HD Clinical Trials

SIGNAL Clinical Trial for Huntington’s Disease

Another Sponsor at the Huntington’s Disease Society of America in Boston last June was Vaccinex, Inc

Vaccinex and the Huntington Study Group (HSG) launched a trial for people with the gene mutation that causes HD who are either early in the progression of the disease or are not yet diagnosed with the disease.

SIGNAL, a Phase 2, multi-center, randomized, double-blind, placebo controlled study in subjects with early manifest and late prodromal Huntington’s disease (HD) to assess the safety, tolerability, pharmacokinetics, and efficacy of VX15/2503 (pepinemab). Pepinemab a monoclonal antibody that is a potential treatment for Huntington’s disease (HD).  What-is-hd/overview-of-huntingtons-disease/

On January 16, 2019, Vaccinex, Inc. announced the enrollment was complete and includes two cohorts with a total of 265 Huntington’s Disease subjects – 179 in group 1 (B1) who have early manifest disease and 86 in group 2 (B2) who are late prodromal.  All subjects are randomized to receive monthly infusions of either VX15/2503 (pepinemab) or placebo for 18 months in double-blind fashion without crossover.

To read more about the SIGNAL trial, visit http://ir.vaccinex.com/news-releases/news-release-details/vaccinex-inc-announces-completion-enrollment-its-signal

To read FAQ regarding about the trial visit: https://huntingtonstudygroup.org/signal-faq/

Please visit my Author Website to read my blogs about Huntington’s disease and my personal experience  https://www.theresecrutchermarin.com

We Can Never Lose HOPE……………….

Author Therese Crutcher-Marin

HD Advocates, Love

Huntington’s Disease Advocate Marjorie Guthrie

    

Phyllis Marin

The following is a letter I received from Marjorie, Woody Guthrie’s wife in 1978 when my then fiance, John Marin and his three (3) sisters discovered the family secret; their mother, Phyllis Iva Cahoon Marin, had Huntington’s disease (HD). What is HD?

Marjorie and five other volunteers succeeded in forming the Committee to Combat Huntington’ s Disease(CCHD), which was incorporated in the state of New York on September 18, 1967, as a nonprofit voluntary health agency. To read about Marjorie, go to https://hdsa.org/about-hdsa/hdsa-history/  The CCHD, later evolved into the nonprofit, HDSA, after her death in 1983. http://www.hdsa.org    

___________________________________________________________________________________________________

Dear Therese, 

I wanted to answer your letter to us personally because I really do appreciate your thoughts and the fact that you are not deserting your young man and are looking for ways to HELP!  That is a wonderful attitude….and I am sending you a packet of information about CCHD and the work we are doing.

I hope that after you read all this you might be “inspired” to JOIN WITH US….and perhaps in time….you might event help us build a chapter in your area.  The closest to you is in San Francisco.  But…for now…read…and learn what is going on and when you can…get involved. 

We need FRIENDS!  No one asks the cancer patients to go out and help…and we must realize that most of the HD families who are burdened with the problems of HD can not do all that must be done!  We look for friends, relatives, people who really care and you just might  be one of those!   I will be anxious to hear from you…..Until then….

LOVE & PEACE! In the NEW YEAR!

Signature of Marjorie Guthrie

___________________________________________________________________________________________________

We Can Never Lose HOPE…………

Therese at the 2018 HDSA Annual Convention in LA.

I became an HD Advocate the day I decided to marry John.  I wrote the nonfiction book, WatchingWatching the Dance Huntingtons Disease Their Dance: Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s to help in the fight against HD.

100% of the profits from the book is being donated to the nonprofit, Huntington’s Disease Society of America (HDSA). My author website is:  https://www.theresecrutchermarin.com

 

 

Palliative Care

The Confusing Word” Palliative Care”

The term “Palliative Care” has been confusing to folks for a long time. The last ten years of my healthcare career was in hospice at our community hospital and we used “palliative care” synonymously with “hospice care”.  As I was retiring, a change was happening in the healthcare world and the change could help folks with chronic illnesses, like Huntington’s disease (HD).

When attending the Huntington’s Disease Society of America (HDSA) Annual Convention in Boston Massachusetts last June, I loved that there was a session on Palliative Care.  If you would like to watch the session, it’s about 55 minutes long, please visit:  https://vimeo.com/345957809

Andrew Esch, MD was the presenter and he is board certified in Internal Medicine with a subspecialty in Palliative and Hospice Care.

