A free teleconference series offered by UsAgainstAlzheimer's Network covering a wide range of topics with leaders in the Alzheimer's community.
Dr. Lawrence Friedhoff from Axovant Sciences was our guest for this Alzheimer’s Talks. Dr. Friedhoff has worked in pharmaceutical research and development for more than thirty years. He has led teams that developed and obtained approval for six new drugs, including donepezil (commonly known as Aricept) and he is also the author of the book New Drugs: An Insider’s Guide to the FDA Approval Process for Scientists, Investors, and Patients. Dr. Friedhoff described the clinical trial called MINDSET, which is currently enrolling participants.
Key highlights from the call:
MINDSET is a new trial that is testing a promising new drug
MINDSET is a Phase-III clinical study evaluating whether an investigational Alzheimer’s treatment known as RVT-101 could improve cognition and ability to perform daily living tasks for those with mild-to-moderate Alzheimer’s disease. RVT-101 works by raising levels of an important neurotransmitter, acetylcholine, in the brain, and is being developed as a once-daily oral pill. The MINDSET study is currently enrolling patients. To find out more, visit www.alzheimersglobalstudy.com or call 1-855-241-6288.
Clinical trials are the key to drug development
Because scientists still don’t understand the cause of Alzheimer’s disease, there are not good models in test tubes or animals, so clinical trials with human subjects are the best way to see if drugs can help or not. By participating you receive access to potential new medications, study-related medical care from expert doctors, and contribute to research that may help future generations.
Hope in future, practicality at present
Though there may someday be one single medicine to treat Alzheimer’s, Dr. Friedhoff drew comparisons between the treatment for Alzheimer’s and the treatment for other serious illnesses—once acutely fatal, now manageable—that involves combination therapy with multiple drugs, and said that in the shorter term, multiple drugs may be a way to bring benefit to people afflicted with Alzheimer’s.
Thank you to Dr. Lawrence Friedhoff from Axovant for describing his research and answering questions, both about the MINDSET trial and Alzheimer’s in general. If you missed the talk – or if you’d like to hear it again – you can listen to an audio playback or read the transcript of our conversation.
Check out the discussion Storified!
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During our January Alzheimer’s Talks, we were joined by Dan Gasby – the partner in marriage and business with B. Smith, the incredible woman who broke down barriers as a restaurant owner and model and is now facing the challenge of her life: Alzheimer’s disease. The two are doing all they can to make a difference in the fight for a cure.
Dan and B. just published a heartfelt book, Before I Forget: Love, Hope, Help and Acceptance in Our Fight Against Alzheimer’s, and they are sharing their story to let people know that if B. Smith could get Alzheimer’s, it can happen to anyone. Click here to read our co-founder Trish Vradenburg’s wonderful review of the book.
UPDATE: Dan and B.'s New York Times bestselling book is now available in paperback.
Key highlights from our conversation with Dan Gasby:
Caregiving: “It’s gut wrenching every day”
Dan shared with us the experience of being a male caregiver and how he constantly has to remind himself when he is upset or frustrated that “it’s the disease.” Some strategies he has to avoid burnout include working out, reading, meditating and going for walks with the couple’s dogs. Still, he says watching such an amazing woman decline is “gut wrenching every day.”
Increasing clinical trial participation – particularly for African-Americans
Medicines work differently for men and women and different races – so we need a representative sample in trials in order to find a drug that works for everyone. Dan talked about the problem with African Americans not trusting the medical system, the safeguards in place now and why he believes this is a 21st-century civil rights issue. We have to participate so that our children and our children’s children won’t have to worry about Alzheimer’s.
One way that anyone can help accelerate innovative medicines is by signing up for the Brain Health Registry, where you can play brain games online to help researchers and get more information on clinical trials. Click here to sign up for the Brain Health Registry.
We have bright minds working on finding a cure, but they need more funds to try more strategies. The government needs to be behind this effort in conjunction with private industry, and Congress can help speed research by increasing the amount of money spent on research funding. Dan encouraged all of us to be part of the solution by voting, writing your elected officials and being ambassadors to motivate people to make a difference.
Thank you to Axovant for sponsoring this call. Axovant is currently enrolling a clinical trial called MINDSET and actively seeking participants, particularly African Americans. We are so grateful for their contribution to bring you this fascinating conversation.
Thank you to Dan Gasby for this honest and informative discussion. If you missed the talk – or if you’d like to hear it again – you can listen to an audio playback, read the transcript of our conversation, and check out the Storify.
Stem cells, cells that have the ability to develop into different cell types, hold great promise for the treatment and prevention of Alzheimer’s disease. Dr. Larry Goldstein – Distinguished Professor in the Department of Cellular and Molecular Medicine at University of California, San Diego; Director of the UC San Diego Stem Cell Program; Director of the Sanford Stem Cell Clinical Center; and Scientific Director of the Sanford Consortium for Regenerative Medicine – joined us for November’s Alzheimer’s Talks. He described some of the groundbreaking research being done and offered a glimpse of potential future therapies and treatments for Alzheimer’s disease using stem cell technology.
Key highlights from the call:
Stem cell technology is potentially disruptive in the fight against Alzheimer’s. Stem cells provide new ways to attack the problem, and there’s hope that one day they can replace lost brain cells.
Two methods are being studied with stem cells. One is to use stem cells to replace or repair lost or damaged brain cells. The other is to use stems cells to create brain cells in the lab, sometimes referred to as ‘Alzheimer’s in a dish,’ in order to study the process of Alzheimer’s disease as well as develop and test drugs.
It’s important to try a variety of approaches. Dr. Goldstein is hopeful, as there are many promising leads. To get a cure as fast as possible, we need to try many different approaches. It is also possible that successes in research for other diseases could help inform the research into Alzheimer’s disease.
