Gene therapy focuses on preventing or curing genetic conditions by manipulating the genetic material in living cells. For people with certain medical conditions, gene therapy can be life-changing — and often life-saving. While most gene therapies are currently only available as part of clinical trials, the field holds promise for treating a number of serious conditions, ranging from cancer, heart disease, and diabetes to cystic fibrosis, sickle cell disease, and AIDS. Because it targets the root cause of certain conditions or diseases, one of the most notable benefits of gene therapy is that it offers a potential avenue to treat diseases previously considered incurable.
Progress in Gene Therapies
Only a few gene therapies have been fully approved by the U.S. Food and Drug Administration (FDA). Those that have been approved include treatments for a rare form of inherited vision loss, treatments for certain cancers, and a recent breakthrough treatment approved for Duchenne Muscular Dystrophy (DMD) in pediatric patients 4 to 5 years of age.
But the number of approved gene therapies is only expected to grow. More than 1,500 active clinical trials are currently in process worldwide, and some have already begun to demonstrate success in using gene therapy to treat diseases like severe combined immune deficiency, hemophilia, blindness caused by retinitis pigmentosa, and leukemia. With the rapid pace of technological progress and research breakthroughs, we’re only just beginning to understand the curative possibilities and benefits of gene therapy.
Exploring the Many Benefits of Gene Therapy
Targeted, Long-Lasting Treatment
Traditional pharmaceutical interventions often focus on short-term, symptomatic relief. Gene therapy, on the other hand, offers the promise of effective, long-lasting solutions by targeting the problem at the cellular level. Indeed, for some conditions, gene therapy offers the potential to eliminate a person’s symptoms for life. And in some cases, a lifetime of symptom relief can be accomplished in a single dose.
Treating Inherited Conditions
Gene therapy has the potential to treat a number of inherited genetic conditions. Researchers have enjoyed some clinical success using gene therapy to treat the following conditions:
- Severe Combined Immune Deficiency (SCID)
- Adenosine deaminase (ADA) deficiency
- Certain types of hereditary blindness
- Cystic Fibrosis
- Sickle Cell Disease
For some conditions, gene therapy benefits don’t just extend throughout a patient’s lifetime — the positive effects may be passed down through generations. Germline gene editing techniques affect all of a person’s cells, including egg and sperm cells. That means removing a faulty gene from an eventual parent won’t just treat the parent’s condition — it could also prevent them from passing that gene down to their children.
Hope for Cancer
Gene therapy may be an important key in the fight against cancer. One gene therapy approach that has had early success is immunotherapy, a technique that uses genetically modified cells and viral particles to trigger the immune system to destroy cancer cells. Immunotherapy has the potential to treat lung cancer, pancreatic cancer, prostate cancer, and malignant melanoma.
Emerging research points to two other promising approaches:
- Oncolytic virotherapy, which utilizes viruses to target and destroy cancer cells selectively
- Gene transfer, which introduces new genes into a cancerous cell (or the tissue surrounding it) to either slow the cancer’s growth or cause cell death
While these are emerging treatment modalities, early clinical data is promising. Oncolytic virotherapy appears to have notable potential to treat metastatic cancers. And researchers point to the flexibility of gene transfer techniques, which can be used to treat a range of genes and vectors, as one of the most promising benefits of that particular treatment method.
Fighting HIV, Parkinson’s, and More
The benefits of gene therapy extend beyond their potential to treat genetic disorders and cancer. Research suggests that gene therapy may eventually also help to treat conditions ranging from Parkinson’s disease to human immunodeficiency virus (HIV) and more.
The gene therapies available today are the direct result of the seismic technological advances that have occurred in the field over the past few decades. As technology continues to improve, so will gene therapy. We’re likely only at the beginning of understanding the therapeutic potential and life-saving benefits of gene therapy.
QPS is a GLP/GCP-compliant CRO delivering the highest grade of discovery, preclinical, and clinical drug development services. Since 1995, it has rapidly expanded from a bioanalysis shop to a full-service CRO with 1,200+ employees in the US, Europe, India, and Asia. Today, QPS offers expanded pharmaceutical contract R&D services with special expertise in neuropharmacology, DMPK, toxicology, bioanalysis, translational medicine, and all phases of clinical development. QPS has CLIA-certified and GLP-compliant laboratories ready to fast-track gene therapy, RT-qPCR/QPCR, serological assays, and vaccine development programs. An award-winning leader focused on bioanalysis and clinical trials, QPS is known for proven quality standards, technical expertise, a flexible approach to research, client satisfaction, and turnkey laboratories and facilities. For more information, visit www.qps.com or email [email protected].