CANCER BREAKTHROUGH: First Ever Personalized Cellular Therapy Developed at Penn
Robert Vonderheide, MD, director of the Abramson Cancer Center, and Carl June, MD, director of the Center for Cellular Immunotherapies
An FDA advisory committee recently unanimously approved a personalized cellular therapy for pediatric and young adult leukemia – developed right here at Penn Medicine's Abramson Cancer Center.
The vote is the last step before the therapy is formally approved for sale by Novartis, our collaborator in the research and development of this and other similar therapies. It will be the first gene therapy ever to reach the market.
Read about this major milestone in medicine in the New York Times.
Finding Tomorrow's Cure Today
The Abramson Cancer Center at Penn Medicine is leading the world in cancer immunotherapy research, including developing cancer vaccines, immune modulatory drugs and cell-based therapies such as CAR-T.
As pioneers in immunotherapy, we've forged the way with key advances, and our discoveries continue to drive progress. Every day, Penn’s award-winning doctors and scientists are hard at work, coming up with new ways to use the immune system to fight cancer.
This short documentary firm details the story of the first pediatric patient to receive Penn Medicine’s personalized cellular therapy:
What is immunotherapy?
The immune system is one of the most powerful forces in the human body. When it's healthy, it protects us from disease and fights infection. Yet, this protection is imperfect. Disease reaches us. In the case of cancer, it overpowers the immune system and allows unhealthy cells to grow and expand.
Imagine a drug or therapy that could treat disease by helping the immune system do its job better. That is the idea behind immunotherapy – treatments designed to boost the immune system.
Learn about our personalized cellular therapy:
How does immunotherapy work?
The immune system relies on a powerful army of “T cells.” They on are on the front line of our body’s defenses. Their mission is to search and destroy anything that causes illness or infection.
With cancer, the immune system often fails to deploy T cells right away or at all. When it does, the attack is ineffective.
This is where immunotherapy comes in. This new class of drugs works either by empowering the T cell army so that it is bigger, stronger or smarter, or by taking down the obstacles in its path.
Immunotherapy Research at Penn
From left to right: David Porter, MD, Bruce Levine, PhD, Carl June, MD, Stephan Grupp, MD, PhD, and Michael Kalos, PhD
The Abramson Cancer Center's dynamic research program is always growing. Major areas of focus include CAR T therapy, vaccine therapy and checkpoint inhibitors.
Chimeric Antigen Receptor T Cell Therapy (CAR T Therapy)
CAR T therapies are a form of personalized cellular therapies that aim to transform your T cells into an elite force better able to fight cancer. A growing number of clinical trials are in place to test this groundbreaking approach.
This treatment has three key steps:
- Remove some of a patient’s white blood cells.
- Send the blood cells to a special Penn facility for re-engineering. The goal is to improve their ability to target cancer in a more specific and aggressive way.
- Return the blood cells to the patient’s body to go to work.
In 2011, Penn's CAR T cell therapy research first made headlines. Study results showed success in treating an aggressive form of leukemia.
Since then, CAR T research has grown considerably. Studies focus on both blood and solid tumor cancers. This research is a high priority for the coming years. In 2016, Penn opened the Novartis-Penn Center for Advanced Cellular Therapeutics to support its growth.
Vaccine Therapy
The miracle of vaccines: The best ones are unstoppable.
What if vaccine technology could be used to cure cancer – or stop it from happening in the first place? Penn’s pioneering cancer vaccines aim to do just that.
This approach works on the same general principle as other forms of immunotherapy, boosting the body’s natural immunity to fight cancer.
DNA vaccines lead the way. They show great promise in treating cancers that have responded to standard therapies but are at high risk of relapse. Current studies focus on pancreatic, lung, head and neck, and recurrent breast cancers.
Learn more about vaccine therapy
Checkpoint Inhibitors
When the body struggles against cancer, even the most vital T cells can face trouble – resistance that blocks their attack. This resistance takes the form of molecules on the surface of cancer cells. These molecules, called immune checkpoints, act like a brake, stopping the T cell attack.
Checkpoint inhibitors are drugs designed to disable the checkpoint and let the immune system do its job. The drugs have been used to treat melanoma and lung cancer, and are being tested in other cancers, including ovarian cancer.
What the Future Holds
Cancer is too complex for any one doctor or lab to solve alone. In 2016, Penn was one of six top medical institutions chosen to form the new Parker Institute for Cancer Immunotherapy.
This national team of top cancer researchers regularly shares ideas and findings. By working together, we aim to speed up the pace of science.