Ashvattha Therapeutics Announces Presentation of Preclinical Data at the 2022 Neurofibromatosis (NF) Conference Demonstrating the Potential of Hydroxyl Dendrimer Therapeutics to Reduce Toxicity in Targeted Treatment of Plexiform Neurofibroma
REDWOOD CITY, Calif., June 21, 2022 (GLOBE NEWSWIRE) -- Ashvattha Therapeutics (“Ashvattha”), a clinical-stage company developing novel hydroxyl dendrimer therapeutics, today announced a poster presentation of preclinical data demonstrating the potential of hydroxyl dendrimer therapeutics (HDTs) to reduce toxicity in the targeted treatment of plexiform neurofibroma, a slow-growing tumor associated with neurofibromatosis type 1 (NF1), at the 2022 Neurofibromatosis (NF) Conference hosted by the Children’s Tumor Foundation and taking place at Loews Philadelphia Hotel in Philadelphia, PA, June 18–21, 2022.
The data was presented by Emma C. Mazurek, a scientific collaborator at Indiana University School of Medicine, in a poster titled, “Hydroxyl Dendrimer Therapeutics Reduce Toxicity in Targeted Delivery to Plexiform Neurofibroma.”
The study investigated the uptake of HDs varying in size. The data showed selective uptake of HDs only by tumor associated macrophages and microglia, but not adjacent neurons, in an NF1-associated PNF mouse model. Notably, the smaller HD (~14 kDa) showed greater uptake in tumor associated macrophages and microglia, than the larger HD (~56 kDa). In vivo evaluation of HD therapeutics is currently underway in an NF1 mouse model of PNF to assess efficacy as measured by a reduction in tumor burden. These results lay the groundwork for future HD therapies towards treatment of NF1-associated PNF.
“Systemic toxicity remains a challenge in treating PNF, limiting the use of pharmacologic agents. These results suggest that by conjugating drgs to HDs there’s potential to improve safety and reduce toxicity associated with current treatments for NF. HDs allow for the uptake of highly toxic drugs specifically in immune cells within the PNF microenvironment impairing tumor growth,” said Jeffrey Cleland, Ph.D., Chairman, CEO and President of Ashvattha Therapeutics. “Studies are currently underway to evaluate novel HDTs in this mouse model of disease to further demonstrate the potential of HDTs in NF1-associated PNF.”
Chronic activation of macrophages and microglia plays a critical role in the progression of many neurological diseases, including NF1. Due to the high inoperability of neurofibromas, pharmacological therapeutics are the main strategy in treatment. Current therapies cause systemic toxicity that is not tolerated by patients leading to discontinuation of treatment. For patients with NF1, there is a need for safer treatments to achieve greater efficacy and durability. Previous data suggests that the combined targeting of tumorigenic pathways and immunosuppressive cells within the PNF tumor microenvironment (TME) may be an effective strategy to overcome these challenges. HDs can be conjugated to more than one drug, enabling one HDT to affect multiple pathways.
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