Photo: Dr. Vakoc (Professor), Junwei Shi (Graduate Student)
Your donations at work! In a groundbreaking achievement, the Vakoc Lab at Cold Spring Harbor Laboratory, led by Dr. Christopher Vakoc, has unveiled a promising breakthrough in the battle against pediatric cancer, specifically Rhabdomyosarcoma (RMS). This connective tissue cancer affects 400-500 Americans each year, with over half receiving their diagnosis before the age of 10.
Dr. Vakoc and his dedicated team have successfully transformed cancer cells into healthy muscle cells, a monumental discovery that opens the door to potential implementation of differentiation therapy. This marks a significant stride in childhood cancer research and brings hope to families affected by pediatric cancer.
Differentiation therapy is a type of medical treatment that aims to induce cancer cells to take on the features of normal cells, losing their malignant characteristics and restoring some normal functioning to the affected tissues. In the context of pediatric cancer, Dr. Christopher Vakoc and his team, are exploring ways to reprogram cancer cells, in this case, RMS cells, into becoming normal, functional cells, specifically muscle cells. This approach is different from traditional cancer treatments, like chemotherapy, which primarily focus on killing rapidly dividing cells.
The potential safety advantage of differentiation therapy lies in its specificity. By targeting the transformation of cancer cells into normal cells, this approach may have fewer side effects on healthy tissues compared to traditional treatments that can affect both cancerous and normal cells.
Dr. Vakoc’s team successfully disrupted a signal in a protein called NF-Y, leading to the differentiation of cancerous cells into normal muscle cells. The hope is that this differentiation process could offer a safer and more effective therapeutic approach for treating pediatric cancers. This incredible discovery was published in the August edition of the Proceedings of the National Academy of Sciences.
Dr. Vakoc and his team have made a significant step forward for pediatric cancer treatment, but we are still a long way from creating a new treatment. While the initial results are promising, differentiation therapy is still a relatively experimental and complex field, and more research and clinical studies are needed to determine its safety and efficacy in treating various types of cancers.
This is why one of the core missions at the Daniela Conte Foundation is to bridging the research funding gap. Childhood cancer receives a mere 4 percent of federal cancer research funds, hindering progress in developing treatments for the leading cause of death by disease in children worldwide. At DCF we believe that every child deserves the very best, and your support directly fuels research that can revolutionize their lives, including the work of Dr. Vakoc and his team.
Looking ahead, Dr. Vakoc plans to expand on this discovery by using advanced techniques to further exploring the entire human genome. Each step forward lights the path of hope and progress for families battling childhood cancer.
Today we celebrate the incredible work of Dr. Vakoc's team, and we thank our generous supporters for helping to make their work possible. It fills us with immense joy to witness this step towards a brighter future for children bravely battling pediatric cancer.
If you want to dive deeper into Dr. Vakoc’s work and this groundbreaking moment, the full PNAS article is available at: https://www.pnas.org/doi/10.1073/pnas.2303859120
Written by: Katia Conte, President/Co-Founder