How a clinical trial gave me my life back after MDS and AML

Five years ago, my spouse and I had settled into our dreamed-of retirement. Although our 28 years as professors at the University of Arizona were rich and rewarding, retirement was much better.

But on Feb. 6, 2012, I was diagnosed with myelodysplastic syndrome (MDS), a rare blood cancer.

Because I was in my early 70s, a bone marrow transplant wasn’t my best option. Instead, I received chemo infusions for seven days every 28 days to improve my bone marrow and blood cell function.

But after nearly 3 1/2 years of this, I learned the chemo was no longer working.

A subsequent bone marrow biopsy demonstrated progression of my MDS, with the identification of an IDH1 mutation.    

I sought a second opinion and got a grim prognosis. The oncologist gave me only five to seven months to live. He said I needed to find a clinical trial soon.

Choosing a clinical trial at MD Anderson

During my search, I learned about a Phase II clinical trial at MD Anderson using an experimental drug called AG120. About a week after I applied, Courtney DiNardo, M.D., asked me to travel from my home in Tucson for testing.

Between MD Anderson’s huge campus and the battery of medical tests, our first visit was overwhelming. Yet, when Dr. DiNardo entered the room, she immediately made us feel like we were long-time patients or even friends. She was so cool, young and confident.

Only 24 hours after my spouse and I returned home, Dr. DiNardo called and asked us to return right away. We canceled our holiday plans, packed our motorcoach and arrived in Houston on Dec. 12, 2015.

At MD Anderson, we learned that my MDS had progressed to acute myeloid leukemia (AML). This was shocking, but I felt a strong sense of hope. We were right where we needed to be. People come to MD Anderson from all over the world, and I was grateful to be there with so many other AML patients.    

On December 23, I took my first pills for the clinical trial. Then came endless EKGs and every-other-day blood tests to check my blood cell counts.

My amazing AG120 results

Two weeks into the clinical trial, my white cell count was higher than it had been in two years. My spouse and I were amazed.

But the biggest surprise was my blast count. When I’d arrived at MD Anderson, it was at 30% — extremely high. At the end of the first 28-day cycle, it was just 2%, which is normal. 

Unlike chemo, which tries to kill the blasts and everything else in the bone marrow, AG120 blocks the mutant IDH1 protein that caused my AML. It allows the blasts to mature properly into normal white cells of the immune system. The bone marrow is no longer crowded out by AML, and the normal red cells and platelets return, too.

I’m now beginning my 12th cycle of AG120, and my blood values, red blood cells, white blood cells and platelets have all reached normal range. I’m in complete remission, but I will continue to take AG120 indefinitely. Whenever Dr. DiNardo’s team asks about side effects, I can’t come up with anything.

‘A small price to pay’

I am so grateful and praise God every day for giving me my life back through the AG120 clinical trial.  

I used to always say you have to be your own advocate because no one else will. But I was unable to take charge of my cancer until I met Dr. DiNardo. I’ll always remember what she said the day before I enrolled in the clinical trial: “You are in the right place at the right time with the right mutation.”

She was absolutely right. And the ongoing research that Dr. DiNardo is doing as part of MD Anderson’s MDS/AML Moon Shot is helping to ensure other patients will have this same opportunity.   

I still fly back to Houston every 28 days to receive my next cycle of pills. But when the FDA approves AG120, I’ll be able to fill my prescription at my local pharmacy. Until then, returning to Houston is a small price to pay for getting my life back.