The biopharmaceutical industry is one of the most research-intensive of all sectors in the U.S. economy and supports a broad range of STEM jobs through R&D and manufacturing. At the backbone of the biopharmaceutical ecosystem are its researchers. Each day, in labs across the country, these individuals work tirelessly on the front lines of research and development to help bring novel medicines to patients. This challenge is far from easy. More often than not, the process of researching and developing innovative treatments is fraught with more setbacks than successes. Despite this, hearing first-hand testimonies from researchers on the successes and future of innovative medicines delivers a sense of hope for patients around the globe.
In a new Axios series that spotlights medicines in development, researchers from Bristol Myers Squibb (BMS) discuss the application of gene-modified cell therapies in treating blood cancers, including lymphoma and multiple myeloma. During the interview, Teri Foy, Ph.D. and Justine Dell’Aringa — who each lead a group of Seattle-based researchers — shared their optimism that gene-modified cell therapies like CAR-T cells will help address this area of significant unmet medical need. These treatments employ the latest in scientific advances and may transform current treatment standards for the benefit of patients for years to come.
Here are some key takeaways:
- Gene-modified cell therapies use a unique approach to treating blood cancers. When a patient receives a gene-modified cell therapy, their T-cells are temporarily removed from the blood stream and engineered in a way that enables the immune system to identify and target cancerous cells. By targeting cancer at the source, these treatments have the potential to treat previously untreatable forms of cancers.
- Continuing advances in manufacturing capabilities and greater understanding of genomics and disease mechanisms mean the future of cell and gene therapies is promising for a wide range of blood and other cancers. Currently, gene-modified cell therapies are used to treat blood cancers such as diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL), multiple myeloma (MM) and acute lymphoblastic leukemia (ALL). The next challenge will be finding ways to make these treatments more effective against solid tumors. Furthermore, researchers are looking at “off-the-shelf" approaches that use cells from a healthy donor instead of the patient. This approach will streamline the administration process and allow these therapies to reach more patients across the globe.
- Gene-modified cell therapies that use a one-time infusion to treat blood cancers can spare patients from the tribulations that often come with current standards of care. The one-time administration approach of gene-modified cell therapies differentiates them from other treatment regimens that often require repeated cycles of chemotherapies or medications taken throughout a patient’s lifetime. Eliminating these burdens positions gene-modified cell therapies to have the potential to significantly improve a patient’s quality of life.
Looking ahead, there are over 500 medicines in development for disorders of the blood, including blood cancers. This pipeline underscores our industry’s commitment to building on learnings from fundamental biology and overcoming the challenge of translating these findings into medicines that address the unmet needs of patients. Despite significant advances in the understanding of cell and gene therapies, a policy environment that fosters biopharmaceutical innovation is still needed to ensure patients have access to these novel treatments.
Learn more about medicines in development here.
Matthew Norawong
Matthew is a senior manager on PhRMA’s Public Affairs team focusing on Medicare, Medicaid, importation and more. Prior to coming to PhRMA, he previously worked as a health policy analyst for National Journal’s Network Science Initiative, primarily supporting companies in the life sciences industry. Matthew completed his undergraduate studies in political science at the University of Washington and subsequently earned a Master of Public Health from the George Washington University. A native of the Pacific Northwest, Matthew currently resides in Washington, DC. Outside of the office, Matthew is an avid runner and enjoys playing bass guitar with his indie rock band.
Matthew Norawong
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Stories from the lab: Researchers discuss innovative treatments for cancers of the blood
Cancer
Cell and Gene Therapy
Medicines in Development
PhRMA Member Company
Research and Development
Stories from the lab: Researchers discuss innovative treatments for cancers of the blood
Stories from the lab: Researchers discuss innovative treatments for cancers of the blood