Monthly Archives: July 2024

Quantifying love

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This audience does not have to be told that caring for a loved one with PSP can be a major burden.  Two CurePSP Centers of Care have now quantified this using the Zarit Caregiver Burden Scale completed from 2014 to 2022 by 139 caregivers of 131 persons with atypical Parkinsonian disorders.  The results are published in Clinical Parkinsonism & Related Disorders.  Here’s the scale:

Ninety (65%) of the caregivers were women.  Of the 131 patients they were caring for, 59% had PSP, 28% MSA and 13% CBS.  The patients were on average about 4.5 years into their illnesses.  (All three disorders have about the same average rate of progression and survival duration.)  Here are the salient statistically significant results, adjusted for potential confounders as necessary:

  • The average (i.e., mean) total score was 28.8 of a possible 88.
  • Of the four disorders, PSP, CBS and MSA-cerebellar gave similar average scores, but scores for MSA-Parkinson were milder by a margin of about 8 points.
  • Female patients regardless of diagnosis involved a greater caregiver burden than males, regardless of the caregiver gender.
  • Female caregivers reported greater burden than males regardless of the patient’s gender or diagnosis.

The authors list several weaknesses of the analysis and propose a prospective study to avoid these.  They include the absence of consideration of:

  • the relationship between patient and caregiver
  • age and any chronic illness of the caregivers
  • changes in the score over time
  • measures of the patient’s disability, especially falling frequency

The authors’ subjective opinion is that the patient’s cognitive and behavioral deficits are perhaps the most important determinant of the caregiver’s burden.  A future study should examine this hypothesis in a formal way.

Adding these variables to the analysis would require a larger number of patient/caregiver pairs.  This might be best accomplished in the context of a large treatment trial, where “secondary” and “exploratory” measures always accompany the “primary” measure of the drug’s efficacy.  Alternatively, all 36 CurePSP Centers of Care could undertake such a project outside of any treatment trial, assuming adequate funding is available.

The first author of the new paper is Jessica Shurer, a medical social worker who serves as CurePSP’s Director of Clinical Affairs and Advocacy.  The two senior authors are neurologists Alexander Pantelyat and Miriam Sklerov, directors of the Centers of Care at Johns Hopkins and the University of North Caroline at Chapel Hill, respectively, The three are credited with having conceptualized the study along with the second-named author, Margaret Ivancic, a medical social worker at UNC.

The Feds pitch in

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I’m happy to report that the National Plan to End Parkinson’s Act was signed into law by President Biden this week. It covers not only Parkinson’s disease itself, but also “all other neurodegenerative Parkinsonisms, including multiple system atrophy, corticobasal degeneration, progressive supranuclear palsy, and Parkinson’s-related dementia.”

The bill is named after two people: Dr. Emmanuel Bilirakis, who died with PD last year and was the brother of the act’s original sponsor, Rep. Gus Bilirakis, Republican of Florida; and Rep. Jennifer Wexton, Democrat of Virginia, who announced in September 2023 that she has PSP. Both representatives have been critical proponents of the act throughout the legislative process. Here’s the President signing the act on July 2, 2024, seated between Rep. Wexton and Rep. Bilirakis.

Here’s a copy of the act. It requires the Department of Health and Human Services to develop and periodically update a coordinated Federal plan to find the causes and cures of the Parkinsonisms and to make recommendation for future Federal support of those efforts. The act requires the DHHS to appoint an Advisory Council of at least 23 members from multiple stakeholders to meet at least quarterly. It also requires the DHHS to organize an annual research conference on its efforts in this area. Both conferences are to be open to the public. Although the act itself provides no funding — not even for the conferences it requires — the Advisory Council and DHHS are tasked with generating recommendations for future Federal funding.

I’m not sure that this is a step forward for PD, where there’s already excellent public awareness, plenty of research conferences, and robust, ongoing programs in the academic, lay-led non-profit, philanthropic, and pharmaceutical sectors. I don’t know that “coordination” by a Federal agency is going to improve on the current system, which is driven by the inherently powerful, albeit messy, force of our capitalist system and by the collective voices of the 1 million Americans with PD.

But for PSP and the other atypical Parkinsonisms, it’s a different story. With only about 5,000 to 10,000 diagnosed affected people in the US and no major celebrity as its champion, PSP could benefit greatly from the awareness this act could bring. That could help turn the attention of all of those existing PD-interested players toward the atypicals.

And another thing: For years — no, decades — I’ve felt frustration over how promising preliminary findings in PSP often can’t gain traction because of personalities, scientific “fashion,” funding issues or other immediate practicalities. A potentially longer-term view of the Advisory Council could help change that, but only if the group avoids the traditional pressures hampering progress for the atypical Parkinsonisms up to this point. The potential, however, is definitely there.