VUB launches fund for research into Duchenne muscular dystrophy

On the eve of World Duchenne Awareness Day on September 7, the Vrije Universiteit Brussel launched its own fund for Duchenne. Through the VUB Duchenne Fund, the university invests in promising gene therapy research led by Prof. Dr. Thierry VandenDriessche and Prof. Dr. Marinee Chuah as a potential remedy for the muscle disease, which affects 1 in 3,500 boys worldwide. The preclinical phase has shown that the developed technology prevents muscle deterioration in mice, effectively enabling recovery.

A DNA error destroys lives

Duchenne muscular dystrophy is a hereditary disease that affects the muscles, eventually making independent movement impossible. The condition is caused by a defect in the dystrophin gene on the X chromosome. At birth, the disease is not visible, but from early childhood motor problems begin to appear. Children have difficulty standing up, and running or climbing stairs is challenging. A wheelchair becomes essential from around age 8. In addition, the disease also affects the heart and respiratory system, which can ultimately be fatal.

"Ruben was a very calm baby and toddler. He never climbed on anything and did everything very cautiously. When he fell, he had trouble getting up. I had visited several orthopedic and physical doctors, but according to them, I was an overprotective mom. Ruben was in first grade when we received the diagnosis," says Lieve, mother of a Duchenne patient.

Gene therapy brings hope

At the Vrije Universiteit Brussel, Prof. Dr. Thierry VandenDriessche and Prof. Dr. Marinee Chuah, from the Department of Gene Therapy and Regenerative Medicine, made a scientific discovery that opens the door to a potential cure. They have been conducting gene therapy research for over 30 years. The professors confirm that “this new gene therapy technology prevents muscle deterioration in mice with Duchenne disease, effectively enabling recovery. This represents a first important step toward further clinical applications in patients with Duchenne.”

Prof. Dr. Thierry VandenDriessche: “We would like to sincerely thank the patients, their families, and friends for their financial support of our research. With these funds, we will continue to work on improving the efficacy and safety of gene therapy for the treatment of muscular dystrophy. We are the only research group in Belgium with over 30 years of experience in this field, which we can build upon to fulfill our mission.”

The research team sought a way to introduce a copy of a functional dystrophin gene into the genetic material of muscle cells to compensate for the defective gene. To activate the healthy gene, they developed a naturally occurring molecular “turbo engine.” This turbo ensures that the healthy piece of DNA works efficiently within the muscle cells.

Philanthropy enables sustainable research

Safety and efficacy are major challenges in gene therapy. The funds are used to improve these aspects and further test the therapy in animal models, which have already shown positive results. This represents a first and necessary step toward testing in patients. In addition, the technique can also be applied to other hereditary muscle diseases, such as Steinert’s disease.

The establishment of the fund was made possible thanks to the support of Duchenne Parent Project Belgium and the commitment of Luc Vandevyvere, initiator of Cycling Against Duchenne.

Prof. Dr. Ir. Hugo Thienpont, Vice-Rector for Innovation & Valorization: “After the many years of commitment from our researchers and donors, this fund is the cherry on top. Through the fund, VUB aims to give gene therapy research a boost with a view toward clinical testing. In short, thanks to philanthropy, we can make a difference for Duchenne patients and their families.”