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Det svenska biofarmabolaget TikoMed och The Division of Clinical Neuroscience vid Oslo universitetssjukhus har nyligen undertecknat ett forskningssamarbetsavtal inom Amyotrofisk lateralskleros – ALS.

TikoMed har tidigare genomfört och rapporterat om två kliniska fas 2a-studier på ALS patienter, båda med lovande resultat.^1,2 Såväl säkerhet som tolerabilitet för läkemedelskandidaten ILB® hos patienterna kunde verifieras. Dessutom pekar resultaten på ILB®s framtida potential som det första sjukdomsmodifierande läkemedlet för behandling av både familjär- och sporadisk ALS med minimala biverkningar.

Lars Bruce, som ligger bakom utvecklingen av ILB® och leder programmet kommenterar:
”Min hypotes är att ILB® representerar «The Missing Link» som orkestrerar kroppens signaler för reparation av vävnader. ILB® aktiverar kroppens naturliga reparationssystem och är ett läkemedel som behandlar neurodegenerativa och neuroinflammatoriska processer genom endogen aktivering av en repertoar av tillväxtfaktorer. Jag sätter stort hopp till att samarbetet med Oslo Universitetssjukhus ska leda till mer kunskap och bättre behandlingsalternativ för patienter med ALS.”

”Vi är mycket exalterade över den kliniska potentialen för ILB®, en behandling som kan visa sig vara banbrytande för patienter inom flera förödande neurodegenerativa sjukdomar”, säger TikoMeds vetenskapliga rådgivare, professor Ann Logan som är hedersprofessor i regenerativ medicin vid University of Warwick.

Publikationer:

1. Logan A, Nagy Z, Barnes NM,Belli A, Di Pietro V, Tavazzi B, Lazzarino G, Lazzarino G, Bruce L, Persson LI. A phase II open label clinical study of the safety, tolerability and efficacy of ILB® for Amyotrophic Lateral Sclerosis. PLoS One. 2022 May 25;17(5):e0267183. doi: 10.1371/journal.pone.0267183. PMID: 35613082; PMCID: PMC9132272.

2. Srinivasan V, Homer V, Barton D, Clutterbuck-James A, Jenkins S, Potter C, Brock K, Logan A, Smith D, Bruce L, Nagy Z, Bach SP. A low molecular weight dextran sulphate, ILB®, for the treatment of Amyotrophic Lateral Sclerosis (ALS): an open-label, single-arm, single-centre, phase II trial. PLoS One. 2024 (in press).

Fortsatt positiv utveckling inom nya behandlingar, ökade krav på lagerhållning och fokus på konkurrenskraftiga kliniska prövningar. Det är tydliga trender i Läkemedelsverkets årliga omvärldsrapport.

En av trenderna som lyfts i Läkemedelsverkets omvärldsrapport är den fortsatt positiva utvecklingen inom nya behandlingar. Foto: Johnér

– Samverkan i EU och globalt kommer att bli avgörande för hur vi lyckas möta de hot vi ser, mot folkhälsan och mot stabiliteten i vår omvärld. Det beredskapsarbete som nu pågår i Sverige är viktigare än någonsin. Samtidigt ser vi en explosionsartad utveckling av nya behandlingar, kommenterar generaldirektör Björn Eriksson.

Utveckling av nya behandlingar

Utveckling av avancerade terapier och biologiska läkemedel inklusive vacciner ökar, både inom human- och veterinärmedicinen. Behandlingar blir alltmer individualiserade, såsom vissa läkemedel för avancerade terapier. Statistik från EMA visar på att ansökningar inom terapiområdena onkologi, hematologi och neurologi kommer fortsatt att vara aktuella. Infektions- och inflammatoriska sjukdomar, demenssjukdomar och oftalmologi är andra centrala områden.

Försörjningsberedskap och lagerhållning en viktig samhällsfråga

Erfarenheterna från pandemin och numera också den geopolitiska situationen har lett till omfattande diskussion om Sveriges försörjningsberedskap rörande läkemedel och medicintekniska produkter. En trend är att bygga lager i samhället på flera olika nivåer. Statliga utredningar föreslår utökade krav på apotekens lagerhållning, ökad hemberedskap, en lag om lagerhållningsskyldighet och inrättandet av statliga säkerhetslager. Andra länder ser ett ökat behov av lagerhållning på olika nivåer för att öka sin beredskap och säkerställa läkemedelstillgänglighet.

Bättre förutsättningar för kliniska prövningar

Förutsättningarna för kliniska prövningar behöver förändras. Utredningar visar tydligt på behovet av att nationellt samla kliniska prövningar och skapa en effektiv infrastruktur, som också avlastar en redan överbelastad hälso- och sjukvård. Det kommer att krävas ansträngningar för att öka antalet kliniska prövningar där flera aktörer måste samverka för att överbrygga klyftan mellan forskning, sjukvård och marknad. Nära samverkan mellan hälso- och sjukvård, företag och universitet och högskolor är en förutsättning för att kliniska prövningar ska kunna genomföras på bästa sätt.

