Being an advocate for your loved one with LBD and other disorders – UCSF webinar

Editor’s Note: Although the focus of this webinar was advocacy for those with Lewy body dementia, this webinar applies to those who have loved ones with any neurological disorder (not just LBD and not just dementias). This is one of the most comprehensive webinars I’ve ever heard. This should be required reading for every caregiver!

In April 2020, the UCSF Memory and Aging Center (MAC) hosted a webinar on advocacy in healthcare and community settings — the fourth in its ongoing series on Lewy body dementia (LBD).  The two wonderful speakers were Sarah Dulaney, RN, clinical nurse specialist with UCSF, and Helen Medsger, long-time Brain Support Network LBD support group member.  Helen cared for her sister with LBD.  They discussed the importance of establishing a daily routine and preventing falls, plus provided activity suggestions and tips for communication.

Thanks to Helen Medsger for alerting us to the MAC’s LBD webinar series.  You can find the list of the full series here:

UCSF Lewy Body Dementia Caregiver Webinar Series

The April 28th webinar featuring Sarah Dulaney and Helen Medsger was recorded and is available on YouTube:

www.youtube.com/watch?v=BsKMUCcfehk

The MAC is asking that everyone who views the recorded webinar provide feedback through a brief survey here:

https://ucsf.co1.qualtrics.com/jfe/form/SV_3gCsLd4Ed4CRaOF

This webinar is worth listening to and/or reading the notes (see below)!

If you missed the first three webinars in this series, check out some notes here:

What is Lewy body dementia?
www.brainsupportnetwork.org/what-is-lewy-body-dementia-and-how-is-it-treated-ucsf-webinar-notes/

Behavior and mood symptoms in Lewy body dementia
www.brainsupportnetwork.org/behavior-and-mood-symptoms-in-lewy-body-dementia-ucsf-webinar-notes/

Managing daily activities and fall prevention
www.brainsupportnetwork.org/managing-daily-activities-and-fall-prevention-in-lewy-body-dementia-ucsf-webinar-notes/

For further information on Lewy body dementia, look through Brain Support Network’s list of resources:

Brain Support Network’s Top Resources on LBD

Lauren Stroshane, with Stanford Parkinson’s Community Outreach, listened to the webinar and shared her notes.  See below.

Robin

. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .

Advocacy in healthcare and community settings – Webinar notes
Presented by the UCSF Memory and Aging Center
April 28, 2020
Summary by Lauren Stroshane, Stanford Parkinson’s Community Outreach

“As family caregivers, we often play many roles, including scheduler, financial manager, housecleaner, encourager, nurse, navigator, nurturer, and more. Perhaps the most important role, though, is advocate, as we ensure the best life possible for our family and friends when they are vulnerable.”

– Amy Goyer, AARP’s Family and Caregiving Expert, 2016

Whether you know it or not, you are already a healthcare advocate in many ways. Lewy body dementia (LBD) is a progressive neuro-degenerative illness that will require a high level of care over time.

A sample dementia caregiving trajectory, from the National Academy of Sciences (2016):

  1. Awareness – Checking in and monitoring. Light errands. Communication with medical providers. Advance care planning (medical & legal).
  2. Unfolding responsibility – Monitoring symptoms and medications. Managing finances and household tasks. Hiring, training, and managing caregivers. Coordinating medical care. Providing emotional support.
  3. Increasing care demands – Monitoring safety risks and behavior. Symptom monitoring and management. Personal care. Acute care transitions. Insurance and public benefits.
  4. End of life care – Communication of the person’s values and preferences. Advocacy and surrogate decision making. Comfort and sensory stimulation. Personal care.

What are some tasks/responsibilities of LBD caregivers?

  •     Medications, appointments, diet, and lifestyle
  •     Monitoring, responding, and alerting
  •     Learning & sharing information
  •     Communication & documentation
  •     Record keeping, insurance, finances, & public benefits
  •     Negotiation, advocacy, & surrogate decision-making

The volume of responsibilities can be overwhelming, but you don’t have to go it alone. Sit down and talk with loved ones to see what responsibilities can be shared or delegated.

Carrying the burden of caregiving alone can be dangerous for your health, with up to a 63% higher mortality rate.  Respite care, such as home care, a day program, housekeeping, or meal delivery can be very beneficial, both for you and for the person with LBD.

Other sources of help might be family, friends, or a neighbor; a private care manager or fiduciary; a publicly appointed conservator or guardian; or a county case manager if the individual is low-income. When looking to hire a professional, seek recommendations from a trusted source, such as the local chapter of an advocacy group such as the Alzheimer’s Association, members of a support group, a senior program, or your attorney.

