Research News

The ALS Association

  • To help accelerate drug development and get promising treatments closer to regulatory evaluation, we are investing more than $2.9 million over the next two years through our Lawrence and Isabel Barnett Drug Development Program to help move six emerging ALS therapies from the laboratory into the clinic. We also have committed $1 million through our Hoffman ALS Clinical Trial Awards Program to support a small, early-stage trial of a nasal spray designed to dampen inflammation and potentially slow or stop ALS progression.We are connecting people with ALS to clinical trial opportunities through our partnership with myTomorrows and improving trial accessibility through our Trial Capacity Awards. Initiatives like these not only help more people who want to participate in clinical trials do so, but also increase the efficiency and speed at which clinical research is conducted.

  • We joined with the National Institutes of Health, Food and Drug Administration, and 13 other private partners to help accelerate ALS diagnosis and treatment through the Accelerating Medicines Partnership® in ALS (AMP® ALS). This partnership will create the largest open data platform for ALS research and help enable discovery and development of diagnostic tools, biomarkers, and better treatments.

  • Researchers at UC Davis supported by our Seed Grant and Assistive Technology Grant Programs developed a new brain-computer interface that translates brain signals into speech with up to 97% accuracy—the most accurate system of its kind. By combining this technology with AI, one person with ALS who needed others to help interpret for him was able to speak again by just trying to think about what he wanted to say. The words were read aloud by a computer in a voice that was constructed from audio samples of his pre-ALS voice.

  • We celebrated 20 years of our Milton Safenowitz Postdoctoral Fellowship Program by welcoming five talented young scientists to the program. It is through programs like this that an exciting trend has emerged in recent years: approximately 80% of applications to our research funding programs now come from early-career or new investigators, including scientists from fields outside traditional ALS research.

  • Go to https://www.als.org/2024-year-end-report#research_tech for more research news.

Alzheimer’s Association - Nebraska Chapter

  • UNMC: Kelly Stauch, PhD, neurological sciences, received a grant of $200,000 from the Alzheimer’s Association for the study “The Role of APOE4 in Synaptic Mitochondrial Dysfunction.”

  • Donanemab (Kisunla™) is an anti-amyloid antibody intravenous (IV) infusion therapy that is delivered every four weeks. It has received traditional approval from the U.S. Food and Drug Administration (FDA) to treat early Alzheimer's disease, including people living with mild cognitive impairment (MCI) or mild dementia due to Alzheimer's disease who have confirmation of elevated beta-amyloid in the brain.

    By slowing progression of the disease when taken in the early stages of Alzheimer's, donanemab can allow people to have more time to participate in daily life and live independently. Individuals should talk with their health care provider to develop an Alzheimer's treatment plan that is right for them, including weighing the benefits and risks of all approved medications and therapies.

  • Contact the Alzheimer’s Association 24/7 Helpline at 800.272.3900 for support or with additional questions.

Arthritis Foundation Nebraska

  • UNMC is continuing a clinical Osteoarthritis trial funded by a $200,000 grant from the Arthritis Foundation.

  • The Arthritis Foundation Invests $1.1 M in Refractory Rheumatoid Arthritis Research.

Brain Injury Association of Nebraska

  • A growing body of research highlights the positive impact of exercise on brain structure and cognitive function following a traumatic brain injury. Evidence suggests that exercise assessment and aerobic training may help mitigate concussion-related physiological dysfunction by restoring autonomic balance and improving cerebral blood flow regulation.

  • The National Institutes of Health (NIH) has awarded grant funding to UNMC’s College of Public Health, with funding allocated to the Brain Injury Association of Nebraska (BIA-NE) through 2027. This funding will support the implementation of a brain injury screening tool across 12 domestic violence programs in Nebraska. In collaboration with UNMC’s College of Public Health, BIA-NE will provide brain injury training, facilitate the use of the screening tool, and offer resource support services.

