World’s First Clinical Trial for anti-Abeta Vaccine Targeting Alzheimer’s Disease-like Characteristics in People with Down Syndrome

PRESS RELEASE – Issued by AC Immune

• Studies AC Immune’s ACI-24, the first anti-amyloid vaccine for treatment of Alzheimer’s disease-like characteristics in people with Down syndrome
• Clinical Study is done in collaboration with University of California San Diego
• US NIH provides significant funding with an additional grant from the LuMind Research Down Syndrome Foundation
• Alzheimer’s disease-like characteristics develop in virtually all people with Down syndrome over age 40; majority develops associated dementia over
  age 60

Lausanne, Switzerland, San Diego, CA and Marlborough, MA USA – January 7, 2016 – Today plans were announced to conduct the world’s first clinical trial for a vaccine targeting Alzheimer’s disease-like characteristics in those with Down syndrome. The study will test AC Immune’s vaccine ACI-24 and is being conducted in collaboration with the University of California, San Diego (UC San Diego) Down Syndrome Research and Treatment Center. Funding is provided by a significant grant from the US National Institutes of Health (NIH) and an additional grant from the LuMind Research Down Syndrome Foundation. This is the first public/private collaboration for a clinical trial in the field of Down syndrome.

Individuals with Down syndrome (DS) have an extra copy of chromosome 21 which carries the gene for APP encoding the precursor protein of Abeta, one of the hallmarks of Alzheimer’s disease (AD). An important consequence is that individuals with DS develop AD-like characteristics at a rate three to five times greater than that of the general population and at a much younger age. Further, AD-like characteristics develop in more than 98% of people with DS over age 40 with up to 80% developing associated dementia over the age of 60. It is estimated that there are 6 million people with DS worldwide, with 400,000 in the United States.

Trial participants will be adults with DS. The objectives of the trial include studying safety and tolerability of ACI-24, its effect on induction of antibodies against Abeta, clinical and cognitive measures in adults with DS and its effect on biomarkers of AD-like pathology in DS. Participants in the study will be treated for 12 months, with 12 months follow up.

Prof. Andrea Pfeifer, CEO of AC Immune said: “We are very pleased to bring this potentially disease modifying treatment for Alzheimer’s disease into the vulnerable, genetically predisposed Down syndrome population. The combined knowledge and resources of AC Immune, UC San Diego, NIH and the LuMind Research Down Syndrome Foundation should generate much needed insight for treating the Alzheimer’s-like characteristics of those with Down syndrome. Additionally, this ground-breaking clinical trial could enhance our understanding of early intervention and prevention of Alzheimer’s in general.”

Dr. William Mobley, Executive Director of the UC San Diego Down Syndrome Research and Treatment Center, commented: “We are delighted to join our colleagues at AC Immune and the LuMind Research Down Syndrome Foundation in this exciting study, the first step in a process whose ultimate goal is preventing Alzheimer’s disease in people with Down syndrome.  That both public and private funding sources support the study signifies the importance attached to Alzheimer’s disease and the valuable insights that will come from studies of this disorder in Down syndrome.  We wish to thank our colleagues as we eagerly look forward to helping people with Down syndrome and their families and loved-ones.”

Dr. Michael Harpold,  LuMind Research Down Syndrome Foundation’s Chief Scientific Officer, stated: “We are very excited the LuMind Research Down Syndrome Foundation has been able to work and join together with AC Immune, UC San Diego and NIH in establishing the first ever private-public partnership for a clinical trial in the field of Down syndrome. Accelerating research and the development of new potential therapies to address the developmental intellectual disabilities and earlier onset of Alzheimer’s disease in people with Down syndrome represents a major part of our foundation’s mission and commitment to prevent the earlier decline and loss of important gains they have attained throughout their lives.”

