Frequently Asked Questions

With Matt Might, PhD, Director, Hugh Kaul Personalized Medicine Institute, University of Alabama at Birmingham

Q: How can cell- and gene-based medicines benefit me?

A: Cell and gene medicine are new kinds of therapies in which we’re using products from the body itself as medicines. When a lot of people hear “cell- and gene-based medicine,” they’re thinking, “Oh, this is for genetic disorders,” but it also applies to cancers and even has the potential for things like Alzheimer’s, Parkinson’s and osteoarthritis. With gene therapy, the medicine in this case is a gene. Your body already contains 20,000 or so genes, and in some cases, we need to put a gene back in to heal you. So within gene therapy, there’s a variety of things that we actually do, but what they really boil down to is introducing a gene, replacing a gene, or silencing a gene.


Q: Can we use cell- and gene-based medicine to heal?

A: In the case of cell therapy, you’ve already probably heard of several. (An example would be a bone marrow transplant.) Every one of us already has stem cells in our body in the form of bone marrow. Now, bone marrow isn’t the most flexible form of stem cell, but it is possible now to take your cells and turn them into induced pluripotent stem cells. Pluripotent stem cells have the ability to become any other cell type in the body, which means that they could potentially regenerate any damaged tissue you have.


Q: What is gene-modified cell therapy?

 With gene-modified cell therapy, we take your cells, usually from your blood. We modify them to give them some enhanced capacity, and then we put them back into you. So, for example, we could take out immune cells, modify them so that they’re better able to fight cancer, and put them back into you.


Q: What is gene editing?

A: With gene editing, we’re not introducing a new gene. We’re modifying an existing gene. If you think of DNA as a book that’s kind of like an instruction manual to build and operate you, sometimes that book has a typo. With gene editing, we can go in and correct that typo.


Q: How are cell- and gene-based medicines given to patients?

A: There are a variety of ways to deliver cell- and gene-based medicines. Some may be as simple as a shot or an injection. Some might take an IV. And what’s amazing is that we now have the capacity in some cases to deliver these directly to the brain.


Q: How frequently do you have to go back to get more treatments?

A: With most cell and gene-based therapies, you might need only one treatment. Because it’s fundamentally modifying your DNA, you only need to get it once.


Q: Could cell- and gene-medicine hurt healthy areas of my body?

A: Cell- and gene-based medicines won’t modify every cell in your body. They only modify the cells that matter for your particular disease.


Q: Do current cell- and gene-based medicines pass altered genes to future children?

A: No. We don’t modify your reproductive cells.


Q: How can I know this medicine is safe?

A: Cell and gene therapies, just like any other medication, have to go through a rigorous clinical trials process with the U.S. Food and Drug Administration. So just like any other medication, we have the same confidence in the safety and efficacy of these drugs. During the clinical trials process, physicians are closely monitoring both the safety and effectiveness of cell- and gene-based therapies. Some cell- and gene-based therapies are already available, and many more are in clinical trials.

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