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MD Anderson and Sibylla Biotech announce strategic collaboration to discover and develop small-molecule protein degraders

The University of Texas MD Anderson Cancer Center and Sibylla Biotech today announced a strategic collaboration agreement to discover and develop novel small-molecule cancer therapies known as folding interfering degraders (FIDs), which disrupt the proper folding of target proteins and lead to their degradation.

Under the agreement, Sibylla and MD Anderson will jointly conduct discovery and development work from target identification through drug candidate nomination on selected proteins, with the potential to continue further pre-clinical and clinical development. The collaboration brings together Sibylla’s Pharmacological Protein Inactivation by Folding Intermediates Targeting (PPI-FIT) technology with the drug development expertise and capabilities of MD Anderson’s Therapeutics Discovery division.

“The Sibylla team is committed to expanding the applications of our PPI-FIT technology and FIDs to provide new treatment options for hard-to-treat indications. This collaboration builds upon the progress we have achieved to date and the applicability of our technology and know-how,” said Lidia Pieri, PhD, co-founder and chief executive officer of Sibylla Biotech. “We value the opportunity to work with MD Anderson’s team of drug development experts in order to foster our vision of treating patients with high unmet medical need.”

Sibylla’s proprietary PPI-FIT technology is used to discover and develop FIDs, small molecules that induce the degradation of target proteins by interfering with the folding pathway. Notably, PPI-FIT can be applied to target proteins currently considered “undruggable” due to the absence of suitable pockets in their native state.

“Folding interfering degraders represent an exciting new modality to target key cancer drivers, and we look forward to opportunities to advance compelling candidates forward as novel therapeutic options,” said Tim Heffernan, Ph.D., vice president and head of Therapeutics Discovery at MD Anderson. “By aligning Sibylla’s innovative PPI-FIT technology with our drug development engine, we hope to create impactful new medicines that will expand options for patients in need of more effective therapies.”

By uniting scientists, clinicians and drug development experts together within MD Anderson, the institution’s Therapeutics Discovery division is designed to eliminate the bottlenecks slowing traditional drug discovery. Seamless integration with MD Anderson physicians allows Therapeutics Discovery to develop impactful cancer therapies inspired directly by patient needs and clinical insights.

Once a drug candidate is nominated, Sibylla and MD Anderson may consider further drug development, translational, and clinical activities to advance the candidate for patients in need.

Disclosure
MD Anderson has an institutional conflict of interest with Sibylla and will implement an Institutional Conflict of Interest Management and Monitoring Plan to manage this relationship.

Belfer family’s $20 million donation invigorates neurodegeneration research at MD Anderson

HOUSTON ― Laurence Belfer, on behalf of Robert Belfer and the Belfer family, today announced a $20 million gift to The University of Texas MD Anderson Cancer Center to strengthen neurodegeneration research through the Belfer Neurodegeneration Consortium (BNDC), a transformative multi-institutional initiative to advance the study and treatment of Alzheimer’s disease.

This $20 million commitment brings the Belfer family’s total giving to MD Anderson to $53.5 million in just over 10 years. Their generous gift will be matched through institutional philanthropic efforts, bringing an additional $20 million to neurodegeneration and other age-associated disease research efforts at MD Anderson.

The research of the BNDC, which is part of MD Anderson’s Therapeutics Discovery division, may also benefit patients with cancer, as cancer shares biological similarities with neurodegenerative diseases and certain cancer treatments are known to cause neural and cognitive impairments.

“Transformative gifts like this have the power to achieve more than we could have ever imagined,” said Peter WT Pisters, M.D., president of MD Anderson. “We are grateful to the Belfer family for their forward-thinking vision and for their renewed investment in the Belfer Neurodegeneration Consortium. Their generosity has and will continue to impact countless lives.”

To commemorate the Belfer family’s generosity, MD Anderson’s South Campus Research Building 4, a 208,000 sq.-foot research facility located at 1901 East Road in Houston, will be renamed “The Robert A. and Renée E. Belfer Research Building.” The Belfer Building houses laboratories and offices of the BNDC as well as the Therapeutic Discovery division and the departments of Experimental Therapeutics, Cancer Biology, Genomic Medicine and Surgical Oncology Research.