Läkemedelsverkets omvärldsrapport 2024

Eli Lillys Kisunla™ (donanemab-azbt) är nu godkänd av FDA för behandling av tidig symtomatisk Alzheimers sjukdom.

Kisunla slowed cognitive and functional decline by up to 35% compared to placebo at 18 months in its pivotal Phase 3 study and reduced participants’ risk of progressing to the next clinical stage of disease by up to 39% .
Kisunla is the first and only amyloid plaque-targeting therapy that used a limited-duration treatment regimen based on amyloid plaque removal; nearly half of study participants completed their course of treatment with Kisunla in 12 months.
Once-monthly infusions of 30 minutes reduced amyloid plaques on average by 84% compared to the start of the study.

The U.S. Food and Drug Administration (FDA) approved Kisunla™ (donanemab-azbt, 350 mg/20 mL once-monthly injection for IV infusion), Eli Lilly and Company’s (NYSE: LLY) Alzheimer’s treatment for adults with early symptomatic Alzheimer’s disease (AD), which includes people with mild cognitive impairment (MCI) as well as people with the mild dementia stage of AD, with confirmed amyloid pathology.1, 2 Once-monthly Kisunla is the first and only amyloid plaque-targeting therapy with evidence to support stopping therapy when amyloid plaques are removed, which can result in lower treatment costs and fewer infusions.3-6

”Kisunla demonstrated very meaningful results for people with early symptomatic Alzheimer’s disease, who urgently need effective treatment options. We know these medicines have the greatest potential benefit when people are treated earlier in their disease, and we are working hard in partnership with others to improve detection and diagnosis,” said Anne White, executive vice president and president of Lilly Neuroscience, Eli Lilly and Company. ”Our deepest thanks to the patients and their loved ones for participating in our clinical programs and to Lilly scientists and collaborators persevering over decades of research. Each year, more and more people are at risk for this disease, and we are determined to make life better for them.”

Amyloid is a protein produced naturally in the body that can clump together to create amyloid plaques. The excessive buildup of amyloid plaques in the brain may lead to memory and thinking issues associated with Alzheimer’s disease.7, 8 Kisunla can help the body remove the excessive buildup of amyloid plaques and slow the decline that may diminish people’s ability to remember new information, important dates, and appointments; plan and organize; make meals; use household appliances; manage finances; and be left alone.1, 7-9

In the TRAILBLAZER-ALZ 2 Phase 3 study, people who were the least advanced in the disease experienced the strongest results with Kisunla. Trial participants were analyzed over 18 months in two groupings: one group who was less advanced in their disease (those with low to medium levels of tau protein) and the overall population, which also included participants with high tau levels.1, 10, 11 Treatment with Kisunla significantly slowed clinical decline in both groups.1 Those individuals treated with Kisunla who were less advanced in their disease showed a significant slowing of decline of 35% compared with placebo on the integrated Alzheimer’s Disease Rating Scale (iADRS), which measures memory, thinking, and daily functioning. In the overall population, the response to treatment was also statistically significant using the iADRS at 22%.1, 12 Among the two groups analyzed, participants treated with Kisunla had up to a 39% lower risk of progressing to the next clinical stage of disease than those taking placebo.13

Among the overall population of participants, Kisunla reduced amyloid plaques on average by 61% at 6 months, 80% at 12 months, and 84% at 18 months compared to the start of the study.1, 14 One of the treatment goals of the study was to remove amyloid plaques to minimal levels consistent with a visually negative scan using amyloid positron emission tomography (PET). If participants were confirmed to have reached these levels, they were able to complete treatment with Kisunla and switch to placebo for the remainder of the study.

Kisunla can cause amyloid-related imaging abnormalities (ARIA), which is a potential side effect with amyloid plaque-targeting therapies that does not usually cause symptoms. It can be detected via magnetic resonance imaging (MRI) scans and, when it does occur, may present as temporary swelling in an area or areas of the brain, which usually resolves over time, or as small spots of bleeding in or on the surface of the brain. Infrequently, larger areas of bleeding in the brain can occur.1, 2 ARIA can be serious, and life-threatening events can occur. Kisunla can also cause certain types of allergic reactions, some of which may be serious and life-threatening, that typically occur during infusion or within 30 minutes post-infusion. Headache is another commonly reported side effect. See the Indication and Safety Summary with Warnings below for additional information.