Breakthrough T1D

In 2024, we can reflect upon the incredible progress we’ve made in advancing breakthroughs toward cures and improving everyday life with T1D.  

This wouldn’t have been possible without each and every one of you and your continued support of our mission as we drive toward cures for T1D.

Top 11 advances that together, we made happen in 2024: Article & Video

  •  Project ACT

    This year we launched Project Accelerate Cell Therapies, or Project ACT—an initiative to fast-track the development of and access to T1D cell therapies. It’s among the most promising pathways we have toward cures.  

    By safely accelerating their development, we can ensure more people have access to these treatments as soon as possible.

  •  Updates in Cell Therapy

Among this year’s biggest breakthroughs are the early findings from Vertex's cell therapy in their ongoing clinical trials. VX-880 is a stem cell-derived islet therapy to restore the body’s ability to produce insulin for people with T1D and hypoglycemia unawareness and severe hypoglycemic events.

So far, 11 out of 12 participants in the clinical trial have reduced or eliminated the need for external insulin altogether. And, all 4 participants who received the full dose of cells with a follow-up after more than one year met the primary endpoint of eliminating severe hypoglycemic episodes and achieved the secondary endpoint of insulin independence. In other words, they no longer need to administer insulin. 

 Vertex has invested heavily in T1D, and they recently broke ground on a manufacturing facility to produce these cells. We’re proud to partner with them to pursue this progress. 

  •  T1D Autoantibody Detection: The first clinical guidelines are here

In a major effort spearheaded by Breakthrough T1D, the first internationally recognized clinical guidelines for those who test positive for T1D autoantibodies have been published. These include guidance on monitoring frequency, education, and psychosocial support in addition to recommended actions for healthcare professionals (HCPs) when the risk of T1D progression is high. The guidelines were cooperatively developed with over 60 international experts spanning ten countries.

Watch a recording featuring three Breakthrough T1D experts discussing how we’re tackling early detection through research, clinical implementation, and health policy.

Crohn’s & Colitis Foundation, Nebraska/Iowa Chapter

  • We grew IBD Plexus®, our scientific network and transformational platform that makes millions of data points and hundreds of thousands of biosamples available to researchers to help speed the pace of discovery.

  • We announced plans to launch a groundbreaking research project—MyIBD Health Study. This new patient registry, which aims to recruit more than 100,000 patients, will help accelerate IBD research.

  • We discovered a marker in the blood that could indicate which kids living with IBD will go on to develop strictures in their intestines.

  • We uncovered how mutations in a specific gene increase the risk of developing ulcerative colitis.

  • We supported research concluding that a protein in the intestine correlates with disease severity that doesn’t respond to biologics therapy.

  • We leveraged technology to invest in a "smart pill" that delivers medicine directly to diseased areas in the intestines.

  • We made progress on a new blood test that predicts which IBD patients are at high risk of having multiple relapses within the following 12-18 months.

  • We led studies on how diet and nutrition can help you manage your IBD symptoms.  We supported research that is evaluating shifts in the gut microbiome and how they correlate to brain changes and the likelihood of developing disease flares in response to perceived stress.

  • We announced plans to launch a groundbreaking research project—MyIBD Health Study. This new patient registry, which aims to recruit more than 100,000 patients, will help accelerate IBD research.

Cystic Fibrosis Foundation Nebraska Chapter

  • $155,000 has been pledged to the University of Nebraska Adult CF Care Center. This grant supports the general operations of the CF Therapeutics Development Center at the University of Nebraska, which conducts clinical trials.

  • $180,000 has been pledged to the University of Nebraska Pediatric CF Care Center. This grant supports the general operations of the pediatric CF care center at the University of Nebraska, which provides specialized medical care for all children living with CF in Nebraska.

  • $30,000 has been pledged to the University of Nebraska Therapeutics Development Center. This grant supports the general operations of the CF Therapeutics Development Center at the University of Nebraska, which conducts clinical trials.