About ACI-24

ACI-24 is a liposomal therapeutic anti-Abeta vaccine candidate, which is owned by AC Immune and was discovered utilizing the Company’s proprietary SupraAntigen^TM technology platform. The vaccine is designed to stimulate a patient’s immune system to produce antibodies that specifically target the oligomeric and fibrillary Abeta proteins to prevent beta amyloid plaque accumulation and to enhance plaque clearance. Preclinical data demonstrated a significant activity in plaque reduction and memory restoration as well as a favorable safety profile characterized by a lack of local inflammation and a mode of action independent of inflammatory T-cells. The vaccine is currently also being studied in a phase 1/2a clinical trial in patients with mild to moderate AD, in which no significant safety issues have been detected to date.

About Down syndrome

Down syndrome, or trisomy 21, is the most common genetic cause of intellectual disability and developmental delay, and affects one in 700 newborns. This condition results when an individual has three, rather than two, copies of the 21st chromosome. This additional genetic material causes impairment of cognitive ability and physical growth, and is associated with other medical issues ranging from neurological and cardiac defects to hearing and vision problems as well as earlier development of Alzheimer’s disease. The average life expectancy for people with DS has increased from 25 years in the 1980’s to approximately 60 years today.

About Alzheimer’s disease

It is becoming increasingly clear that Alzheimer’s disease develops because of a complex series of events that take place in the brain over a long period of time. Two proteins – Tau and beta-amyloid (Abeta) – are recognized as major hallmarks of neurodegeneration: tangles and other abnormal forms of Tau protein accumulate inside the brain cells and spread between cells, while plaques and oligomers formed by beta-amyloid occur outside the brain cells of people with AD.

AD is one of the biggest burdens of society with a dramatic and growing worldwide incidence rate of one new case every three seconds, or 9.9 million new cases of dementia each year. Since the incidence and prevalence of AD increase with age, the number of patients will grow significantly as society ages. Worldwide in 2015 there are 46.8 million people living with dementia and by 2050 it is expected that global patient numbers will triple to 131.5 million. It is estimated that the annual societal and economic cost of dementia has risen from US$ 604 billion in 2010 to US$ 818 billion in 2015.  In the US, AD is now the 6th leading cause of death across all ages and is the fifth leading cause of death for those aged 65 and older.

About AC Immune

AC Immune is a leading Swiss-based biopharmaceutical company focused on neurodegenerative diseases with three product candidates in clinical trials.  The Company designs, discovers and develops therapeutic and diagnostic products to prevent and modify diseases caused by misfolding proteins. AC Immune’s two proprietary technology platforms create antibodies, small molecules and vaccines to address large markets across a broad spectrum of neurodegenerative indications. Alzheimer’s disease (AD) is the largest indication addressed by its products but the company’s innovative, differentiated and disease-modifying therapies are designed to shift the paradigm in the treatment of other neurodegenerative diseases such as Parkinson’s, Down syndrome, and Glaucoma. The Company has a large, diversified and promising pipeline featuring seven therapeutic and three diagnostic product candidates. The most advanced of these is crenezumab, an anti-Abeta antibody that is licensed to Genentech entering phase 3 clinical trials. Crenezumab was chosen by the US National Institute of Health for use in the first-ever AD prevention trial. The company has partnered three programs targeting Tau: ACI-35 with Janssen (therapeutic vaccine, phase 1b), Tau-PET imaging agent with Piramal (Alzheimer’s diagnostic agent) and anti-Tau-antibodies with Genentech (preclinical). The anti-Abeta vaccine ACI-24 phase 1/2a trial is run in house.

About UC San Diego Down Syndrome Research and Treatment Center

Established in 2009, the Center’s efforts focus on defining the genes and mechanisms responsible for the cognitive challenges faced by people with Down syndrome. Studies are carried out in both mouse models and in mouse and human cellular models.  The insights derived support translation of basic science findings into new treatments, using either existing drugs or through drug discovery. The Center’s work has resulted in conceptual innovations and several novel treatment targets and has inspired existing trials as well as the clinical study announced in this press release (supported by an NIH grant under award number R01AG047922). The Center is supported by the NIH and private foundations, including the LuMind Research Down Syndrome Foundation, the Alzheimer’s Association, the Tau Consortium and the Cure Alzheimer Fund.