“Since the consortium was formed, we have made tremendous progress in our understanding of the molecular and genetic basis of neurodegenerative diseases and in translating those findings into effective targeted drugs and diagnostics for patients,” said Jim Ray, Ph.D., executive director, Belfer Neurodegeneration Consortium. “Yet, we still have more work to do. Alzheimer's disease is already the most expensive disease in the United States. As our population continues to age, addressing quality-of-life issues and other challenges of treating and living with age-associated diseases must become a priority.”

With this latest gift, the Belfer family aims to do just that.

“We want to help aging adults lead better, longer lives,” said Belfer. “My family strongly believes in the work being done at MD Anderson through the Belfer Neurodegeneration Consortium to help those with Alzheimer’s and other forms of neurodegenerative diseases. We are proud to put our name behind this collaborative effort that will impact generations to come.”

Belfer Neurodegeneration Consortium to advance novel therapeutic strategies
Formed in 2012, the BNDC is a multi-institutional initiative between MD Anderson, Massachusetts Institute of Technology (MIT), Icahn School of Medicine at Mount Sinai, Baylor College of Medicine, among others. The consortium was established to gain a deeper understanding of neurodegenerative disease biology and translate those findings into effective therapeutic interventions.

The BNDC is a core component of MD Anderson’s Therapeutics Discovery division, which unites scientists, clinicians and drug development experts to eliminate the bottlenecks stifling traditional drug discovery. Driven by unparalleled proximity to patients and unmatched clinical and research expertise, Therapeutics Discovery and the BNDC can advance effective new therapies in ways not possible elsewhere.

Building upon early successes, the BNDC aims to develop five new medicines for Alzheimer’s disease and related disorders over the next 10 years, with the goal for two treatments to demonstrate clinical efficacy.

“Our goal is ambitious, but having access to the vast clinical trial expertise at MD Anderson ensures our therapeutics can improve the lives of patients everywhere,” Ray said. “The key elements for success are in place: a powerful research model, a winning collaborative team and a robust translational pipeline, all in the right place at the right time.”

MD Anderson researchers are working through its Cancer Neuroscience Program to explore the intersection between neurological disease and cancer. The work of the BNDC helps inform the Cancer Neuroscience Program, positioning the institution to make maximal impact on patients with cancer.

Advances in cancer neuroscience and drug discovery could benefit patients with Alzheimer’s disease

MD Anderson is known for its cancer care and research. But did you know that for more than a decade, MD Anderson researchers have been working on better treatments for Alzheimer’s disease and other age-related neurodegenerative diseases, too?

Launched in 2012 through a $25 million gift from Robert Belfer and the Belfer family, the Belfer Neurodegeneration Consortium (BNDC) is a multi-institutional initiative between MD Anderson, the Massachusetts Institute of Technology (MIT), the Icahn School of Medicine at Mount Sinai in New York, Baylor College of Medicine and New York University, among others. The consortium was established to gain a deeper understanding of neurodegenerative diseases and translate those findings into effective therapeutic interventions.

Unparalleled drug discovery and development expertise

The Belfer Neurodegeneration Consortium is a core part of MD Anderson’s Therapeutics Discovery division, which brings together scientists, clinicians and drug development experts to eliminate the bottlenecks stifling traditional drug discovery and advance effective new therapies in ways only possible here at MD Anderson.

In Fiscal Year 2023, MD Anderson had more NCI-funded projects than any other U.S. institution and the world’s largest cancer clinical trials program. MD Anderson also helped to evaluate nearly 60% of the new drugs approved by the Food and Drug Administration last year.

“Very few places have this breadth and scope of drug discovery,” says Jim Ray, Ph.D., executive director of the Belfer Neurodegeneration Consortium. “The Therapeutics Discovery division is essentially a mid-sized biotech company within an academic hospital.”