When is a publicly-appointed conservator required? When the individual can no longer take care of themselves and does not have a family member or close friend to act on their behalf.

Tips & strategies for healthcare advocacy

Each of these are explored in depth throughout the rest of the summary:

  1. Compile & organize critical documents and information. 
  2. Educate yourself about LBD.
  3. Understand the person’s needs, preferences, goals, and values to understand what is important to them.
  4. Build a “village of care” team.

Compile & organize critical documents and information

A three-ring binder can be portable and easy to update, or virtual options as preferred. Provide copies for others who serve a primary caregiver role for the individual.

  • Medical information for the patient
    • Medical history, current diagnoses
    • Current list of medications (name, reason, dose, route, schedule)
    • List of allergies or contraindicated medications
  • Emergency contacts (name, email, phone #)
  • Patient’s ID, insurance card, and medical record #
  • Names and contact information for the patient’s medical providers
  • Legal documents
  • HIPAA Release of Information form (one for each health setting)
    • Every health delivery system will have their own form and requires it to be signed by the individual, if they are able, before they will release health information to you.
  • Advance Healthcare Directive or Durable Power of Attorney for Healthcare: a legal document that allows an individual to spell out their preferences for life-saving medical interventions and end of life care, ahead of time, and designates an individual who is empowered to make healthcare decisions on their behalf, if necessary.
    • Prepare for Your Care
    • This requires that you speak with the loved one about their wishes while they are still able to consider such decisions.
  • POLST (Physician’s Order for Life Sustaining Treatment) tells emergency professionals what to do in a crisis when the patient cannot speak for themselves.
  • Durable Power of Attorney for Finances: a legal document that establishes who is permitted to make financial decisions on behalf of the individual.
    • It is important to consult with an attorney while the individual still has mental capacity, to ensure this is set up correctly and that there is no concern about fraud or financial abuse.
    • The Consumer Financial Protection Bureau offers a helpful guide on managing someone else’s money.
  • Representative Payee (Social Security): if your loved one is eligible for Social Security, visit the Social Security Administration website to learn about how you can manage these benefits on behalf of your loved one.
  • VA Fiduciary (Veteran’s benefits): if your loved one is eligible for Veteran’s benefits, read this handout from the Veterans Affairs website to learn about how you can manage these benefits on behalf of your loved one.

Educate yourself about Lewy body dementia

It’s important to understand the common symptoms and risks associated with LBD, to prepare yourself to be an effective and empathetic caregiver.

Those with LBD commonly experience:

  • Cognitive and functional decline
  • Lack of safety awareness
  • Mobility problems and fall risks
  • Swallowing problems and weight loss
  • Mood and behavioral symptoms
  • Sleep disruption
  • Dysfunction of the autonomic systems in the body, affecting urination, digestion, and blood pressure regulation.

How to accomplish all of this?

Amassing all of this documentation and knowledge can feel like an insurmountable task at first! Prioritize needs and goals to help yourself determine what to tackle first:

  • Physical/medical
  • Emotional/psychological
  • Social/familial
  • Legal/financial

Most importantly, learn to ask for (and accept) help from others! Whether family, friends, or professionals, reach out to others and try to build a “village of care” team to help support you and your loved one.

Members of a village of care team may include:

  •     Primary care provider
  •     Specialist provider, such as a movement disorders specialist
  •     Friends, family, neighbors
  •     Therapist, support group
  •     Respite care providers
  •     Elder law attorney, financial planner, or case manager

What to expect in different medical settings?

Let’s say you have all your documents organized and your “village of care” team in place. Next it’s helpful to know what to expect at different levels of medical care for your loved one. The second half of the webinar focused on common care settings and what to expect at each one, in terms of the team you will be communicating with, the types of help available, and the best ways to advocate for your loved one.

Care in ambulatory clinics

Ambulatory clinics include doctors’ offices, urgent care clinics, outpatient surgery settings, dialysis units, and many others. The team will likely include a clinic coordinator or medical assistant, a social worker, triage nurse, and provider (physician, advanced practice nurse, or physician assistant).

Types of help that are available in a clinic setting typically include:

  • Triage urgent care needs
  •  Diagnostic and routine follow-up appointments
  •  Medication refills and prior authorization
  •  Help with forms
  •  Referrals for specialty care, home health, outpatient rehab, and durable medical equipment such as a walker or wheelchair
  •  Information and resources

Consider the best way to communicate

Who should you be communicating with when a need arises? In many clinics, a care coordinator or nurse will confer with you first, to determine if input from the doctor is needed. Find out how the communication workflow operates in this clinic – how do they prefer that you contact the office? Does the doctor or medical group have a phone app or online messaging? An online portal or secure email? Or are they “old school” and prefer phone or fax?

