Thank You - We Did It
In this journey called “life” we all experience tears of joy, pain and sadness. Three years ago my wife and I experienced tears of immense grief, sadness and despair when we found out that our youngest son Michael was diagnosed with a terrible neurodegenerative disease, SPG50. It is a disease with no treatment options available of any kind.
All our hopes and dreams for the life we envisioned for our son were wiped out within a second after the doctors told us the disease would slowly take away both his mobility and mind. It was at that moment a piece of our souls were taken from us. Like any parent, we knew instantly that “We would do anything, Go Anywhere and Give Up Everything To Save Him.”
As soon as we got home we began researching the disease, finding other families whose children were afflicted and started mapping out a plan. A month later we began flying around the world, meeting with experts that specialized in Michael’s disease and leaders in innovative treatment options. That journey led us to fortunately meet and collaborate with two amazing doctors, Dr. Steven Gray and Dr. Xin Chen. Over the course of 3 years these amazing doctors, in addition to the team at UT SouthWestern Medical Centre Dallas led by Dr. Minassian along with Dr. Bonnificiano’s Lab at the NIH and Dr. Shahin’s Lab at Boston Children’s Hospital moved heaven and earth, even throughout the Covid pandemic to ensure that the experiments, tests and designs required to get to a clinical trial, were performed.
At the same time that this was happening, back home in Toronto our incredible community was helping us raise funds so that we could afford to make this Gene Therapy from scratch.
Through Golf Tournaments, Gala’s, Soccer Tournaments, BBQ’s, Beer Market, Gyro Night, Bowling Tournaments, Pop Up Clothing stands, Bike Rides, Bottle Bob, Hackathons, Church Donations, Danceathons, GTACC Conferences, Marathons/Triathalons, Bingo/Raffles Nights, Lemonade Stands, Garage Sales, local and worldwide events, money boxes and so much more.
The incredible love and support the community showed our family and the children afflicted with SPG50 helped lift our spirits and gave us strength during our darkest days.
Once the team at UTSW, NIH and Boston Children’s proved that the drug could work and had the potential to change the lives of children affected by SPG50, we hired consultants, worked with companies for safety testing, made raw material for the drug and manufactured the treatment into an approved first-ever human Gene Therapy for SPG50 in Canada.
This achievement was made possible by the hard work of the following talented teams from the below labs, companies and individuals:
Dr. Gray’s Lab - UTSW (Dr. Chen, Dr. Rodriguez, Roxana Ploski, Eric Shih, Ben Eckert, Yuhui Hu, Dr. Dong)
Dr. Bonnificiano’s Lab - NIH(Dr. De Pace, Dr. Mattera, Dr. Mardones),
Dr. Shahin’s Lab - Boston Children’s (Dr. Ebrahimi-Fakhari, Dr. Angelica D’Amore, Helena, Dr. Wardiya Afshar Saber, Dr. Marvin Ziegler, Hellen Jumo, Catherine Jordan, Dr. Afshin Saffari)
Dr. Robinson’s Lab - Cambridge University (Dr. Hirst, Dr. Davies)
Dr. Ostrowski’s Lab – University Of Toronto (Dr. Samaan, Dr. Yue)
Jax Labs (Dr. Lutz, Dr. Zuberi)
Aldevron (Dr. Berg, Dr. Chambers, Shiney Mathews, Sharon King)
Ultragenyx (Dr. Kakkis, Dr. Bedrosian, Dr. Flagella, Dr. Schutten, Dr. Weiss, Dr. Fyffe-Maricich, Dr. McKeever, Paul Wickman)
Spark Therapeutics (Dr. Levy, Dr. Savola, Dr. Mingozzi)
Viralgen (Dr. Samulski, Dr. Garcia, Dr. Pignataro, Renee Hermer, Becky Raymond, María Orio, Ana Vázquez, Andy Holt, Jone Herrero de Miguel, Dr. Trigueros Fernandez)
Columbus Children’s Foundation (Laura Hameed, Dr. Samulski, Dr. Garcia)
SickKids Hospital (Dr. Dowling, Dr. Cohn, Keshini Devakandan, Chris Viney, Eriskay Liston, Tomasz Czarny, Nisha Mechery, Dr. Malkin, Dr. Subbarao, Dr. Miller)
Charles River (Dr. Black, Dr. Cinquino, Dr. Douville, Zach Bousies, Barbara Killian, Frank Alleruzzo)
Cellular Technology Limited (Dr, Lehmann, Dr. Tary-Lehmann, Dr. Kirchenbaum, Dr. Subbramanian)
Dr. Balderson, Dr. Putnam, Dr. Boshoff, Dr. Bonnemann, Dr. Saade, Dr. Brooks, Dr. Parise, Dr. Galasinski, Dr. Ekins, Dr. Kher, Deanna Portero, Dr. Austin, Dr. Kampranis and so many others.