About LuMind Research Down Syndrome Foundation

LuMind Research Down Syndrome Foundation, formerly the Down Syndrome Research and Treatment Foundation (DSRTF) and Research Down Syndrome, is an international non-profit organization with headquarters in Marlborough, Massachusetts, aimed at accelerating the development of treatments to significantly improve cognition, including memory, learning and speech, for individuals with Down syndrome. LuMind RDS Foundation is the leading source of private funding supporting Down syndrome cognition research at major research centers, including Johns Hopkins Medicine, Stanford University, University of California, San Diego, and University of Arizona. Since its founding in 2004, LuMind RDS Foundation has committed more than $13 million to fund results-driven research programs that will benefit children and adults with Down syndrome, and has been instrumental in the initiation of clinical trials now under way.

For further information please contact:

AC Immune

Prof. Andrea Pfeifer Chief Executive Officer Phone: +41-21-693 91 21 E-mail:andrea.pfeifer@acimmune.com	Eva Schier Corporate Communications Manager Phone: +41-21-693 91 34 E-mail: eva.schier@acimmune.com
Nick Miles Senior Consultant Cabinet Privé de Conseils s.a. Mobile : +41 79 678 76 26 E-mail : miles@cpc-pr.com	In the US Ted Agne The Communications Strategy Group Inc. Phone: +1 781 631 3117 E-mail: edagne@comstratgroup.comed

 

UC San Diego

William C Mobley, M.D., Ph.D. Professor of Neurosciences, and Executive Director, Down Syndrome Research and Treatment Center Phone: +1 858-534-9434 Email: wmobley@ucsd.edu

Scott LaFee Director, Media Relations Marketing and Communications UC San Diego Health Sciences Phone : +1 619-543-6163 Email : mailto:slafee@ucsd.edu

 

LuMind Research Down Syndrome Foundation

Carolyn Cronin President/Chief Executive Officer Phone: (508) 630-2178 Email: ccronin@lumindrds.org	Ellen Oliver Marketing Director Phone: (508) 630-2179 Email: eoliver@lumindrds.org
Michael M. Harpold, PhD Chief Scientific Officer Phone: (520) 297-3105 Email: mharpold@lumindrds.org

 

Federal Budget Updates Related to Down Syndrome Research

Our Dr. Harpold stays closely connected to and works in the world of research, not only Down syndrome and Alzheimer’s disease, but also relevant research endeavors at a broad scale. Here’s his take on the Federal 2016 Omnibus Budget Bill.

By Dr. Michael Harpold, LuMind RDS Chief Scientific Officer

The just enacted Federal 2016 Omnibus Budget Bill, which includes a $2 billion increase for NIH, represents significantly good news for advancing biomedical research including Down syndrome research.

Over approximately the past decade, the budget for NIH has remained essentially flat, translating to a more than 25% decline in actual NIH “research-buying” power. This has made securing funding for NIH research grants by researchers extremely difficult and, closer to home, created significant challenges in gaining increased NIH funding dedicated to Down syndrome research.

This newly enacted increase in NIH funding will enable funding for more NIH grants as well as significantly increased funding to address Alzheimer’s disease… all potentially good news for Down syndrome research.

In addition to work focused on NIH funding for Down syndrome throughout this year, LuMind RDS contributed to a recent final push for this increased funding, especially Alzheimer’s disease research, through our continuing membership and work together with Leaders Engaged in Alzheimer’s Disease leveraging together 80 member organizations, as a signatory on advocacy letters, which also specifically included Down syndrome reference, to the respective US House and Senate Appropriations committees’ leadership.

Among other important relevant appropriations in this new Federal budget:

• National Institutes on Aging (NIA) Alzheimer’s disease and related dementias research funding will increase to $936 million, a $350 million, or almost 60%, increase above Fiscal Year 2015
• NIA’s overall funding will increase by $400 million, more than 85% of that for dementia
• The Center for Disease Control (CDC) will have 3.5 million for its Alzheimer’s Disease (brain health) program, and
• Food and Drug Administration (FDA) funding will increase 5%, roughly $90-100 million more than House and Senate appropriators passed earlier this year.

Thank you for the momentum you’ve helped to create to bring the importance of increasing funding for all types of research to the attention of the government.

Please consider continuing to show your support for Down syndrome research with a donation during our Annual Appeal.

