The Latest on CAR T-Cell Therapy

What is the current state of CAR T-cell therapy at the CU Cancer Center?

Cell therapies in general, and CAR T cells more specifically, are clearly a tremendous growth area in the world of blood disorders and blood cancers. There's a lot of excitement and enthusiasm about them, and here at the CU Cancer Center, we are leading the way. There are now several of them that are FDA-approved, and we have all of those available to our patient population.

We are leading clinical research trials of CAR T drugs sponsored by various pharmaceutical companies, and we have put the effort into developing the in-house capacity on campus to develop these drugs. We are very excited to have two clinical trials opened, including a first-in-human study of a novel CAR, using CARs that we manufactured on campus. We are also very excited that a $200 million philanthropic commitment has just established the Gates Institute at CU, which will give scientists across the CU Anschutz campus a place to develop cell and gene therapeutics more effectively.

Is CAR T a new treatment option for blood cancers that wasn't there before, or is it more about a treatment with fewer side effects and things like that?

Generally speaking, CAR T-cell therapies are for patients who are going to die of their cancer if we don't do it. Many of the cancers that we use these CAR T cells for have other treatments, and the treatments are effective for the majority of people. But for those patients in whom the available treatments aren't successful, this is an incredibly exciting, novel strategy that results in profoundly improved outcomes as compared to the historical alternatives. Having established that, we now are trying to see if we give it earlier in therapy, will it do better? That might become the new way of doing things.

The standard therapies for these blood cancers are also very toxic. And while the CAR T therapies have a definite and distinct toxicity profile, primarily related to immune reactions that occur very early after the infusion of the cells, those reactions are generally very manageable compared to the side effects from more standard therapies.

You mentioned the FDA-approved CAR products that CU Cancer Center researchers helped to pioneer. Are those now available to cancer patients at any institution?

They are FDA-approved, but CAR T-cell therapy is an extremely complicated thing, even with an FDA-approved agent. Only very specialized centers can do it, under any circumstances. We are at the forefront with our participation, even in the approved agents. This is not a therapy where you can just go to your doctor down the street and get it. You can only get it at centers that have the resources to do it. You have to collect cells from a patient, then you have to be able to send those out to the company that manufactures the product; you have to have a stem cell lab or cell therapy lab that can receive the product back; you have to be able to store the product; and you have to give the patient the therapy. The toxicity profile that these drugs have requires the capacity for 24-7 monitoring with specialized training and understanding of the potential toxicity profile. It's not easy stuff to do.

What does it mean to people who come to the CU Cancer Center with these specific blood cancers, to now have access to these kinds of trials?

I think it's very exciting for patients. Clinical trials are very important, and we're grateful to our patients who participate in them. These trials are addressing an unmet need in the patient population. These are patients whose diseases are very bad, and for whom we don't have great alternatives. Those are the kinds of patients we deal with all the time; it's just we have better options for them now. Many community physicians are well aware of this option, so they refer patients to us as well.

What’s the future for CAR T-cell therapy?

I think this is the tip of the spear of this general set of therapies that is likely going to be extended into all kinds of other places, including solid tumors. I am medical director of the cell therapy program at UCHealth University of Colorado Hospital, and I'm getting inquiries with increasing frequency from rheumatologists asking about using these for autoimmune diseases, or neurologists and pulmonologists looking to use CAR T therapy in their specialties. This is a modality of therapy that has lots and lots of applications beyond just blood cancers. And the success we've seen in blood cancers is paving the way for consideration in a lot of other places.