Soliris

Soliris^® (eculizumab) is a first-in-class terminal complement inhibitor discovered, developed and commercialized by Alexion. Soliris works by inhibiting terminal complement, a normal part of the immune system that, when activated inappropriately, plays a role in serious diseases like PNH and aHUS.

Soliris is also approved for the treatment of patients with atypical hemolytic uremic syndrome (aHUS), a catastrophic, ultra-rare genetic disease in which a deficiency in one or more of the complement regulatory genes causes chronic, uncontrolled complement activation that can result in sudden and progressive damage to vital organs, leading to stroke, heart attack, kidney failure and premature death. 

Soliris is the first and only therapy approved for the treatment of patients with aHUS, and is specifically indicated in this population to inhibit complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body. 

Gliadel Wafer; FDA approved treatment for brain cancer

Gliadel Wafer (polifeprosan 20 with carmustine implant) is indicated in newly diagnosed patients with high-grade malignant glioma as an adjunct to surgery and radiation. Gliadel is also indicated in recurrent glioblastoma multiforme patients as an adjunct to surgery. Gliadel provides localized delivery of chemotherapy directly to the site of the tumor and is the only FDA approved brain cancer treatment capable of doing so. 

Gliadel is a white, dime-sized wafer made up of a biocompatible polymer that contains the cancer chemotherapeutic drug, carmustine (BCNU). After a neurosurgeon removes a high-grade malignant glioma, up to eight wafers can be implanted in the cavity where the tumor resided. Once implanted, Gliadel slowly dissolves, releasing high concentrations of BCNU into the tumor site. The specificity of Gliadel minimizes drug exposure to other areas of the body.

 

Reference:

 1. Dang, Wenbin, et al. "Effects of GLIADEL® wafer initial molecular weight on the erosion of wafer and release of BCNU." Journal of Controlled Release 42.1 (1996): 83-92.

2. www.gliadel.com

 

Science isn't about why...It's about why not?

 

RNA interference; Novel strategy in treatment of cancer

CALAA-01

The siRNA nanoparticle is based on a polymer of cationically derivatized cyclodextrin complexed with the anionic siRNA payload. The nanoparticles are stabilized by binding of hydrophobically terminated PEG to the cyclodextrin rings, which occurs via the interaction of the hydrophobe (guest) within the core of the cyclodextrin (host). Delivery of cyclodextrin-bound-PEG further grafted with transferrin — a protein for which many tumour cells over-express a receptor — resulted in knockdown of target messenger RNA and the reduction of protein levels in tumours, opening the door for further clinical investigations focused on antitumour activity.

Reference: Hubbell, Jeffrey A., and Robert Langer. "Translating materials design to the clinic." Nature materials 12.11 (2013): 963-966.

The whole of science is nothing more than a refinement of everyday thinking; Albert Einstein