We have all read about people who smoke a pack of cigarettes and drink a bottle of whiskey a day and somehow manage to live a long, healthy life. Then there are people like Sandra Dillon. She lived as healthy a life as you can imagine; she exercised a lot, ate a healthy diet and didn’t smoke. Yet at the age of 28 she was diagnosed with a rare and deadly form of blood cancer called myelofibrosis.
Sandra underwent the traditional forms of treatment but those proved ineffective and time seemed to be running out. Then she heard about a clinical trial for a new, experimental stem cell therapy, with Dr. Catriona Jamieson at the University of California San Diego.
Sandra says she wasn’t looking forward to it, but she was in a lot of pain, was getting much sicker and none of the treatments she tried was working.
“At the time I was actually quite afraid of seeing doctors or going to medical institutions. My experience had been rough, and I knew that I had to overcome my fear of going to hospitals and being treated. But it was a chance to have hope and to be on something that might work when there was nothing else available.”
Dr. Jamieson’s approach (CIRM helped support her early work in this area) had led to her identifying how abnormal gene activity was responsible for the progression of this form of blood cancer. With that knowledge she then identified a specific small molecule known to inhibit this mutant gene activity, and how it could halt the disease.
That’s what happened with Sandra. She says after years of pain and exhaustion, of fearing that she was running out of time, the treatment produced impressive results.
“It was pretty amazing. I had really low expectations from how sick I was and that this was experimental, and it was cancer and you expect it to be awful. And my experience was the opposite of what I’d expected. I started to feel incredible. The pain, after a few months, the side effects from my cancer started to come down.”
Today Sandra’s cancer is still in remission. She is back to her old, healthy, energetic self. She says she doesn’t consider herself a stem cell pioneer but is glad her participation in the trial might also benefit others.
“It’s helped me but the opportunity that it could also help other people is truly meaningful.”
The treatment she received was approved by the US Food and Drug Administration in 2019, the first approval for a therapy that had CIRM support.
I recently had the great pleasure of interviewing Sandra as part of our CIRM 2020 Grantee Meeting.