The Personal and Professional Drive Behind Life Sciences Innovation

By Chelli Miller Biotech Lead Partner @ Content Carnivores |

We all say it, but it’s true: the drive behind life sciences innovation, the relentless pursuit of cures, is deeply personal. You don’t just work in this field; you invest your heart and soul. My connection to Pacylex Pharmaceuticals and the journey they’re on with AML is a perfect example of a rare intersection of the personal and the professional.

Professionally, this journey started with Pacylex’s CEO, Michael Weickert, Ph.D, someone I’ve known since the very beginning of my two decades in healthcare. I remember his work at Sea Medical, inspired by his family’s experience with extensive hospitalization of their new baby. His emotional personal experience, coupled with his scientific knowledge, helped make the Sea Medical story resonate with me. That same passion fueled Pacylex’s work on a novel approach to treating acute myeloid leukemia (AML), a particularly brutal cancer, especially for children.

AML, as I’ve come to understand it more deeply, is a cancer that affects the blood and bone marrow. It’s an aggressive disease where immature blood cells rapidly accumulate, hindering the production of normal blood cells. It’s particularly devastating in children. Pediatric AML, pediatric cancer in general, presents unique treatment challenges. Children’s bodies metabolize drugs differently than adults, making it difficult to adapt adult treatment protocols. AML in children can be especially aggressive, and relapse is a significant concern. Finding effective therapies for relapsed or refractory AML – when the cancer doesn’t respond to initial treatment – is a major hurdle. Even then, adults are treated first, and only when safety and efficacy is determined, would a child be treated with a new treatment modality. The harsh reality is that the toxicity of existing chemotherapies takes a greater toll on growing children. This is a huge reason we need novel therapies that can also treat our young!

Content Carnivores started working with Pacylex when they were in the preclinical stage. They were developing a completely new way to attack AML. Their lead drug, zelenirstat, is a first-in-class N-myristoyltransferase inhibitor (NMTI). It works by inhibiting myristoylation, a fatty acid modification crucial for certain proteins to function in cancer cell survival and proliferation. In simpler terms, zelenirstat targets key cancer-driving processes that depend on myristoylation, which kills cancer cells at drug levels that don’t harm normal cells. Think of it like this: Cancer cells use myristoylation to help them grow and spread. Zelenirstat is designed to stop that. It’s a novel approach, targeting the disease in ways other treatments don’t.

At about this time, life threw me a cruel curveball. A dear friend, someone I’ve known since I was practically a kid myself, called me. This was a friend whose family I adore – I even named my son after her daughter and son. She told me her 12-year-old granddaughter had been diagnosed with AML. As a new grandmother, myself, the news hit me like a ton of bricks.

My friends are a brilliant family living in the Chicago area. They assembled a team of researchers and experts, leaving no stone unturned. They fought tooth and nail to get their granddaughter into every treatment modality and clinical trial possible. We watched as her little body endured round after round of treatments, hoping against hope for a positive response.

A few devastating years passed for my friends, and Pacylex continued its work. I watched every avenue and every potential treatment be exhausted. Then, just before this beautiful girl’s 16th birthday, she, with heartbreaking grace and acceptance, planned her 16th birthday party or her funeral. There was nothing left to try. Her body was just too weak. I tear up just thinking about this!

And that’s where Pacylex’s work became even more significant to me. Their novel mechanism was something her body hadn’t encountered before. Preclinical studies suggested AML cells, including AML stem cells, are particularly sensitive to zelenirstat. It’s designed to trigger apoptosis and cell death in AML. But it was too late. She had run out of time. We attended her 16th birthday, which was also her funeral. That experience, that loss, is seared into my memory and my heart.

Personally, this child’s diagnosis and battle with AML created a powerful connection to my professional work with Pacylex. I wish that the treatments being developed by Pacylex Pharmaceuticals had been advanced to the point where she might have participated in their trials. This was a perfect case for a Compassionate Use Designation – an FDA designation that allows patients with serious or life-threatening conditions to access investigational drugs or medical devices outside of clinical trials when no other treatment options are available. Zelenirstat data continues to indicate the need for the designation for AML as well as other tumor types.

Fortunately, the company is advancing, meaning others like her will have a fighting chance against this devastating disease.

Today, Pacylex is making real progress. They have clinical data. A published Phase 1 dose escalation safety and tolerability study in 29 heavily pre-treated patients showed that zelenirstat was well-tolerated, with most adverse events being mild gastrointestinal side effects. The study also showed that zelenirstat prolonged cancer progression-free and overall survival in solid tumor patients receiving the recommended Phase 2 dose. They’re moving into Phase 2, and the results are promising.

I still see my friends on Facebook, still actively supporting other families battling AML, still organizing blood drives in their daughter’s name. It’s both heartbreaking and inspiring. Every time I see this, I am reminded of the family’s desperate quest to find trials, novel treatments—anything to help the medical team save their loved one.

This isn’t just a professional interest for me anymore. It’s deeply personal. It reinforces why we do what we do in healthcare. Cancer is personal. We all know someone who’s lost the battle, and we all know countless lives could be saved with the right innovation. We need multiple innovations because each body, at every age, is different.

I’m incredibly proud to be involved in getting the word out about Pacylex’s work. It’s more than just marketing and PR; it’s about advocating for the funding and support companies and families desperately need. We need to accelerate the pace and funding of innovation because every day counts. Every child, every parent, deserves a chance. And that’s why I’m here, telling this story, because it’s not just their story; it’s part of mine.