HD Clinical Trials

  Disappointing news for the Huntington’s disease community from Novartis Pharmaceutical Company regarding stopping the clinical trial VIBRANT-HD.  Read full article here

“Tucked into its latest earnings report, Novartis confirmed to investors that it has stopped developing an experimental drug for Huntington’s disease.

The reason, according to Novartis, was an “overall assessment of the risk-benefit profile” observed in a mid-stage clinical trial named VIBRANT-HD. In August, Novartis said it had temporarily suspended dosing in the study, after discovering that some patients treated with the drug, known as branaplam, were experiencing nerve damage.”

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We Can Never Lose Hope…           

Annexon, a clinical-stage biopharmaceutical company, located in the San Francisco Bay Area, is a company with good news for the Huntington’s disease  (HD) community.  HD is a rare, fatal, genetic brain disorder that strikes young and has symptoms of ALS, Parkinson’s disease and Alzheimers at the same time.             

The local biotech company is developing a new class of complement medicines for patients with classical complement-mediated  autoimmune, neurodegenerative and ophthalmic disorders announced on June 7, 2022, the final data from its open-label Phase 2 clinical trial of ANX005 in patients with Huntington’s disease (HD).  Read the full news release here

The progress on ANX005 for Huntington’s disease is one of the green arrows below.

The following is a summary of the results of the Phase 2 clinical trail of ANX005.

-ANX005 Demonstrated Full C1q Target Inhibition and Was Generally Well-Tolerated

-Disease Progression Stabilized in Overall Patient Population Through the Nine-month Study

-Rapid Improvement in Clinical Outcome Measures Maintained in Patients with High Baseline Complement Activity Through the Nine-month Study

I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author.         

Purchase my book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s.  Find it on:  Amazon, and many online book stores.  100% of book sales is donated to Huntington’s Disease Society of America.

Some good news for the Huntingtons disease community around the world from the company Bayer. 

Huntington’s disease is a rare, fatal, genetic brain disorder that has symptoms of ALS, Parkinson’s and Alzheimers at the same time and has NO CURE.

Bayer aspirin, a name familiar to me that resided in our medicine cabinet when I was growing up in Kansas.  When I was very young and had a fever, I remember my mother touching my forehead, picking me up and setting me on the kitchen countertop and giving me a spoonful of sugar with a dissolving Bayer aspirin in it. (not recommended today for young children because it is linked with Reye’s syndrome) My father took a low dose of Bayer aspirin every morning for years for his heart, as it is recommended by many physicians.  Bayer aspirin is one of several brand names that have become generic words for a type of product; i.e. xerox, Google.

After receiving sad news a couple of weeks ago from Novartis, they temporary suspended the clinical trial on Liquid Branaplam, a drug for Huntington’s disease, we can use some good news. 

On August 23, 2022, this article was release in Pharmaphorum: 

“France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease“.

I’m a Huntington’s disease (HD) advocate, Chair for the HDSA San Francisco Bay Area Affiliate, a blogger and an author.         

Purchase my book, Watching Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s.  Find it on:  Amazon, and many online book stores.  100% of book sales is donated to Huntington’s Disease Society of America.

Author Therese Crutcher-Marin, HD Advocate

Last Thursday, August 11th, I watched a HDSA Research Webinar: HD Clinical Trial Update with Arik Johnson, PsyD (HDSA’s Chief Mission Officer), Leora Fox, PhD (HDSA’s Assistant Director of Research & Patient Engagement), Jennifer Simpson, LCSW (HDSA’s Assistant Director of Youth & Community Services) and Victor Sung, MD (HDSA’s Chairman of the Board of Trustees) discussed recent Huntington’s disease (HD) clinical trial news. 

Last week Novartis Pharmaceutical Company announced VIBRANT-HD trial of the oral huntingtin-lowering drug, branaplam was temporarily suspended for the safety of participants.

Also last week, UniQure Biotech Company announced that it has paused enrollment in its higher-dose cohort receiving AMT-130 due to the occurrence of suspected unexpected severe adverse reactions.

Over the next several weeks I will update the progress on the drugs in clinical trials that are being conducted around the world for Huntington’s disease (HD).                               

The companies/drugs I will be sharing  are:

Prilenia-HD Proof Improves day-to-day function, movement & behavior in HD

Roche-Tominersen  May help younger people with HD

PTC Therapeutics-PTC-518 Designed to selectively lower huntingtin mRNA and protein

Novartis-Vibrant-HD (branaplam) Lowers levels of the huntingtin protein 

UniQure-AMT-130 Gene therapy

Neurocrine-Kinect HD Treatment of chorea

Sage-Sage-718 Cognitive function

We Can Never Lose HOPE……

Author Therese Crutcher-Marin, HD Advocate

If you are considering joining a clinical trial for Huntington’s disease (HD) but don’t know how  to find the right clinical trial for you, please go to HDSA  HD Trial Finder sponsored by the nonprofit Huntington’s Disease Society of America (HDSA).

HD Trial Finder is a clinical trials matching service: a way for individuals with Huntington’s disease, caregivers, healthy volunteers, and physicians to connect with current research studies. It includes an easy-to-use website and free call center staffed by trained HD clinical trial navigators.

Hope for tomorrow begins with your involvement today!

People with Huntington’s Disease, care partners, and healthy volunteers are all needed today to participate in different aspects of HD research. By creating a profile and answering some basic questions about yourself or a friend, family member, or patient who is impacted by HD, you can match to studies and find contact information for nearby sites. You can also use HD Trial Finder to read about ongoing trials without entering any information.

We Can Never Lose HOPE….

Silencing the mutant huntingtin gene 

A promising gene therapy for a rare, devastating disease             

uniQure has begun dosing patients in a dose-escalating, randomized and controlled Phase I/II clinical study to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington’s disease (HD).

This investigational treatment is the first one-time administered gene therapy to enter clinical testing for the treatment of HD. AMT-130 is administered only once by neurosurgical procedure.

If you live in the San Francisco Bay Area, the University of San Francisco Medical Center is involved in this clinical trial.

Read here:  UniQure press release with clinical update on HD patients in the trial Dec. 16,2021.   

Frequently asked questions about AMT-130

Animated illustration of how AMT-130 is administered into the caudate and striatum regions of the brain via a micro-catheter   Watch it here

We Can Never Lose HOPE……