Author Archives: Magdalena Kegel

Spinal Muscular Atrophy Researchers Identify Spinraza Ethical Challenges

Although Spinraza has the potential to change the course of spinal muscular atrophy, a number of ethical challenges revolve around its use, a group of American researchers argues. In an article in the journal JAMA Pediatrics, they maintained that the healthcare system needs to address six issues to ensure that SMA patients “benefit from treatment, are protected from harm, and are treated fairly.” The title of the piece is “Ethical Challenges Confronted When Providing Nusinersen Treatment for Spinal Muscular Atrophy,” The group, led by Dr. Alyssa M. Burgart of Stanford University, said the biggest challenge is cost. Spinraza's pricetag is $750,000 the first year, and $375,000 each year thereafter. A cost that hefty can make insurers reluctant to cover the treatment, the group said. It also creates the possibility of hospitals bearing partial or full costs if an insurer refuses to re-imburse them. In fact, this is a major reason why some hospitals are not offering Spinraza (nusinersen) treatments, the team wrote. A second ethical challenge is the possibility that people with similar levels of disease severity will receive different treatments because of cost. This generates "concerns for the just distribution of healthcare," the scientists wrote. While unequal health insurance coverage is an inherent flaw of the U.S. healthcare system, Spinraza's enormous cost makes the problem particularly daunting. Healthcare insurers and hospitals are not the only ones bearing the cost burden, the team wrote. The lifelong cost of the therapy may force families to opt out of treatment or become impoverished. The second ethical challenge is dealing with limited information on Spinraza's long-term effectiveness. Since Spinraza trials involved small patient samples and were relatively short term, the verdict is out on whether most patients will benefit long term. It is also not clear if the improvements seen in the trials will translate into improvements in muscle strength and function when doctors treat patients.   While patients and their families may be prepared to accept these uncertainties, health insurers may not be. This may lead to situations in which insurers approve a treatment only if patients can prove with arbitrarily determined measurements that it is effective. Limited access to such testing may further disadvantage a patient, the team argued. In addition, there is no agreement on what a treatment benefit is. This has a bearing on a third ethical issue — informed consent. If a treatment is failing to provide benefits, a doctor may decide that it should be dropped. Since there is no consensus on what a treatment benefit is, patients, families and physicians may find themselves holding differing views on the issue. The best way to deal with this is to discuss it before a patient starts treatment, the team contended.

Cytokinetics’ New Muscle Activator May Have Better Tolerability, Effectiveness Than Tarasemtiv

Cytokinetics’ new muscle activator compound shows promising safety, tolerability, and effectiveness in three early clinical trials in healthy volunteers. The data supports the ongoing Phase 2 trial of the drug in patients with spinal muscular atrophy. Importantly, the findings indicate that the therapy is better tolerated and more potent than the company’s earlier muscle activator, tirasemtiv. CK-212 7107 is an investigational next-generation therapy that Cytokinetics is developing in collaboration with Astellas Pharma. The compound aims to act as a muscle activator by slowing calcium signaling in so-called fast skeletal muscle fibers. The drug was explored in three separate Phase 1 trials, adding to data from two earlier studies. The studies showed that CK-212 7107 triggered a muscle force more than double of that seen with tirasemtiv. All three trials also concluded that the drug was relatively well tolerated — all adverse events were mild or moderate. Laboratory values, neurological examinations, vital signs, brain waves, walk tests, and blood oxygen levels were all normal after the treatment. Researchers also concluded that higher doses gave rise to higher blood concentrations of the drug — a desirable feature of any new drug. CK-212 7107 is currently being assessed in a Phase 2 trial in patients with SMA types 2–4. The trial is still recruiting participants in the U.S. and Canada. Interested patients can find more information, including contact details, at the trial’s registration page. The drug is intended for the treatment of patients with muscle fatigue or weakness. In addition to SMA, the compound is being tested in Phase 2 trials in patients with ALS, elderly people with mobility limitations, and patients with COPD.

People in the UK Support Newborn SMA Screening, Study Shows

The majority of people that completed a U.K. survey support the idea of newborn screening for spinal muscular atrophy , according to a report published in the journal Molecular Genetics & Genomic Medicine. With Spinraza now available as an SMA treatment option, people in favor of screening believe that a diagnosis at birth may now offer better healthcare choices and improved life expectancy for those affected. The study is an important contribution to the debate of a potential implementation of SMA screening, as public support is key to its evaluation, said researchers at Warwick Medical School and the University of Warwick in the U.K. A number of reasons have prevented newborn SMA screening from being introduced in the U.K., researchers said. For instance, it is difficult to anticipate whether the disease course will be mild or severe, based only on screening results. Before Spinraza’s approval — in 2016 in the U.S. and 2017 in Europe —  there was also no treatment for the disease. But now there is, and research in the past year has demonstrated that Spinraza is most effective when given before infants start showing symptoms. This has renewed debate of the feasibility of newborn screening both in Europe and the U.S. Eighty-four percent of the study participants favored newborn screening — a number significantly higher than the 70 percent reported among SMA-affected families. Those who favored screening stated better healthcare and support as key reasons, but also mentioned the potential to advance SMA research and ease enrollment into clinical trials. A diagnosis at birth would also make it easier for parents to make informed decisions about future pregnancies, people argued. The majority, or 82 percent, found that a diagnosis at birth was important even with the current inability to predict disease type and severity. People who were against screening stated that a diagnosis at birth may interfere with the bonding process and cause unnecessary stress. Researchers, however, noted that families with SMA-affected children did not agree with the idea that a diagnosis would affect bonding. Researchers also noted that the support for newborn screening was similar to that of other screening methods, including preconception and prenatal screening. The study demonstrates that a large part of the general population favors the introduction of newborn screening for SMA.