Brampton father Mandeep Chandi is trying to raise $5.5 million for a gene therapy treatment for his son, Zorawar. (Photos: GoFundMe)<\/p>\n
Expensive gene therapy considered experimental in Canada could help a Brampton toddler who is unable to walk due to rare and serious genetic disorder.<\/p>\n
\u201cIt affects his nervous system, so his body is not able to produce dopamine and serotonin,\u201d Mandeep Chandi said of his son Zorawar in an interview with INSAUGA Canada.<\/p>\n
Zorawar has Aromatic L-amino acid decarboxylase (AADC) deficiency. It\u2019s a rare genetic disorder that affects the brain\u2019s ability to produce an enzyme needed to make neurotransmitters \u2013 chemical messengers that help control mood, movement, and other essential functions.<\/p>\n
At two-and-a-half years old, most toddlers are walking and talking. But Chandi says his son hasn\u2019t hit any of those milestones due to the rare genetic disease.<\/p>\n
\u201c(Zorawar\u2019s) just in a newborn situation right now, his brain isn\u2019t able to get the proper function,\u201d Chandi says. \u201cHe\u2019s not able to walk, talk or sit. He can\u2019t do anything, he just sits in his mom\u2019s lap 24\/7.\u201d<\/p>\n
Chandi moved to Canada from India in 2015 and says English is his second language, and says becoming first-time parents dealing with the medical system has been stressful for him and his wife Ramandeep. She\u2019s been his constant caregiver, with help from Mandeep\u2019s mother.<\/p>\n
And while Mandeep says there\u2019s a treatment that\u2019s been approved in the U.S. which could have Zorawar up and walking within a year, the treatment isn\u2019t approved in Canada.<\/p>\n
It\u2019s called KEBILIDI, and is the first AADC deficiency approved by the U.S. Food and Drug Administration, and the first approved gene therapy to be given directly into the brain, according to the Boston Children\u2019s Hospital.<\/p>\n
Mandeep says an application for out-of-country funding to the Ontario Ministry of Health was denied, and it will cost the family more than $5.5 million to make the trip to Boston for the treatment.<\/p>\n
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\u201cWhile treatment requires specialist assessment and results cannot be guaranteed, we are fighting for Zorawar to have the opportunity to be evaluated for this therapy and receive it if he is eligible,\u201d reads a GoFundMe page started by the family to raise funds for the procedure<\/a>.<\/p>\n The therapy only lasts about eight hours, and could see results in four months, Mandeep says.<\/p>\n The Boston Children\u2019s Hospital says that, without treatment, AADC deficiency \u201cis a progressive, life-shortening disorder that can lead to severe disability.\u201d<\/p>\n But with early diagnosis and gene therapy, \u201cthe outlook has improved for some children.\u201d<\/p>\n \u201cWhile gene therapy has shown positive results in clinical trials, the long-term benefits are still being evaluated,\u201d the hospital\u2019s website reads.<\/p>\n The family is hoping to raise $5.5 million, and had collected more than $23,000 as of Nov. 4. Donations will help with medical assessments, treatment-related expenses, travel, accommodation, transportation and necessary follow-up care, the family says.<\/p>\n