Gurmoh is a joyful three-year-old boy from Surrey who dreams of becoming a firefighter one day and drives his toy truck around the house while “proudly wearing his fire hat,” but time is not on his side, his parents say.
Gurmoh was diagnosed with spastic paraplegia, which is caused by a de novo mutation (meaning a genetic mutation not present in either parent). This mutation is often associated with severe early onset of the disease, and kids with it can experience progressive muscle stiffness, speech, and weakness in the legs. It affects nerves in the spinal cord that control movement.
Gurmoh’s parents, Stalin and Navpreet Gill, are working to raise $2.7 million to help pay for the creation of personalized gene therapy for Gurmoh, who has the only known case of the rare and progressive neurodegenerative disease in Canada.
Navpreet told the Now-Leader that the condition impacts Gurmoh’s ability to walk, speak and maintain cognitive function. He is currently independent but struggles with stairs and uneven ground.
“Despite this, Gurmoh remains a joyful child who loves music, especially ‘Wheels on the Bus,’” she said. “Time, however, is not on his side. Without urgent treatment, his condition will continue to progress, leading to severe and irreversible loss of abilities.”
There is currently no approved therapy due to the rarity of his condition, which is why the family is raising funds for personalized treatment.
The funds raised would go towards the estimated costs of developing and delivering the gene therapy. Navpreet said that McGill University has already committed to developing a personalized gene therapy for their son.
The family is working to raise $2.7 million “to initiate and accelerate the development of his gene therapy.”
But the total costs will be around $6 to $8 million to develop, research, and run pre-clinical and clinical trials.
“Gene therapy has already been successfully developed for similar conditions, such as SPG50, demonstrating that treatment is possible,” said Navpreet.
Gurmoh’s care currently costs the family $6,000 to $8,00 a month, which includes therapies, specialized equipment, and frequent travel for intensive treatment.
“After this one-time gene therapy treatment, he will be cured of this disease and could lead a normal life,” she said.
“Every second his nerves are dying because of this disease and they cannot be recovered back. He needs the treatment as soon as possible. If the funding doesn’t come in time, it will not be possible to save him from this disease,” Navpreet said.
The family has also created a website where they share their journey: spg4hope.org.