Author Archives: Joana Fernandes, PhD

Kezar Life Sciences Raises $50M to Advance Clinical Testing of Potential MS Treatment

Kezar Life Sciences announced that it is planning to move ahead with clinical testing of KZR-616, a potential treatment for multiple sclerosis (MS) and other autoimmune diseases and inflammatory disorders. The company recently concluded a Phase 1 safety study of the treatment, and raised $50 million in investment funding to support its development. KZR-616 is a first-in-class selective immunoproteasome inhibitor, meaning it works by blocking abnormal protein degradation. Cells eliminate proteins by sending them to a specialized cell compartment known as the proteasome. In immune cells, the proteasome is called immunoproteasome, and it regulates several selective inhibitors and participates in the regulation of the immune response associated with inflammatory diseases such as MS, rheumatoid arthritis, Crohn's disease, and lupus. "We are pleased with the results of our healthy volunteer study, and grateful for the support of such an excellent group of investors to finance our upcoming clinical trials," John Fowler, Kezar Life Sciences’ CEO, said in a news release. "The strong demand for this financing reflects growing excitement for the potential of immunoproteasome inhibition in treating autoimmune disorders and recognizes the clear leadership position enjoyed by Kezar." The Phase 1 trial enrolled 82 healthy subjects, who were assigned to receive either KZR-616 or placebo. In total, 61 volunteers were given KZR-616 as single or multiple doses at varying dose levels to identify the optimal dose for both tolerability and proteasome inhibition. Results will be presented at the American College of Rheumatology's Annual Meeting to be held in San Diego in November. "These initial clinical trial results demonstrate that KZR-616 is achieving the desired levels of immunoproteasome inhibition that correlate with anti-inflammatory activity seen in laboratory models,” said Christopher Kirk, PhD, company president and CSO. “By selectively targeting the immunoproteasome, we believe we can avoid the toxicities associated with dual proteasome inhibitors like Velcade and Kyprolis." The Series B financing round was led by Cormorant Asset Management and Morningside Venture and raised $50 million to support the development of KZR-616. Kezar announced it has the support of new investors, including Cowen Healthcare Investments, Pappas Ventures, Qiming Venture Partners, and Bay City Capital. "Cormorant is pleased to support Kezar as it enters an exciting series of patient studies, the first ever with a selective immunoproteasome inhibitor," said Bihua Chen, founder of Cormorant Asset Management. "While much work remains, I believe KZR-616 has the potential to be a transformative treatment in autoimmunity."

Vitamin D Supplements Fail to Prevent Bone Loss in MS Patients, Study Reports

A new study reports that vitamin D supplements do not prevent bone loss in multiple sclerosis patients who are not vitamin-D-deficient. Previous research has suggested that low levels of vitamin D increase the risk of a person developing MS. In addition, Vitamin D prevents loss of bone density. That loss can lead to fractures and osteoporosis, a condition that many MS patients experience as their disease progresses. Researchers decided to investigate the effect of weekly doses of vitamin D3 on patients with relapsing-remitting multiple sclerosis, versus patients receiving a placebo. All 68 participants in the Phase 4 clinical trial also received 500 mg a day of calcium, a compound that is also important for bone health. The team measured the effectiveness of the supplemental vitamin D by analyzing biomarkers of bone health in blood. These included levels of the proteins PINP, or procollagen type I N propeptide, and CTX1, or C-terminal cross-linking telopeptide. At the start of the study, levels of PINP and CTX1 were not significantly different between the two groups. And that continued to be true at week 48 and week 96 of the study. The bottom line was that vitamin D supplementation did not change bone health in patients with MS after 96 weeks. “Our results do not support that high dose weekly vitamin D supplementation is beneficial for bone health in ambulatory persons with MS, and suggest that weekly vitamin D supplementation alone is not sufficient to prevent bone loss in persons with MS who are not vitamin D deficient,” the researchers concluded. The results were different from previous studies supporting the beneficial effects of vitamin D supplementation in MS patients. The researchers said they believed the discrepancy was due to differences in the studies' patient characteristics, sample size, and duration of follow-up.