The Leukemia & Lymphoma Society – Nebraska

The Latest in Blood Cancer Treatment Patients with blood cancer need more options now. We made great strides in blood cancer treatment over the past year. MORE RESEARCH. BETTER TREATMENTS. OPTIMAL OUTCOMES.

Here are five ways LLS moved the needle in 2024:

  • Expanded Access to Data for Pediatric AML Research - LLS funded University of Chicago’s creation of the INTERACT Pediatric AML Data Commons, which collects clinical data from around the world into a single unified platform for research. Because acute myeloid leukemia (AML) is a rare cancer, it is
    vital that scientists gather as much data as possible in one place so they can make informed decisions about AML research and treatment. This partnership not only makes thousands of pediatric AML patient records available—from Germany, Japan, and North America, including St. Jude Children’s Research Hospital—but it also standardizes the data into a consistent language that can help advance pediatric AML treatment. It activated with 3,200 records, with many more global data sets to be added going forward. This was made possible by LLS’s commitment to innovation and ability to convene key players from around the world in pursuit of blood cancer research. This will speed the development of more effective, less toxic treatments for children with acute leukemia.

  • United with Partners to Accelerate Research LLS TAP is an expert in identifying the most impactful investment opportunities for blood cancer innovation, and because of its track record and established credibility, is a leader in venture philanthropy. More than $100 million in investment returns have gone right back into LLS research funding and patient support. LLS TAP hosted an event in May highlighting the pivotal role of venture philanthropy and investments in accelerating cures. More than 150 people attended, including panelists from companies with several TAP-supported therapies. Two of these therapies are moving toward filing for FDA approval. Pelabresib, a promising new treatment for myelofibrosis from MorphoSys, improves disease symptoms and reduces scarring in the bone marrow. Kura Oncology’s phase 3 ziftomenib trial recently announced its full enrollment, with final data expected early next year. TAP is proof that venture philanthropy can help companies make significant strides in blood cancer treatment and cures.

  • Committed to CMML Research - There are only about 1,100 cases of chronic myelomonocytic leukemia (CMML) diagnosed each year, giving researchers few opportunities to study it—but at LLS, we’re committed to improving treatments and outcomes for all blood cancer patients. Thanks to a generous $17 million gift from the Mike & Sofia Segal Foundation, we can fund more research to accelerate this commitment, including five new research grants this year to help us learn more about:

    • CMML biology and ways to target and kill cancerous CMML cells

    • Relapse and resistance to treatment

    • How we can optimize outcomes for patients living with CMML

    This research may also result in treatment options that benefit
    patients with other forms of blood cancer, including AML and
    myelodysplastic syndromes.

  • The LLS Therapy Acceleration Program® (TAP) speeds the pace of drug development through strategic investments in promising projects by biotech companies. Through these partnerships, LLS TAP advances novel therapies to
    address urgent patient needs and helps build momentum for companies and investors to join our mission.

  • Blood cancer researchers and biotech companies strive to transform the way we treat blood cancer, but they can’t do this work alone. Donors and philanthropists unite with LLS to lead this charge by funding projects focused on deep understanding that can be applied to clinical studies—which can bring patients much needed new treatments. Researchers have coupled their findings with technological advancements to dramatically accelerate the way we have treated blood cancer over the last ten years!

  • Treatment IN ACTION
    *MDS: myelodysplastic syndromes, MPN: myeloproliferative neoplasms

  • LLS currently supports 277 research projects led by innovative scientists worldwide to find new treatments for all types of blood cancer, including:

    • Fahmin Basher, MD, PhD | Duke University Medical Center: Working on an immune-based solution to graft-versus-host-disease, which can limit the effectiveness of stem cell transplants for blood cancer patients

    • Pierre Belichard, PhD, MBA | Enterome: Developing an innovative vaccine immunotherapy for certain types of lymphoma.

