Indian-origin family in Canada seeks $5.5 million for US gene therapy they hope will help their toddler with rare genetic disorder
Despite being 2.5 years old, Zorawar Singh Chandi remains like a newborn as he is suffering from a rare genetic disease. Family turns to US
Despite being 2.5 years old, Zorawar Singh Chandi remains like a newborn as he is suffering from a rare genetic disease. Family turns to US for gene therapy Join conversation View All Comments → Share your thoughts in the comments Post Comment Be respectful · TOI community guidelines $5.5 million fundraising appeal An Indian-origin family in Canada's Brampton is trying to raise $5.5 million to take their two-year-old son to the United States for a specialized gene therapy that they hope could transform his life after he was diagnosed with a rare genetic disorder. Mandeep Chandi and his wife Ramandeep are seeking funds for their son, Zorawar, who suffers from Aromatic L-amino Acid Decarboxylase (AADC) deficiency, a rare genetic condition that affects the nervous system and prevents the brain from producing an enzyme needed to make essential neurotransmitters.
The disorder affects the production of dopamine and serotonin, chemical messengers that play a crucial role in controlling movement and other bodily functions.At two-and-a-half years old, Zorawar has been unable to achieve basic developmental milestones such as sitting, walking and talking.Ramandeep Kaur, his mother, told CTV News that every day is a challenge for Zorawar. Several times a week, his eyes will stay rolled upward for up to 30 minutes, which is painful for him. And he can't chew food. So he is still on a liquid diet. “Sometimes he is very irritated, cry all the day, and he is not able to — like he can’t say anything about what is going on with him,” Kaur said."He's just in a newborn situation right now, his brain isn't able to get the proper function," his father Mandeep told INSAUGA Canada.
"He's not able to walk, talk or sit. He can't do anything, he just sits in his mom's lap 24/7."Mandeep, who moved to Canada from India in 2015, said caring for their son has been an emotionally and financially challenging experience for the family.The family is hoping to access a treatment called KEBILIDI, a gene therapy approved by the US Food and Drug Administration (FDA) for patients with AADC deficiency. The treatment, developed for the rare disorder, is administered directly into the brain and is designed to address the underlying genetic problem.According to Boston Children's Hospital, KEBILIDI became the first FDA-approved gene therapy for AADC deficiency. However, the treatment is not approved in Canada.Mandeep believes the therapy could give his son a chance at achieving developmental milestones that have so far remained out of reach.The family says the procedure itself takes approximately eight hours, with potential improvements becoming noticeable over the following months.
However, treatment requires specialist assessment, and outcomes cannot be guaranteed.The family had approached Ontario's Ministry of Health seeking funding for treatment outside Canada, but its application was denied.With no other viable option currently available to them, the parents have turned to crowdfunding to raise the money needed to pursue treatment in Boston.The family has launched a GoFundMe campaign with a target of $5.5 million Canadian dollars. As of November 14, the fundraiser had collected more than $1.5 million, with approximately 29% of its target reached.The family has appealed to the community to support their efforts, saying they want to give their son an opportunity to access treatment that could potentially improve his quality of life.
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