  •  Electronic medical record messaging
  •  Secure email
  •  Phone
  •  Fax
  •  Scheduled phone or video visit
  •  Routine or urgent in-person visit
  •  Quarterly care planning meetings (at long-term care facilities)

How to get the most out of visits with providers

  1. Prepare and prioritize 3-4 concerns to bring up during the visit. More may not be practical to address in one appointment. What is most important? If you have kept notes since the last visit about how your loved one is doing, that’s great! If not, sit down a day or two before the appointment and write down some bullet points about how they’ve been, any changes and concerns.
  2.  Stay focused – describe symptoms or changes briefly, including the most important information such as when it started, how often and when it occurs, and if it is improving or worsening.
  3. Be honest with the care provider, even if it is uncomfortable. If you are truly unable to speak openly in front of your loved one, send a message ahead of time to the provider so they can bring it up in person, if appropriate.
  4. Advocate if you feel your concerns are not being addressed.
  5. Accept that sometimes providers are not able to provide the answers you seek. If you feel your concerns are being ignored, it may be appropriate to seek a second opinion.

Learn what is covered by your insurance

To help avoid surprising or potentially devastating medical bills, it’s a good idea to know what your insurance covers as far as appointments, procedures, and medications. Contact your insurance company to review your coverage, or check what benefits you are eligible for on government and insurance websites:

If you don’t have comprehensive drug coverage, the website www.GoodRx.com is a helpful resource to compare out-of-pocket drug prices among different pharmacies, and often provides coupons as well. 

Emergency room visits and hospitalizations

One of the largest challenges in caring for someone with LBD is when they require treatment in a hospital or emergency department. Providers you may interact with include:

  • Patient care assistant or nursing assistant
  • Shift nurse
  • Charge nurse
  • Rehabilitation therapist (speech, physical, or occupational therapy)
  • Discharge planner (typically a nurse case manager or social worker)
  • Attending physician

Since your loved one is likely injured or ill, if you are in a hospital setting, do your best to be prepared: have all your emergency information with you, have a list of their current medications with you, introduce yourself to the team, and do not be afraid to ask questions if you need clarification.

Types of care that are available:

  • Evaluation and treatment of severe symptoms, acute conditions, and serious injuries
  • Scheduled operations and procedures
  • Discharge plan
    • Follow-up care instructions
    • Follow-up care appointment
    • Medications and referrals
    • Home health
    • Skilled nursing facility

How does Medicare handle payment for a hospital admission?

Let’s say you came through the emergency room and your loved one was admitted to the hospital. Is Medicare going to reimburse the cost?

“Observation status” or “social admission” means Medicare will not cover a post-hospitalization stay in a skilled nursing facility.

To be eligible for skilled nursing coverage under Medicare, typically a 3-day hospitalization with an “admitting diagnosis” is required.

In a hypothetical scenario, your loved one with LBD has become really lethargic suddenly. They aren’t eating or taking their medications, and their regular doctor advises that you take them to the emergency department (ED). After you arrive in the ED, the doctor there orders testing to see if there is an underlying infection, such as a urinary tract infection (UTI), that would need treatment.

What’s tricky with LBD is that symptom fluctuation is a core feature of the disease. This could be an instance of that. In our hypothetical scenario, the test results in the hospital don’t indicate any infection, and an LBD fluctuation seems most likely.

As the day goes on, your loved one does not improve, however, and they are admitted to the hospital for further monitoring. The attending physician feels that they might benefit from staying in a skilled nursing facility (SNF) for a period of time to help bring them back to their previous baseline.

At this point, it is very important to know their original hospital admission status. Were they there under observation, or social admission status? If so, Medicare will NOT cover the skilled nursing stay afterward. If they had an admitting diagnosis, such as a UTI or sepsis, and required 3 days of hospitalization or more, then typically Medicare will cover it.

It’s essential that you speak with the assigned discharge planner – who may be a case manager or social worker – and review the options before your loved one is discharged from the hospital.

As you get closer to discharge, if you feel like their condition has not improved and warrants more care than you or the rest of the existing care team can provide, then you may have the right to challenge the discharge as unsafe. Before taking this step, however, it’s important to know your rights, and carefully consider whether this is truly warranted.

The California Advocates for Nursing Home Reform provides a factsheet with useful information about this process.

Long-term care settings

Heading into this care setting, it’s important to know whether you expect this to be a short-term rehab stay for or a long-term “custodial” stay. This will determine if the stay is covered under Medicare or Medicaid, or if you will be responsible for payment. To avoid surprises, make sure to discuss all these options carefully with the facility’s billing office, at the beginning of placement.