I apologize for anyone that I have mistakenly left out.
Thank you to the incredible team at SickKids (Dr. Dowling, Dr. Cohn, Keshini Devakandan, Chris Viney, Eriskay Liston, Tomasz Czarny , Nisha Mechery, Dr. Malkin, Dr. Subbarao, Dr. Miller, IRB Board and all the nurses, Drs. and staff) who not only took care of Michael, but stood by us from the day he was diagnosed to the day he received his Gene Therapy.
On March 24th Michael received the Gene Therapy that we all worked so hard to create. Thank you to everyone’s unwavering support, encouragement, and belief that the impossible can be made possible! We would like to extend a special thank you to Health Canada and the FDA for working with us and being so very understanding and accommodating.
Our journey, however, does not end with Michael but is only the beginning! Thank you to the Heros at Columbus Children’s Foundation & Viralgen for being able to make 6 doses with a commitment to manufacture another batch to potentially treat 10 or more children in total. Thank you to the unwavering commitment from the team at University Of Texas SouthWestern Medical Centre (Dr. Minassian, Dr. Iannaconne, Dr. Gray, Dr. Chen, Dr. Messahel, Dr. Greenberg, Dr. Batley, Dr. Christie, Dr. Bordes Edgar, Dr. Dr. Varadarajan and so many others). With your dedication and expertise children should begin to get treated later this year.
Due to the incredible generosity of over 23,000 donors, companies and foundations from around the world we were able to raise just over $3.5M CDN. The fact that in under 3 years we were able to build a drug from scratch, safety test, manufacture and getting our clinical trial approved by Health Canada and the FDA is truly incredible. However, all of this could not of happened without YOU!
Proof Of Concept & Natural History Study = $400K USD
Raw Drug Material & Manufacturing = $1.5M USD
Safety Testing & Studies = $690K USD
Consulting & Other Costs = $82K USD
Donation & Credit Card Fees = 50K USD
Total Raised/Spent = $2.722M USD or $3.53M CDN
Although we raised enough money to create and manufacturer the drug for Michael – there are many more children with SPG50 that need to be treated. In order for us to be able to treat at least 10 more children we will need to raise US$250,000 per child to cover the 5 year study and hospital costs.
We are asking you to please open your hearts one last time and donate what you can to our cause to ensure other children are able to receive the treatment and live a better life. Our journey began as “A Journey To Cure Michael” but together it can be “A Journey To Cure Every Child with SPG50!”
Canadian Citizens – https://www.curespg50.org/donate
Rest Of The World - https://www.gofundme.com/f/cure-michael-curespg50-spg50
To Dr. Gray, Dr. Kakkis & Jason Wolkove we want to thank you for always being there for us no matter the ask, no matter the time!
Words cannot express our gratitude, thank you to our friends, family, parents and everyone that has been there for us, supported us, cared for us and prayed for us along this crazy journey. Michael is still not out of the woods and will have a long road ahead with therapies, but with a lot of luck and prayers we are hoping he will grow up to lead a normal life.
May God bless you and your families with health and happiness
The Pirovolakis Family
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