 Click here to read about Dr. Andrew Esch

Palliative Care is not just a concern for Huntington’s Disease (HD) families, it’s information we all need when family, friends are facing their mortality.

So what is Palliative Care?  The Mayo Clinic definition is: “Specialized medical care that focuses on providing patients relief from pain and other symptoms of a serious illness, no matter the diagnosis or stage of disease. Palliative care teams aim to improve the quality of life for both patients and their families. This form of care is offered alongside curative or other treatments you may be receiving.”

Palliative care is provided by a team of doctors, nurses and other specially trained people. They work with you, your family and your other doctors to provide an extra layer of support that complements your ongoing care.

The Difference Between Palliative Care and Hospice. Both palliative care and hospice care provide comfort. But palliative care can begin at diagnosis, and at the same time as treatment.

Kaiser Permanente has a palliative care program.  https://healthy.kaiserpermanente.org/palliative-care

Sutter Health has a program:  https://www.sutterhealth.org/services/palliative-advanced-illness-management

Dignity Health:  https://www.dignityhealth.org/sacramento/services/palliative-care

Mercy:  https://www.mercy.net/service/palliative-care/

Check with your hospital system or talk with a Huntington’s Disease Social Worker in your area to find out more about when palliative care is appropriate for your loved one.

We Can Never Lose HOPE…………….

   100% of the profits from the book goes to the nonprofit HDSA.  I’ve gifted over $14,000.00 to HDSA since I published in 2017.  You can find it on many book websites like Amazon:

https://www.amazon.com/Watching-Their-Dance-Marrying-Huntingtons/dp/0998442208

 

 

 

 

HD Research, Helping Others

Lundbeck

After I returned from the 2019 Huntington’s Disease Society of America  Convention in Boston in late June, I began wondering how and what the partners (sponsors) of the event contribute to the Huntington’s disease community.

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

On the back of the Convention Guide Book is a list of some of the partners. Today I’ll post about LUNDBECK.   lundbeck.com/us/about-us

Insight from Lundbeck’s Patient-Focused Approach to Business:

“Lundbeck offers financial support, and does it with a real understanding of what patient communities value and need: Patient groups are often strained for financial resources, which is why it’s not only important that we offer support generously, but in places where it can make the biggest impact. From many years of listening to patients, we have seen time and again how important it is for people to be able to connect with each other at patient group events and conventions. That’s why we introduced annual scholarships to help patients and their families attend the Huntington’s Disease Society of America (HDSA) Convention.”  

To read full article, visit:  lundbeck.com/us/our-commitment/community-involvement/moving-together-for-hd

Thank you to Lundbeck Pharmaceutical Company for offering scholarships to Huntington’s disease (HD) families so they are able attend the Annual HDSA Convention.  It’s so important for HD families to connect with the HD community so they do not feel alone.

We Can Never Lose HOPE…… 

         

Visit my Author Website at: https://www.theresecrutchermarin.com.  100% of the profits from the book, Watching Their Dance” is being gifted to the nonprofit HDSA.

The Woody Guthrie Family

More About Woodrow Wilson Guthrie

Woody Guthrie was a very interesting and talented man.  I think he accomplished more in his 55 years on this earth than most people do in 80-90 years.

During his short, tumultuous life Guthrie was a writer, union activist, performer, social commentator, story teller, and composer of over 1000 songs.  He was also a poet, and author. When John and I visited the Woody Guthrie Center in Tulsa Oklahoma, we saw his work.  He was a brilliant man.  https://woodyguthriecenter.org/

The timeline of Woody’s life, can be seen here: https://library.uoregon.edu/ec/wguthrie/timeline.html

I had the privilege to present a “book discussion” at the Woody Guthrie Center.  I had just published the nonfiction book, Watching Their Dance.

With that said, I’ll tell you about Woody’s private life.  Woody’s mother, Nora, had Huntington’s disease and was placed in Oklahoma Hospital for the Insane.

At age 19, Guthrie met and married his first wife, Mary Jennings, in Texas in 1931. They had three children together: Gwendolyn, Sue, and Bill.  Bill died at age 23 of an automobile accident. Each daughter died of Huntington’s disease at the age of 41, in the 1970s.  Guthrie and Mary divorced in 1940.

He married twice more, to Marjorie Greenblatt (1945–53), they had daughters Cathy and Nora; and sons Arlo and Joady.  Cathy died in a fire.

