Spencer Haws is on a Quest to Treat Fragile X Syndrome


Spencer Haws might be relatively new to the field of stem cells, but he’s diving in head first to understand a disease with few treatment options: fragile X syndrome.

“Stem cells are the perfect way to study this disease,” he remarked. “To explore it in the context of a real, human patient will be really critical for targeting it at its root. It’s all very exciting.”

We talked with Dr. Haws (a 2022 NYSCF – Druckenmiller Fellow in NYSCF – Robertson Stem Cell Investigator Alumna Jennifer Philips-Cremins’ lab at the University of Pennsylvania) about the power of stem cells, his work to understand fragile X syndrome, and advice for those who want to pursue STEM.

What made you decide to become a scientist?

The first time I was exposed to lab work was between my freshman and sophomore years of my undergraduate degree. I remember being struck by how exciting it was – it was something that I felt like I immediately identified with and enjoyed. I thought chasing a discovery – finding out something that nobody else knew before – was really fun. Once I realized how much was possible through science, my mind was made up.

What made you interested in stem cells?

I’m pretty new to stem cells – I didn’t do stem cell work as part of my PhD. Now, working with Jennifer [Phillips-Cremins, a NYSCF – Robertson Stem Cell Investigator Alumna], who has been leveraging stem cells for a while, I’ve become much more immersed in using stem cells as a model. Stem cells let us do so much: for example, we can study different diseases using patient-derived stem cells that carry all of that patient’s genetic information, which is huge. We can also use stem cells to make all the different cell types in the body, which is also incredibly powerful for understanding how a disease affects patients.

What is fragile X syndrome and what made you want to study it?

Fragile X syndrome is the most common inherited intellectual disability disorder – it has some similarities to autism spectrum disorder. What motivates me in the lab is that there’s no fragile X syndrome-specific treatment right now. All the treatments are really geared towards managing the symptoms, but because there is such a wide breadth of symptoms for the disorder, these treatments aren’t really effective for everyone. It’s better than nothing for sure, but patients really need something specifically geared towards their needs. And we’re hopeful that with some of the insights generated from our ongoing work, and this NYSCF funding, we can start moving in that direction.

How is your lab using stem cells to study fragile X syndrome?

We have a wide range of stem cells made from fragile X patients. The origin of the disease is a repeating sequence of DNA in front of a gene called FMR1. We have stem cells from patients with either short, normal, or long repeats of this DNA sequence, and we can use those to figure out how the DNA expansion actually leads to the disease etiology.

What do you hope to achieve in your research career?

If my work can help push us even one step closer to a new therapeutic or something meaningful for patients, that would be huge for me. This is definitely the most translational project I’ve ever worked on, and that’s very exciting. 

This project is also starting to connect two areas: metabolism and genome organization, which is pretty underexplored but likely relevant to a lot of different diseases and disorders. So I think beyond my postdoc and later in my scientific career, if I could help identify some of these fundamental mechanisms connecting the two disciplines, I’d be more than happy with that too, because I think over time that’s something that’s going to be more greatly appreciated as a driver of different diseases and disorders.

What advice would you give to young people who are interested in pursuing STEM?

I think it’s about finding something that’s exciting to you, because a research career can be very rewarding but also very challenging. It’s kind of a roller coaster, but if you’re working on something that you really feel passionate about, it helps you stay steady and motivated, and it just makes the whole process more fun.

Can you talk a little bit about the role of DEIB in stem and how you’ve seen its importance throughout your career?

Fostering DEIB in STEM is huge, and we know that there are a lot of implicit biases in science. Having dedicated DEIB strategies is a great step in trying to address those. I also think one way of fostering diversity in the field is getting young kids interested in research science. When you can get excited about a subject, it really changes perspectives.

What is most exciting to you about receiving this award and joining the NYSCF innovator community?

As someone who is newer to the stem cell field, I’m looking forward to soaking in knowledge from all the other awesome researchers in the community. I’m also looking forward to the NYSCF Conference and retreat, and gaining feedback about my work and learning about new approaches. Being a part of this community is a big honor.

Diseases & Conditions:

Development, Neurobiology

People mentioned: