Research into the future use of stem cells in providing medical care has opened the door to potential breakthroughs for treating many medical disorders such as degenerative conditions, inflammatory ailments, metabolic diseases, cardiovascular conditions, blood disorders, and more. At the same time, questions have been raised about the safety of these new therapeutics that require diligent and well-planned preclinical testing.
Designing the Right Study
Designing the right study for your stem cell-derived therapy is key. MPI Research partners with you to evaluate
- Proof-of-Concept in select disease models—Once implanted, what effect do the cells have on modifying the disease condition?
- Biodistribution and cell fate—Where do the cells go, do the cells differentiate, and how long do they persist?
- Toxicity—Is the cell product safe?
- Tumorigenicity—Do the cells proliferate or facilitate tumor development?
An effective preclinical evaluation requires careful study design based on scientific expertise, knowledge of regulatory requirements, and an understanding of the complexities of both somatic and stem cell therapeutics. Our scientists apply these strengths to develop relevant studies that take into account GLP regulations, appropriate test systems, dose level and stability (including viability and durability), cell fate (proliferation and differentiation), and cell biodistribution, toxicity, and tumorigenicity.
Selecting the Right Model
Considerable knowledge and experience are applied while selecting the in vivo models that are the best options for biodistribution and safety assessments of stem cell therapeutics. MPI Research works closely with Sponsors to ask—and answer—the right questions to guide the selection of the applicable model based on solid scientific and regulatory understanding.
- Appropriate models for evaluating cell survival, fate, and function
- Genetically immunocompromised or chemically immunosuppressed models [e.g., severe combined immunodeficiency (SCID) mice, nude rats, targeted disease models, immunosuppressed large animals] that provide relevant data
- An array of options to ensure that specific safety evaluation endpoints are met
- Multiple models that meet the needs for clinically relevant surgical procedures, and biological and pharmacologic relevance to human counterparts, sample availability, and appropriate study duration
Applying the Right Science
Stem cell therapeutics represent cutting-edge life science research and development. MPI Research scientists have conducted more than 50 related studies (including key pivotal studies for IND filings) in partnership with more than 20 companies developing novel stem cell therapies.
Our scientists use their experience and expertise to design a program to properly assess your cellular therapeutics. Our scientists use multiple animal models; apply conventional and specialized surgical delivery routes; and assess a range of endpoints, including specialized tests, clinical observations, clinical pathology, histopathology, and biodistribution to demonstrate the efficacy and/or safety of your therapeutic. MPI Research provides
- State-of-the-art research facilities
- Superior surgical, advanced molecular imaging (PET/CT and nanoSPECT/CT), and analytical capabilities
- GLP-compliant necropsy, histology, and histopathology departments
- Board-certified anatomical and clinical pathologists
- Small and large animal in vivo models
- Inflammation, cardiovascular, stroke, metabolic disease, orthopaedic, wound healing, and other therapeutic area models
- Immunocompromised rodent handling, Biosafety Level (BSL) 2 barrier facilities, hyperfiltration, and microisolator caging systems
- Immunosuppressed large animal handling in specialized facilities to ensure prolonged animal health
- Surgical implantations including intraocular, intracranial, intraspinal, intrathecal, intracoronary, myocardial, intraprostatic, topical grafting, infusion, and others
- Specialty endpoints that include behavioral and functional assessments, angiogenesis, allodynia, ocular morphology (OCT, fundic imaging) and function (ERG)
- Board-certified veterinary ophthalmologist
- Quantitative polymerase chain reaction (qPCR) for biodistribution, including GLP-validated methods, compliant facilities, and robotics
- Validated use of immunohistochemical and immunofluorescent staining, immunoassays, and flow cytometry
Studies performed in the past 2 years
Partnerships with Sponsors developing novel stem cell therapies