May 4, 2023 Sai Dhupkar, PharmD, BCMTMS, CSP

Care Team Clinical Pharmacists Help Optimize Treatment for Patients With ALS

As we recognize amyotrophic lateral sclerosis (ALS) Awareness Month in May, the disease remains without a cure. A rare and fatal neurodegenerative disease characterized by a progressive degeneration of motor nerve cells in the brain and spinal cord,1 ALS leads to paralysis and inability to speak or swallow, while leaving cognition and senses intact.1 Identified in 1869,2 ALS became more widely known as a result of baseball star Lou Gehrig’s diagnosis in 1939.2

Today, more than 5,000 new patients are diagnosed each year.3 Life expectancy varies from 20-48 months, with rare long-term survival of up to ten years.4 ALS affects people of any racial or ethnic background, and only about 10% of incidences are thought to be genetically predisposed.5

Treatment is aimed at slowing progression of the disease.6 There are currently four FDA-approved medications for ALS: riluzole, sodium phenylbutyrate/taurursodiol, edaravone,6 and newly approved tofersen.7 Tofersen comes in an injection form and is indicated in patients with the SOD1 mutation.7 In addition to these disease-modifying agents, therapy incorporates supportive care medications, including various muscle relaxers for spasticity and pain, anticholinergics for swallowing difficulties, and dextromethorphan-quinidine for the treatment of pseudobulbar affect.8

As understanding of the disease process has evolved, the number of investigational ALS treatment options has expanded, and several drugs are in clinical trials. While each has a unique mechanism of action, they all aim to slow progression of the disease.

For ALS cases that are genetic, mutations in C9orf72, TARDBP, SOD1 and FUS genes are being investigated for potential treatment options.9 Some treatments currently in clinical trials include:

  • ION363, which targets a genetic form of ALS caused by mutations in the FUS gene;
  • masitinib, a tyrosine kinase inhibitor, and
  • AIT-101, a PIKfyve kinase inhibitor.10

Other unique drugs that offer hope are pridopidine, a sigma 1 receptor agonist that has shown therapeutic potential in several neurodegenerative diseases,10 and CNM-Au8, which reduces the oxidative stress associated with the pathologies of many neurological diseases.11-12 Stem cell research is another area with promise for future therapies.13

While experimental therapies are being explored, optimizing current treatment options is crucial. The care team of an ALS patient—typically a neurologist, mental health professional, physical/occupational therapist, dietician, respiratory therapist, and the primary caregiver—is of paramount importance.14 To maintain and improve quality of life, each member of the team addresses specific symptoms and provides appropriate interventions, such as breathing devices, feeding tubes, and gentle exercises to strengthen functioning muscles and maintain cardiovascular health.14

Another important part of the team is the clinical pharmacist. Shields Health Solutions (Shields) provides a unique care model that allows its highly trained pharmacists and pharmacy liaisons to have all-encompassing visibility into and understanding of a patient’s care. The model gives patients and caregivers abundant access to medication experts who can address important questions regarding medication administration, adherence, drug-drug interactions, and side effect management, while efficiently triaging concerns to the clinic.

Currently, 71 patients with ALS receive pharmacy services with Shields. In addition to adherence, side effect management and lab monitoring, Shields pharmacists capture outcomes data that reflects the quality of life of patients and their caregivers; pharmacists are in a unique position to perform depression screenings and provide additional resources.

While the management of ALS is complex, patients and caregivers alike can rest assured that they are not alone. The clinical pharmacist and specialty pharmacy can provide an additional layer of support for optimizing medication regimens and quality of life.

For more insights into advancing pharmacy care for patients with rare diseases, check out our recent white paper, Advancements in Rare Disease Treatment and Management.



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