    • Bing Carter, PhD MD | Anderson Cancer Center: Working on a way to reactivate a gene that can improve survival of people with acute myeloid leukemia.

    • George Daley, MD, PhD | Boston Children’s Hospital:
      Developing off the shelf CAR T-immunotherapies to make this groundbreaking treatment available to more patients quickly 313 children enrolled in the LLS PedAL.

  • Master Clinical Trial
    30 U.S. states and 16 countries
    Research funded in LLS supports research projects led by innovative scientists worldwide to find new treatments for all types of blood cancer, including...

    • Fahmin Basher, MD, PhD Duke University Medical Center.
      Working on an immune-based solution to graft-versus-host disease.

    • Bing Carter, PhD MD Anderson Cancer Center. Working on a way to activate a gene that can improve survival of people with AML.

    • Pierre Belichard, PhD, MBA Enterome. Developing an innovative vaccine immunotherapy for certain types of
      lymphoma.

    • George Daley, MD, PhD Boston Children’s Hospital. Developing off-the-shelf immunotherapies to be available to more patients quickly.

  • Research Investment
    $332.2 million in multi-year research commitments for all
    forms of blood cancer Myeloma $38.7M MDS/MPN* $52.8M
    Lymphoma $122.2M Leukemia $118.5M

  • 1,500 patients enrolled in the Beat AML®
    master clinical trial.

National MS Society, Mid-America Chapter Society

  • 2023: First generic versions of Aubagio® (teriflunomide) available

  • 2023: A team funded by the Society reports that MS can be prevalent in any population in the U.S.

  • 2023: The first-ever Pathways to Cures Global Summit held in New York City, convening nearly 200 participants from 15 countries to review the roadmap

  • 2023: Multiple sclerosis organizations worldwide jointly declare their collective commitment to a global research strategy to cure MS

  • 2023: Scientists pinpoint possible genetic basis for progressive MS

  • 2023: An international consortium updates guidelines on the complex process of telling MS from lookalike disorders (“differential diagnosis”)

  • 2023: FDA approves Tyruko® (natalizumab-sztn), similar to Tysabri; Tyruko is the first biosimilar disease modifying therapy for adults living with relapsing forms of MS

  • 2023: FDA approves Portable Neuromodulation Stimulator (PoNS®), a device for use during physical therapy to treat walking problems in people with MS

  • 2024: Refined Pathways to Cures Roadmap is published, accounting for recent scientific advances and providing recommendations to accelerate progress toward MS cures

  • 2024: Global Landscape Assessment of MS research published, highlighting the distribution and topics of more than 2,300 research projects valued at nearly $2 billion

Nebraska Chapter of the National bleeding Disorders Foundation

  • NHF has created Community Voices in Research (CVR) which is a community based registry that gathers surveys offering researchers a 360- degree view of what it is like to live with a bleeding disorder. Open to all persons affected by an inherited bleeding disorder as well as their non- affected family members (Parents, spouses, grandparents and siblings), it provides all an opportunity to participate in patient- reported outcome research.

Nebraska Kidney Association

  • Home Hemodialysis has a better mortality rate with more frequency than in center treatments. It also frees up some of the dietary and fluid restrictions.

Susan G. Komen®

WHY DOES ENDING BREAST CANCER NEED RESEARCH?

 It’s a question we like to ask ourselves here at Komen, because it reminds us why we are here every day, doing this work. Research is the only thing that will find the cures for breast cancer. Research means hope and is the key to better future treaments that will help us end breast cancer forever.

 We know breast cancer is much more complex than the biology of a single cancer cell or gene and believe in a future where breast cancer care is holistic and personalized for each individual. That’s why we invest in studies and clinical trials to develop more that can be tailored to address the unique needs of every person. To date, Komen has invested more than $360 million in over 770 research grants and nearly 140 clinical trials focused on precision medicine. In fact, in 2024 65% of Komen’s $10 million research investment focused on advancing precision medicine.