Short-term rehab stay:
– Full coverage for up to 20 days, then partial coverage by Medicare for 80 more days
– Requires the ability to make progress towards rehab goals

“Custodial” long-term care:
– Private pay
– Medicaid, if eligible

Team members that you may interact with include:

  • Nursing assistant
  • Shift nurse
  • Activities coordinator
  • Rehabilitation therapist (PT, OT, ST)
  • Social worker
  • Director of nursing
  • Provider (physician, advanced practice nurse, or physician assistant)
  • Administrator

How to advocate in long-term care settings?

If your loved one is placed in a facility – whether for a short-term rehab stay or for long-term care – and you need to advocate for their safety and well-being, it’s important to familiarize yourself with residents’ rights (sometimes called patients’ rights). By law, facilities must post these rights on the wall in a visible place; it’s a good idea to locate and read these documents. You can take a photo to read in more detail later, if need be.

Recognize workforce challenges the facility staff are facing. What is the staff-to-patient ratio? Do they meet or exceed state regulations? Maybe the staff were unable to meet your loved one’s needs because they were busy taking care of many residents. Sometimes it is necessary to let a few things go or lower some expectations, but never at the risk of your loved one’s safety.

Prioritize needs and communicate your concerns concisely to the staff. Open the lines of communication and offer to work with staff to address any issues. Participate in quarterly care planning meetings to touch base with the care team. If necessary, you may need to escalate concerns that are related to safety and well-being, if they are not being addressed.

Contact the local Long Term Care Ombudsmen, a trained patients’ rights advocate. Their phone number is also required to be posted visibly in the facility.

Home health care

If the person you are caring for is able to stay at home, but needs additional medical care in that setting, this is called home health care. Examples of this type of care include:

  • Skilled nursing (wound care, infusions, medication management, teaching)
  • Rehabilitation (PT, OT, ST)
  • Medical social services
  • Durable medical equipment (DME), such as a walker, wheelchair, or brace
  • Limited home care assistance (such as toileting or bathing)

Medicare coverage typically requires a referral from a provider and ongoing oversight. The individual must also be homebound. Coverage is typically intermittent, such as fewer than 7 days a week and less than 8 hours a day, over a period of 21 days. There is sometimes a 20% copay.

Palliative or Hospice care

While palliative care has historically been associated with the end stages of life, it can actually provide assistance at almost any stage of illness. The goal of palliative care is to reduce suffering and improve quality of life. Hospice care is appropriate for advanced or terminal illness, when the person likely has less than six months to live. In some cases, patients go on hospice and survive longer than anticipated; they then “retire” from hospice but can go back on it if their condition worsens again. The fluctuations experienced by dementia patients means this is common in those with LBD.

Hospice services are covered by Medicare; palliative care typically is not. Availability of palliative care varies greatly by location and health plan coverage. The support services for patients and caregivers can be greatly beneficial. Check with the primary care provider for more guidance.

A palliative care or hospice team typically includes:

  • Home health aide
  • Social worker
  • Nurse
  • Chaplain
  • Attending provider (may be a primary care doctor or a hospice/palliative care doctor)

Types of care provided:

  • Various palliative care programs
  • Medications
  • DME such as a hospital bed
  • Help with bathing
  • Incontinence supplies
  • 24/7 on-call triage nurse
  • Weekly in-home nurse visits
  • Ancillary services
  • Limited respite (varies)

. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .

Advocacy in community care settings

In the United States, medical care and social care are considered quite separate, more so than in some other countries. Legal and financial assistance was mentioned earlier in the presentation, but falls under this category as well, and is essential to consider, particularly as the disease progresses and the costs of care may or may not be covered entirely. An attorney with expertise in elder law can be very helpful for this process.

The Community Resource Finder, funded by the Alzheimer’s Association and AARP, can be helpful for finding local options.

Psychosocial caregiving is a bigger responsibility than we often give it credit for. It often arises early in the disease and is an important role that the caregiver plays, in helping the individual but also the family and broader community to understand their changing needs and abilities.

  •     Balancing activity and rest
  •     Maintaining connections and managing conflict
  •     Informing and preparing
  •     Planning and scheduling social events
  •     Adapting communication
  •     Simplifying social engagements

Care management services

If you are feeling (understandably) overwhelmed by all these responsibilities, there is often help available in the form of care needs assessments and recommendation, coordination of care and services, and assistance with home care arrangements and placement.

There are private pay options, for those who can afford it. Fortunately, California also has subsidized programs:

The Family Caregiver Alliance, which is part of the National Center on Caregiving, provides many resources such as a list of nonprofit Caregiver Resource Centers in California.

