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Accelerating Alzheimer’s Treatment: Fast and Effective Preclinical Development

In this blog post, we dive into how the InVivo Biosystems preclinical development platform is closing the gaps in finding successful treatments for Alzheimer’s disease and other Central Nervous System (CNS) diseases.

Alzheimer’s disease (AD) is one of the most challenging neurodegenerative disorders of our time, affecting millions of individuals and their families globally. This complex disease, characterized by memory loss, cognitive decline, and personality changes currently has no cure. However, the relentless pursuit of scientific discovery is paving the way for new treatments. One of the crucial stages in the pursuit of any new treatment is preclinical development, a vital phase that takes a potential drug lead and provides the foundational research needed before a new therapeutic enters clinical trials.

What is Preclinical Development?

Preclinical development is the critical phase of research that occurs before clinical trials involving human participants. It encompasses a series of laboratory and animal studies designed to evaluate the safety, efficacy, and biological mechanisms of a potential treatment. For Alzheimer’s disease, this stage is especially significant due to the intricate nature of the disease and the need for innovative approaches to tackle it.

The Importance of Preclinical Models in Alzheimer's Research

The path to effective Alzheimer’s treatments begins with the right models. At InVivo Biosystems, we create model organisms that mimic the genetic and pathological features of human diseases, including Alzheimer’s. These models, ranging from live alternative models to complex cellular systems, provide invaluable insights into disease mechanisms and therapeutic potential.

  1. Induced Pluripotent Stem Cells (iPSCs): At InVivo Biosystems, our partners offer iPSCs which are often used in mechanistic discovery studies prior to moving to in vivo model studies. iPSCs are derived from adult cells which have been reprogrammed to an embryonic-like state. These cells can differentiate into various cell types, including neurons, astrocytes, and oligodendrocytes, providing a powerful tool for modeling neurodegeneration. iPSC-derived neurons from patients with Alzheimer’s disease allow researchers to study disease mechanisms in a human cellular context and test potential therapies on patient-specific models. 
  2. Nematodes (C. elegans): These tiny worms are genetic powerhouses. Their short lifespan and well-mapped nervous system make them ideal for high-throughput screening of potential drugs. In Alzheimer’s research, C. elegans models expressing human amyloid-beta and tau proteins help scientists understand how these proteins aggregate and cause neurodegeneration. At InVivo Biosystems, we utilize our patented Whole-gene Humanized Animal Models (WHAM) technology to create C. elegans models with human-equivalent ortholog genes. Our WHAM technology has enabled us to generate numerous reliable models for Alzheimer’s Disease. Click here to learn more about our Alzheimer’s Disease models
  3. Zebrafish: Zebrafish offer a unique blend of simplicity and complexity. Their transparent embryos allow real-time observation of brain development and pathology. With our patented rapid CRISPR technology, we are able to produce Alzheimer’s genetic models in zebrafish in less than 4 weeks. Our high throughput phenotypic assay technologies also provide us the capability to study the disease’s progression, screen and validate compounds that may prevent or reverse cognitive decline, and more. Click here to learn more about our rapid zebrafish CRISPR technology
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Key Stages of Preclinical Development

1. Target Identification and Validation: The journey begins with identifying potential targets—molecules or pathways involved in Alzheimer’s pathology. Once identified, these targets are validated in preclinical models to ensure they play a critical role in the disease process.

2. Compound Screening and Optimization: With validated targets, researchers screen large libraries of compounds to identify potential drug candidates. This stage involves extensive testing to find compounds that interact with the targets effectively. Promising candidates undergo further optimization to enhance their efficacy and reduce potential side effects.

3. In Vitro and In Vivo Testing: Selected compounds are tested in cell cultures (in vitro) and/or in live organisms (in vivo) to assess their impact on cellular processes as well as effects on disease progression and cognitive function.

4. Pharmacokinetics and Toxicology: Understanding how a potential drug is absorbed, distributed, metabolized, and excreted (pharmacokinetics) is crucial. Equally important is assessing its safety profile through toxicology studies. These evaluations ensure that the candidate drug is safe for further development.

Overcoming Challenges in Alzheimer's Preclinical Research

Alzheimer’s disease poses unique challenges for preclinical development. The complex interplay of genetic, environmental, and lifestyle factors in AD requires multifaceted approaches. Additionally, the blood-brain barrier complicates drug delivery to the central nervous system. Traditional models used for clinical development are known to not be reliable for neurodegenerative diseases such as Alzheimer’s due to the susceptibility rodent models have to genetic drift when modeling for protein aggregation phenotypes.

At InVivo Biosystems, we embrace these challenges with innovative solutions. By leveraging advanced genetic tools, we create alternative models that reliably capture the diversity of Alzheimer’s pathology. Our cutting-edge in vivo imaging technologies allow us to visualize disease processes in real-time, accelerating the discovery of effective treatments.

The Road Ahead: From Bench to Bedside

The ultimate goal of preclinical development is to transition promising therapies from the laboratory to the clinic, where they can benefit patients. This journey is a collaborative effort involving researchers, pharmaceutical companies, regulatory agencies, and, most importantly, patients and their families.

While the path to effective Alzheimer’s treatments is long and arduous, each step in preclinical development brings us closer to breakthroughs that can transform lives. At InVivo Biosystems, we are committed to advancing Alzheimer’s research through innovative model organisms and cutting-edge technologies. Together, we can unlock the future of Alzheimer’s treatment and offer hope to millions affected by this devastating disease.

Join Us in the Fight Against Alzheimer's

As we continue to explore the fascinating world of genetics and neurobiology, we invite you to join us in our mission. Whether you’re a fellow researcher, a healthcare professional, or someone passionate about making a difference, your support and collaboration are invaluable.

Stay tuned for more updates on our exciting developments in Alzheimer’s research. Together, we can turn the tide against this formidable disease and build a brighter future for all.

References and Further Reading:

C. elegans Alzheimer’s Disease Model Publications:

Zebrafish Alzheimer’s Disease Model Publications

About The Author

Ally Wimberly

Ally Wimberly is the Manager of Marketing and Sales Operations at InVivo Biosystems. Ally has diverse experience in both academia and biotech industry, and in her industry experience has been successful in both Operations and R&D roles. She leverages her product development, project management, and quality assurance expertise in her current role at IVB.

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