Advances in blood disorders research continue despite COVID-19

Expanding use of CAR T-cell therapies, gene editing lead to insights at virtual annual meeting of the American Society of Hematology
graphic showing red blood cells and large white leukemia cells in a blood vessel
At the annual meeting of the American Society of Hematology, researchers presented the latest news about blood cancers like leukemia (white cells in illustration), disorders of red blood cells (red cells) and more. Illustration by Getty Images

Just as the COVID-19 pandemic forced the annual meeting of the American Society of Hematology to go virtual to keep its 30,000 participants safe, it threw up roadblocks to research in labs and clinics. But the 3,500 research abstracts presented at the virtual meeting proved that the coronavirus couldn't stop science altogether.

The 62nd ASH Annual Meeting & Exposition, held online from Dec. 5 through Dec. 8, featured presentations on the latest discoveries in blood cancers like leukemia, lymphoma and myeloma, and in blood clots, sickle cell disease, bleeding disorders, blood cell development and more.  

COVID-19's hematologic impact

The pandemic loomed large over ASH 2020, and not just because of its impact on the format.

Outgoing ASH President Dr. Stephanie Lee, who is the associate director of the Clinical Research Division at Fred Hutchinson Cancer Research Center, opened the meeting’s first big session with a moment of silence to “remember the human devastation of this pandemic.” As the meeting opened, there had been about 68 million cases of COVID-19 around the world and 1.5 million deaths.  

Fred Hutch research at #ASH20

COVID-19 is of particular concern to hematologists. It causes blood clots, it is sometimes treated with a blood product — convalescent plasma — and people with blood cancers or other problems of the blood and immune system are at high risk of the disease. Accordingly, ASH held several special sessions with the latest in what hematologists need to know on the disease.  

The meeting also featured a special Q&A with Lee and Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases and the U.S.’s most visible coronavirus expert.

The pair held a wide-ranging conversation that touched on the similarities between the COVID-19 and HIV pandemics — they are both viruses that jumped from animals to people but that’s about where it ends, Fauci said — how long immunity to this coronavirus might last — maybe months, hopefully years, but we will definitely be finding out — and what Fauci thinks of his newfound celebrity. (It’s good that all scientists and health professionals are getting recognition and appreciation during this time, he said.)

Lee will spend the last two weeks of December in the hospital caring for people getting blood and marrow transplants, and she remarked that this might put her near the front of the line to get one of the new coronavirus vaccines expected to be available in the coming days. She asked Fauci whether he’s worried that people will let their guard down as the vaccines start to roll out.

“I hope not,” he answered. “It will be months and months and months before enough people get vaccinated to get that umbrella of protection.” He warned that no one should be expecting life to go back to normal any time in 2021. A return to normalcy is “doable, but not in a few months,” he said, given the massive effort required to vaccinate billions of people and the continued public health vigilance that will be necessary to control the virus’s spread until herd immunity is achieved.

Dr. Anthony Fauci, NIAID director, participated in a special session at the ASH 2020 annual meeting on Dec. 5 with ASH President Dr. Stephanie Lee, who holds the David and Patricia Giuliani/Oliver Press Endowed Chair in Cancer Research at Fred Hutch.
Dr. Anthony Fauci, NIAID director, participated in a special session at the ASH 2020 annual meeting on Dec. 5 with ASH President Dr. Stephanie Lee, who holds the David and Patricia Giuliani/Oliver Press Endowed Chair in Cancer Research at Fred Hutch.

Pool photo by Graeme Jennings / Getty Images (Fauci); Fred Hutch file (Lee)

Lee noted that she and her colleagues treat people who are immunocompromised, either by their disease or its treatment (or both), but none of the people enrolled in any of the vaccine trials to this point were immune-compromised. So should people with damaged immune systems receive the vaccine when it’s available to them? 

“I think we should recommend they should get vaccinated,” Fauci said. “If you’re immunosuppressed, history tells us you won’t have as big of a protective response. But some is better than none.” 

Other COVID-19 highlights at ASH 2020: 

  • Why do some people who contract the coronavirus develop severe COVID-19 but others do not? A study led by researchers in Greece found a high rate of gene variants associated with a propensity for blood clotting among people who developed severe disease. The differences in coronavirus outcomes between people with and without the gene variants was “quite profound,” Lee said in a Nov. 24 media preview. 

