Therapeutics

Stable core crosslinked poly (amino-acid) based particles as biodegradable delivery materials

RCSI and DCU have developed a scalable emulsion polymerisation process for the synthesis of cross-linked nanoparticles based on α-amino acids. The nanoparticles are expected to be fully biodegradable, non-toxic and amenable to a wide range of functionalisation via standard peptide chemistry. This materials platform is thus highly adaptable for various applications in the pharmaceutical, cosmetic and personal care sectors.

ChemoGel: A thermoresponsive drug delivery platform for intratumoral delivery of chemotherapeutics in pancreatic cancer

ChemoGel is a unique, thermoresponsive, hydrogel drug delivery platform for the site-specific delivery and sustained release of chemotherapeutics at solid tumour sites. ChemoGel is a platform technology which enables the sustained local delivery of chemotherapeutics agents and is intended for use in the chemical ablation of solid tumours alone or in conjunction with other treatment approaches. The technology is now in pre-clinical development at RCSI for its first clinical indication in pancreatic cancer.

Novel therapeutics for type-2 diabetes and maturity-onset of diabetes of the young (MODY)

Research scientists at RCSI have identified several miRNAs implicated in both MODY and Type 2 diabetes which may provide novel druggable targets for therapeutic intervention. The miRNA targets identified at RCSI may have applications in treating symptoms of metabolic disorders characterised by deficiencies in insulin secretion such as increased blood glucose levels and insulin sensitivity.

Disease-modifying treatment for epilepsy

Researchers at RCSI's Department of Physiology & Medical Physics discovered that levels of microRNA-134 are abnormally high in human epilepsy and silencing microRNA-134 expression in vivo using a microRNA-134 antagomir (‘AntmiR134’) rendered mice refractory to both seizures and hippocampal injury caused by status epilepticus (Nature Medicine 18, 1087 to 1094, 2012). Subsequent research by the RCSI research group has shown the antagomirs work in other seizure models and, most recently, has shown that a single systemic injection of AntmiR134 (30 mg/kg) resulted in: (a) a significant reduction in the total number of epileptic seizures; and (b) the total time spent in seizures.

ADAM22 and LGI1 mimetic peptide: A complete solution as a directed therapeutic strategy for endocrine related tumour metastasis

ADAM22 is a novel therapeutic target for metastatic disease, with a peptide drug based on its natural ligand, LGI1. The technology compromises of a clinically validated and commercially ready immunohistochemical assay for the detection of ADAM22 in breast cancer tissue to be utilised as (i) as a biomarker for predicting the metastatic potential of breast tumours in endocrine treated breast cancer patients and (ii) as a companion diagnostic for a novel therapeutic (LGI1) to treat metastatic ER positive breast cancer.

Ground-breaking discovery into the causes of amyotrophic lateral sclerosis (ALS). Opportunity for the development of new therapeutic strategies for neurodegenerative diseases

Amyotrophic lateral sclerosis (ALS) is the most common neurodegenerative disorder of young and middle aged adults that is incurable and invariably fatal. Novel disease- specific mutations in a particular gene have recently been identified in ALS patients. This gene has not previously been associated with ALS, thereby opening up a whole new avenue of possible treatments to slow down or even reverse the process of cell degeneration in this condition and potentially other neurodegenerative disorders. Our novel technology is a therapy of pre-formulation sterile, recombinant preparations of human angiogenin protein.

Single-step encapsulation technology for drug formulation

MicroPET is a new high yielding method of producing microcapsules for application in the controlled release, sustained release and/or targeted delivery of pharmacologic agents including biologicals. The process, based on spray drying, uses a specifically designed concentric nozzle system so that particles emerge ready-formed from a single-step, scalable process. The resulting microcapsules can be further processed into other dose forms.

InnovoSep – A novel method of treating or preventing sepsis

Sepsis is a major challenge in the intensive care unit, where it is one of the leading causes of death. It arises unpredictability and can progress rapidly. Staphylococcus aureus and Escherichia coli are among the most common microorganisms isolated from sepsis patients. The Cardiovascular Infection Research Group in RCSI has demonstrated that cilengitide prevents both S. aureus and E. coli from binding to the endothelium thus inhibiting early signal generation that results in endothelial cell dysfunction in sepsis.