Building on decades of work, these scientists have used cutting-edge technology to quickly and efficiently insert beneficial genes at exact locations within human immune cells. Previously, this process was time-consuming, expensive and imprecise, introducing toxic viruses that could damage the cells.
“This new technology creates an opportunity to speed development and testing of therapeutic strategies in lupus by offering researchers new ways to modify particular genes that contribute to the disease,” comments Gerald Nepom, MD, PhD, Co-chair of the Lupus Research Alliance Scientific Advisory Board and Director of the Immune Tolerance Network (ITN), sponsored by the National Institute of Allergy and Infectious Diseases.
“The remarkable study describes improvements in genetic engineering that allow the modification of multiple genes simultaneously in specific immune system cells,” said Ward Wakeland, PhD, Edwin L. Cox Distinguished Chair in Immunology and Genetics at UT Southwestern Medical Center. “As a result, proposing to change several defective genes simultaneously in a single therapeutic treatment is now feasible. By coupling this technology with a detailed understanding of the specific genetic variations that cause lupus in individual patients, it should be technically possible to design patient-specific therapies that suppress autoimmune disease without completely impairing the immune system.”
Richard DeScherer, Co-chair of the Lupus Research Alliance Board of Directors expressed the organization’s excitement at these new findings. “This is exactly the level of modern immunology and powerful new technology that we seek. This is the revolutionary science that can address the complexities of the disease and advance understanding of lupus at the molecular level, to carve out the path to the most effective new treatments and transform the lives of people with lupus.”
Lupus is a chronic, complex autoimmune disease that affects millions of people worldwide. More than 90% of people with lupus are women; lupus most often strikes during the childbearing years of 15-45. African Americans, Latin Americans, Asians and Native Americans are two to three times at greater risk than Caucasians. In lupus, the immune system, which is designed to protect against infection, creates antibodies that can attack any part of the body including the kidneys, brain, heart, lungs, blood, skin, and joints.
About the Lupus Research Alliance
The Lupus Research Alliance aims to transform treatment while advancing toward a cure by funding the most innovative lupus research in the world. The organization’s stringent peer review grant process fosters diverse scientific talent who are driving discovery toward better diagnostics, improved treatments and ultimately a cure for lupus. Because the Lupus Research Alliance’s Board of Directors fund all administrative and fundraising costs, 100% of all donations goes to support lupus research programs.
With limited treatment options currently available for patients diagnosed with systemic lupus erythematosus (SLE), known more commonly as lupus — a chronic autoimmune disease1 — a potential new medicine could be a game-changer for patients suffering from the frustrating and debilitating symptoms of lupus.
The immune system of a patient with a chronic immune disease cannot distinguish between healthy tissue and foreign invaders and produces antibodies to attack the body’s own tissue instead of fighting infection, leading to systemic inflammation in tissues and organs across the body.1,2 As a result, lupus can affect the skin, joints, kidneys, brain, and other organs, resulting in a wide variety of signs and symptoms.1,2 SLE accounts for approximately 70% of all cases of lupus,3 which can be difficult to diagnose as its symptoms mimic many other diseases and can change over the course of time.4
There is currently no cure for lupus1 and treatments that are approved currently aim to control symptoms. In fact, only one drug for SLE has been approved by the FDA in the last 60 years.5 RemeGen, Ltd., a leading biopharmaceutical company in China, recently announced positive data from a Phase 2b study for the treatment of lupus, pointing to a new wave of hope for those struggling with the disease.
RemeGen’s investigational candidate RC18 (telitacicept) is a novel recombinant TACI-Fc (transmembrane activator and calcium modulator and cyclophilin ligand interactor) fusion protein that has the potential to address significant unmet medical needs in the treatment of autoimmune diseases.
RC18 (telitacicept) is a dual-targeting fusion antibody that works by binding to two cell-signaling molecules, B lymphocyte stimulator (BLyS), and a proliferation-inducing ligand (APRIL). By only affecting mature B cells, RC18 has minimal impact on early and memory B cells, which are important for normal body immune function.6
Recent data presented at the American College of Rheumatology’s Annual Meeting unveiled positive results confirming the Phase 2b clinical trial met its primary endpoint of a greater than 4-point reduction in the SLE Responder Index (SRI4). At a dosage of RC18 240 mg, 75.8% of patients achieved clinically meaningful disease activity improvement (p<0.001), as compared to placebo (33.9%).6
“These data show the promise of RC18 to precisely target lupus with its novel dual-target mechanism and become a first-in-class and best-in-class treatment,” said Jianmin Fang, Ph.D., founder and CEO of RemeGen, Ltd. “We are excited about the potential this medicine has to effectively treat symptoms of lupus — especially in those patients who are currently struggling to manage them.”
The SLE treatment landscape is desolate — clinical trials focusing on the condition have faced many difficulties and the need for an effective treatment option is paramount. RemeGen looks forward to continuing research in the space to bring a new efficient treatment alternative for people living with SLE. The company will be working with regulatory authorities around the world to start Phase 3 trials in 2020 in an effort to provide treatments to patients as soon as possible.
So these are the latest technological advancements for lupus (λυκος) treatment.