Thank you to everyone who has donated to continue the Alzheimer’s Talks teleconference series, including the Zickler Family Foundation, Karen and Chris Segal, Tom Pheasant and the Richard and Ruth Lavine Family Foundation. We are so grateful for these contributions, along with the many smaller contributions, so that we can continue to bring you the latest in Alzheimer’s research from leading scientists.
Thank you to Dr. Larry Goldstein for describing his research and answering questions. If you missed the talk – or if you’d like to hear it again – you can listen to an audio playback or read the transcript of our conversation.
There is a strong connection between Down syndrome and Alzheimer’s disease, and understanding this connection has the potential to lead to major advances in preventing or treating Alzheimer’s – both for individuals with Down syndrome and for the population as a whole.
Dr. Michael Harpold is Chief Scientific Officer of the LuMind Research Down Syndrome Foundation. He joined us for this month’s Alzheimer’s Talks call to discuss why individuals with Down syndrome experience Alzheimer’s disease earlier and at a much higher incidence and to talk about the latest research advances, including a groundbreaking new clinical trial and how this work can hopefully help find a treatment for everyone.
Key highlights from the call:
People with Down syndrome are now living longer. Life expectancy for people with Down syndrome has increased dramatically since the 1980s from fewer than 30 years to 60 years, due to medical advances and more inclusionary interventions and policies.
There is a strong connection between Down syndrome and Alzheimer’s disease. The amyloid precursor protein (APP), which plays a major role in the development of Alzheimer’s disease, is located on chromosome 21, the extra chromosome that causes Down syndrome. This could represent an effective potential drug target for Alzheimer’s disease.
People with Down syndrome could provide key information about Alzheimer’s. Many researchers now believe that early intervention will be crucial to prevent or treat Alzheimer’s. Virtually everyone with Down syndrome develops the neuropathology of Alzheimer’s disease by their 40s. There are more than 350,000 people in the U.S. with Down syndrome who will develop or are living with Alzheimer’s disease. They develop the pathology and symptoms 20 to 25 years earlier than the general population.
A groundbreaking new clinical trial for Alzheimer’s in individuals with Down syndrome is about to begin. The trial will look at ACI-24, an anti-Abeta vaccine that was developed by AC Immune and overcame characteristic neurodegeneration and restored memory deficits in a mouse model with no inflammatory side effects. There is a Phase I/II trial of this vaccine with Alzheimer’s patients in Scandinavian countries, but this will be the first trial specifically for individuals with Down syndrome.
Thank you to Dr. Michael Harpold for sharing his important research. For more information, listen to the audio playback or read the transcript of our conversation.
Thank you also to the Zickler Family Foundation and Karen and Chris Segal for their generous support, which made this call possible.
On Wednesday, November 18, Alzheimer’s Talks will welcome Dr. Larry Goldstein, Director of the Sanford Stem Cell Center and Distinguished Professor in the Department of Cellular and Molecular Medicine at University of California, San Diego. He will discuss the stem cell-based models of disease and his work in using stem cells to fight against Alzheimer’s. Click here to register for the November Alzheimer’s Talks.
Though only about one percent of the population have a rare, genetically inherited form of Alzheimer’s disease, studies with this population give scientists great insight into how the disease works as well as ways to prevent and treat it in the general Alzheimer’s population.
Dr. Randall Bateman is the Charles F. and Joanne Knight Distinguished Professor of Neurology at the Washington University School of Medicine and Director of the Dominantly Inherited Alzheimer’s Network Trials Unit (DIAN-TU), which aims to prevent the onset of cognitive impairment by launching the first clinical trials for this population. He joined us for this month’s call to describe the study, and discuss its implications for the broader population of people who have, or may get, Alzheimer’s disease.
Key highlights from the call:
- Alzheimer’s was historically studied as a rare disease that occurred in younger people. In the 1970s and ‘80s, researchers realized the same disease was also occurring in older people as well, and in a much larger percentage of the population.
- DIAN-TU is the first ever prevention trial in Alzheimer’s disease. In this global network of patients with dominantly inherited Alzheimer’s disease, researchers are able to predict not just who will get the disease but when they will get it, which has allowed them to launch the first prevention trial in Alzheimer’s disease. The main goal is to try to prevent cognitive loss, and information from this study will have applications for all people with Alzheimer’s.
- Different biomarkers are being tested in this trial. This may lead to shortcuts that will allow us to test many more drugs.
- People in this study may respond better to treatments. Dominantly inherited Alzheimer’s strikes at a young age, so patients are less likely to have other diseases or health issues. This makes them a pure population to study and gives excellent information about drug efficacy.
- Researchers are optimistic that we are close to a treatment. Even penicillin failed its first few clinical trials, meaning it is important to find the right dose and the right patients. We have information that is pointing us in the right direction, so it is important to double down efforts to bring together researchers and public-private partnerships and increase the number of trials. With increased funding and research, we could have a possible treatment as soon as a few years from now.
People who come from families who have these mutations are eligible to join the trial. If you think that your family has a high prevalence of Alzheimer’s disease that strikes people at a young age, typically less than age 55, visit www.DIANexr.org, to learn more about this study, or you can contact them directly at 1-844-342-6397 or by email at Dianexr@wustl.edu.
If you don’t qualify for this specific trial, other resources mentioned by Dr. Bateman include: The Alzheimer’s Prevention Registry and The A4 Study. Dr. Bateman also provided the information for the National Society for Genetic Counselors, which has a ‘find a genetic counselor’ tool.
Thank you to Dr. Randy Bateman for sharing his important research. And a very special thank you to donations from Karen and Chris Segal and the Zickler family foundation, which made this call possible.
If you missed the talk – or if you’d like to hear it again – you can listen to an audio playback or read the transcript of our conversation.