Medicaid waiver county-based case management programs.

Program for All-inclusive Care for the Elderly (PACE).

Other community support services

  • Housekeeping
  • Meal or grocery delivery
  • Transportation – Paratransit, ride-sharing apps, specialized ride-share services such as Go Go Grandparent
  • Money management
  • Home modification programs – these are sometimes available through your local county

Congregate respite programs

The speakers encourage caregivers to consider these types of programs for their loved one with LBD earlier in the illness, not later.

  • Senior centers
    • Free or low cost
    • Meals and activities
    • May not be appropriate for those with cognitive impairment
  • Social day programs
    • Private pay
    • Typically includes transportation, meals, and activities
    • Medication administration
  • Adult day health centers
    • Private pay and Medicaid
    • Typically includes transportation, meals, and activities
    • Medication administration
    • Rehabilitation services (PT, OT, ST)
    • Nursing and social work services

To find these kinds of resources in your own community, you can search the following websites to see what is located near you:

In-home care

Not to be confused with home health care, in-home care refers to general assistance in the home and is not covered by Medicare. This includes help such as companion care, light housekeeping, supervision, and personal care such as toileting, bathing, and dressing.

The Family Caregiver Alliance has a useful article on hiring in-home help.

Those who can afford to do so often use a home care agency, which handles background checks, liability insurance, staff training, and scheduling.

A private caregiver is typically cheaper, but carries potentially higher risk (such as taxes, liability, and labor laws).

A live-in caregiver typically provides services in exchange for reduced rent; this type of arrangement can sometimes be subject to liability and labor laws.

Medicaid community-based in-home care waiver programs vary by state:

CA Department of Social Services In-Home Supportive Services Program.

Care in an assisted living facility

These types of facilities are for individuals who require less medical intervention than in skilled nursing or a nursing home. Instead, they provide the types of assistance that in-home care might help with: meals, help with medications, personal care, activities, and sometimes dementia care.

  • Board and care home – typically 6-8 residents, often lower-cost options
  • Assisted living facility – can have hundreds of residents, varying levels of assistance
  • Memory care facility

Some retirement communities might provide all levels of care, from assisted living to skilled nursing. The cost of these facilities varies greatly. The Family Caregiver Alliance has a helpful web page with tip sheets comparing the different residential care options.

. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .

Other ways to advocate

Participation in research can be deeply rewarding for those with LBD. You can learn more about what clinical trials are currently recruiting at the national database of clinical trials.

There are many different beliefs around organ and brain donation. For some, brain donation can be a rewarding way to feel they are leaving a legacy and helping to further our understanding of neurodegenerative illness. Brain Support Network is a nonprofit organization that facilitates brain donations, educates families, and organizes caregiver support groups.

Consider volunteering with an advocacy organization such as the Lewy Body Dementia Association.

. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .

Question & Answer Session

Q: In this time of Covid-19, how can we advocate for loved ones’ needs even if we may not be allowed to visit them in the hospital or facility? How can we make sure their needs are advocated for remotely?

A: The possibilities will be largely dependent on the facility and staff. It’s generally a matter of calling the facility and hopefully finding a staff member who is willing and able to facilitate contact (via phone or a video call) with the resident. This might be a social worker, an activities coordinator, or the director of nursing. They have heard of some families purchasing an Amazon Echo Show device for their loved one with LBD, which allows the family to make video calls easily. Scheduling a time each day or week with facility staff to check in with the individual and/or the staff can be very helpful, both for the staff to make time regularly and for the family to get regular updates. Sometimes calling at mealtimes may be beneficial.

Others have organized “window visits” to arrange for a staff member to help the resident to a designated window while the family waves to them from outside. Finding ways to support the staff, who may be under tremendous stress – such as helping coordinate access to personal protective equipment (PPE) that may be in short supply, or arranging meals to be delivered for staff members – may help the staff have the bandwidth to assist with resident contacts as well.

For those who end up in the hospital, the inability to visit or be present for someone with LBD can be extremely challenging. The change of environment and routine, the potential for developing delirium, and the lack of familiar faces can be especially difficult for LBD patients. The hospital’s supply of PPE is likely what will determine their ability to permit visitors.

Q: I am having a hard time finding assisted living facilities whose staff are experienced with LBD. Do you have any tips about how to educate the staff about working with this patient population?

A: Helen has taught classes to student nursing assistants before, regarding this exact topic. She would bring educational materials to pass around, such as booklets and leaflets, and recommends the Lewy Body Dementia Association (LBDA) website as a good resource. Those on the front lines will be better able to care for someone with LBD if they have the information they need. Offer to facilitate instruction if need be. Also keep in mind that the staff needs to know about “your” person specifically; LBD can present very differently among different people.