Alzheimer’s disease and age-related diseases could affect future cancer research and care

When the Belfer Neurodegeneration Consortium was formed, Alzheimer’s was rapidly becoming a major health care crisis and was projected to account for 50% of the total U.S. health care economy by 2050. Today, nearly 7 million people are estimated to be living with Alzheimer’s disease, and 1 in 3 adults over age 65 will develop dementia. And, the risk for Alzheimer’s and other neurodegenerative diseases goes up drastically after age 85.

“In addition to the tremendous suffering it causes, Alzheimer's is also the most expensive disease in the United States. Patients need full-time family support or round-the-clock care in specialized centers,” Ray says. “As the population ages, we won't be able to support the number of people who need care.”

MD Anderson’s leadership and donors foresaw how this diversion of health care resources could affect future cancer patients. If limited health care resources were tied up with caring for patients with neurodegenerative diseases, that could affect future funding for cancer research and care.

Alzheimer’s disease and cancer have unexpected connections

Given the brain’s unique environment and the fact that cancer and Alzheimer’s disease share risk factors and have similar biological pathways of disease, many therapies the Belfer Neurodegeneration Consortium studies for Alzheimer’s also may have potential for treating cancer.

“We are constantly discovering unexpected connections between Alzheimer’s and cancer,” Ray says. “By studying the fundamental causes of neurodegenerative diseases, we hope to develop therapies that promote nervous system health and function in cancer patients, too.”

For example, he points to the similarities between the memory-related side effects of chemotherapy, commonly referred to as chemobrain, and the dementia experienced by Alzheimer’s patients. The Belfer Neurodegeneration Consortium observed cancer and Alzheimer’s patients’ brains both exhibited the activation of a protein called DLK, which senses injury to neurons and kills damaged brain cells, leading to cognitive impairment. Similarly, patients also share similar inflammatory responses in the brain’s immune cells called microglia, which differ from immune cells found in the rest of the body. Together, these biological responses contribute to the memory loss and nerve damage experienced by both cancer patients and Alzheimer’s patients.

That’s why the consortium is helping launch the NeuroHealth Initiative at MD Anderson to study the brain and the nervous system – beginning with ovarian cancer patients – to understand what happens when patients develop memory loss and neuropathy during chemotherapy and radiation treatment.

Ray says this provides a unique lens for studying the early stages and underlying mechanisms of neurodegeneration because, unlike with Alzheimer’s, researchers will be able to study cancer patients before their first treatment and thus, before the onset of their first symptoms.

Early breakthroughs show promise for next-generation Alzheimer’s therapies

Just as the last decade saw immunotherapy, targeted therapy and advanced diagnostics extend the lives of cancer patients, Ray believes now is also a critical time for treatment advances for Alzheimer’s.

“For the first time, we have agreement in the field that it is possible to slow the disease,” he says. “To do this, we need multiple ways to attack the disease, as well as a deep understanding of what's driving each patient's dementia, so that we can have personalized medicine for Alzheimer’s.”

Over the past decade, the Belfer Neurodegeneration Consortium has pursued over 40 drug discovery projects, with five advancing to the next stage. From this research, they have established four keys to treating Alzheimer’s:

Introduce neuroprotective agents to keep brain cells from dying
Address genetic risk factors like APOE4 before patients show symptoms of Alzheimer’s disease
Lessen the inflammatory response of microglia, the brain’s immune cells
Combat the toxicity of tau proteins that cause amyloid plaque tangles in the brain

Encouraged by the Belfer Neurodegeneration Consortium’s early success, the Belfer family recently made an additional $20 million gift to advance the consortium’s goals for the next 10 years: to develop five new drugs to treat Alzheimer’s and related diseases, and for two of those drugs to show meaningful evidence of changing the course of the disease. The Belfer family’s $20 million gift will be matched through MD Anderson’s institutional fundraising efforts, bringing a total of $40 million to the consortium’s work.