  • How does COVID-19 affect cancer patients? Groups of researchers are collecting real-world data to understand how the coronavirus interacts with cancers. Data was presented at the meeting by multiple cross-institutional research teams, including the ASH Research Collaborative Data Hub focused on COVID-19 in blood cancer patients, and the COVID-19 and Cancer Consortium Registry, who studied the infection’s severity in blood cancer patients and the risk of blood clots in cancer patients with COVID-19

  • Researchers presented data on both existing and possible future treatments for COVID-19, from convalescent plasma (antibody-containing plasma from the blood of recovered people), which has received an emergency use authorization from the Food and Drug Administration, to a potential immune cell-based treatment strategy for those with severe disease. 

Gene editing and cell therapy 

Gene-edited immune cell therapies for certain blood cancers — CAR T-cell therapies — are now FDA-approved and standards of care for people with certain advanced blood cancers. ASH 2020 featured a slew of advances in applying existing immune cell therapies to new groups of patients or in new combinations. Researchers also presented on the development of totally new immune cell therapies for cancers. 

For example, among presentations from researchers at Fred Hutch were clinical trial data on a new CAR T-cell strategy for lymphoma aimed at a different target than the FDA-approved CAR T-cell therapies and on a novel engineered T-cell strategy for children and adults whose leukemia relapsed after bone marrow transplant. Hutch scientists previewed potential new engineered cell therapies, still in the lab, for acute myeloid  leukemia and lymphoma. They led sessions on ongoing challenges in engineered T-cell therapies, like cancer’s ability to escape from therapy, and presented data helping illuminate the factors linked to engineered T cells’ effectiveness and side effects. 

photo of Stephanie Lee sitting at her computer desk at home, seen from behind. The back of a backdrop behind her is visible, as is a cat sitting on a chair next to her.
Dr. Stephanie Lee moderates a session at the ASH 2020 virtual annual meeting from home in the company of her cat. (Click to view on Twitter.) Photo from Twitter / Dr. Stephanie Lee

But the biggest buzz was about a different group of gene-editing-based therapies: new strategies to correct inborn disorders of blood cells, such as the debilitating and deadly sickle cell disease and hemophilia B, a hereditary bleeding disorder.  

A pair of trials of a CRISPR-based gene therapy called CTX001 provides “clinical support that this is a potential functional cure” for people with sickle cell disease and its relative, β-thalassemia, said the meeting’s scientific program co-chair, Dr. Alisa Wolberg of the University of North Carolina, in a “Best of ASH” presentation at the meeting’s close. 

At ASH 2020, the trials’ global research team presented evidence that the experimental therapy provides lasting effects, eliminating β-thalassemia patients’ need for transfusions and greatly reducing the frequency of sickle cell patients’ excruciating pain crises. 

The meeting featured the first presentation of data from the industry-sponsored Phase 3 HOPE-B trial, which used a gene therapy method to correct a deficiency in more than 50 people with hemophilia B. The data so far show that the strategy appears safe, and nearly all of the patients in the trial have been able to stop taking their regular anti-bleeding drugs. 

“We’re getting very close to the point where gene therapy is possibly going to be considered a standard of care for certain forms of hemophilia,” said ASH Secretary Dr. Robert Brodsky of Johns Hopkins in the media preview. 

“Collectively these gene therapy advances are exciting because they bring to fruition decades of basic and clinical work to cure these devastating hematologic disorders."

Many research groups continue to develop new forms of gene therapies for non-cancerous blood disorders like these and the ASH meeting featured presentations on many different strategies being developed in labs and clinics by teams at Fred Hutch and institutions around the world. 

“Collectively these gene therapy advances are exciting because they bring to fruition decades of basic and clinical work to cure these devastating hematologic disorders,” Wolberg said. 

New standards of care 

The meeting also offered numerous presentations that experts expect will change how patients with blood cancers and other blood disorders are treated. Some examples: 

Grappling with racial bias and disparities in medicine 

This was also a year for a major reckoning across society about racism and its impacts. ASH 2020 picked up this topic and examined it from several angles. 

Speakers highlighted why a diverse, inclusive medical and scientific workforce makes for better medicine and better science, and exhorted physicians and researchers to examine their own inherent biases, embrace humility about their own cultural backgrounds, and implement deliberate countermeasures to racism in their own institutions and work. And scientific talks documented some of the disparities that cause many people of color in the U.S. to experience poorer health than white Americans in areas from prenatal care to cancer-associated blood clots to leukemia treatment.  

“2020 really brought a lot of attention to these issues and really has spurred a nationwide dialogue about the problem and potential solutions,” Lee said. 

Susan Keown was a staff editor and writer at Fred Hutchinson Cancer Center from 2014-2022 who has written about health and research topics for a variety of research institutions. Find her on Twitter @sejkeown.

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