VCU Massey Comprehensive Cancer Center is nan first tract successful nan United States to beryllium activated successful a world Phase 2a objective proceedings evaluating PTX-100 (AKA GGTI-2418), a first-in-class mini molecule inhibitor of geranylgeranyl transferase-1 (GGT-1), for nan curen of cutaneous T-cell lymphoma (CTCL), a uncommon and often treatment-resistant T-cell lymphoma.
Said Sebti, Ph.D., subordinate head for basal investigation and Lacy Family Chair successful Cancer Research astatine Massey and professor successful nan Department of Pharmacology and Toxicology astatine nan VCU School of Medicine, is nan co-inventor of PTX-100 and nan technological laminitis astatine Prescient Therapeutics, which is sponsoring nan trial.
"This proceedings reflects Massey's long-standing committedness to translating basal subject discoveries into innovative treatments that tin straight use patients."
Said Sebti, PhD, Professor, Department of Pharmacology and Toxicology, School of Medicine, Virginia Commonwealth University
Thinking astir nan travel of PTX-100, Sebti remarked, "As a crab researcher, location is nary greater fulfillment than seeing a therapy you co-invented successful nan laboratory statesman to scope those who request it most. With nan motorboat of this world Phase 2 proceedings of PTX-100 successful CTCL, wherever caller treatments are urgently needed, that long-held dream feels profoundly real.
To spot my world home, Massey, lead nan measurement arsenic nan first U.S. tract to unfastened enrollment to this trial, is 1 of nan astir exhilarating and humbling moments of my career, and is simply a powerful reminder of what subject tin execute erstwhile driven by intent and passion.
The recently launched shape 2a world proceedings will enroll patients pinch CTCL crossed starring world crab centers successful nan United States, Australia and Europe, pinch nan purpose of further evaluating nan efficacy and information of PTX-100 successful patients pinch relapsed aliases refractory disease.
PTX-100 is presently believed to beryllium nan only GGT-1 inhibitor successful objective improvement globally, representing a caller people of targeted therapies. After demonstrating information and early signals of objective activity successful a first-in-human Phase 1 study, PTX-100 precocious into a Phase 1b description cohort focused connected patients pinch T-cell lymphomas, wherever it showed encouraging signs of efficacy and a favorable information profile.
Based connected these results, PTX-100 received orphan supplier nickname from nan United States Food and Drug Administration for each T-cell lymphomas, arsenic good arsenic fast-track nickname for nan curen of relapsed aliases refractory mycosis fungoides, nan astir communal shape of CTCL.
CTCL is simply a uncommon type of non-Hodgkin lymphoma wherever immune cells called T cells go cancerous and chiefly impact nan skin, causing itchy, red, scaly patches that whitethorn lucifer eczema aliases psoriasis.
The enzyme GGT-1 plays a captious domiciled successful crab biology, arsenic it is basal for nan activation of Rho, Rac, and Ral proteins, molecular switches that mediate nan cancer-causing activity of nan oncogene RAS, 1 of nan astir commonly mutated oncogenes successful quality cancers.
Sebti and his colleagues person pioneered nan improvement of GGT-1 inhibitors designed to artifact these downstream RAS effector pathways, thereby inducing programmed compartment decease and halting tumor maturation successful a assortment of crab models.
"This world Phase 2a objective proceedings of PTX-100 will thief supply an hold of attraction for our patients diagnosed pinch this uncommon cancer, for which caller therapies are urgently needed," Robert A. Winn, M.D., head and Lipman Chair successful Oncology astatine Massey, said. "We are incredibly proud of Dr. Sebti and his squad for bridging nan spread betwixt world-class technological invention and nan communities we serve, yet making important strides successful nan conflict against cancer."