The Belfer Neurodegeneration Consortium is currently running 12 projects targeting various aspects of Alzheimer’s, with one project already proceeding to a Phase I clinical trial. Efforts underway include the:

Choline trial, which is testing whether a safe dietary intervention can reduce the risk of dementia in carriers of the APOE4 gene.
DLK project, designed to develop a neuroprotective therapy for Alzheimer’s, which was awarded $20 million from the State of Texas to be developed also as a treatment for cancer patients who experience nerve damage due to chemotherapy.
RIPK1 project, which aims to prevent microglial inflammation in the brain during neurodegeneration and could also prevent brain inflammation following radiation therapy.
PU.1 project, which has identified PU.1 as a genetic risk factor not only for Alzheimer’s disease but also for leukemia.
MS4A project, which targets the MS4A4A and MS4A6A genes for the prevention of dementia. These genes have also been shown to play a role in brain cancer.

The consortium hopes these projects will contribute to a multi-pronged approach for treating Alzheimer’s and neurodegenerative diseases at an earlier stage, ideally halting the disease’s progress before patients begin experiencing cognitive impairment.

“We are confident our goals are achievable,” Ray says. “The key elements for success are in place — a powerful research model, a winning collaborative team and a robust translational pipeline, all in the right place at the right time.”

Make a gift to support the Belfer Neurodegeneration Consortium.

MD Anderson and CureVac enter strategic collaboration to develop novel cancer vaccines

The University of Texas MD Anderson Cancer Center and CureVac N.V. today announced a co-development and licensing agreement to develop novel mRNA-based cancer vaccines.

The collaboration creates strong synergies between CureVac’s unique end-to-end capabilities for cancer antigen discovery, mRNA design, and manufacturing and MD Anderson’s expertise in cancer antigen discovery and validation, translational drug development, and clinical research. The collaboration will focus on the development of differentiated cancer vaccine candidates in selected hematological and solid tumor indications with high unmet medical need.

"We look forward to collaborating with the team at MD Anderson to push the boundaries of mRNA technology and develop impactful therapeutic options for patients in need," said Alexander Zehnder, M.D., chief executive officer of CureVac. “In combining our respective expertise, we believe we can go further and faster to develop novel, off-the-shelf, mRNA-based cancer vaccines that have the potential to significantly improve patient outcomes.”

Both parties will contribute to the identification of differentiated cancer antigens based on whole genome sequencing combined with long- and short-read RNA sequencing and cutting-edge bioinformatics. Joint preclinical validation of the highest-quality cancer antigens will be supported by Sachet Shukla, Ph.D., assistant professor of Hematopoietic Biology and Malignancy and director of the department’s cancer vaccine program, and by MD Anderson’s ECLIPSE (Evolution of Cancer, Leukemia, and Immunity Post Stem cEll transplant) platform, part of the institution’s Therapeutics Discovery division.

“We are excited for cancer vaccines to potentially emerge as an essential therapeutic tool in the future,” Shukla said. “This collaboration with CureVac is an important milestone in our efforts and brings together complementary strengths toward our goal of developing transformative vaccines for cancer.”

Following selection of the most promising clinical-lead vaccine candidates and completion of Investigational New Drug (IND) approvals, MD Anderson will be responsible for conducting initial Phase I/II studies in appropriate clinical indications.

“Our ECLIPSE team uses proprietary high-throughput technology to identify and validate immune targets, and we are driven to advance impactful immunotherapies with the potential to transform the lives of patients with cancer,” said Jeffrey Molldrem, M.D., chair of Hematopoietic Biology and Malignancy and leader of the ECLIPSE platform at MD Anderson. “Together with CureVac, we hope to embrace this exciting area of drug discovery and development in pursuit of mRNA vaccines that will address significant unmet medical need.”

Under the terms of the collaboration agreement, CureVac and MD Anderson will jointly contribute to and support development of those programs designated to move forward. CureVac has worldwide exclusive rights to late-stage development, commercialization, or partnering of the cancer vaccine candidates. MD Anderson is eligible for certain downstream payments based on potential future commercialization.