Alzheimer's Research UK’s Post

Wash. Rinse. Repeat. How can two parts of the same #healthsystem not work together to make sure that #Alzheimers patients have access to the newest therapeutic #innovations? Of course, we should make sure that the patients that will benefit most are the beneficiaries with #earlydetection and #accuratediagnosis, but not only for people with the ability to pay out of pocket - this is a detriment to #healthequity and #healthsystempreparedness for all. #TeamUK, let’s unify and (quickly) take another stab at this, shall we?

Putting reporting of rare diseases into perspective. So in this report a drug is toted as prolonging the life in a patient with a neurological disorder. The drug mentioned is a form of cholic acid which is a bile acid we make in our livers every day. It has never been proven to have any effect on neurological disease (how it would get in the brain is beyond explanation) and it has not impacted neurological outcomes in people with a CNS disorders like Zellweger spectrum (which is what I suspect the person has). No claim is made that it prolongs life by the manufacturer - that’s a fake claim in the report. But the reporter gives the impression that it’s life saving therapy for $600,000 a year that everyone is denying. So the reporters claim is false. I’m all for value and when a drug is expensive sometimes it’s needed - when it does something significant. It is doubtful it will do anything here except modify some liver results. Why the reporter didn’t talk to someone who knows about the drug - I don’t know. It brings to discussion how we pay for rare disease drugs - do we pay to correct just lab values or when there is a demonstrable impact on health ? https://lnkd.in/grkGNprn

#ISPEP A call-to-action to all members of ISPEP here. And all #PatientEngagementExperts trying to elevate the importance of #PED to regulatory authorities and develop #COA strategies. Patient data must be elevated and routine as part of #HTA reviews and regulatory authorities must stop neglecting the expertise of Patient Organisations — who’s Scientific Boards have the same knowledge of clinical trial design (flaws and all) as #biopharma. It is long past time for a new algorithm for #HTA assessments. This is no longer an ‘ask’.

This is heartbreaking, but even more than that, it's infuriating. Most people think of the respiratory symptoms when they think of cystic fibrosis. In the 1950s, when Dr. Dorothy Hansine Andersen discovered and characterized Cystic Fibrosis, it wasn't primarily a respiratory condition. The children with cystic fibrosis died in their infancy from the pancreatic symptoms before the lung issues had a chance to develop. (https://a.co/d/dcKeBqS) Cystic fibrosis is one of the great successes in medicine and pharmacology. In only a few decades, it has gone from being a death sentence in infancy, to having a close-to-normal life expectancy with a near-normal quality of life thanks to modulator therapy. The miracle of CFTR modulators (Kalydeco/ivakaftor, or Kaftrio, a.k.a., Trikafta in USA) is that instead of treating the symptoms, they restore the function of the patients' CFTR. The medications are a simple pill and generally well-tolerated with minimal side effects (in stark comparison to treatment of thalassemia which involves blood transfusions, chelator therapy infusions, and numerous side effects from the treatments). Cystic fibrosis and CFTR are my areas of expertise. I have studied all aspects of CFTR from compiling a full exon map of protein domains/important residues/pathogenic variants, taking over twenty credit hours of IUSM courses of the clinical aspects of cystic fibrosis, to learning all I can about the history of cystic fibrosis and the amazing contributions of Dr. Andersen. I am also half-English. To know that young children are being denied a readily available cure for a disease that I study passionately in a place I consider home is too much to bear. I hope this decision is reversed quickly and all these vulnerable patients are given treatment before they start to accumulate lung damage. #cysticfibrosis #NHS #Kaftrio #Trikafta #CFTR2 #CFF #CysticFibrosisTrust https://lnkd.in/evNrdZTk

Big study. Serious complications. Caregivers should think twice...or three times prior to prescribing this class of drugs to dementia patients. Look into Montessori methods to work with patients suffering mental decline. Ignoring these findings may subject caregivers to litigation. www.lifespanhealth.me #dementia #pharmaceutical #montessori #aging #longtermcare #Healthcare #insurance #risk #exercise

Leqembi: A New Era in Alzheimer's Care FDA approval of Leqembi (lecanemab) is a major advance in Alzheimer's treatment. Here's what you need to know: 1. Targeted for Early Stages: Leqembi is approved for early-stage Alzheimer’s patients, mainly 65 and older. 2. Leqembi slows the progression of the disease, reducing memory and cognitive decline by 30% over an 18-month period. 3. How It Works: The drug targets brain amyloid plaques, a key Alzheimer’s marker, to slow disease progression. 4. Eligibility and Administration: For mild Alzheimer’s cases, the drug is given via bi-weekly IV infusions. Efficacy for severe cases is unknown. 5. Accessibility challenges include limited initial access, especially in rural or underserved areas, and potential difficulties with brain imaging for side effect monitoring. 6. Mild infusion-related anxiety or shortness of breath are common. Serious side effects, such as brain swelling or microhemorrhages, occur in about 25% of cases. 7. Medicare covers 80% of the $26,500 annual cost, but out-of-pocket expenses may apply for imaging scans. Leqembi provides accessible and affordable care, but understanding its limitations is crucial. #AlzheimersCare #Leqembi #HealthcareInnovation #SeniorHealth #MedicalAdvancements