Woody married Anneke Van Kirkand (1953–56) and had a daughter; having a total of eight children.  https://en.m.wikipedia.org/wiki/Woody_Guthrie

Woody was hospitalized at Greystone Park Psychiatric Hospital in Morris County, New Jersey, from 1956 to 1961; at Brooklyn State Hospital (now Kingsboro Psychiatric Center) in East Flatbush until 1966; and finally at Creedmoor Psychiatric Center in Queens Village, New York, until his death in 1967.

 

We Can Never Lose HOPE……

100% of the profits from Watching Their Dance is being donated to the nonprofit, Huntington’s Disease Society of America. You can purchase it on Amazon and many other book websites.  My author website is https://www.theresecrutchermarin.com

 

Therese-Author

Miracles Happen Every Day

Sometimes we witness a miracle, a wonder, and overlook the meaning/purpose/impact that the experience can have on our lives. I think folks think of a miracle as a tremendous, jaw dropping event.  To appreciate these special moments in my life, I have changed my perception of what a miracle is.

An encounter I experienced this week at the Homeless Center was about a young man who I’ve seen many times.  I’m an interviewer, asking what services they need that day and I feel I’m a sounding board acknowledging their story, plans, and goals.  The young man,  who I have seen many times, shared that he had been sober for 14 days and that he was being admitted into a 90-day rehab program in a couple of weeks.  I consider this a miracle, so, I congratulated him, gave him a hug, and wished him well.

A while back, one of John’s cousin’s made a statement that made me ponder for a moment, and recognize that what happened in my life was indeed a miracle. The conversation was about divorces in our world today.  The cousin said, “It’s a miracle that you and John didn’t divorce with all you have been through”.  I had to agree.

                              

We Can Never Lose HOPE………….

I’m a Huntington’s Disease advocate having lost my three sisters-in-law to this horrific disease that has no cure.  100% of the proceeds from the book is being donated to the nonprofit, Huntington’s Disease Society of America (HDSA), to help in the fight against the disease.  What is Huntington’s disease:  http://www.hdsa.org

To purchase the book, go to https://www.amazon.com/Watching-Their-Dance-Marrying-Huntingtons.  It’s also available on many other book websites.

 

HD Clinical Trials

WAVE Life Sciences-An Update on PRECISION-HD Clinical Program

WAVE Life Sciences had a table at the Huntington’s Disease Society of America (HDSA) Convention in Boston last month and I spoke with a rep for quite awhile.  It’s very exciting what they are doing for Huntington’s disease (HD).

“We have designed stereopure oligonucleotides to selectively target the mutant HTT allele to lower the production of mHTT protein while leaving healthy HTT protein relatively intact.” https://www.wavelifesciences.com/

Huntington’s disease (HD) is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities usually during their prime working years and has no cure.  http://www.hdsa.org

A little bit of information:  We all have two Huntingtin genes; one from your father, one from your mother.  Say, you have a CAG repeat of 42 from your father and a CAG count of 17 from your mother.

The drug WAVE life Science is conducting clinical trials on, Phase 1b/2a, only lowers the mutated Huntingtin gene and leaves the healthy one alone.

                                                                                                                                                                        

IMPORTANT NEWS

WAVE is currently enrolling adults with early manifest Huntington’s disease (HD) who carry a SNP at the rs362307 (“SNP1”) location for a Phase 1b/2a clinical trial.

Learn more about Wave trials for HD at clinicaltrials.gov 
You may also find information about clinical trials in HD at hdtrialfinder.org 

WAVE is currently enrolling adults with early manifest Huntington’s disease (HD) who carry a SNP at the rs362331 (“SNP2”) location for a Phase 1b/2a clinical trial.

Learn more about Wave trials for HD at clinicaltrials.gov 
You may also find information about clinical trials in HD at hdtrialfinder.org 

We Can Never Lose HOPE…………….

100% of the profits from my book is being donated to the nonprofit, HDSA.  Since publication in 2017, I’ve donated over $14,000.00.  It can be found on many book websites.

 

Hope

The Human Condition and HOPE

The Human Condition: “the characteristics, key events, and situations which compose the essentials of human existence, such as birth, growth, emotionality, aspiration, conflict, and mortality”.  It is a very broad topic which has been and continues to be pondered and analyzed from many perspectives.

I believe HOPE lives in each element of the Human Condition and HOPE lives in the heart of every Huntington’s Disease (HD) family member. HD is a rare brain disorder with symptoms of ALS, Alzheimers and Parkinson’s that no cure.  http://www.hdsa.org

Dr.’s Ed Wild from the U.K. & Jeff Carroll from the U.S.

