Inform, Empower, Connect

We were delighted to award Dr Tanya Heim a travel grant to present her work at FACTOR. Find out more about her work and FACTOR in her guest blog post.

I have been a biomedical research scientist for over a decade. I have not always studied cancer, but I have always been interested in rare diseases. For the last 5 years, my research has focused on finding novel druggable targets for patients with metastatic osteosarcoma (OS). Although rare with around 1,000 new annual cases in the United States, OS is the most common primary bone cancer. Most patients are diagnosed with this rare cancer during puberty.

I was very honoured to receive the invitation to speak at the MIB FACTOR 2023 conference. FACTOR is a conference hosted by the MIB Agents. MIB Agents is a non-profit dedicated to ‘Making It Better’ for patients with OS and the support systems around them. This was the 6th annual FACTOR conference but my first time attending. I knew the conference was unique not only for its niche focus but because of patient involvement, as well. I was overwhelmed with the thoughtful curation of this conference. It was hard not to collaborate with everyone you met. Many researchers and clinicians had similar interests but differing levels of experience. This naturally led to fruitful connections. FACTOR included scientific and patient panels and the knowledge and inspiration gained from these sessions is priceless. Most importantly, all attendees showed a passion for continuing to understand and cure OS.

I presented our work which targets androgen receptor (AR) in OS. AR’s are structures on the surface of cells that respond to sex hormones. We have found a high amount of AR in primary OS tissues and cells compared to metastatic OS samples. We also have evidence to believe that ALDH1A1, a target elevated in metastatic OS, may be linked to AR activity. Using this knowledge, we have been investigating the effects of AR and ALDH1A1 inhibition via gene and drug therapies. Neither drug was effective at inhibiting these targets alone. But we saw significantly reduced cell survival and spread in our more metastatic cell type, called SaOS-LM2, when the treatments were combined. Furthermore, we were able to achieve significant results with lower doses of each inhibitor with the combination therapy. While it is strongly believed sex and growth hormones are involved in OS, it has been difficult to prove. Results from previous studies have been inconsistent with one another. We hope our new discovery of AR and ALDH1A1’s possible mechanistic link will provide a better way to study the role of this sex hormone receptor in OS. We recognize that hormone therapy may be risky for the average OS patients, However, we believe there is true knowledge to be gained by looking at the influence AR has on ALDH1A1.

It has always been our lab’s hope to find a treatment for OS that could be used in the clinic. Treatment options for metastatic OS are lacking and patients are suffering from low survival rates. Our research uses drug repurposing to avoid possible delays that could arise if using a new drug which still requires FDA-approval. Our lab understands the importance of developing better treatments for the patients we serve and the need to make these treatments accessible quickly. Attending the FACTOR conference this year has made this clearer than ever. It inspired me to push harder to make it better for patients with OS.

We would like to thank Tanya for sharing her work. You can find out more about her lab’s work here. You can also read more about FACTOR in our recent blog.