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A Treatment for Henry
While there historically has not been a treatment for AHC, Henry’s parents have discovered a treatment for Henry with the support of his medical team at Boston Children’s Hospital. After developing this treatment, called an ASO, doctors would be able to use it as a blueprint to treat others with AHC and other rare neurological diseases.
Antisense oligonucleotides, or ASOs, are short strings of DNA or RNA letters that can bind to the mRNA to modify gene expression in the nervous system. ASOs can knockdown (reduce) or upregulate (increase) protein expression, depending on the functional consequence of the mutation and how to correct it.
We believe a knockdown ASO will be most useful to treat Henry as they may be particularly useful for mutations that are dominant negative or which make a toxic protein which adversely affect the wild type (or healthy gene) product in the same cell. These ASOs can “knockdown” such mutations and eliminate the adverse effects. To date, the FDA has approved about ten ASO treatments for genetic disorders and there is a precedent for such treatments to go from idea to injection in under one year.
This means Henry could be treated with an ASO in the next 12-18 months, changing his life forever.
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Treatment Update Newsletter - August 2023
Written by Henry's Parents on 8/16/2023​
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Dear Dedicated For Henry AHC Friends,
It’s hard to believe that it’s been a year and a half since we embarked on this rollercoaster journey to save Henry’s life! As Summer 2023 comes to a close, we wanted to share this update with you as supporters and friends of Henry because you too are a part of his life!
Ok, some complicated science stuff is coming up but it’s hugely important to finding Henry’s cure!
With your support, we sponsored work in Dr. Alfred George's lab at Northwestern University to conduct two proof of concept in vitro experiments to determine if an ASO is a suitable treatment option for AHC and to test ASOs in patient neurons to see if we can achieve a therapeutic result (therapeutic = lifesaving!) We collaborated with our three other US AHC organizations -- AHCF, CureAHC, and Hope for Annabel – cosponsoring the first experiment, while we underwrote the costs of the second experiment which was conducted specifically in Henry's neurons.
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And we have awesome news!! Dr. George and his team have found that indeed, a knockdown ASO is a suitable strategy to treat AHC, which is truly amazing news! This finding not only impacts Henry but others in the AHC community as well. This means that your support of Henry could very well treat other kids too!
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While Dr. George was busy with this first proof of concept experiment, Charles River Laboratories designed ASO sequences for Henry (these are all of the different types of ASOs that could work to knock down his bad copy and preserve his good one). Dr. George then took these ASO sequences and is testing them in Henry’s neurons to see which ones are the most effective.
Dr. George is still in the process of testing the ASOs in Henry's neurons and we hope to have news on the top candidates in the next month. Most significant, Dr. George has found that our gene is amenable to a knockdown ASO in practice, NOT just in theory. We can’t wait to find out the results of Dr. George’s experiments and look forward to sharing with you soon.
March brought incredible, non-scientific news when CNN did a story about Henry both on their website and through two interviews which can be found here and here. This media coverage raised us a whopping $600K in just over 24 hours and reminded us just how many truly good people there are in the world who are so devoted to being our partners to help Henry. Even months later, we remain humbled that not only Henry, but we as his parents have received such an outpouring of love and support from people like you from across the globe.
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As for Henry, he has been enjoying a summer with family on the lake in Vermont. He continues to amaze us with his resilience, persistence, and joy. He began attending preschool when he turned three in January and it has been wonderful to see him march us hurriedly and excitedly into school each morning he is well enough to go.
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We are, of course, so excited to share the wonderful news. But (we’re so looking forward to that “but” to totally disappear), Henry still experiences life threatening seizures every few days, averaging 2-5 a week! He stops breathing with every seizure – as parents, they’re terrifying even though we’re pretty much used to them by now. Paralysis and apneas have also made a comeback, and the sight of Henry losing the ability to move an arm, or his entire body, or to not be able to breathe breaks our hearts every time.
We WILL stop AHC in its tracks. We have to! We have to work as quickly as possible to treat Henry before he suffers permanent brain damage from this awful disease.
Despite these awful symptoms, Henry continues to progress. He is able to walk now holding just one of our hands, he has begun to use a special iPad to help him communicate in addition to learning new words, and his fine motor skills continue to progress.
While AHC continues to threaten Henry's life way too often, there is much hope that we can treat Henry in the next year and stop the progression of this disease.
So far, with your remarkable help, we have raised $1.27 million. We need to raise another $1.73 million to take us through toxicology, manufacturing, and get to a clinical trial to dose Henry. It’s a long way off, but we’ll get there! We have to!
This October, we’re going to be doing something big! And we’re going to need your help to get it done! Stay tuned for details to follow. If you’re interested in stepping up for Henry and making a difference in his little life, email me at mary@forhenryahc.org so we can chat, and I can fill you in on the details.
Thank you for continuing to share Henry's story with everyone you know, for your messages of kind support, and for your love of Henry. We are so grateful.
Mary and Anthony
& Team For Henry AHC