John and I had HOPE for healthy children with a normal CAG count. HOPE keeps me emotionally grounded and passionate about offering HD information on all my social media platforms so no one fights HD alone. The HD community has  HOPE that researchers will find a cure/therapy.  HOPE keeps my heart open to forgiveness and HOPE allows me to use my mortality to keep me mindful everyday of all the all the blessings I have in my life. 

Lora, Cindy, Marcia Marin

HOPE is what John and I clung to, along with each other, as we watched his three sisters, Marcia, Lora, and Cindy, battle Huntington’s disease over a 24 year period of time. 

HDSA 2019 Convention

I embraced  HOPE, praying everyday John didn’t carry the mutated huntingtin gene.

We must remember, there is power in the emotion of HOPE that can help us through tough times.  Read about /the-power-hope  

 

“The road that is built in HOPE is more pleasant to the traveller than the road built in despair, even though they both lead to the same destination.” — Marion Zimmer Bradley, The Fall of Atlantis

 

HD Clinical Trials

Gene Therapy AMT-130 for Huntington’s Disease

The Huntington’s disease (HD) community has never been so hopeful that a therapy will be found for HD in the next few years.

A new development in gene therapy was announced by uniQure, whose focus is to advance the future of medicine through gene therapy. http://www.uniqure.com/about/corporate-mission.php

“The U.S. Food and Drug Administration (FDA) has completed its review of our Investigational New Drug (IND) application for AMT-130, allowing us to begin our planned Phase I/II study.  We expect that in the second half of 2019 AMT-130 will be the first ONE-TIME administered AAV gene therapy to enter clinical testing for the treatment of Huntington’s disease.”  To read about the clinical trial, visit http://uniqure.com/gene-therapy/huntingtons-disease.php

uniQure’s gene therapy candidate for Huntington’s disease is differentiated in that:

  • AMT-130 targets the deep brain structures known for the disease pathology onset.
  • AMT-130 silences mutant huntingtin protein at levels not demonstrated in other studies.
  • AMT-130 targets the accumulation of the exon 1 HTT fragment, the most toxic source of abnormal protein aggregation in Huntington’s disease.

The AMT-130 trial will be based at HD clinical sites in the Unites States. It’s not known at this time what sites or how many, yet. These will be publicly announced when they come online. UniQure hopes to begin enrolling patients before the end of 2019.  It is brain surgery.  https://en.hdbuzz.net/274


Please sign up for EnrollHD if you would like to participate in a HD clinical trial study.  https://www.enroll-hd.org/

We Can Never Lose HOPE….. 

 

 

 

HD Advocates

Trey Gray-A Drummer, Huntington’s Disease Advocate, Father, Philanthropist & an Inspirational Guy

    

Paul Erdman Band

I love to post about Trey Gray because he is such an inspiration, a kind person and, often says, he is GRATEFUL!   He graciously wrote a book jacket comment that appears on the back of my nonfiction book, Watching Their Dance:  Three Sisters, a Genetic Disease and Marrying into a Family At Risk for Huntington’s. 

I asked him not too long ago when he would come out West so the Huntington’s disease (HD) community could meet him and listen to his music.  Unfortunately, it’s not in the cards right now.

Trey’s HD diagnosis came in 2003, and he has beaten the odds because he is asymptomatic after 19 years.  Wow is all I can say!  (Trey’s mom, Cindy, had HD).

What is Huntington’s disease?  https://www.huntingtonsdiseasefoundation.org/life-with-hd-2

Marcia and Cindy, my two sisters-in-law to die from HD complications, struggled with HD for approximately 16-17 years and their quality of life the last five years was not good.

Trey’s son Max.

I hope Trey keeps pounding on those drums, doing what he loves, and keeps us all inspired by his HD advocacy, positiveness, philanthropy work he accomplishes through the Huntington’s Disease Foundation he and two friends started.  https://www.huntingtonsdiseasefoundation.org/

Trey’s Facebook wall is:  https://www.facebook.com/trey.gray

We Can Never Lose HOPE….

 

Watching Their Dance can be found on Amazon around the world & many other book websites. It’s available as an e-book also.   100% of the profits are being donated to Huntington’s Disease Society of America (HDSA).  I’ve donated over $14,000.00 so far from the book to HDSA.

Read a book review from Kirkus:  https://www.kirkusreviews.com/book-reviews/unknown/watching-their-dance/