Clinton Campaign Announces Investment Plan for Alzheimer’s Disease

LuMind Research Down Syndrome Foundation applauds the development and announcement by the Clinton presidential campaign of a formal plan for new investment to prevent, treat, and make an Alzheimer’s disease cure possible by 2025, which includes a commitment to reach the $2 billion annual funding level for NIH’s dementia research. You can read more details on Clinton’s announced plan in the following article.

We join with many of our other colleagues and partners in the Down syndrome and Alzheimer’s disease communities in encouraging all of the presidential campaigns to develop and support formal proposals for advancing Alzheimer’s disease and dementia research.

This research is especially important for individuals with Down syndrome since virtually all develop the brain characteristics of Alzheimer’s disease earlier, by their 40’s, and the majority subsequently progress to earlier onset of the associated dementia. In November, NIH announced significant new grant awards to find Alzheimer’s biomarkers in Down syndrome.

LuMind RDS is the leading source of private funding for Down syndrome cognition research, including funding initiatives to identify and develop effective new therapies to prevent and halt the progression of the earlier onset Alzheimer’s disease in people with Down syndrome and help avoid the loss of gains they achieve throughout their lives. If you would like to support this research, please consider a donation during our Annual Appeal.

Awaken A Force

By Alan Gard, Devoted Dad (and Jedi Knight Battling Darth Dementia)

Aspiring Jedi Alijah and others with Ds need your help to defeat Darth Dementia!

As December greets us this year, many are in a festive move as a special event approaches…namely the release of Star Wars: The Force Awakens. Over $50 million in presale tickets have already been sold. When was the last time you bought a ticket in advance to go see a movie? I didn’t think so. In other words, this movie is going to be HUGE. There is extreme interest in checking in on Han, Luke, and Leia as well as meeting new characters Rey, Finn, and Kylo Ren.

The Force Awakens will be the seventh entry in the Star Wars saga. In a broad sense, the saga is a simple story of Good vs. Evil, or maybe more accurately it is a story of our individual choices to be Good or Evil and how those choices can change over the course of time and circumstance.

If one concentrates on the original trilogy, Episodes IV – VI, the story is Luke Skywalker’s Hero Journey.  It starts with the Call to Adventure from humble beginnings. In Luke’s case, he is reticent at first. He is too busy with the harvest to get involved. That sounds familiar to most of us in the frenzy of our day-to-day lives! He eventually heeds the call and over the course of this story arc redeems his father and brings down the leadership of the Galactic Empire.

Of course, now the story won’t end there and much of the interest in The Force Awakens is in what becomes of Luke after the story left off 32 years ago. With one Hero’s Journey complete, we now will learn what happens when there is another chapter:

• Does Luke stay a hero?
• Does he turn to the Dark Side?
• Does he become a character more gray than at the black or white end of the Dark vs. Light spectrum?

As one who grew up with the original trilogy, Luke is the character in which I have the most interest as the new saga begins.

The story could also be an ongoing story of The Force, a metaphysical energy that binds everything in the universe together and gives those who are genetically and spiritually gifted to tap into it magical powers. It has its basis in the concept of Prana, meaning “life force” from Hindu culture. This view makes the story one about how we are collectively part of something bigger and all must play our role in that story.

If viewed over the entirety of the six episodes released to date, it is the Story of Anakin. He starts as a slave, learns he is strong with The Force, is trained, becomes a Jedi, turns to the Dark Side becoming a Sith Lord named Darth Vader, gets severely injured in a duel with his original mentor Obi-Wan, gets saved by the Emperor and technology, serves as the Emperor’s sergeant-at-arms in oppressing the galaxy, vanquishes Obi-Wan, fails to crush the Rebel Alliance, discovers he has a son, cuts son’s hand off in a duel, takes his son to the Emperor to be turned to the Dark Side, turns on the Emperor and kills him, and then dies having returned back to Good. And that’s the simple version!

In my view, Episodes I – VI are really a rise and fall story of the Sith, an order that derives power from the Dark Side of the Force. This order sought to elevate the strong and eliminate the weak. But throughout the Star Wars story, the Sith are foiled by those they might consider weak working together to overcome them.