Leqembi: A New Era in Alzheimer's Care FDA approval of Leqembi (lecanemab) is a major advance in Alzheimer's treatment. Here's what you need to know: 1. Targeted for Early Stages: Leqembi is approved for early-stage Alzheimer’s patients, mainly 65 and older. 2. Leqembi slows the progression of the disease, reducing memory and cognitive decline by 30% over an 18-month period. 3. How It Works: The drug targets brain amyloid plaques, a key Alzheimer’s marker, to slow disease progression. 4. Eligibility and Administration: For mild Alzheimer’s cases, the drug is given via bi-weekly IV infusions. Efficacy for severe cases is unknown. 5. Accessibility challenges include limited initial access, especially in rural or underserved areas, and potential difficulties with brain imaging for side effect monitoring. 6. Mild infusion-related anxiety or shortness of breath are common. Serious side effects, such as brain swelling or microhemorrhages, occur in about 25% of cases. 7. Medicare covers 80% of the $26,500 annual cost, but out-of-pocket expenses may apply for imaging scans. Leqembi provides accessible and affordable care, but understanding its limitations is crucial. #AlzheimersCare #Leqembi #HealthcareInnovation #SeniorHealth #MedicalAdvancements

While this is news coverage from the US, I don’t think the headline really answers the question it poses. There are several reasons I think the drug is off to slow start including prelim cost, poor health system preparedness particularly imaging needs, and effectiveness vs cost-benefit. If you want to dive in, more details below…. Partly it was the extremely high cost that its predecessor Aducanumab was introduced to the market at (subsequently prices slashed and then completely discontinued) which created major sticker shock given the number of people expected to access it. Secondly, most health systems are woefully underprepared to adequately screen and manage patients on the drug given the number of imaging tests they would need to monitor for side effects - the more serious ones being ARIA which includes brain swelling or bleeding. With limited MRI machines patients would need to wait much longer than the protocol time would recommend if they even get access to them at all. We haven’t even touched on really how under prepared physicians are to diagnose appropriately that early. And lastly the window of effect is in essence to slow down memory decline by about five months. To some patients, this is a huge window of opportunity, for others the cost benefit may not be worth it.

### Key Takeaway New Alzheimer's treatments, like lecanemab and donanemab, shift treatment discussions, urging consideration of quality of life alongside cognitive benefits. ### Summary - Patient Robert seeks Alzheimer's treatments, signaling a perspective shift. - FDA approval of lecanemab and anticipation for donanemab signal significant advancements. - Traditional treatments focused on limited cognitive enhancements. - Lecanemab and donanemab offer potential breakthroughs in Alzheimer's treatment. - Emphasis on considering quality of life in treatment discussions. - Raises questions about individuals wanting to live longer with Alzheimer's. - Upcoming treatments prompt broader discussions beyond cognitive benefits. - Article suggests a paradigm shift in approaching Alzheimer's care. - Part of a health and wellness series covering diverse topics. - The evolving Alzheimer's treatment landscape requires a reevaluation of priorities, balancing cognitive benefits and quality of life.

🌟 **Spotlight on Alzheimer's Treatment: Donepezil** 🌟 As a pharmacist, staying informed about the latest and most effective treatments is crucial. Today, I want to shed light on Donepezil, the drug of choice for Alzheimer's disease. This medication has been a game-changer in managing this challenging condition, helping to improve cognitive function and slow the progression of symptoms. What is Donepezil? Donepezil is a cholinesterase inhibitor that works by increasing the levels of acetylcholine in the brain. This neurotransmitter is essential for memory and learning, which are severely impacted in Alzheimer's disease. Dosage **Adults:** - **Initial dose:** 5 mg once daily at bedtime - **Maintenance dose:** After 4-6 weeks, may increase to 10 mg once daily **Children:** - Not typically prescribed for children. Always consult with a healthcare provider for specific cases. Importance of Monitoring Regular monitoring is crucial to assess the effectiveness and side effects. This includes: - Cognitive assessments - Monitoring for gastrointestinal symptoms - Checking for any heart-related issues Side Effects Common side effects include: - Nausea - Diarrhea - Insomnia - Muscle cramps - Fatigue Drug Interactions Donepezil can interact with: - Anticholinergic medications - Nonsteroidal anti-inflammatory drugs (NSAIDs) - Other medications that affect heart rhythm Contraindications - Known hypersensitivity to Donepezil - Pre-existing heart conditions like bradycardia Patient Counseling - Take Donepezil exactly as prescribed, usually at bedtime. - Do not crush or chew the tablets. - Inform your doctor about any other medications or supplements you are taking. - Report any severe side effects, such as difficulty breathing, severe nausea, or irregular heartbeats, immediately. Disclaimer This post is for educational purposes only. Always consult your healthcare provider for personalized medical advice. 💡 **Stay informed, stay healthy, and keep supporting each other!** #Pharmacy #AlzheimersDisease #Healthcare #Pharmacists #PatientCare #Medicine #PharmacyLife #AlzheimersAwareness #Donepezil Let's make a difference together! 💊💙