Avoid Stem Cell Tourism

I’ve mentioned this caveat before, but here are some more official warnings, as summarized well by Neurology Today. Look how many UNAPPROVED stem cell procedure facilities are in the US alone! This goes way beyond laetrile for cancerOriginal.00132985-201607210-00001.FF2

Bottom line: although the rationale sounds good for hoping that stem cells will cure neurodegenerative disorders, there are no FDA approved stem cell therapies available yet!

 

 

Shining the Symptom Searchlight on Behavior

Over the weekend, Dr. Zahinoor Ismail started to get terrific coverage about a new questionnaire he’s developing for helping primary care providers and families to recognize Alzheimer’s disease cases that have other features presenting PRIOR to memory loss. The NY Times picked this up from AAIC on Saturday, and he’s been taking local and national interviews from where we’re staying (our former CAMH boss’ home) during the conference. 

I’m proud of his work and especially his resistance to making more of his creation than has yet to be validated. This can be tempting when media calls, but he appreciates the feedback and optimistic caution expressed by colleagues in that NY Times piece. Emails he’s received in the last few days from caregivers are prompting these notes for you:

  • The take home point for this week is that families often note a mood or behavior change before memory loss becomes overt. If this has been going on consistently for 6 months or more, it may be an indicator of incipient Alzheimer’s disease
  • Folks familiar with the world of frontotemporal dementia have known that memory loss and Alzheimer’s disease are not the only word on dementia. Items on Dr. Zahinoor’s MBI overlap with some of the key features of FTD. 
  • He’s collected data and will analyze before stating cutoff points for scores with reliable clinical significance, so if you feel compelled to go through the scoring process right now, it’s better as a means of communicating symptoms to your treating physician than self-diagnosing! I am hoping it will go further soon, but let’s see. 
  • Please don’t use the MBI contact information to ask personal case questions. That link is mainly for other researchers to connect and help work on the development.  
  • DO call this to the attention of treating physicians – this may not be useful for patients already diagnosed with dementia, but to raise awareness of new potential diagnostic aids serves us all better
  • On a personal note, I’m hoping there’s a day that Z’s MBI will help us recruit the people with earliest symptoms into our clinical drug trials. As you’ve been hearing in the recent past and from this week’s AAIC, much of disease-modifying drug development has gone the way of prevention or earliest intervention.

App Advocacy

AcTo Dementia is working to identify existing apps for devices with touchscreens (tablets) with which patients with dementia demonstrate beneficial engagement. They’re also contacting developers to advise on adaptations that could increase the available options.

Their observational research has shown that 79% of auto-prompts do NOT help advance play. Also of note, both drag-and-drop and single tap activations don’t work well for patients, and the menu bar can be too distracting. Developers were able to take this info and create a new version of a solitaire game that allows users to opt out of these patient-plaguing challenges.

MAB Trials in the Toronto Star

The local press have been getting right into announcements from this year’s Alzheimer’s Association International Conference. Saturday the Toronto Star wrote about 3 studies of note.

1. There is a specially funded study group that focuses on families with genetic mutations that can grant the early-onset, presenilin-related version of Alzheimer’s disease (AD). Its acronym is DIAN (Dominantly Inherited Alzheimer Network). By early-onset, we mean patients with symptoms that begin before age 60. The most common genetic mutation that associates with or causes early-onset AD involves a protein called presenilin, but more rarely some other genes are involved and may not be excluded from entry into DIAN.

2. Clinical trial testing of one monoclonal antibody (MAB) called gantenerumab.

3. Clinical trial testing one monoclonal antibody (MAB) called solanezumab.

You don’t have to have early-onset Alzheimer’s disease to participate in a trial of solanezumab. Click here for more info. We are halfway into recruiting to this study; USC ATRI is the coordinating center (HQ) for this study.

Researchers are actually splitting trials that recruit people at risk for early- and late-onset AD, because the progressions for these are different and merit separation.

One of the take home points from the Toronto Star article is that we are at an exciting time in clinical trials for Alzheimer’s disease. We are recruiting participants who don’t yet have memory loss.¬†

[Shoutout to Barry and RoseAnn, for calling my attention to this article]

Do You Need a “Partner in Crime”?

butchnsundance

The topic today is Trial Partners. Sometimes we researchers refer to them as Study Partners or Informants, the latter of which can cast a negative connotation onto the role.

Before this decade, clinical drug trials for Alzheimer’s disease that had proceeded to Phase 2 recruited patients who had clearly developed symptoms of this type of dementia. The same held true for trials for patients with the other types of dementia: vascular dementia, frontotemporal dementia, dementia with Lewy bodies. By definition then, these participants were ethically vulnerable to proscription into trials. If you can’t remember what the study entails, are you qualified to consent to participation? If you lack the ability to weigh the consequences of your decisions based on other cognitive deficits, are you really able to understand that your participation in the study is voluntary? Could you get yourself out of the study if it proved unsafe or inconvenient or even painful for you?

To help protect participants with cognitive impairment severe enough to qualify as dementia, researchers enlist a trial partner for each vulnerable participant. Not only could the trial partner vouch that the researcher had not coerced the patient into participating, but that partner was likely a more reliable historian about possible side effects or disease progression over the course of the study. Recall that some types of dementia reduce the patient’s ability to perceive his/her own deficits or to remember them long enough to report at scheduled visits to the trial site.