Unfortunately, the Sith are not confined to a galaxy far, far away. We have a Sith Lord among us here, and his name is Darth Dementia. He is a particularly ruthless enemy. He steals the souls of his victims and inflicts pain on suffering on all those around them.

I have witnessed Darth Dementia firsthand. Growing up, visits with my Great Aunt Catherine showed me what Alzheimer’s related dementia could do to one’s ability to know their family. Imagine how sad an existence it would be to not be able to connect with your loved ones from both sides of that diagnosis. As a young adult, I saw two of my uncles suffer from Parkinson’s disease and the dementia that can eventually accompany that disease.

Now I am a parent to a child with Down syndrome. While my wife and my current energies are spent trying to help Alijah develop and reach his potential, Darth Dementia is an ever-present, dark-cloaked figure in the corners of our minds. As one who works with numbers, I know just how stacked the odds are against Alijah in his fight to hold off Darth Dementia. To think that all the gains Alijah is making could be taken away so early in his life is a heartbreaking concept. But, as of today, that is a very likely scenario.

Luckily, there are already a few Jedi taking on this powerful Sith Lord. But as the movies have shown, the only real victory comes when the multitude work together. So if you are an aspiring Jedi, you have an opportunity to be a hero in this story. Or if you see yourself as more of a Wookie or an Ewok, you can be a hero in this story too. Imagine if everyone who has already pre-ordered their The Force Awakens ticket got so inspired by the movie and the fight against Darth Dementia that they came home and made a donation in the amount of their movie ticket. $50 million would serve notice to Darth Dementia that that his power was at risk.

If all those that see the movie would do the same thing, it would change the conversation from IF we can do it to WHEN. And asking when is a MUCH better question than asking if – not just for those with Down syndrome, but for each and every one of us that want to keep ourselves and our families safe from Darth Dementia’s soul-stealing clutches. While Darth Dementia may play favorites toward victims with an extra chromosome, they are not his only prey.

Think back to where the Star Wars phenomenon began. It began with A New Hope. Any donation now—big or small, the price of a movie ticket, whatever—will in fact make A New Hope come true in the lives of those most at risk and their loved ones. Don’t let the supposed constraints of the “harvest” in your life deny you your own Hero’s Journey. And while we—at least us non-insiders—don’t know where the story goes after The Force Awakens, I am positive there is a place in this story for us all to help Awaken A Force to defeat Darth Dementia once and for all. I definitely want that to be a part of my story. I hope you do too.

Thank you for your consideration. And may The Force be with you!

NIH supports new studies to find Alzheimer’s biomarkers in Down syndrome

The National Institutes of Health announced a groundbreaking initiative to track dementia onset and the progress of Alzheimer’s disease in people with Down syndrome.

The studies will be funded by the National Institute on Aging (NIA) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), both part of NIH.

“We are very excited and grateful that the NIH Biomarkers of Alzheimer’s Disease in Adults with Down Syndrome Initiative has now been established with funding by the National Institute on Aging and the Eunice Kennedy Shriver National Institute of Child Health and Human Development,” said Dr. Michael Harpold LuMind RDS Chief Scientific Officer.

This initiative represents one of the major recommendations deriving from the 2013 NIH Workshop “Advancing Treatments for Alzheimer’s Disease in Individuals with Down Syndrome” with the participation, co-sponsorship and co-support by the LuMind RDS Foundation. This recommendation was also subsequently incorporated into the Federal National Plan to Address Alzheimer’s Disease.

“This is an extremely important and much needed research initiative to more deeply understand the earlier onset and course of Alzheimer’s disease in individuals with Down syndrome, and the results from this new five year research initiative will also be important in further accelerating clinical trials to develop effective new therapies,” said Dr. Harpold.

View the full press release from NIH here.

21st Century Cures Act Passes in US House – What It Means for Down Syndrome Research

By Dr. Michael Harpold, LuMind RDS Chief Scientific Officer

The 21st Century Cures Act, originating and developed out of the US House Energy and Commerce Committee, has been working its way through to a vote in the US House of Representatives. LuMind RDS signed the letter of support organized by National Health Council, in conjunction with our membership with the LEAD Coalition (Leaders Engaged on Alzheimer’s Disease), which had more than 250 organizations as signatories.

