Canadian Family Races to Fund Personalized Gene Therapy for Son's Rare Neurodegenerative Disease
Key Insights
A three-year-old Surrey boy has been diagnosed with Hereditary Spastic Paraplegia Type 4 (search) (HSP), a rare progressive neurodegenerative disease with no known cure.
McGill University's Dr. Ziv Gan-Or (search) has committed to developing a personalized gene therapy, with total costs estimated between $6 and $8 million.
The family has raised $2.4 million toward the initial $2.7 million needed to kickstart therapy development, including a 120-km walk from Vancouver to Victoria.
A Surrey, British Columbia family is in a desperate race against time to fund a personalized gene therapy that could preserve their three-year-old son's ability to walk, talk, and connect with the world. Gurmoh Gill was diagnosed earlier this year with Hereditary Spastic Paraplegia Type 4 (search) (HSP), a rare, progressive neurodegenerative disease caused by a de novo mutation — a genetic alteration not present in either parent.
The diagnosis came after a long and frustrating medical journey. When Gurmoh struggled to walk independently at 1.5 years of age, his parents, Dr. Stalinjeet Gill and Dr. Navpreet Sangha, were initially told the issue would resolve on its own. Instead, his condition worsened. After his legs completely gave out, he was rushed to B.C. Children's Hospital and initially diagnosed with cerebral palsy. The correct diagnosis of HSP Type 4 was not confirmed until April of this year.
A Race Against Progressive Decline
Hereditary Spastic Paraplegia Type 4 (search) is the most common form of HSP, a disease that affects approximately five out of every 100,000 people — translating to roughly 2,000 patients across Canada. The condition causes progressive weakness and stiffness in the legs, and in Gurmoh's case, the de novo mutation is associated with severe early onset.
"He is expected, as this disease progresses, to lose his ability to walk, his ability to talk, his ability to connect with people and he would be completely dependent on his environment and his caretakers," said Dr. Ziv Gan-Or (search), clinical director at The Neuro (Montreal Neurological Institute-Hospital) at McGill University.
Personalized Gene Therapy in Development
Faced with no known cure, Navpreet Sangha sent approximately 2,000 emails to researchers around the globe. Dr. Gan-Or responded and committed to developing a personalized gene therapy for Gurmoh at McGill University. The total cost to develop, research, and run pre-clinical and clinical trials is estimated between $6 and $8 million.
The family reached out to both the B.C. provincial government and the federal government seeking coverage, but the province informed them it could not fund the treatment. Undeterred, the Gills launched an intensive fundraising campaign.
Community Mobilization and Fundraising Progress
In just seven weeks, the family raised $2.2 million through an online fundraiser, community support, radiothons, and a 120-kilometer walk from Vancouver to the B.C. Legislature in Victoria. As of the latest reports, total funds raised have reached $2.4 million, approaching the initial $2.7 million goal required to initiate and accelerate the development of Gurmoh's gene therapy.
"This is the only thing we have on our mind, it's all we do, all we care about," Stalin Gill said.
Advocacy on Parliament Hill
The Gill family is now taking their fight to the nation's capital. From June 8 to 10, they will be at Parliament Hill near the Centennial Flame in Ottawa, advocating for "faster support and access to life-saving gene therapies in Canada." Dr. Gan-Or will join the family on June 8.
On the morning of June 8, Gurmoh and his family will meet at the Terry Fox memorial sculpture at 8:30 a.m. and walk to the Centennial Flame, arriving around 9 a.m. Members of the public are welcome to join the family on June 8, as well as at 9 a.m. on June 9 and 10 near the flame.
"Our message is simple: rare kids matter," said Gurmoh's parents. "Every child deserves an equal chance at life, hope, and health care."