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iwNHL 2018 | The challenging field of hem-onc therapeutics

The field of blood cancer therapeutics has seen enormous progression. Here, Steven Rosen, MD, from the City of Hope, Duarte, CA, gives an overview of the exciting trials and research being conducted in the field of hematological malignancies at the City of Hope. The center is home to facilities where CAR T-cell and stem cell trials are underway, and targeted small molecules are being produced and trialed, eliminating the need for support from pharmaceutical companies. At the International Workshop on Non-Hodgkin Lymphoma (iwNHL) 2018 meeting, held in Nice, France, Dr Rosen reflects upon the success of the studies that have taken place so far this year.

Transcript (edited for clarity)

The City of Hope is fortunate to have some of the leading figures in blood cancer research and we pride ourselves on being able to take discoveries all the way to patients, because on campus we have GMP facilities that allow us to make both small molecules, biologics and stem cells, without having to go through a pharmaceutical company.

So, at the present time, we have a number of CAR-T therapy trials in lymphoma, myeloma, leukemia… We have stem cell trials with the stem cells actually hone into malignancies, that we can load them with cytotoxic agents and then we actually have small molecules we produce on campus...

The City of Hope is fortunate to have some of the leading figures in blood cancer research and we pride ourselves on being able to take discoveries all the way to patients, because on campus we have GMP facilities that allow us to make both small molecules, biologics and stem cells, without having to go through a pharmaceutical company.

So, at the present time, we have a number of CAR-T therapy trials in lymphoma, myeloma, leukemia… We have stem cell trials with the stem cells actually hone into malignancies, that we can load them with cytotoxic agents and then we actually have small molecules we produce on campus. I myself have one that’s an RNA-directed nucleoside analog, where we’ve treated now more than a dozen patients. So, it’s a very interesting place. We’ve had 750 stem cell transplants this year, we’ve treated 250 patients with CAR-T and with the Beckman Research Institute and with now our affiliate TGen (Translational Genomics Research Institute). We’re really in the forefront of precision medicine.

There are so many unanswered questions related to blood cancer therapeutics. It’s been rewarding to see the advances and to see diseases where, historically there were limited options, where now we can reduce remissions with well-tolerated therapies: some immunologic, some small molecules affecting pathways. But still there isn’t, and maybe there will never be a gold standard – outside of if we could prevent the actual malignancies, which is a real challenge for a therapy to be incredibly well-tolerated, (meaning no toxicity) that effectively controls the disease, where the individual can live a normal life expectancy. We also have to deal with the reality of the costs of all of these treatments.

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