Hi friends 👋🏼,

You might have noticed I disappeared from social for a few weeks. A lot happened: I got a new job, moved into a new house, and went on vacation. Here’s a silly photo of my wife and me on vacation that was supposed to be a piggyback ride, but she had other ideas 🙄.

But this newsletter’s not about me; it’s about you, so let’s dive in…

• Paraplegic man walks again with brain-spine bridge

• Atrial Fibrillation, Blood Clots, and Factor XI ^{READER REQUEST}

• Antibody Therapy for Wegeners Granulomatosis ^{READER REQUEST}

• Custom Gene Therapy for PYCR2? ^{READER REQUEST}

🦼Paraplegic Man Walks Again with Brain-Spine Bridge

Paraplegia is a loss of muscle function in part of your body. For example, Gert-Jan Oskam (pictured above) lived with paralyzed legs and partially paralyzed arms after an accident 12 years ago damaged the spinal cord in his neck.

But now, he is back on his feet and walking, thanks to a device developed by NeuroRestore, that creates a ‘digital bridge’ between his brain and the nerves below his injury.

How does the brain-spine interface work?

1. First, they used advanced imaging technology to locate the parts of his brain that lit up due to the intention to want to move his legs. Additionally, they imaged his spine to get a 3D structure.

2. These images were used to predict the optimal locations for cortical implants and a spine paddle, which were surgically implanted into his brain and spine.

3. The technology used advanced computational models to read the brain signals and predict the intention to walk, as well as the scale and directionality of movement.

4. The signals were then sent to a computer Oskam wore as a backpack that relayed the call to stimulate his lower spine and ultimately create movement in his legs.

@crisprclassroom

Imagine wiggling your toes again…. 🤯 #paraplegia #biomedicalscience #science

❤️ Atrial Fibrillation, Blood Clots, and Factor XI

Atrial Fibrillation is a problem with the speed or rhythm of the heart that can lead to an increased risk of blood clots and subsequent stroke.

1/ While several treatments for Atrial Fibrillation are available, from antiarrhythmics to surgery, they are often accompanied by oral anticoagulants to prevent the formation of blood clots and stroke.

2/ Oral anticoagulants prevent blood clots from forming in all cases, even when necessary, like when you get a cut. As a result, patients with atrial fibrillation who are put on oral anticoagulants are at significant risk of bleeding.

3/ Anthos Therapeutics has developed a new antibody treatment that targets a protein in the clotting pathway called Factor XI. Interestingly, recent research has shown that Factor XI is essential to form blood clots internally but does not participate in wound repair/bleeding control at the surface. As a result, by targeting Factor XI, Anthos is attempting to develop a new class of anticoagulants that will not significantly increase the risk of bleeding but will prevent blood clots from forming within blood vessels that ultimately can lead to stroke.

4/ While this new treatment is not directly a “cure” for atrial fibrillation, it does help manage symptoms to prevent stroke.

5/ This new antibody targeting Factor XI is being tested in the LILAC-TIMI clinical trials (NCT05712200). Talk to your doctor or contact Janeen at Anthos ([email protected]) for more information.

🌬️ Antibody Therapy for Wegeners Granulomatosis

Wegeners Granulomatosis is a rare disorder where blood vessels in the lungs, kidneys, nose, sinuses, and ears become inflamed. While we don’t know the cause, it is an autoimmune condition.

1/ GSK is comparing two proprietary treatments, Depemokimab and Mepolizumab, head to head for their effect on Wegeners Granulomatosis.

2/ Both Mepolizumab and Depemokimab are monoclonal antibodies that target an immune stimulatory factor called interleukin-5 (IL-5) and inhibit its activity. As a result, they may help to reduce the overactive immune system part of Wegeners.

3/ Mepolizumab is already an approved therapy for treating severe asthma and is sold under the name Nucala. Interestingly, Depemokimab is in late clinical development and may soon be approved for asthma treatment.

4/ Now, in clinical trials, GSK is testing whether these treatments can be applied to other autoimmune conditions, specifically Wegeners Granulomatosis (NCT05263934). To get involved, talk to your doctor or contact GSK at [email protected].

❓Custom Gene Therapy for PYCR2?

PYCR2-related disorders are very rare. Probably the rarest we’ve ever written about here at The Cure. In fact, after extensive searching, we found no clinical trials related to the disorder. Even so, is there no hope? It’s tough to say; here is what we do know:

1/ What does the PYCR2 gene do? PYCR2 is part of the synthesis pathway that allows our body to make proline, an essential amino acid. Defects of proline synthesis significantly harm health, leading to intellectual disability, skin and joint hyperelasticity, osteopenia, and cataracts.

2/ One option is to develop a personalized PYCR2 treatment for a specific individual. This is very hard because it relies on the patient's family to recruit scientists/doctors and fund the research. But it has been done before:

• Mila Makovec was born with Batten disease, a horrible and deadly condition. Her mother created a non-profit, raised funds, and recruited scientists/doctors who successfully developed a personalized gene therapy for Mila called Milasen. Nevertheless, even after treatment with Milasen, Mila did pass away.

• Terry Horgan was born with Duchenne Muscular Dystrophy. His brother, Rich, created a non-profit, raised funds, and recruited scientists/doctors who successfully developed personalized gene therapy for Terry. Unfortunately, Terry passed away shortly after receiving it.

3/ You can notice some common themes in these stories: While it’s possible to develop custom gene therapies, it takes a lot of commitment from the family without any guarantee of success.

To the person who requested this topic, I am very sorry we could not provide better news. However, we have our Google alert set, and if any new research is published on PYCR2, we will be the first to inform you.

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