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Comprehensive Evaluation of the Effectiveness and Safety of Placenta-Targeted Drug Delivery Using Three Complementary Methods
Published on: September 10, 2018
Preeclampsia - will orphan drug status facilitate innovative biological therapies?
1Laboratory for Prenatal Medicine, Department of Biomedicine, University Hospital Basel , Basel , Switzerland.
Abstract:
It is generally accepted that the development of novel therapies to treat pregnancy-related disorders, such as preeclampsia, is hampered by the paucity of research funding. Hence, it is with great interest to become aware of at least three novel therapeutic approaches for the treatment of this disorder: exploiting either the anticoagulant activity of antithrombin, the free radical scavenging activity of alpha-1-microglobulin, or the regenerative capacity of placenta-derived mesenchymal stem cells. As these projects are being carried out by small biotech enterprises, the question arises of how they are able to fund such undertakings. A novel strategy adopted by two of these companies is that they successfully petitioned US and EU agencies in order that preeclampsia is accepted in the register of rare or orphan diseases. This provides a number of benefits including market exclusivity, assistance with clinical trials, and dedicated funding schemes. Other strategies to supplement meager research funds, especially to test novel approaches, could be crowdfunding, a venture that relies on intimate interaction with advocacy groups. In other words, preeclampsia meets Facebook. Perhaps similar strategies can be adopted to examine novel therapies targeting either the imbalance in pro- or anti-angiogenic growth factors, complement activation, reduced levels of placenta protein 13, or excessive neutrophil activation evident in preeclampsia.
Insights
Novel therapies for preeclampsia, a pregnancy disorder, are emerging. Companies are using orphan disease designations and crowdfunding to fund research and development for these treatments.
Area of Science:
- Obstetrics and Gynecology
- Biotechnology
- Pharmacology
Background:
- Development of novel therapies for pregnancy-related disorders like preeclampsia is limited by insufficient research funding.
- Preeclampsia is a serious condition affecting pregnant individuals, necessitating innovative treatment strategies.
Purpose of the Study:
- To highlight novel therapeutic approaches for preeclampsia, including antithrombin, alpha-1-microglobulin, and mesenchymal stem cells.
- To explore innovative funding strategies for small biotech enterprises developing preeclampsia treatments.
- To discuss the potential of designating preeclampsia as a rare or orphan disease to secure funding and support.
Main Methods:
- Review of emerging therapeutic targets for preeclampsia.
- Analysis of funding strategies employed by biotech companies, including orphan disease designation and crowdfunding.
- Examination of the benefits associated with rare/orphan disease status, such as market exclusivity and dedicated funding.
Main Results:
- Three novel therapeutic avenues for preeclampsia are identified: antithrombin, alpha-1-microglobulin, and placenta-derived mesenchymal stem cells.
- Biotech companies are successfully leveraging orphan disease designation to access funding and clinical trial support.
- Crowdfunding and advocacy group collaboration are presented as supplementary funding mechanisms.
Conclusions:
- Securing funding for preeclampsia research is crucial for advancing novel therapies.
- Orphan disease designation and innovative financial strategies like crowdfunding can significantly aid small biotech enterprises.
- Further research into preeclampsia's underlying mechanisms, including angiogenic factors and immune responses, is warranted, potentially benefiting from similar funding approaches.
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