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. But on Feb. 6, 2012, I was diagnosed with myelodysplastic syndrome (MDS). Because I was in my early 70s, a bone marrow transplant wasn’t my best option.

Shirley O’Brien

What happened when the chemo stopped working

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.

CAR T cell therapy is currently being evaluated in the clinic at MSK for certain types of leukemia and lymphoma. In this approach, T cells are genetically engineered to recognize a protein called CD19, which is found on the surface of blood cells called B cells. In the largest study reported so far, for adult patients with B cell acute lymphoblastic leukemia — a rapidly progressing form of blood cancer — a report published by MSK researchers last year found that 88 percent of patients responded to the therapy. In late 2014, the US Food and Drug Administration granted MSK Breakthrough Therapy Designation for its CD19 CAR therapy.

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.

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.”

Take a look at current MDS clinical trials

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