We’re now at an exciting milestone for Alzheimer’s clinical trials. We have begun to test medications that would stop the disease process before significant losses occur. The recruitment of participants to these studies therefore does NOT include those who won’t remember the study drug dose instructions or who can’t sign informed consent themselves. Do these independently functioning, non-demented participants need to have trial partners?

It may depend on the nature of the clinical trial you are considering. If you are fully independent in daily life, maybe a bit forgetful but doing great with a smartphone to keep you on track with reminders and appointment alerts, you could probably handle signing informed consent and participating in a brief (1 year or less) study. The study would end before you could not make notes and keep appointments by yourself. But some of the new trials are designed to go beyond a single year. One we’ll soon launch in North America will last 4.5 years. Given that recruitment is geared toward elderly people at a higher risk for Alzheimer’s disease than their age-peers, chances are good that cognition will change over that period of time. And we will need a person who is objective and qualified by the participant to give us a sense of that change over time.

Some things to keep in mind when you choose a trial partner:

  • Being a trial partner means accompanying the participant to some or all visits. If your potential trial partner has a busy schedule or health considerations that make traveling with you difficult, it’s better to pick someone else. The trial partner does not have to live in your home with you.
  • It’s not an automatic assumption that your spouse/partner will make the best trial bonniepartner. If you tend to disagree with that person’s impression of you or if your relationship is the type that doesn’t tolerate taking instruction, you might want to choose someone else. Trial partners often serve as another pair of ears for the study instructions, and therefore they may act as “deputies” to help make sure the study protocol is followed.
  • If you will be counting on the trial partner to help you stay organized (what pill pack this week? When do we go back in for a brain scan? Which location do we go to this time?), pick someone who you think can fulfill that assignment. The study team will do their best to send reminders, but a disorganized trial partner can derail your completion of study-related tasks.
  • Who’s your back-up? We have seen some trial partners “fall by the wayside” during a study. Some of this is due to other life matters having to take priority or unexpected health issues or even death. Most of us will be happy to sign you on with notes about who might act as your second trial partner, even if this manifests as a short-term, transient occurrence.
  • Not all studies will allow two life partners to be each other’s trial partners. If one develops dementia despite participation in the study, that brings us back to “Who’s your back-up?” above. Research teams may benefit when you reach out to the rest of your social circle – getting others outside of your household involved helps spread the word and speed up recruitment. Speeding up recruitment speeds up the time we’re all waiting to reveal whether the study drug worked the way we wanted it to.

New Brief Study – Older Volunteers Needed

mindspa-2

We are seeking 5 individuals over the age of 65 to test the tolerability of a light therapy non-medical device worn as sunglasses for 20 minutes daily. Trial length: 5 consecutive days.

To volunteer or for more information, please email Dr. Tiffany Chow, tchow@usc.edu or 858-964-0462

Study conducted by Tiffany Chow, MD (USC Dept. of Clinical Neurology)

USC IRB HS-16-00440
Date of Preparation: 6/29/2016

Poetry Corner

One Afternoon at Teatime

Marilyn Hammick

Arthur stops close to where we sit waiting
for the person you call the activities lady
to serve us drinks and biscuits.
He moves his wheelchair with slippered feet,
so we become another group.
You introduce me, This is my sister,
I nod to Arthur and watch his mouth form words
that seem reluctant to reach me, hang
in the air unsteady, diminished.

He continues to speak, I continue to nod,
I think he’s asking about my name,
you seem to understand, or do you guess?
I’m trying to work out if there’s a knack
I’ve yet to grasp, a way to hear
the hush and lisp of his voice, because
all the time you’ve been here, where
you don’t want to be, after all these months
Arthur is the first person you’ve introduced me to.

We choose our biscuits, I drink tea, you have coffee
Arthur has half milk, half coffee and continues to speak.
I think he’s talking about his family, two daughters, a son,
I’m unsure so I ask is he watching the rugby?
No, his game is football and there’s something
about a golden goal. I say what position did you play
and hear, clearly, outside left.
Something rights itself inside me.

About the poet:

Marilyn Hammick retired in 2013 from her international consultancy in medical and healthcare professional education, specializing in evidence-informed education, systematic reviews and interprofessional education. Previously she was in clinical practice as a therapeutic radiographer. She writes (and reads) when traveling, during still moments at home in England and France, and when recalling her childhood in New Zealand and years living in Iran. Her poems have appeared in Obsessed With Pipework, Prole, The Interpreter’s House, The Linnet’s Wings, The Journal, I am not a silent poet and Nutshells and Nuggets. She was named a “Spotlights poet” in Paragram’s 2015 pamphlet competition.

About the poem:

“My sixty-year-old brother lives, somewhat reluctantly, in a residential-care home. He is now safe, well fed and on the correct medication, and has help with personal care. But he feels that the independence he highly valued as a person with a disability has been taken away from him. His brain damage means that he’s resistant to explanations of why he is living in the right place for him at this time of his life. Most days he sits alone or remains in his room. This poem came from the joy and relief I felt after visiting him as described. Poetry gave me the opportunity to voice my emotions about a small, positive change in his attitude to his new home, and the importance of that to me.”

Poetry editors of Pulse