by Gus Iversen
, Editor in Chief | July 24, 2023
Cell and gene therapies are being developed at record pace, but getting them to market and into patients has proven challenging.
The development of these so-called “living drugs”, which work on a cellular and genetic level, not only hold promise in treating diseases like cancer, rheumatoid arthritis and heart disease, but could potentially address genetic diseases people are born with that currently have no cure.
HealthCare Business News spoke to Fran Gregory, vice president of Emerging Therapies at Cardinal Health, to better understand the potential of gene and cell therapies, and also to talk about the hurdles limiting access to these treatments.
HCB News: How do you describe gene and cell therapies to people who are not familiar with them?
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Cell and gene therapies are personalized medicines that are designed to be specific to an individual patient. Cell and gene therapies are often grouped together when we talk about innovative therapies that are truly cutting edge. However, these product categories are quite different.
Cell therapies are personalized treatments that use human cells (either from the patient directly or from donors), and those cells are then modified in some way outside of the patient. These modified cells are then infused back into the patient, and the new cells are now therapeutic cells which are designed to attack specific targets or diseases. Examples of conditions treated by cell therapies today include multiple myeloma, leukemia and thalassemia, among others.
Gene therapies are also created using biologic material, but they directly impact a gene that is causing a condition or disease. Gene therapies can modify a targeted gene by correcting, eliminating or replacing a gene that is causing a disease. This can be done in a variety of ways, but many gene therapies use vectors, or “vehicles” to get this new genetic material to the existing gene. Gene therapies are already on the market today, they treat oncologic conditions and many other complex and rare diseases such as hemophilia, spinal muscular atrophy and Duchenne muscular dystrophy.
Cell and gene therapies can also be combined into a single therapy.
HCB News: What are some real-world applications for gene and cell therapies today?
These medications are often very impactful and are even sometimes referred to as curative. Early CAR-T cell therapies saved the lives of many children with late-stage pediatric acute lymphocytic leukemia (ALL). More recently, a gene therapy to treat hemophilia was approved, which is hugely impactful for patients living with that disease. These patients have a high risk of complications from their disease and they require constant treatment with incredibly expensive medication. Ultimately, these new gene therapies take a significant burden away from patients and drastically improve patient outcomes.