MS Symptoms, Coupled with Poor Self-Esteem, Can Lead to Serious Depression, Study Says

Fatigue, limited mobility, and poor self-esteem or resiliency were found to be associated with periods of serious depression among multiple sclerosis patients, according to a recent study. Previous research has suggested that MS patients are at risk of major depression, with potentially profound impact on their quality of life. But only a few studies have addressed the incidence of depression among MS patients or the risk factors that may underlie its occurrence. Researchers in Canada enrolled 188 MS patients being treated an Alberta clinic, who were interviewed to assess potential risk factors for depression: namely, socioeconomic status, disease-related factors, childhood risk factors, psychosocial factors, and health behaviors. Participants were also asked to complete the Patient Health Questionnaire every two weeks for six months to identify depressive symptoms in real-time. Over a six-month follow-up, 36 cases of depression were reported among the group of MS patients analyzed. The incidence of depression was 0.019 for women, but higher — 0.044 — for men. Importantly, several factors seemed to be associated with depression in these patients — fatigue, limited mobility, and low resiliency, self-esteem, and self-efficacy, as well as poor coping skills. Results also showed that gender and income were associated with depression. Overall, the researchers concluded that "depression in MS exhibits a risk factor profile similar to that of depression in the general population, with the additional impact of MS illness-related factors. Potentially modifiable risk factors, such as coping with stress and resiliency, present opportunities for focus of further research in depression in MS treatment and prevention efforts." Concerning treatment, the team also emphasized that "while there is evidence in the clinical context that supports the efficacy for pharmacologic and non-pharmacologic treatments for depression in the general population, there is currently insufficient evidence to support/or refute the efficacy of depression treatment for individuals with MS ... Clearly this is an area that requires additional research."

GeneFo to Host July 26 Webinar on Sex and Intimacy Issues by MS Advocate Tuppy Owens

GeneFo, an online multiple sclerosis community that offers support, advice and educational resources to patients, will conduct a free webinar July 26 for those interested in knowing more about how MS affects sex and intimacy. The webinar, hosted by renowned MS expert Dr. Tuppy Owens, follows a GeneFo survey showing that sex and intimacy are two main concerns for patients living with the disease. Owens will talk about self-acceptance, how to overcome MS symptoms, such as fatigue and movement deficits, and how to create intimacy beyond sex. Registration for the webinar is free and can be done here. "We are very proud to be offering this important session for the MS community, to provide a useful and valuable resource and point of encouragement,” GeneFo CEO Neer Ziskind said in a press release provided to Multiple Sclerosis News Today. “Dr. Tuppy has been at the forefront of campaigning for and supporting people with chronic conditions/disabilities and has helped many patients find healthier and happier lives while rediscovering intimacy and sexuality. Her years of experience, openness and profound sensitivity will resonate with those seeking answers and will address their unmet needs." Studies have, in fact, demonstrated that MS may limit patients' sexual life. Many couples affected by MS encounter anxiety, depression and anger when trying to achieve a healthy sex life. Patients frequently feel vulnerable and embarrassed with certain medical procedures, reducing their desire for intimacy with their partners. According to GeneFo, male patients often suffer erectile dysfunction, decreased libido and trouble achieving orgasm. Complaints among female patients include decreased libido, difficulty achieving orgasm, vaginal dryness and painful sexual intercourse. Caregivers may also be affected by feeling guilty for wanting to have sex or for not feeling attracted to their partners the same way as before the disease. For more information about the webinar, please contact Adi Bein at [email protected]

