Catastrophic Antiphospholipid Syndrome (CAPS)
Catastrophic antiphospholipid syndrome (CAPS) is an uncommon complication associated with a specific type of antiphospholipid syndrome. To further the CAPS investigations, better and faster specialized drug and therapy development services are needed. Our firm can fulfill all your drug and therapy development needs regarding CAPS treatment.
Introduction to Catastrophic Antiphospholipid Syndrome
CAPS is a rare and life-threatening form of acute Antiphospholipid Syndrome (APS), which causes high morbidity because of micro vascular thrombi in multiple organs resulting in failure of two or more systems. Recent evidence shows how uncommon it is with the annual rate of about 0.15 to 1.05 cases occurring in a million people. The complexity of CAPS lies in its highly aggressive nature and poor prognosis; therefore, it must be identified and treated as fast and as effectively as possible to achieve the best possible results.
Pathogenesis of Catastrophic Antiphospholipid Syndrome
Since it is uncommon, the causes for Catastrophic Antiphospholipid Syndrome are not completely known. It is thought that there is a genetic component, presence of anti-phospholipid antibodies, and some environmental factors. CAPS is characterized by a ‘thrombotic storm,’ where a primary thrombophilic risk (genetic susceptibility and aPL positivity) is followed by a secondary inflammatory hit, most times, the inflammatory hit is started through an infection or surgical procedure. This process does create a local area of thrombosis, but it is accompanied by a systemic inflammatory response syndrome (SIRS) in which there is a dramatic release of pro-inflammatory cytokines.
Fig. 1 CAPS: the "two-hit" theory. (Bitsadze, V., et al., 2024)Diagnosis Development of Catastrophic Antiphospholipid Syndrome
The search for CAPS and its more effective differentiation from other thrombotic conditions lies in identifying its specific and sensitive biomarkers. Some of the potential biomarkers are those indicative of endothelial dysfunction activation or systemic inflammation, tissue inflammation, coagulation, complement activation, and tissue destruction. Diagnostics will be more sensitive and specific if multiple markers are combined into panels or analyzed through algorithms, but practice validation studies are necessary.
Therapy Development of Catastrophic Antiphospholipid Syndrome
Managing CAPS utilizes a variety of small molecule drugs targeting different regions of the coagulation cascade. Warfarin, in use for many years as an anticoagulant, works by blocking vitamin K dependent factors. Rivaroxaban is a direct oral anticoagulant that does not require monitoring and works by inhibiting factor Xa. Hydroxychloroquine helps in preventing thrombosis by inhibiting platelet inflammation and aggregation.
There are various cellular therapies which include introduction and manipulation of cells to alter the functionality of immune system. Mesenchymal Stem Cells (MSCs) are known to have immunomodulatory activity which can be useful in treating CAPS by reducing autoimmunity. Studies have shown that MSCs can reduce inflammation and improve prognosis in animal studies. Under development T cell therapies are supposed to control autoreactive T cells in APS and CAPS.
CAPS generally intensifies severe manifestations of antiphospholipid syndrome that could be treated with monoclonal antibodies targeting selected proteins in patients suffering from CAPS. For example, Rituximab targeting CD20 on B lymphocytes in patients reduces autoantibody levels with some encouraging results in resistant cases. Eculizumab, by blocking the cleavage of the complement protein C5, ameliorates unwanted downstream vocalization of clotting and inflammation and is providing promising results in the management of CAPS.
CAPS gene therapies seek to modify the genetic elements responsible for CAPS. There is current research into the use of CRISPR-Cas9 technology to attempt targeting autoimmune genes in APS and its subtypes. It could be curative. Gene therapies using viral vectors involve the addition of functional genes to immune modulators, but this is still at an early stage in APS and CAPS.
Our Services
We have flexible models of business around partners and clients. CAPS therapy techniques development and service delivery is done in close collaboration with clients to provide guaranteed effective therapy support.
Platforms of Catastrophic Antiphospholipid Syndrome Therapy Development
Animal Models of Catastrophic Antiphospholipid Syndrome
We have already built the necessary animal models of the disease that are needed to run the therapy tests for its safety and effectiveness.
| Non-Genetically Engineering Models | ||
| We provide diverse model choices customized to meet specific research needs related to CAPS. These models allow researchers to simulate and study the complex biological processes associated with CAPS. | ||
| Biologics/Chemical Induced Models | ||
| Optional Models |
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| Genetically Engineered Models | ||
| Our expertise in genetic engineering techniques, allows us to generate accurate and reliable models that recapitulate the genetic alterations observed in human CAPS. | ||
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| Optional Species | Mice, Rats, Non-human primates, Others | |
In addition to these models, our comprehensive services encompass other models that target specific signaling pathways and molecular targets.
If our services align with your goals, please contact us for more details.
References
- Bitsadze, V., et al., "Catastrophic Antiphospholipid Syndrome." Int J Mol Sci, (2024). 25(1).
- Martin, E., et al., "Catastrophic antiphospholipid syndrome in a community-acquired methicillin-resistant Staphylococcus aureus infection: a review of pathogenesis with a case for molecular mimicry." Autoimmun Rev, (2011). 10(4): p. 181-188.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.