 Komen also works to eliminate breast cancer disparities and inequities by investing in research focused on the root causes of these disparities and supporting the development of a more diverse scientific workforce who will reflect the communities we serve. We believe every person should have a just opportunity to be as healthy as possible, which is why we have already invested nearly $140 million in over 320 research grants and nearly 190 clinical trials focused on breast cancer disparities& inequities.

 This year, we continue to make metastatic breast cancer (MBC) and other deadly and aggressive breast cancers a top research priority, with 70% of this year’s Komen-funded research projects focused on MBC and other deadly and aggressive breast cancers. To date, Komen has invested more than $410 million in nearly 900 research grants and over 130 clinical trials focused on MBC and other deadly and aggressive breast cancers.

Team Jack

  • A $350,262 Grant has been awarded to Dr. Sutapa Ray at the University of Nebraska Medical Center. The project is to study the role of Coactivator-Associated Arginine Methyltransferase 1 (CARM1) in Pediatric Brain Cancer Medulloblastoma

  • A $164,434 grant has been awarded to Dr. A. Angie Rizzino at the University of Nebraska Medical Center. The project is to study SOX2 Levels Control Transcriptional Dynamics in Tumor Cells

  • In 2024, the Team Jack Foundation awarded $514,696 in grant funding for two investigators at the University of Nebraska Medical Center as part of the Power 5 Childhood Brain Tumor Initiative which is a $5 million commitment to UNMC and Children’s Nebraska for research, pain management and education.

  • To date, the Foundation has awarded 15 grants totaling over $2.5 million to researchers at the University of Nebraska Medical Center as part of the Power 5 initiative.

  • Visit https://teamjackfoundation.org/nebraintumorprogram/

  • In 2017, the Team Jack Foundation joined other national non-profits to fund basic/translational science and subsequently the clinical trial phases of a project entitled TAK580 lead by Principal Investigator, Dr. Karen Wright, at Dana-Farber Cancer Institute in Boston, MA.  This promising therapy is an oral treatment taken once a week for children battling relapsed glioma brain tumors. The Team Jack Foundation invested $800,000 to this project.  In May 2020, Day One Biopharmaceuticals, a pharmaceutical company whose focus is to bring new treatments to market that benefit children, announced that they had acquired TAK580 (DAY101), and it will be their top priority. Day One developed DAY101, now referred to as tovorafenib, as a targeted treatment for children with brain cancer.  On April 23, 2024, DAY101, now OJEMDA, received approval from the U.S. Food and Drug Administration (FDA) for the treatment of patients six months of age and older with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. pLGG is the most common brain tumor diagnosed in children, with patients suffering profound tumor- and treatment-associated morbidities that can impact their life trajectory. BRAF is the most commonly altered gene in pLGG, with up to 75 percent of children having a BRAF alteration. Until now, there had been no medicines approved for patients with pLGG driven by BRAF fusions.

  • In December 2024, the Team Jack Foundation awarded $2,275,000 to three national research projects at eight different institutions world-wide.

  • For updates on currently funded projects and updates, visit: https://teamjackfoundation.org/our-impact/

UCP

  • From Boys Town National Research Hospital: Igniting Mobility in Adolescents & Young Adults with CP… The Institute for Human Neuroscience at Boys Town National Research Hospital is conducting a study to determine the magnitude of the mobility improvements seen in individuals with cerebral palsy after undergoing a novel gait training approach. Click here for more information.

  • The Cerebral Palsy Research Network launched a Dystonia in CP (DCP) Toolkit, one of the first comprehensive resources about DCP for people with CP and their families and caregivers. The DCP Toolkit provides an overview of dystonia in cerebral palsy, a movement disorder that is part of the dyskinetic CP group (dystonia, chorea and athetosis) and affects 70% of people with CP.  The Toolkit includes current information about screening, general care and treatments for dystonia, as well as targeted research efforts.

    Check it out here.