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All brains on deck to fight neurodegenerative disease

Neurodegenerative diseases affect more than 62 million people worldwide. The Center for Innovation in Brain Science (CIBS) at the University of Arizona merges translational and clinical research to advance the creation of precision therapeutics. Tight integration with faculty operated biotech startups and the development industry partnerships ensures the translation and delivery of innovative precision therapeutics. A testament to the model’s success is a recent $37.5 million grant from the National Institutes of Health for a phase 2 clinical trial testing a potential regenerative therapeutic forAlzheimer’s Disease.

What is the main challenge that CIBS addresses?

There is not a single cure for a single neurodegenerative disease. Our focus is on the four major age-associated neurodegenerative diseases: Alzheimer’s, Parkinson’s, Multiple sclerosis and ALS (Amyotrophic Lateral Sclerosis).Enormous intellectual and financial resources have been brought to bear on these; yet cures remain elusive. We must think differently about the origin and trajectory of these diseases. A common factor in each is advanced agewhich is the most significant factor for these diseases. Our approach focuses on understanding that ageing is a dynamic process, comprisingmultiple distinct transition states and that these diseases involvemultiple systems of biology.

How do you achieve this goal?

CIBS is an integrated translational ecosystem focused on the four major age-associated neurodegenerative diseases. The expertise of our faculty and research staff spans the spectrum of translational innovation: frombasic disease biology, brain bioenergetics, computational systems biology, bioinformatics, structural biology, medicinal chemistry, translational animalmodel development, to clinical trial design. Collaborations within the centre take the form of dedicated design teams, comprising faculty from differentdisciplines focused on finding and validating new targets and developing therapeutics.

At CIBS, once a promising candidate has been identified, a team of regulatory and clinical experts ensures that all Investigational New Drug (IND)-enabling requirements are met to ensure it moves through the pipeline.

Why is this approach unique?

CIBS is a hybrid between academic research and biotech translational knowhow. We have an ‘all brains on deck’ culture where we shareintelligence and resources. Because we are all located together, we can discuss issues, answer questions and advance our research in real time.Part of our success is that we are nimble and can integrate enabling innovations across our research endeavors. This is one of the most importantaspects of our success, we can test innovation in one disease category, and once proven successful we can seamlessly expand that innovation acrossmultiple neurodegenerative diseases. We understand that to achieve our goal of delivering precision therapeutics we will have to both develop startupsand partner with existing companies. In fact, most of our investigators have launched one or more startups.

What advancements has CIBS made recently?

A major effort is the development of the first regenerative therapeutic forAlzheimer’s disease. Our research into the regenerative potential of the neurosteroid allopregnanolone has transitioned from mechanistic discoveryscience to IND-enabling translational research, and then to a phase 1 clinical trial in people with Alzheimer’s. Based on outcomes from our mechanistic,translational and phase 1 clinical trial research, we are advancing a precision medicine approach in our National Institute on Ageing funded Phase 2 clinical trial of – allopregnanolone for patients that carry the APOE4 geneticrisk factor.

How does this advancement in Alzheimer’s treatment apply to other neurodegenerative diseases?

Our regenerative therapeutic for Alzheimer’s is one example ofthe therapeutics being developed by CIBS investigators, and itssuccess is a huge endorsement of our model. Our portfolio includesnovel therapeutics that target pathways for regeneration, proteinaggregation, neuroinflammation and brain bioenergetics. I amvery excited that since launching in 2016 we have been ableto identify therapeutics for Alzheimer’s, Parkinson’s, Multiplesclerosis, and ALS, whose stage in our pipeline ranges from early development to clinical trials. The University of Arizona has an excellent intellectual property office and we have been able to file patents for our discoveries.

What impact does your work have on patients?

We are living longer, but not necessarily better. Most neurodegenerative diseases are chronic conditions. They are an unrelenting physical and financialchallenge for the person with the disease as well as their family, community and nation. Curing one person of one of these diseases has a broad rippleeffect; when a person is cured of a neurodegenerative disease, thatperson is freed of the disease, as are their family and community.

What’s next?

These diseases are personal, and our therapeutics should be too, while we have a promising portfolio, our pipeline of therapeutics will expand toadvance precision medicine for neurodegenerative disease. Ourapproach has been successful. Going forward, we will continueto innovate and expand our therapeutic horizon to bringinnovations in brain science of the future to those who need acure today.


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