”This approval marks another step forward in evolving the standard of care for people living with Alzheimer’s disease that will ultimately include an arsenal of novel treatments, providing much needed hope to the Alzheimer’s community. As a physician, I am encouraged by the potential to stop treatment, which could reduce out-of-pocket costs and infusion burden for eligible patients,” said Howard Fillit, M.D., Co-Founder and Chief Science Officer at the Alzheimer’s Drug Discovery Foundation (ADDF). ”Diagnosing and treating Alzheimer’s sooner than we do today has the potential to meaningfully slow disease progression, giving patients invaluable time to maintain their independence for longer.”

Läs hela pressmeddelandet från Eli Lilly här.

Call to Action: Davos Alzheimer’s Collaborative Calls for the Implementation of Innovative, Global Strategies for Alzheimer’s Accurate Diagnosis.

We are entering a new, critical period for the global response to Alzheimer’s disease with novel treatments and diagnostic tests poised to transform care. However, in order for patients, clinicians, and families to realize the benefits of these innovations, health systems, governments, and other stakeholders must prioritize implementation to speed clinical adoption and broaden access.

On June 18th, the Davos Alzheimer’s Collaborative (DAC) Healthcare System Preparedness Learning Laboratory convened 450+ stakeholders from 45 countries and 270+ organizations to explore the current landscape for clinical adoption of blood biomarkers, introduce DAC Healthcare System Preparedness’s (DAC-SP) newly launched Accurate Diagnosis (AccDx) program, and discuss ongoing collaborations with organizations around the world to speed and scale early detection and accurate diagnosis of Alzheimer’s disease and related dementias.

The discussion featured perspectives from: Karen Blackmon, Imarisha Center for Healthy Brain Aging, Brain and Mind Institute, Aga Khan University; Jeff Burns, University of Kansas Alzheimer’s Disease Research Center; Amy Deckert, Davos Alzheimer’s Collaborative; Ishtar Govia, Amagi Health, Ltd.; Atsushi Iwata, Tokyo Metropolitan Institute for Geriatrics and Gerontology; Tim MacLeod, Davos Alzheimer’s Collaborative; Lefkos Middleton, School of Public Health, Imperial College London; Michelle Mielke, Wake Forest University School of Medicine; Suzanne Schindler, Washington University; and George Vradenburg, Davos Alzheimer’s Collaborative.

As next steps, DAC calls on governments, healthcare systems, global organizations, the private sector, and other stakeholders to implement several key actions:

1 Accelerate clinical adoption of blood biomarker tests and other innovative diagnostic tools. Blood tests for amyloid pathology are now available in multiple countries and hold great promise for early detection and accurate diagnosis. However, there is a need to translate research into pragmatic guidance for healthcare systems and clinicians to determine appropriate tests for their context of use, interpret results, and how to integrate tests into routine clinical practice in real world care settings. The recent CEOi Blood Biomarker Working Group publication in Nature Reviews Neurology represents an important step forward, laying out performance standards to guide health systems as they select and adopt blood tests in the United States.

1 Bridge healthcare system gaps to drive towards accessible, early, and accurate diagnosis. The first-of-its-kind DAC-SP AccDx program recently launched with 8 sites from 5 countries, to adopt innovative blood biomarker tests in the diagnostic pathway. Partners are already working together through a Community of Practice to share learnings and find solutions to common challenges. Findings will be shared at future DAC events, including the Learning Laboratory and build on the DAC-SP Early Detection Blueprint.

1 Prioritize education and support for primary care providers. Clinician education related to new diagnostic screening and tests is essential, especially at the primary care level. Today, the majority of people with cognitive impairment are still not identified, resulting in individuals not having access to critical care and support services in a timely manner. For new tests, primary care providers need help understanding the difference between Alzheimer’s disease and dementia, when to provide the tests, how to interpret and disclose results, what steps to take after diagnosis, and how best to engage with specialists.

2 Advance, fund, and scale global implementation and innovation. Governments, global organizations, and the Alzheimer’s disease community cannot afford to wait on the challenges faced by LMICs, home to the majority of people with dementia. Our collective efforts in high resource settings will help inform infrastructure needs in LMICs. At the same time, innovations from LMICs have the potential to deliver important benefits to the globe, with opportunities of lower costs and greater scalability. DAC-funded programs in Jamaica to identify and address reversible causes of cognitive impairment and research on fluid biomarkers and cognitive assessments in Kenya are part of this journey. Programs such as these should be a policy and funding priority for governments, foundations, multilateral organizations, and others, given the size of the challenge, the immense economic impact, and promising advancements on the horizon.

After decades of effort, we are finally seeing vital new diagnostics and treatments poised to move from the lab to the clinic. Our challenge now is to accelerate this process, far faster than the typical 17-20 years for routine use of clinical innovations, while ensuring new tools are used appropriately for the millions of families affected by Alzheimer’s disease in every country and community around the world.

DAC Healthcare System Preparedness Program will continue collaborating with our partners to advance these goals, share findings through our Learning Laboratory, and equip healthcare systems to implement adaptable, proven solutions.