Pioneering New CLL Treatments for All: Bita Fakhri’s Innovative Approach

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Bita Fakhri, MD, MPH

In the last decade, nearly everything about how clinicians treat chronic lymphocytic leukemia (CLL) has changed. The most common leukemia in adults, CLL is a cancer of blood-forming cells in the bone marrow. For many years, the disease was treated with the same chemotherapies as other cancers, which indiscriminately kill all quickly growing cells in the body. But recently, scientists developed more targeted ways of treating CLL by attacking specific proteins that CLL cells rely on or by using the power of the immune system. These drugs have proved to be more effective – and have a better side effect profile – than conventional chemotherapies. 

During the early years of her career, Assistant Professor of Hematology Bita Fakhri, MD, MPH, was involved in many of the seminal clinical trials showing just how effective the new generation of drugs was. She watched experimental drugs become commercially available options to extend the duration and quality of patients’ lives. 

“The advances in this field over the last 10 years have been truly mind-blowing,” says Fakhri. “Seeing the success of these drugs, and just how dynamic the field has been, made me want to keep working on CLL.”

Broad Clinical Trial Options

In 2022, Fakhri became the director of the CLL clinical trial portfolio at Stanford after the passing of Steven Coutre, MD, who had established the clinical research program in the Division of Hematology. Since joining, she has launched five new clinical trials for patients with CLL. She is also hard at work to open clinical trials benefiting patients with Richter’s transformation – a condition in which CLL transforms to a more aggressive lymphoma with currently very poor outcomes. Those trials, she says, range from testing new front-line options for patients who have new CLL diagnoses to comparing treatments for people whose recurrent cancers are not responding to newer targeted agents in the field.

“One of my priorities at Stanford is making sure that we always have trials in both of these settings,” says Fakhri. “Despite all the advances in CLL, there are still a subset of patients with high-risk features that need new treatment options, and we want to meet their needs.”

About 88% of patients newly diagnosed with CLL will survive for at least five years, according to the latest data from the National Cancer Institute. That represents a large increase from the 70% to 75% five-year survival rate in the 1990s and early 2000s. But patients who have recurrence of their cancer, even years later, often fare less well – that is one of the populations Fakhri hopes to help with new clinical trials.

Fakhri adds that the most effective CLL drugs have come out of a detailed molecular understanding of how CLL impacts cells, and this kind of basic research must continue. To that end, she is collaborating with Stanford scientists including Sydney Lu, MD, PhD, who studies the underlying biology of CLL and other cancers. Lu and Fakhri are studying the implications of a gene mutation, known as SF3B1, that is found in about one in 10 CLL cancers and is associated with worse outcomes for patients. If they can understand the molecular consequences of SF3B1, Fakhri says, they may be able to develop new drugs to counteract the mutation or develop a better understanding of the clinical behavior and response to different therapies in patients harboring the SF3B1 mutation.

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“I’m not involved in DEI efforts because I think it’s a trendy topic. This is the right thing to do, morally, ethically, and scientifically. If we don’t have a diverse patient population in our studies, then we don’t know if our results are applicable to all our patients.”

– Bita Fakhri, MD, MPH

Equal Access for All

Among the many clinical trials that Fakhri is involved in, one thing ties them all together: an emphasis on equity. As the head of diversity, equity, and inclusion (DEI) efforts in the Division of Hematology, Fakhri is passionate about making sure that patients of all backgrounds, identities, and socioeconomic statuses are represented in her research. 

“I’m not involved in DEI efforts because I think it’s a trendy topic,” says Fakhri. “This is the right thing to do, morally, ethically, and scientifically. If we don’t have a diverse patient population in our studies, then we don’t know if our results are applicable to all our patients.”

As more CLL treatments emerge, and each patient’s path to remission becomes more personalized, it is especially important to include a diverse set of patients in every clinical trial. Ultimately, clinicians’ decisions about which drugs will work best for a particular patient may be based on not only clinical data but demographic information as well – from race and ethnicity to gender and education. 

“What I want is to create the machinery that eases enrollment in clinical trials and makes access to these trials feasible for everyone, not only for the most privileged patients,” says Fakhri. 

Fakhri and her colleagues are currently analyzing data on the diversity of Stanford clinical trials in hematology over the last decade to identify which patients are underrepresented. This information, she says, will help guide future clinical trial recruitment efforts.  

“The beauty of medicine is that we are all physiologically different,” says Fakhri. “We need that diversity captured in our trials.”

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