Prior to the vote on the overall Bill in the House of Representatives, there was a problematic “Brat et al. amendment” that had been introduced which would have jeopardized the increased funding for the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) provided in the Bill as well as the wide bipartisan support. Through the National Health Council/United for Medical Research, LuMind RDS, along with more than 270 other organizations, signed on in opposition to the “Brat amendment” (http://www.no2brat.com/).

I am quite pleased to update that Friday July 10, 2015, the 21st Century Cures Act passed the US House by 344-77, and the “Brat et al amendment” was defeated by vote of 141-281. Among other aspects that are important for Down syndrome, the Bill increases NIH’s budget by additional $8.75 billion over five years. 

This is good news because the flat (in actuality, significantly declining in real dollars) NIH budget over the past five years or so has had a major impact in reducing the number and size of NIH research grants. The increased funding should increase overall NIH grant applications’ funding success and increased numbers of NIH grants, including hopefully more grants for Down syndrome research.

But before that increase is realized, the Bill needs to now make its way through and pass the Senate… which will be an additional effort for continuing support from the Down syndrome community.

My Giving Story: We Need Research AND Advocacy

 – Stephanie Hartman shared this story at our inaugural Sky’s the Limit Awards Gala where we honored Dr. Jamie Edgin, Dr. William Mobley, Sarah Wernikoff and Jim and Patty White. Thank you, Stephanie, for supporting LuMind RDS Foundation and for sharing your personal reasons for supporting Down syndrome cognition research. 

By Stephanie Hartman

Even from a young age, I knew that I was meant to be a mother. I could see through the experience of others and particularly with the closeness within my family growing up, that good parenting is hard work, but immeasurably rewarding and the most important job to be done.

I became pregnant just before turning 34. My husband Ryan and I learned that our baby boy had heart issues that would require heart surgery in early childhood. We were told that these heart issues were commonly found in children born with Down syndrome, so that was a possibility for our son.

For many reasons, including this, my doctor strongly encouraged that I get an amniocentesis. At that time, it was the only method to determine genetic variances with any certainty.

I was informed that conducting the amniocentesis itself presented a slight risk to my baby. The information would have been helpful for preparation. However, I was unwilling to take the risk.

I viewed this differently than other choices that just affected me. This was a tiny unborn person who only had me to count on to keep him safe and healthy. Also, due to strongly held personal beliefs, I knew that regardless of the results of any test, I was going to give birth to and care for and unconditionally love any child growing inside my body.

In utero, the doctors took measurements of my son’s head, torso, arms and legs. We were told that that our child’s proportions seemed typical and didn’t indicate Down syndrome.

That was enough for us to believe what we thought was ‘’best case” scenario, where our child would have the certain heart defects and require surgery but would otherwise be typical. So we put our heads in the sand and continued our dreams for the future of our family.

Not long after midnight on February 26, 2008, at University Medical Center in Tucson, Taylor Glen Hartman was born.  He was tiny, under six pounds….not the nine pounds that had been predicted. Many things were not as predicted.

Soon after the umbilical cord was cut, I could hear the people in the room whispering to each other. I had never had a baby before but I could tell that something was not right. UMC is a teaching hospital, so the room was filled with interns and residents. After cleaning the baby, one of the doctors explained that our son had features consistent with Down syndrome.

What a strange experience. We shouldn’t have been, but we were stunned.

I was trying to simultaneously process this information that would change the course of my life and manage the political correctness of my reaction, given the room full of strangers witnessing this very personal moment.

The hours and days that followed were the most confusing and challenging I had ever experienced.

The instant and beautiful bond between mother and child shown in the movies was not my experience. I understood though that this tiny gift from God in unexpected wrapping needed me. So through the tears, I snuggled him, I sang to him and I nursed him like our lives and our relationship depended on it. It did.

Meanwhile, Ryan left the hospital. It seemed like he was never coming back.

