New hope for leukemia sufferers

Wallenberg Clinical Scholar Eva Hellström-Lindberg believes collaboration between the research world and health care providers is essential. Humility and curiosity are her drivers as she strives to improve the long-term prognosis for patients suffering from various forms of blood cancer grouped under the umbrella term MDS – myelodysplastic syndrome.

Eva Hellström-Lindberg

Chief physician and Professor of Hematology

Wallenberg Clinical Scholar 2018

Institution:
Karolinska Institutet

Research field:
Developing new therapies for myelodysplastic syndrome – MDS.

It is just a short walk from the lab at the Neo research facility to patients at Karolinska University Hospital in Huddinge, south of Stockholm. Hellström-Lindberg has changed into her hospital wear, and makes her way along the corridors leading to the hematology clinic.

She has spent over three decades researching into MDS alongside her role as a doctor. MDS is a group of serious blood cancers that frequently develop into an aggressive form of leukemia. About 300 people in Sweden are diagnosed every year.

“When I started working in this field 35 years ago there wasn’t even a name for the condition. I met patients with really low blood counts, and a high risk of developing leukemia. They were elderly people who were seriously ill, but no one understood why. It was then that I set up the first Swedish, and then the first Nordic, MDS Group to conduct research into the condition.”

The more it has become possible to describe the syndrome, and to begin trying out various therapies, the more research yielding biological and molecular understanding has evolved. Hellström-Lindberg explains:

“Enormous strides have been made in this field, and the Nordic MDS Group is pre-eminent. It’s a privilege to be involved.”

“I want to make high-quality long-term studies, with ambitious targets for improving the outlook for patients. The grant gives us the courage to take greater steps and initiate projects that I know will take a long time to complete.”

Two main goals

At the clinic she meets patients for evaluation and diagnosis.

“We see eighty to ninety patients a year here. They come from primary health care, and for a second opinion, from the whole of Sweden and elsewhere.”

The project includes a large biobank. Blood och bone marrow from MDS patients are saved and studied, so the right treatment can be given, and also for use in various research projects.

The interface with patients gives Hellström-Lindberg inspiration and ideas for her research.

“I learn something new from every patient. As a Wallenberg Clinical Scholar, I have two clear goals. One is to cure more patients with MDS. This is a key objective, with many components designed to improve the long-term prognosis for those with MDS. The other primary goal is to understand and treat the severe anemia associated with MDS.”

Gene panel providing answers

The only cure available to patients with high-risk MDS is a bone marrow transplant, or stem cell transplant as it is also called. Hellström-Lindberg elaborates:

­“Without treatment, these patients are expected to survive no more than a year. We can never promise anything, but my aim is always to cure them.”

MDS is caused by mutations in up to 50 genes. Usually the patient has between one and six genetic mutations. The genetic information is a useful tool in evaluating patients.

“We use the gene panel that we have helped develop to evaluate each patient. We make a prognosis based on the result, which can also guide us in our choice of therapy.”

One serious problem with MDS is that the risk of a relapse is over 30 percent; for some, it approaches 50 percent. A major Nordic study led by Hellström-Lindberg is developing a promising new method of predicting the risk of relapse.

“This is done by studying the patient’s own genetic markers using a method capable of finding them at a very early stage. Our pilot study has shown that a relapse can be predicted many months before it can be detected using normal clinical methods.”

The method is now being tested on 200 patients. We hope it can then be used as a routine method of monitoring all patients following a transplant. The idea is that there will be far fewer relapses if we can find and treat those at risk of a relapse at any earlier stage.

Finding new ways to treat MDS requires a broad, long-term approach. Among other things, Hellström-Lindberg is collaborating with Professor Sten Eirik Jacobsen at Karolinska Institutet in a project aiming to improve understanding of blood cancer stem cells.

“We are working together to learn how to target cancer stem cells effectively, since they are notoriously resistant to normal chemotherapy.”

Essential collaboration

In cases of low-risk MDS it is usually a question of treating the patient’s anemia.

“Here, the treatment of choice is a hormone called erythropoietin, EPO, which stimulates the body to produce more red blood cells. The Nordic MDS Group developed this therapy in the mid-1990s.”

In the laboratory at the Neo facility, the research team is trying to understand the blood cell-forming stem cells in these patients, and identify concepts that can either promote healthy stem cells or inhibit those that are diseased.

Hellström-Lindberg reiterates the importance of working both with patients at the clinic, and in the lab, and of collaborating in wider contexts. She stresses that no one should ever think they have all the answers:

“I believe we can substantially improve the outlook for patients with these complex diseases if we make a truly concerted effort. And to do so requires the full spectrum, from understanding stem cells to clinical studies.”

Text Susanne Rosén
Translation Maxwell Arding
Photo Magnus Bergström