Shaina Willen, MD

, Shaina Willen, MD, of UC Davis Health, shared more insights from her ‘Pediatric Year in Review’ presentation at the 

American Thoracic Society (ATS) 2025 International Conference

 regarding newer sickle cell disease therapies.

Willen, known for her work as a board-certified provider in pediatric hematology/oncology and pediatric pulmonology, spoke about newer therapies in a previous interview. In this segment, she was asked about barriers to access therapies among many patients with sickle cell disease.

Looking at Barriers to Access for New Sickle Cell Disease Therapies, with Shaina Willen, MD

“I think that's been a huge concern,” Willen explained. “I mean, for one, the access for children is still limited. The lower age of FDA approval is age 12, so for younger children who have severe complications, it's still not an option. Those studies are ongoing, and hopefully that will change in the next couple of years, but still the lower age is 12. It's also a very expensive and very involved therapy, so right now, even though we've had FDA approval for about a year and a half, access is still pretty limited to very large academic centers.”

Willen highlighted that families of patients frequently need to travel extensively in order to get access to such therapies. She added that the process can be long and arduous with stem cell collection and the preparation of families and patients for the treatment.

“You know, we talk a lot about what we're what we're doing in FDA approvals and about what's happening in our country, but the majority of patients with sickle cell still live in other countries, and particularly Sub-Saharan Africa,” Willen said. “20 million children with sickle cell disease are living in Sub-Saharan Africa, where this treatment is not an option. So as far as access to the majority of patients with the disease, we're still a very long way from that.”

Willen was also asked about whether or not sickle cell disease treatments are at an inflection point for those with access to such therapies.

“I do think we're still a ways away from truly pivoting the disease, because of access and even the other therapies that I mentioned outside of gene therapy still have some tricky barriers, meaning they're not being used as universally as hydroxyurea,” Willen said. “So my hope is that it's a turning point, and that now that we have other things and other mechanisms and other people interested in the disease, it will be coming to have a bigger inflection point. I don't want to, I don't want to completely minimize enthusiasm for gene therapy. I think it's exciting, and I think having other options is an exciting time for the disease, but as far as truly changing the landscape…in the lives of the majority of children, I think we're still a little ways away.”

For more on Willen’s talk presented at ATS, view the full video segment posted above. To learn more about the data presented at ATS, view our 

The quotes used in this video summary were edited for clarity. Editor’s note: Willen reported no relevant disclosures.

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In her interview at ATS, Willen further described elements highlighted in her presentation on new treatments for sickle cell disease.

 spoke with Shaina Willen, MD, a board-certified provider in Pediatric Hematology/Oncology as well as Pediatric Pulmonology from UC Davis Health, about her talk at ATS titled ‘Pediatric Year in Review.’

In the talk, Willen described a variety of new therapies for sickle cell disease and their relevance to the conference itself, highlighting the recent explosion in options for patients with this condition. Willen was first asked what prompted the discussion in the first place.

Pediatric Year in Review: New Therapies in Sickle Cell Disease, with Shaina Willen, MD

“Historically, since 1998 for adults and to 2017 for children, we've only had one therapy for sickle cell disease,” Willen explained. “So the only disease-modifying therapy drug related is hydroxyurea, which works very well and is a wonderful therapy. But having one option in such a prevalent and difficult-to-manage disease has been very tricky, particularly for children. That's been the only medication since 2017 that's FDA-approved to treat complications of sickle cell disease. Then, first in 2017, 2019, and now, with the advent of gene therapy in 2023, we have more options.”

Willen was asked about some of the therapies for sickle cell that she highlighted within her presentation at ATS.

“Since hydroxyurea, we now have Endari, or L-glutamine, which we talked a little bit about in the talk,” Willen said. “We have crizanlizumab, which is for teenagers aged 16 and up. And then we had voxelotor, unfortunately, that was pulled from the market in December or September of 2024. Now with the 2 gene therapy options, disease-modifying therapies, there are a lot more choices than there used to be, particularly for children. So we were just wanting to highlight those and the trials and tribulations with those new therapies, but it is exciting that now we have options that we didn't have before.”

To find out more from Willen’s presentation at ATS, view the full interview segment posted above. For more from ATS, including late-breaking data, view our 

The quotes contained in the above summary were edited for clarity. Editor’s note: Willen reported no relevant disclosures.

This interview at ATS 2025 features a discussion of a ‘Pediatric Year in Review’ presentation on new therapies for sickle cell disease.

Shaina Willen, MD

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