Entries in First in Human (1)

Tuesday
Aug222017

First-in-Human: the moment research turns to medicine

 If you have not been watching the recent three-part series on the Discovery channel entitled “First-in-human,” I highly recommend checking it out. As a medical researcher who works in a translational lab, it is both refreshing and inspiring to get a glimpse into the moment where all of the basic research and development translates into a treatment for human disease. The documentary, which is targeted for a wide audience, provides rare insight into this critical first stage of drug development when a drug is first put into a human body, and includes the point of view of clinicians, researchers, and patients.

Three patients are introduced in the first episode who each have been left with little to no treatment options for their incurable diseases, two of which are types of cancer. Each patient has traveled to the hospital in building 10 at the NIH, which houses labs and clinical treatment facilities to support bench-to-bedside, first-in-human clinical trials. While these patients are quite sick, their remarkable courage is apparent as they listen to the doctor explain the risks and unknowns associated with taking part in a first-in-human study. The episode then offers an intimate portrait of how dynamic and emotional this first drug treatment can be for both the clinicians and the family members present, with the patient’s desire to maximize the potential for treatment success battling with the clinician’s oath to keep the patient out of unnecessary harm. This humanization of the scientific process of drug development was eye opening. As researchers, it is easy to hyper focus on the data and the statistics of drug success, so it is important to see the other side of the development process.

In addition to portraying the patient/clinician experience in a first-in-human trial, the documentary highlighted two novel immuno-oncology treatments, CAR-T (chimeric antigen receptor) therapy and TILS (tumor infiltrating lymphocytes). Both of these new technologies utilize the patient’s own immune cells to fight their cancer using a true bench-to-bedside technique. In CAR-T therapy, blood cells are harvested from a patient (in the documentary, the patient has relapsing leukemia), engineered and grown in the lab to recognize a target specifically expressed on leukemia cells, CD22, and then reintroduced into the patient’s body to attack the leukemia. Similarly, TIL therapy works by extracting a tumor from a patient’s body, slicing up the tumor and amplifying the endogenous immune cells already working to fight the tumor (lymphocytes), and reintroducing the immune cells back into the body to fight the cancer. Both of these therapeutic mechanisms amplify the patients’ own immune system responses to tackle the malignancy in their bodies. There is a unique aspect compared to small molecule treatments as these therapeutics require a concerted effort between the lab and the clinicians to develop a patient-specific treatment that will be successful.

It is no small feat to drive these types of projects forward in medicine, and it takes the researchers, clinicians, families, and patients to succeed.

 

MMR

Watch full episodes of First in Human here https://www.discovery.com/tv-shows/first-in-human/