He finally returned with a gift of tulips and a story about a trip to Holland. He also had information. While I was contemplating how to best support the social and educational components of the future advocacy of our child, Ryan gained comfort, strength and direction, reading the work of Dr. Mobley and others. He quickly absorbed so much about Down syndrome, the details of recent advances, and research underway that would maximize our son’s intellectual capabilities.

Thank goodness for Taylor, he has both of us. My narrow focus on advocacy for acceptance and inclusion would have prevented me from even considering medical cognitive interventions. I feared that in so doing, I was in someway rejecting Taylor or trying to cure his Down syndrome like a sickness.

We love our little rascal. Ds is part of what makes him who he is. Ryan and I wouldn’t take away the Down syndrome even if we could. But I now understand that cognition research is about maximizing the potential of individuals living with Down syndrome, not attempting to cure them of it. It is about helping our loved ones to:

• think more clearly,
• rest more peacefully,
• express themselves fully, and
• be as independent as possible.

Isn’t that what any loving parent wants for his or her child?

If Ryan hadn’t come across the valuable work funded by the LuMind Research Down Syndrome Foundation, we may have focused our attention on advocacy, at the exclusion of research.

Taylor would grow to the age that I am today and suffer from the cognitive degeneration that occurs in Alzheimer’s. If later I learned that I could have taken simple actions today to prevent such an onset, I would never forgive myself.

LuMind Research Down Syndrome Foundation is funding research that is very real, tangible, and applicable sooner than later – for children and adults with Down syndrome.

• Dr. Jamie Edgin’s sleep and cognition studies provide insight and possible solutions to the sleep-related issues faced by many people with Down syndrome – and their parents!
• Dr. William Mobley and others are working to help Taylor and others living with Down syndrome to retain the cognitive gains they’ve made through their lives.
• And thanks to the recent revelation of the connection between Down syndrome and Alzheimer’s, both communities can benefit from the research synergy.  

I know there are many causes looking for answers from research, but unlike federal funding for conditions such as Cystic Fibrosis at over $2500 per affected person, Down syndrome research only receives $60 per person from the National Institutes of Health.

Thanks to the vision of people like Jim and Patty White and the dedicated volunteers, like Sarah Wernikoff, who have supported private funding for cognition research, there have been many recent amazing breakthroughs.

But, we still have so much more work to do. There is fantastic, groundbreaking research waiting to be conducted by these and other distinguished researchers and rising stars. The only barrier today is lack of funding.

Now more than ever I believe that good parenting is the most important job to be done.

I urge you to be as generous as possible to help ignite new discoveries and propel this important research forward. Please join me in one of the most important jobs we can have as parents – supporting the futures of our loved ones with Down syndrome by funding these research advances.

Let’s Do This: Mobilizing the Cognition Research Army

By Maureen Wallace

The funny thing is, I’ve always had this unexplainable foreboding that I would develop Alzheimer’s disease. I guess that’s not so funny, is it.

The fear lurks in my mind, so much so that I visited a research facility once, thinking I’d be able to learn if I was “predisposed.” Turns out, the facility couldn’t do that.

Recently, I got an answer. My mom is starting to struggle with memory loss. Sure, we all do as we age. But a few incidents emerged that whispered, “Wait. Be still. Listen. Pay attention to me. This is more than lost keys.”

Suddenly, the possibility of Alzheimer’s smacked me in the face.

Let’s fuel the “army of researchers” ready to develop opportunities to improve cognition for Charlie and people who have or may get Alzheimer’s disease (photo credit Stephanie Stum).

Except, this was its second, ferocious smack. The first was the day I learned my son, Charlie, who is only 4 years old, is likely to develop Alzheimer’s disease at the same time I do because he already has Down syndrome.

Down syndrome and Alzheimer’s. Wow. My fears sprang to life before my eyes: My mom and my son, two different generations, about to head up the same frightening, rocky, formidable mountain.

That’s why I’m talking to you right now, why I’m writing this when only a handful of people know about my mom’s condition. I asked her if I could. She almost scoffed as she said, “Of course!” She knows why this – what I’m saying – is so important.

Because the link between Alzheimer’s disease and Down syndrome is unavoidable and unequivocal. Down syndrome is the existence of a partial or full copy of the 21^st chromosome. Alzheimer’s occurs on the 21^st chromosome.

