Several people have asked for more specifics about the trial.  So, I’ll try my best to explain it…

JJ started the MK-1775 clinical trial at Memorial Sloan Kettering 2 weeks ago (9/15/14).  It is a phase I study of MK-1775, an experimental medication taken as a pill, for patients with newly diagnosed glioblastoma.

A phase I trial usually means that the treatment is either being tested for the first time in humans or it is a new drug combination that has never been tested before.  MK-1775 has been tested with other types of cancers, but this is the first time it is being combined with Temodar (a chemotherapy taken by pill) to treat glioblastoma.  In a phase I trial, they basically want to determine the highest dosage that can be safely prescribed. They do this by testing a few patients at a lower dosage of the drug.  They close the trial for a few months to monitor how the patients are doing, and they measure the frequency and severity of side effects.  If that dosage seems to be well-tolerated, they then open the trial to a few more patients and close it again for safety monitoring.  Eventually, they open the trial to a few more patients to try a higher dosage and continue safety monitoring.

JJ is in one of the later arms of the study.  They have already tested the frequency and severity of side effects associated with administering the MK-1775 for 3 days out of the 28 day cycle.  Because that dosage appeared to be well-tolerated by patients, they opened up 3 spots for the 5 day treatment.  JJ was fortunate to secure one of the 3 slots opened nationally.  We are hopeful that because JJ is in a later arm of the study, he is getting a dosage high enough to be effective. As part of the study,  MK-1775 pills are given concurrently with Temodar for 5 days and then patients have 23 days off both medications to allow their bodies to recover.  After that 28 day cycle is over, a new cycle starts again with 5 days of both medications and 23 days off.  The 28 day medication schedule will continue for 6 cycles total, if JJ is able to tolerate the medications well.

To take part in the study, JJ agreed to spend several days at Sloan for observation and monitoring.  During the first week of the trial, JJ spent 8 hours at Sloan on Monday and then another 8 hours on Thursday.  During the day-long observations, the nurses took his blood 6 times throughout the day to measure how his body was absorbing the drug.  The rest of the week he only had one blood sample taken each day.  By the end of the week, they had trouble finding any good veins left from which to draw blood.  JJ now has a nasty bruise on his right arm where they had trouble accessing his veins.

Right now, JJ’s side effects have included nausea, fatigue, shortness of breathe, muscle pains and nose bleeds.  The severity peaked towards the end of the first week of treatment and have very gradually started to improve the further away he gets from the five days of treatment. The doctors are monitoring JJ’s blood counts weekly, and he has to be careful around people who are sick because he is now considered immuno-compromised.  If you see him wearing a surgical mask out in public, it’s because he’s trying to be careful not to get sick as it is harder for his body to fight infections right now.

JJ will follow-up with the study team at Sloan for the remaining 5 cycles of MK-1775 study.  After that, his response to the clinical trial drug will continue to be monitored at his 8 week MRI/neuro-oncologist visits.  To be a part of the trial, JJ also agreed to have follow-up visits every six months (following the first year of more frequent monitoring) for the remainder of his life.  However, he has the option of opting out of the trial at any time.  If there are benefits to using MK-1775 without significant side effects, they’ll likely start a phase II trial with a larger number of patients who are randomly assigned to a treatment and a control group.  They are currently conducting a phase II trial with MK-1775 for patients with ovarian cancer.