In facilities with very large numbers of patients, sometimes it can be difficult for staff not to think of your loved one as “just another person.” You can put together a short bio of your loved one: where they grew up, what family they have, their interests and hobbies, to help humanize them so they are not just their diagnosis.

Q: What if the staff at the facility does not understand LBD? I have tried to give websites and pamphlets but staff were not receptive. There is also a lot of staff turnover, complicating these efforts. My family member has a lot of disruptive sleep behaviors and I would really like to help the staff understand; what can I do?

A: Be persistent; there is often turnover in the direct care staff; hopefully less so in the higher-level nursing staff or activities coordinators. Finding someone on the staff who can be a champion for you would be ideal. If someone can’t spend an hour on the phone with you, maybe you can catch them for 15 minutes here and there. Share information in small, digestible chunks. It’s not a straightforward process, particularly right now during the pandemic.

Try to call and establish a care plan; be proactive rather than waiting out an unsafe situation. Plan for the worst; if it’s a good day, then you’ve got it covered.

Sometimes having a letter from a physician stating that it’s okay to let someone sleep if they need it rather than waking them up for mealtimes, for instance, can release the facility from liability for that particular issue and allow them to tailor the care as needed.

Depending on where your loved one is at with their disease, hospice can also be a great advocate for helping to provide good care and keep them comfortable.

Basket Trials and Neurodegenerative Disease – Summary of a PSP, CBS, and AD Study

This research summary, written by medical writer Lauren Stroshane, has been posted here with permission from Stanford’s Parkinson’s Community Outreach.  The summary addresses a drug trial by the UCSF Memory and Aging Center that evaluated an experimental drug, TPI-287 in Alzheimer’s Disease (AD), corticobasal syndrome (CBS), and progressive supranuclear palsy (PSP).  While the drug was not effective, scientists still learned useful things from this “basket trial,” which is a research method used in cancer research.

Here’s Lauren’s summary:

parkinsonsblog.stanford.edu/2019/12/research-summary-basket-trials-and-neurodegenerative-disease/

Research Summary:  Basket Trials and Neurodegenerative Disease
December 5, 2019
by Lauren Stroshane with Stanford’s Parkinson’s Community Outreach

A recent drug trial by the UCSF Memory and Aging Center is the first of its kind for neurodegenerative disorders and represents an exciting new method for evaluating the effectiveness of a single drug on participants with the same underlying biology, but different diagnoses. First used in cancer research, basket trials lump together patients by disease pathology, not by diagnosis or symptoms. The UCSF trial evaluated the effectiveness of a drug, TPI-287, in treating toxic buildup of tau protein in the brains of patients with Alzheimer’s disease (AZ), corticobasal syndrome (CBS), and progressive supranuclear palsy (PSP). While the drug ultimately did not show promise, scientists still drew useful takeaways from the study, and future studies of neurodegenerative disease may benefit from utilizing a basket trial design.

What is a basket trial?

A fairly recent innovation in cancer research, basket trials evaluate the effect of a drug on one underlying mutation that can cause a variety of different cancers [1]. For instance, treatment developed for melanoma has also been shown to treat some forms of lung cancer [2]. Basket trials are an offshoot of precision medicine that helps scientists and clinicians identify which patients are likely to be helped by specific treatments. By lumping patients together into the same “basket,” scientists can evaluate a drug’s effects among a broader group to see if benefits are observed. This is distinct from an “umbrella” trial that contains different arms of study, with patients assigned to separate arms depending on their cancer type [1].

An added benefit of the basket study is that it has the potential to open up future treatment eligibility to a larger patient population.

Why is this important for research in neurodegenerative disorders?

Most patients with a neurologic disease have had the frustrating and time-consuming experience of trying multiple medications before (hopefully) finding one that is helpful for their symptoms. Precision medicine aims to eliminate some of that guesswork, by allowing clinicians to more accurately predict which patients will respond to which medications.

In the neurologic world, basket trials could translate to studying the effect of a drug on a specific brain pathology—rather than on a cancer mutation—among patients with a variety of neurologic diagnoses and symptoms, who nonetheless share the same underlying biology.

The UCSF Memory and Aging Center has adapted this approach to look at different types of dementia which share a common pathology. A type of mis-folded protein called tau is known to accumulate in the brains of people with Alzheimer’s disease (AZ), progressive supranuclear palsy (PSP), and frontotemporal dementia (FTD), among other diseases [3]. Although the symptoms of tau pathology may manifest differently among individuals, the underlying toxic action of the tau protein is thought to be the same.

