Celebrating Clinical Trials Day - May 20 - Cell and Gene Therapy Advancements

In 1747, James Lind conducted what was considered the first randomized clinical trial – a feat recognized and celebrated in the clinical research community known as Clinical Trials Day. While May 20th each year is set aside to honor that moment, it also celebrates the new therapies helping patients around the world live healthier lives.

This year in particular, I’m celebrating the advancements made in cell and gene therapies (C&GT). These life-saving therapies use advanced biologics by adding or turning off genes in the patient’s own immune system. Gene therapy is designed to deliver personalized medicine at scale with the goal of producing one-time curative treatments for patients.

Years of research and development are starting to provide patients with C&GT options, making previously incurable diseases curable. I’m continually struck by this progress and the life-changing implications for patients. With that in mind, I’d like to highlight the potential of this fascinating field and how this area of research and development is growing at a fast pace.

Getting Cures to Market

The pharmaceutical industry rightly touts advancements in new treatments, and while treatments for improving disease symptoms are necessary and exciting by themselves, they oft don’t fix the root of the problem – cures do. Gene therapies don’t just provide patients with incremental improvement and disease management, instead, some of these therapies have the potential to significantly advance human health by preventing or curing diseases or condition. Within our lifetimes, I believe gene therapies are going to fundamentally change the way we think about treatments, and we are going to hopefully see the word “cure” used more often.

While there are obstacles to overcome, such as regulatory approval and funding challenges, C&GT's potential is hard to ignore. Consider the example of a child with a debilitating genetic abnormality such as Spinal Muscular Atrophy (SMA); five years ago, that diagnosis likely would have meant a fatal outcome because the treatment options on the market, such as small molecule drugs, cannot cure this abnormality. Today, there is an approved gene therapy treatment that addresses the underlying genetic abnormality behind SMA, which has allowed children to maintain and even gain motor milestones. This is just one example demonstrating the significant improvements C&GT can make on a patient’s life. 

Currently, a range of diseases have the potential to be treated with gene therapies, including cancer, diabetes, cystic fibrosis, heart disease, and AIDS. In 2023, we are anticipating regulatory decisions related to disease areas in C&GTs, including sickle cell disease, metastatic melanoma, RR Multiple Myeloma, Duchenne muscular dystrophy, hemophilia, and more. For patients, their caregivers, and loved ones, C&GT could make the difference between daily disease management and a cure.

Industry Growth and Advarra’s Role

The C&GT industry is continuing to grow, and the U.S. is leading the sector. According to a recent report, the U.S Food and Drug Administration ( FDA ) anticipates 10-20 new C&GT approvals per year by 2025, with up to 13 new approvals expected this year alone. However, because genetically engineered materials contain unique biosafety risks, there are additional precautions in place, such as institutional biosafety committee (#IBC) review. The The National Institutes of Health (NIH) guidelines are also in effect if a study receives NIH supports or takes place at research sites receiving NIH support.

In order to support the clinical trial process as research into gene therapy-based treatments accelerates, Advarra’s Gene Therapy Ready Site Network has sites ready to perform gene therapy and mRNA-based drug and vaccine trials, all of which are pre-registered, vetted, and standing by to rapidly start research. As one example, at the start of the pandemic, IQVIA (a provider of analytics, technology solutions and clinical research services to the life sciences industry) realized a traditional timeline would not be sufficient to move new COVID-19 vaccines through clinical trials at warp speed. In order to optimize efficiencies, IQVIA partnered with Advarra to provide integrated institutional review board (IRB) and IBC review service combined with the Gene Therapy Ready Site Network. In doing so, IQVIA reduced study startup timelines and delivered trial results to the sponsor quicker than expected.

It's important to note that bringing safe and effective treatments or cures to market takes a certain amount of time; however, delays ultimately affect patients. This is one of many reasons why Advarra invests in coordinating the facility inspections, registration, and more to help sponsors eliminate these steps and get studies up and running. As the industry continues to gain momentum, accelerating study startup can greatly affect timelines.  

Final Thoughts

Clinical research has the potential to change lives, and I can’t imagine a better way to celebrate Clinical Trials Day this year than by highlighting the incredible cell and gene therapy advancements we’ve seen in recent years. I’m proud of the active role Advarra plays to support our industry and academic partners as they pursue advanced genetic engineering to find cures for the world’s most pressing health conditions. This sector is already altering patients’ lives, and I’m hopeful future research and development will have the same impact for other disease areas with patients facing significant unmet needs.

What clinical research advancements will you be celebrating on May 20th or looking forward to in the future?