Up
Answer a few questions. Free TrialNet screening kits are sent directly to you and can be done from the safety of your home.

Stick It
Each kit includes everything you need. All it takes is a few drops using a finger-stick and the provided collection tube.

Ship It
Send your samples back for free using FedEx contactless at home pick up. Get your results in 4-6 weeks.

The future without T1D begins with you!

Get a free screening kit sent to your home today at www.trialnet.org/screen4t1d

Tracy Olsten shares why her daughter Mikayla participated in the landmark Teplizumab prevention trial

ebatts — Sat, 08 Feb 2020 01:56:52 +0000

Tracy Olsten shares why her daughter Mikayla participated in the landmark Teplizumab prevention trial ebatts Fri, 02/07/2020 - 17:56 Participant Spotlight

With landmark results recently announced, the Teplizumab Prevention Study is making headlines across the country. Here, Tracy Olsten shares why her daughter Mikayla decided to join the study.

When Mikayla’s 9-year-old sister went into DKA and spent several days in ICU, mom Tracy Olsten says it totally caught them off guard. To their knowledge, this was the first instance of T1D in their family.

“There was so much stress and grief,” Tracy remembers. “We spent 6 days in the ICU—she was in respiratory and kidney failure. We saw her life slipping away right before us.”

The Olstens lived in Colorado then and their daughter with T1D was being treated at the Barbara Davis Center for Childhood Diabetes at the University of Colorado in Aurora.

Tracy recalls, “The staff at Barbara Davis Center told us about TrialNet and the opportunity to have our family screened. We jumped on it right away! Seeing everything our child went through, we wanted to catch it at the earliest possible stage in our other kids.”

The whole family was screened at Barbara Davis three weeks before their move to Florida. They had just moved into their new home when they got the call with Mikayla’s results. Mikayla, 15 at the time, had 4 out of 5 markers for T1D. The rest of the family tested negative.

“Barbara Davis put us in touch with the University of Florida (UF) in Gainesville,” explains Tracy. “And, UF invited us to come in to hear about the Teplizumab Prevention Study. We told Mikayla, you know you have the markers, you know everything your sister went through, and you have a high probability. She made the decision herself to participate.”

During the treatment phase of the study, Mikayla spent 14 days at UF. “That team is phenomenal,” says Tracy. “They were extremely informative throughout the entire process. From our experience at Barbara Davis to UF, everybody was super helpful and accommodating, addressing all our fears. They’ve all become like family to us. We have no regrets.”

Tracy says having the knowledge is what’s so helpful. “I would not want anyone to go through what our 9-year-old went through with DKA. We had the power to prevent this from ever happening again by simply taking one blood test and knowing our risk factors.”

“It was extremely comforting to my husband and me to know that we were back in control again by having the information we needed regarding our family’s risk. With T1D, you can’t control a lot of things, but with this information, you can control some really important things.”

Now 17, Mikayla says, “My sister could have had this same opportunity but didn’t because we didn’t have the knowledge. I wanted to do this for her. This is going to help me and other people.”

In response to the study findings, Tracy says, “We were pleased to know that Mikayla was one of the participants that received the study medication. Her counts have remained on track, and so far, it appears that this treatment has worked.”

You can help advance type 1 diabetes research

TrialNet is continually planning and launching new studies. And, we always encourage family members of people with T1D to get screened through the Pathway to Prevention study.

teplizumab

Teplizumab is the first drug to delay the diagnosis of T1D for a median of two years

ebatts — Thu, 19 Dec 2019 20:13:04 +0000

Teplizumab is the first drug to delay the diagnosis of T1D for a median of two years ebatts Thu, 12/19/2019 - 12:13 Study Findings

“This is the first study to show any drug can delay type 1 diabetes diagnosis a median of 2 years in people at high risk,” explains Dr. Herold. “As anyone with type 1 diabetes will tell you, every day you can delay this disease is important.”

Teplizumab is an immunotherapy drug designed to interfere with the body’s immune destruction of its own beta cells. While previous studies showed teplizumab prolonged insulin production in people recently diagnosed, this is the first study to test it in people at high risk.

Of the 76 participants in the study, 55 were under age 18 and all had a relative with type 1 diabetes. All participants had two or more type 1 diabetes autoantibodies and abnormal blood sugar levels, as identified by TrialNet screening. These individuals are thought to have a lifetime risk of clinical diagnosis nearing 100%.

Participants were randomly assigned to either the treatment group, which received a 14-day course of teplizumab, or the control group, which received a placebo. All participants regularly received glucose tolerance tests until the study was completed, or until they developed clinical type 1 diabetes – whichever came first.

72% of people in the control group developed clinical diabetes, compared to only 43% of the teplizumab group. The median time for people in the control group to develop clinical diabetes was just over 24 months, while those who developed clinical diabetes in the treatment group had a median of 48 months before progressing to diagnosis.

This study highlights that type 1 diabetes is an autoimmune disease that can be delayed with immunotherapy. Samples collected during the trial are being studied to help researchers understand why certain people responded to teplizumab better than others. Next, TrialNet researchers will look for ways to extend the benefits of teplizumab, while continuing to test other immune therapies.

You can help advance type 1 diabetes research

TrialNet is continually planning and launching new studies. And, we always encourage family members of people with T1D to get screened through the Pathway to Prevention study.

teplizumab

teplizumab

TrialNet Research Leads to FDA Approval of First Drug to Delay Type 1 Diabetes

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Teplizumab is the first drug to delay the diagnosis of T1D for a median of two years

Tracy Olsten shares why her daughter Mikayla participated in the landmark Teplizumab prevention trial

TrialNet Research Continues to Close in on Type 1 Diabetes Prevention

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Teplizumab is the first drug to delay the diagnosis of T1D for a median of two years

Tracy Olsten shares why her daughter Mikayla participated in the landmark Teplizumab prevention trial

TrialNet highlights research advances during Diabetes Awareness Month and World Diabetes Day

Tracy Olsten shares why her daughter Mikayla participated in the landmark Teplizumab prevention trial

Teplizumab is the first drug to delay the diagnosis of T1D for a median of two years

You can help advance type 1 diabetes research