This is real.

It’s already happening in my family. It can happen in yours.

An evil, selfish monster is intent on robbing your loved ones of the not-so-indelible-after-all images of children – grandchildren – giggling and playing on the same beach you did.

This suffocating blob of “science stuff” is determined to rip away the ability for a perfume to conjure a vision of my mom, kissing me goodbye as I bolted out the door for school, already late.

But wait.

We have an army, poised to attack this monster. Researchers are on the cusp of life-changing, life-saving discoveries. That is real, too.

Except those same researchers are standing helplessly, empty-handed. The funding isn’t enough. Researchers cannot continue on passion alone.

And so, it’s up to us. Now. These may be all the warnings we’re going to get. It’s time to build our resources and forge against this horrible, awful disease on all our horizons.

In the meantime, as we take on Alzheimer’s, we will give people with Down syndrome, like my little Charlie (who right now only cares about Elmo and the Wheels on the Bus), some real hope.

Hope for treatments that don’t cure Down syndrome but rather help Charlie with just enough of a cognitive push to understand why we lock our doors at night. Why we don’t talk to strangers. Why it’s important to be able to remember how to do that job that earns him that paycheck that allows him ever-increasing independence.

Money. Turns out money can absolutely buy my child’s future. It may be too late for my mom. We may be at only the beginning of a slow and horrible devolution. Or are we?

I’m never giving up. What if it’s never too late? What if what we do in celebration of  World Down Syndrome Day, actually matters?

Let’s do this.

– Thank you Maureen and her amazing family for sharing their story. To do more to fight off the “evil, selfish monster” consider a donation to LuMind Research Down Syndrome Foundation. Donations from March 18 through March 21, 2015 (WDSD) will be matched 3:1. Yes, let’s do this!

My Giving Story: A Foot in Two Worlds – Ds and AD

By Anna M. Miller, PhD, RN

New developments in the relationship between Alzheimer’s Disease and Down Syndrome are truly exciting, particularly for those of us who have vested interests in both worlds.

Proud Grandma Anna with her lovely granddaughter Stephanie. Anna supports Ds cognition research because she knows therapies that might help Stephanie may also help her, too.

My mother lived with classic AD progressive deterioration for 20 years, ending in total incapacity. Loving care-giving was an ongoing necessity, changing as her needs changed. Much later, my wonderful and beautiful granddaughter was born with Ds. Now 10 years old, she is very engaging, dearly loved, and quite high functioning, and new therapies to enhance her cognitive functioning (and maybe maintain mine!) may expand her potentials as she matures.

Such possibilities on the horizon send expectant shivers of excitement through my entire being!! She and I may come to have more in common than we thought!

My professional background and faith have helped me find gifts and grace through life’s challenges and opportunities, and also help me appreciate the intensive and extensive scientific efforts that have brought us thus far. Many thanks to all!

– Thank you to Anna and the entire Miller family for their support of LuMind Research Down Syndrome Foundation.

Dr. Harpold at the NIH Alzheimer’s Disease Research Summit 2015

We appreciate Dr. Michael Harpold for being such a road warrior on behalf of the Down syndrome community.

On February 9-10, 2015, Dr. Harpold, LuMind Foundation’s Chief Scientific Officer, attended the biennial NIH Alzheimer’s Disease Research Summit 2015, a major forum for the dynamic development of coordinated Alzheimer’s disease research efforts under the Congressionally-mandated National Alzheimer’s Plan.

As stated on the NIH website, the central goal of the AD Research Summit 2015 is to continue the development of an integrated multidisciplinary research agenda necessary to address critical knowledge gaps and accelerate the discovery and delivery of efficacious treatments for AD patients at all stages of disease.

Attending this conference provides an additional major forum to increase support and advocacy for greater inclusion of research on Alzheimer’s disease in individuals with Down syndrome and its contributions to a deeper understanding of Alzheimer’s disease and the development of effective new therapeutics, not only for individuals with Down syndrome, but everyone.

For more information about the summit including access to webcasts of the two-day event, please visit the NIH website.