VCU Massey Comprehensive Cancer Center is the first site in the United States to be activated in a global Phase 2a clinical trial evaluating PTX-100 (AKA GGTI-2418), a first-in-class small molecule inhibitor of geranylgeranyl transferase-1 (GGT-1), for the treatment of cutaneous T-cell lymphoma (CTCL), a rare and often treatment-resistant T-cell lymphome.
According to Sebti, PH.D., deputy director of fundamental research and family of the Lacy family in Cancer Research in Massey and professor in the Department of Pharmacology and Toxicology at the VCU School of Medicine, is the co-inventor of PTX-100 and the scientific founder at Prescient Therapeutics, which sponsors the essay.
“This test reflects Massey’s long -standing commitment to translate fundamental scientific discoveries into innovative treatments that can directly benefit patients. »»
Said Sebti, PHD, professor, department of pharmacology and toxicology, school of medicine, Virginia Commonwealth University
Thinking of the PTX-100 course, Sebti pointed out: “As a cancer researcher, there is no greater accomplishment than to see therapy that you co-invented in the laboratory begins to reach those who need it most. With the launch of this global PTX-100 test in the CTCL, where new treatments are necessary urgent, that the long-term dream feels deeply real.
Seeing my academic house, Massey, opening the way as the first American site to open the inscription for this essay, is one of the most exhilarating and humiliating moments of my career, and is a powerful reminder of what science can achieve when it is motivated by the goal and passion.
The newly launched global phase 2A trial will recruit CTCL patients in the main university cancer centers in the United States, Australia and Europe, in order to further assess the efficiency and safety of the PTX-100 in patients with a relapse or refractory disease.
The PTX-100 would currently be the only GGT-1 inhibitor in clinical development worldwide, representing a new class of targeted therapies. After demonstrating the safety and the first signals of clinical activity in a first phase 1 study of the first human first, the PTX-100 progressed in a cohort of expansion of phase 1B focused on patients with T cell lymphomas, where he showed signs of encouraging efficiency and a favorable safety profile.
Based on these results, the PTX-100 received the designation of orphaned drugs from the United States Food and Drug Administration for all T cells, as well as the accelerated designation for the treatment of re relapse or refractory fungoids, the most common form of CTCL.
CTCL is a rare type of non -hodgkinian lymphoma where immune cells called T cells become cancerous and mainly affect the skin, causing itching, red and squamous that may resemble eczema or psoriasis.
The GGT-1 enzyme plays an essential role in the biology of cancer, because it is essential for the activation of RHO, RAC and RAL proteins, the molecular switches which ensure the mediation of cancer of cancer of the oncogenic ras, one of the most commonly transferred oncogens in human cancers.
Sebti and his colleagues were the pioneers of the development of GGT-1 inhibitors designed to block these effective tracks down downstream, thus inducing programmed cell death and interrupting tumor growth in a variety of cancer models.
“This global clinical trial of phase 2A of PTX-100 will help provide an extension of care to our patients diagnosed with this rare cancer, for which new therapies are urgently necessary,” said Robert A. Winn, MD, director and president of Lipman in oncology in Massey. “We are incredibly proud of Dr Sebti and his team to fill the gap between world -class scientific innovation and the communities we serve, ultimately making significant progress in the fight against cancer. »»