What did the UCSF study find?

In November 2019, UCSF researchers published results of the first-ever basket trial in neurodegenerative disease, which included 95 participants with AZ, PSP, and CBS, and evaluated their response to the drug TPI-287 [3]. This was a safety and feasibility study, to make sure the drug did not harm participants; if well tolerated, it was hoped that TPI-287 would repair some of the damage caused by abnormal tau in the brain.

While results showed that the drug was ultimately not helpful to treat any of these diseases, the researchers did learn some important takeaways [4]:

TPI-287 caused significant allergic reactions in participants with AZ, but not in those with CBS or PSP. This may be due to the difference in risk factors between these diseases: AZ involves the immune system and carries different genetic risk factors than either CBS or PSP.
Some medications that aim to treat tau pathology may not affect all patients with tau disease the same. Further research will help to identify these differences and make such treatments more useful.

What comes next?

The basket approach proved useful in this study, and the UCSF Memory and Aging Center is continuing to deploy it in further research. Another basket trial is currently underway to evaluate the effects of drug BIIB092 (Gosuranemab) on patients with tauopathies including CBS, primary progressive aphasia (nonfluent variant), tau mutation carriers, and chronic traumatic encephalopathy [5].

Researchers are optimistic that continued use of basket trials will help to speed the progress of research into other exciting therapies for neurodegenerative disorders.

 

Reference List:

[1] Clinical trial design and methodology. American Society of Clinical Oncology. https://www.asco.org/research-progress/clinical-trials/clinical-trial-resources/clinical-trial-design-and-methodology Accessed on December 3, 2019.

[2] Weiler N. ‘Basket’ trials for dementia aim to bring precision medicine to neurodegenerative diseases. UCSF press release. https://www.ucsf.edu/news/2019/11/415901/basket-trials-dementia-aim-bring-precision-medicine-neurogenerative-diseases Published on November 11, 2019. Accessed on December 3, 2019.

[3] Medina M. An Overview on the clinical development of tau-based therapeutics. Int J Mol Sci. 2018 Apr; 19(4): 1160. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5979300/ Accessed on December 3, 2019.

[4] Tsai RM, Miller Z, Koestler M, et al. Reactions to multiple ascending doses of the microtubule stabilizer TPI-287 in patients with Alzheimer disease, progressive supranuclear palsy, and corticobasal syndrome. JAMA Neurol. 2019 Nov. https://jamanetwork.com/journals/jamaneurology/article-abstract/2753779 Accessed on December 3, 2019.

[5] Boxer A. BIIB092 in primary tauopathies: CBS, nfvPPA, sMAPT, and TES (TauBasket). ClinicalTrials.gov. https://www.clinicaltrials.gov/ct2/show/NCT03658135 Accessed on December 3, 2019.

 

Nine tips for traveling with family members impacted by dementia (AFA)

The Alzheimer’s Foundation of America (AFA, alzfdn.org) offers nine tips for traveling with family members impacted by dementia.  These tips apply whether you are traveling near or far.

Robin

———————-

https://alzfdn.org/nine-thanksgiving-travel-tips-families-impacted-dementia/

Nine Thanksgiving Travel Tips for Families Impacted by Dementia

AFA offers the following tips for family caregivers to consider:

  • Advise airlines and hotels that you’re traveling with someone who has memory impairment and inform them of safety concerns and special needs.
  • Inquire in advance with airports/train stations about security screening procedures.  This way, you can familiarize the person beforehand about what will happen at the checkpoint to reduce potential anxiety.
  • Plan the travel mode and timing of your trip in a manner that causes the least amount of anxiety and stress.  Account for the person and their needs when making arrangements; if they travel better at a specific time of day, consider planning accordingly.
  • Preserve the person’s routine as best as possible, including eating and sleeping schedules. Small or unfamiliar changes can be overwhelming and stressful to someone with dementia.
  • Take regular breaks on road trips for food, bathroom visits, or rest.
  • Bring snacks, water, activities and other comfort items (i.e., a blanket or the person’s favorite sweater), as well as an extra, comfortable change of clothing to adapt to climate changes.
  • Consider utilizing an identification bracelet and clothing tags with your loved one’s full name and yours to ensure safety.
  • Take important health and legal-related documentation, a list of current medications, and physician information with you.
  • Depending on the trip duration and/or the stage of the person’s illness, consult with their physician to make sure travel is advisable.

