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.

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.

LuMind Research Down Syndrome Named an Official Charity Partner of the 2016 United Airlines NYC Half Set for Sunday, March 20, 2016

New York, November 17, 2015 — LuMind Research Down Syndrome Foundation was named an Official Charity Partner of the 2016 United Airlines NYC Half, it was announced today by Carolyn Cronin president and CEO of LuMind RDS. The race will take place on Sunday, March 20, 2016.

“The opportunity to partner with the United Airlines NYC Half gives LuMind Research Down Syndrome Foundation the ability to raise funds and awareness for valuable Down syndrome cognition research, including unraveling the connections between Down syndrome and Alzheimer’s disease,” said Cronin. “We look forward to having a dedicated team of runners take on the United Airlines NYC Half on behalf of our organization—training, preparing, and ultimately completing the 13.1-mile race for a cause that is both meaningful and close to each and every one of them.”

Over five million dollars was raised by more than 2,800 charity runners during the 2015 United Airlines NYC Half to help more than 100 nonprofit organizations support their missions and services. Approximately 120 official charity partners will be part of the 2016 United Airlines NYC Half.

LuMind RDS Runners will be running in the United Airlines NYC Half Marathon on 3/20/16.

LuMind RDS Runners is a vibrant community of dedicated parents, siblings, family members, and friends devoted to pushing physical limits in racing challenges to fund research on improving cognition in people with Down syndrome. The research they help to fund focuses on improving learning, memory, and sleep for people of all ages with Down syndrome enabling increased opportunities for independence at school and work. People with Down syndrome have an extra copy of the 21st chromosome, so LuMind RDS Runners “race for eXtraordinary,” especially during the month of March in honor of World Down Syndrome Day on March 21. LuMind RDS runners – and walkers will be participating in events all month with the United Airlines NYC Half Marathon as a capstone event.

“We welcome LuMind Research Down Syndrome Foundation as an official charity partner of the 2016 United Airlines NYC Half,” said Michael Capiraso, president and CEO of New York Road Runners. “We are privileged to partner with approximately 120 official charity partners for this event, impacting thousands in need of support for so many significant causes. Our charity runners are an inspirational and integral part of this race and we applaud their efforts and the positive impact they each make.”

The United Airlines NYC Half features some of the most talented American and international professional athlete fields and a vast range of competitive, recreational, and charity runners.  Some 1,500 volunteers and thousands of spectators line the course in support of all participants. 19,454 finishers completed the 2015 United Airlines NYC Half from nearly all of the 50 states and more than 67 countries. For the first time ever, the NYRR Times Square Kids’ Run at the United Airlines NYC Half gave hundreds of kids the opportunity to participate in a race of their own as they completed a 1500-meter out-and-back course through the heart of New York City.

The 2016 United Airlines NYC Half will be broadcast live locally on WABC-TV, Channel 7 for the fifth consecutive year. 

About New York Road Runners

Founded in 1958, New York Road Runners has grown from a local running club to the world’s premier community running organization, whose mission is to help and inspire people through running. NYRR’s commitment to New York City’s five boroughs features races, community events, youth running initiatives, school programs, and training resources that provide hundreds of thousands of people each year, from children to seniors, with the motivation, know-how, and opportunity to Run for Life. NYRR’s premier event, and the largest marathon in the world, is the TCS New York City Marathon. Held annually on the first Sunday in November, the race features 50,000 runners, from the world’s top professional athletes to a vast range of competitive, recreational, and charity runners. To learn more, visit www.nyrr.org.

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 a national non-profit organization headquartered 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 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. To learn more: www.LuMindRDS.org.

Might Normalizing Brain Development Help in Down’s Syndrome?

LuMind Research Down Syndrome Foundation’s Chief Scientific Officer Dr. Michael Harpold was referenced in a discussion from the Society for Neuroscience Annual Meeting. The article discusses if the cognitive impairments associated with Down syndrome could be prevented.

Dr. Harpold was mentioned along with others from Tufts Medical in Boston, Univeristy of Bologna in Italy and Institute for Basic Research’s Genetics Laboratory.

Check out the full article.

 

 

 

3rd Annual Tribute 21 – November 6th, 2015

Research Down Syndrome is now LuMind Research Down Syndrome Foundation! Please visit our new site, http://www.lumindfoundation.org/tribute-21, to learn more and to purchase tickets for the 2015 Tribute 21 event!

Kate Dochelli Runs to LuMind RDS

We are happy to announce that Kate Dochelli has joined LuMind RDS to lead and grow the vibrant Runners program as well as coordinate special events for the organization. Kate has been a member of the RDS Runners program for several years and is poised to engage with runners since she is marathoner herself.

“I joined RDS Runners, now LuMind RDS Runners, two years ago. As soon I signed up for my first event, the Marine Corps Marathon, I received a call from Bob Schoen welcoming me to the community,” explained Kate. “It’s been such a great experience, learning about the research LuMind RDS funds as well as engaging with other runners around the country through the program.”

Kate’s introduction to the Runners program wasn’t without some bumps – literally. While Kate was training for the Marine Corps Marathon she was hit by a truck and suffered significant injuries that prevented her from competing in the event.

“Bob was in constant communication with my mom while I was in the hospital. We hadn’t even met in person yet and he was so concerned,” she said. That experience helped to solidify Kate’s warm feelings for the RDS Runners community, which have grown as she made friends from Texas to Minnesota.

A few weeks after the event while she was still recovering, Kate received an unexpected surprise. One day a shirt and letter arrived from the Marine Corps Marathon event organizers acknowledging all her hard fundraising and training efforts despite not being able to participate in the race.

“I learned that one of the Runners community members, Chad Simon, had arranged for a letter to be sent from the Marine Corps Marathon,” she explained. “It was such a nice, thoughtful gesture. It meant a lot to me during my recovery.”

Kate’s goal is to introduce new runners to this community and inspire and motivate existing runners whether they run 100 miles at a time or struggle to compete a fun run. She’ll also be motivating them to reach fundraising goals as they open opportunities for their loved one with Down syndrome.

“This community is so inspiring and so motivating. It’s a pleasure to be part of welcoming new runners and continuing to work and train with these amazing people,” she said.

The LuMind RDS Runners program welcomes people of all abilities who want to help raise funds for cognition research by competing in fun runs, road races, marathons, triathlons and relay races. “We say any race, any place, any date,” added Kate. “Just let us know your ideas and we’ll set you up with everything you need to prepare and fundraise.”

To learn more visit the LuMind RDS Runners page and request to join the LuMind RDS Runners Facebook group to join in the fun and raise money for Down syndrome cognition research!

#Race4eXtraordinary #Run4Ds

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.

What’s happening in Down syndrome research?

How do you keep up with the latest in Down syndrome research?

We know you love Dr. Harpold and all the LuMind RDS-funded researchers who let you know about the advances in cognition research, but there’s a big world of discoveries out there.

We’ve curated the latest scientifically-credible articles for you on our website. We’ll be keeping this page current, so please send us any articles that interest you. We want to be sure we – and you – see it all!

Check it out: LuMind RDS: Down Syndrome Research News.