Dr. Gerhard Bauer

My name is Gerhard Bauer. I am a professor of Hematology and Oncology here at the UC Davis Medical Center within the School of Medicine and also within the stem cell program. And I'm also the director and designer of the UC Davis Good Manufacturing Practice facility. And I was hired by the University of Maryland at Baltimore to run the HIV laboratory which I did, and there I was able to find a predictor for HIV transmission from mother to child, which at that time was a cell-based assay, which predicted the transmission rate.

And this was correlated with a viral load and the mom's later on, and we could demonstrate that higher viral load leads to more transmission. And I was asked to move over to the Johns Hopkins University, again in Baltimore, and there I started with the development of stem cell gene therapy for HIV.

We were able to demonstrate that we could make stem cells and subsequent HIV-infectable cells resistant to HIV by the insertion of anti-HIV genes, using retroviral vectors. And at Children's Hospital Los Angeles, I was fortunate enough to develop all of the clinical-grade procedures to transport genes into hematopoiatic stem progenitor cells.

That led to the development of a completely new treatment for ADA-deficient SCID, superior combined immunodeficiency, also called the Bubble Boy disease. And I was able to bring it to the regulators with my laboratory procedures that I developed for transactional B-cells, and that finally ended up in a cure of the Bubble Boy disease, which is called Strimvelis and is available in Europe for the cure of ADA-deficient SCID.

So I'm very proud of this development. A very interesting project that we have been tackling also is CAR T cells. Everybody talks about CAR T cells these days because this has turned from an experimental project into a commercialized application.

And I'm very grateful that I have participated in this as well. You may know that with cancer, we have a real problem. Chemotherapy, radiation, only kills the fast-growing tumor cells and then we have a relapse. With CAR T cells, we have developed such a weapon that allows the patient's own immune system to recognize the cancer and completely eliminate it and even sustained eliminate it because the T cells develop memory T cells that will be reactivated when the cancer comes back.

And we are manufacturing such CAR T cells in the laboratory currently, and we are initiating investigational drug applications, I and Ds with the FDA to apply these homegrown CAR T cells, as we call them, to patients in San Francisco and also here at UC Davis. What we do is we devise our strategies of gene modification around regular laboratory equipment that any laboratory has.

However, within a good manufacturing practice laboratory, we need equipment that can be calibrated, that can maintain the operational status in a very precise way, from beginning to end of the process. And we must be able to clean these pieces of equipment appropriately. You know, cells that grow in a laboratory don't have an immune system.

They are susceptible to anything. So we need to keep all of the contaminants away from these cells. We need to incubate our cells at body temperature which is 37 degrees Celsius and the particular atmospheric conditions like appropriate CO2 content and sometimes even low oxygen. So we need incubators that can do all of that.

And I have had a good experience with the incubators that we usually apply here in the laboratories and they had lasted over the last 10 years until we replaced them with newer incubators with a particular feature, and that's a self-sterilizing incubator which has helped us tremendously in our effort. We need to keep the time that we work on maintenance of the equipment low because our technicians are necessary for making the products and they should spend less time on the equipment.

And so these incubators that we have now have a self-sterilizing feature. We just turn that on. And we don't have to take the shelves out, we don't have to do an autoclaving step on the shelves. It has saved us so much time and effort. And our technicians are really grateful for that.

And I am too. And without these incubators, I don't think we would be able to efficiently produce as many products as we produce currently, because if we don't have reliable incubators, we can't make reliable products. And if we have to throw out a patient product, we could potentially put patients' lives at risk. So we have to have a product that is absolutely reliable, and this is what I really need to stress. You need to have reliable laboratory equipment that is usable within a good manufacturing practice environment.

Gene therapy is working, and it's in approved commercialized, clinical applications. I am very proud of that accomplishment because there was this gene therapy crisis in the year 2000 that some people may still remember and it was predicted that gene therapy is too dangerous. No, it's not the case. We were able to overcome these challenges with proper science and with proper engineering of our cellular products.

And we could demonstrate that gene therapy is very safe. Imagine the millions of people we have to provide with cell and gene therapy in the future. Well, in a few years down the road, we will be able to do that. Currently, we are working on it. so it's not there yet, but I'm very hopeful that in a few years down the road, we will have all of that.