3-step Framework for Diagnosing Dementia (Brad Dickerson, MD)

In this five-minute video, Brad Dickerson, MD, a neurologist at Mass General, describes a 3-step framework for diagnosing dementia:

www.mdmag.com/peer-exchange/early-diagnosis-dementia/recommendations-for-diagnosing-alzheimer-disease

Here are excerpts from the transcript:

Currently, I think we advocate for a 3-step framework that starts by describing the person’s overall cognitive functional status. What we mean by that is, does the person have mild cognitive impairment? Does the person have dementia? … What we really need to do is interview the person and, ideally, an informant, and find out what they are lacking in terms of independent functioning. What have they lost? What do they need help with?

[This] ultimately has major implications for the care plan. Establishing whether the person has mild cognitive impairment or dementia is very important, and I think that threshold varies from person to person and can be quite an arbitrary decision that really takes some clinical experience. Ultimately, what I like to ask people is, if you, as the care partner, can leave the person and go on a trip for a weekend or a week, would they function independently at the things that they need to try to get done to get by in daily life? If the care partner says, “No, I would never do that,” you can pretty comfortably say that the person probably has crossed the threshold into dementia. I think that’s the starting point, No. 1.

No. 2 is, what’s the particular cognitive behavioral syndrome that the person is experiencing? …[Is] the main problem memory loss? Is the main problem executive function? Is the main problem language? Are there multiple problems? A lot of times we see, I think, this common presentation of a person who has memory loss. They’re just not holding on to information, and they also have executive dysfunction. They’re not able to reason. They’re not able to perform tasks to the level that they used to be able to in order to get the job done to reach goals in a valid way.

I think that the syndrome is really meant to capture the major symptoms and signs that the person has of their illness. And that communicates important information to our colleagues and to the patient and family about where their problems are. I think it also allows you to highlight…what their strengths still may be. If this person has a primary memory loss syndrome but their executive function is still good, maybe they can make use of strategies to compensate for some of the problems that they’re having with memory. If they have executive dysfunction, they’re probably not going to be able to do that. Ultimately, that cognitive behavioral syndrome, that second level of specificity in our diagnostic formulation, communicates, in shorthand, to us and to others what the person’s problems are and maybe what they can still do.

And then the third level is, what’s the brain disease that is the cause of the problem? Sometimes it’s multiple diseases. Often, it’s compounded by other medical problems or things like medication effects that affect brain function but are not necessarily a disease in and of itself. The most common, I think, is Alzheimer disease mixed with cerebrovascular pathology in an older adult population—people over the age of 70, 75. In the younger people, I think it can sometimes be a more pure condition, whether it’s Alzheimer disease, or frontotemporal degeneration, or Lewy body disease. Those can often be primary diseases, especially in younger people.

That’s really the 3-step formulation that we advocate that we try to follow. It’s not always possible to be 100% confident in any 1 of those levels, and I think that’s where we have to talk about likely due to Alzheimer disease or likely due to cerebrovascular disease, and rate our level of certainty so we can think about whether we need some additional specialty involvement. If so, what does that involve, and how important is that in thinking about the management? We don’t necessarily have to have the sophisticated biomarkers that we talk a lot about in every individual with dementia likely due to Alzheimer disease. I think there are plenty of people we can all diagnose with fairly straightforward assessments and tests and not do the multimillion-dollar work-up on, that we often end up spending time talking about in the more specialized cases.

 

“The Soul of Care: The Moral Education of a Husband and a Doctor” (book review)

This is a review of a new book “The Soul of Care: The Moral Education of a Husband and a Doctor” by Arthur Kleinman, MD, a psychiatrist and anthropologist at Harvard.  It is about the “stressful, harrowing, depressing, and rewarding” aspects of caregiving for a spouse with early-onset Alzheimer’s Disease.

Excerpt from this article:

In his new book, [the author] chronicles the harrowing decade he spent caring for his wife, Joan, after she was diagnosed with early-onset Alzheimer’s disease in her 50s.  He describes the process of navigating a health-care system that seemed to offer plenty of clinical care but little compassionate care as she declined from a lively, witty Sinologist to a woman unable to see, dress herself or recognize loved ones. In moving detail, Kleinman tells the story of a husband trying to honor, in the best way he could, his wife’s ultimate wish: “I will not die without dignity.”

You can read the full article here:

www.washingtonpost.com/health/caregiving-for-a-sick-loved-one-can-be-stressful-harrowing-depressing–and-rewarding/2019/09/27/dbb41cf4-c918-11e9-a4f3-c081a126de70_story.html

Health
Perspective
Caregiving for a sick loved one can be stressful, harrowing, depressing — and rewarding
By Dhruv Khullar
September 30, 2019 at 6:00 a.m. PDT
Washington Post

Robin