Breastfeeding May Be Linked to Reduced Risk of MS, Study Suggests

Women who breastfeed for 15 months or longer may have a lower risk of developing multiple sclerosis (MS) than those who breastfeed for shorter periods or not at all, according to a recent study. The study also suggests that women who had their first period at age 15 or older are less likely to develop MS. Previous studies have shown that breastfeeding is associated with several health benefits for women. “This is another example of a benefit to the mother from breastfeeding,” said Annette Langer-Gould, MD, PhD, the study’s lead author, in a news release. “Other health benefits include a reduced risk of breast cancer, ovarian cancer, type 2 diabetes and heart attack.” Previous studies have reported that certain reproductive factors, such as pregnancy, gestational age, age at first period, and use of contraceptives, may influence the development of MS. According to researchers, MS mainly affects women during childbearing years (it rarely occurs before a woman’s first period or after menopause), but the risk of this disease significantly decreases during pregnancy and breastfeeding. “Many experts have suggested that the levels of sex hormones are responsible for these findings, but we hypothesized that the lack of ovulation may play a role, so we wanted to see if having a longer time of breastfeeding or fewer total years when a woman is ovulating could be associated with the risk of MS,” Langer-Gould said. The study enrolled 397 women newly diagnosed with MS (or its precursor, clinically isolated syndrome, CIS) and 433 age- and race-matched individuals. Researchers asked participants to complete a questionnaire and provide data on pregnancies, breastfeeding, hormonal contraceptive use, age at first period and menopause, and amenorrhea (absence of period). Results showed that among those who had successful deliveries, healthy women who breastfed their children for 15 months or more (85 women) were 53% less prone to developing MS or CIS compared to MS patients (44) who had breastfed for only four months or not at all. “This study provides more evidence that women who are able to breastfeed their infants should be supported in doing so,” Langer-Gould said. “Among the many other benefits to the mother and the baby, breastfeeding may reduce the mother’s future risk of developing MS.” Also, healthy women who had their first period at the age of 15 or older (44 women) had a 44% lower risk of developing MS later in life, compared to those who had their first period at a younger age (27 women with MS). Other reproductive factors, such as number of pregnancies, episodes of amenorrhea, and use of contraceptives were not associated with MS/CIS risk.

Health Questionnaires Can Help to Predict ‘Hard Outcomes’ Like Survival in MS Patients, Study Says

Patient questionnaires can be sensitive to signs of disease progression and worsening in neurological disorders like multiple sclerosis just as they are in other diseases, helping doctors to better predict clinical outcomes in patients, a study reports. Particularly, the study found that MS patients with higher scores on a specific disease questionnaire were nearly six times more likely to die within 10 years than those with lower scores, and that mortality risk also jumped among people whose scores rose on a second taking of same questionnaire. But the researchers cautioned that their study was not a tool for predicting mortality but a way to help patients be more active participants in their care. “Our research shows that by answering a set series of questions, patients can have an important role in predicting long-term prognosis in diseases like MS, and that these types of questionnaire should be used by doctors to get a better idea of the patient’s health,” Joel Raffel, study’s first author, from the Imperial College London, United Kingdom, said in a university news release written by Ryan O'Hare. “We hope that using patient-reported outcomes like these more and more will mean a shift towards empowering patients," he added. "They will be able to provide their own data, so rather than the doctor telling the patient how they are doing, it’s the other way around.” Among tools often used in the clinic are patient-reported outcomes; that is, questionnaires for patients that focus on their disease and treatment. But while these questionnaires have many uses, from screening for symptoms or evaluating treatment response to improving communications, they are often under-utilized when people have MS or other neurological diseases, "in part because it is not clear if PROs [patient-reported outcomes] relate to ‘hard clinical outcomes’ like disability or mortality," the team noted. Researchers wanted to determine whether the Multiple Sclerosis Impact Scale–29 (MSIS-29) — a 29-question survey assessing quality of life and disease impact over the previous two weeks — might serve as a way of predicting a patient's risk of death. The questionnaire was completed by 2,126 people, registered with the MS Society Tissue Bank in the U.K., beginning in 2004. Of these, 872 patients repeated it one year later. By 2014, the researchers reported that 264 of the original group of MS patients (12.4%) had died, and an evaluation revealed that MSIS-29 scores were associated with 10-year mortality risk regardless of age, gender, and disability score at the time the questionnaire was completed. Indeed, patients with high scores on the MSIS-29 questionnaire, indicative of a poor quality of life, were 5.7 times more likely to die within 10 years than those whose scores were lower. The mortality risk rose further among people whose MSIS-29 score worsened between the first and second year of answering the questionnaire. “Ideally, these questionnaires should be administered routinely, once a year in the clinic or online,” Raffel said. “This could help doctors to understand what issues the patients are facing and could also help to answer big research questions around prognosis and which of the available treatments we have for MS are working.” The team believes that questionnaire responses, together with usual clinical assessment tools like imaging data through MRI scans, could help doctors and patients choose the best course of treatment.

RRMS Patients at Risk of PML Can Safely Switch from Tysabri to Lemtrada

Lemtrada (alemtuzumab) may be an effective option for relapsing-remitting multiple sclerosis (RRMS) patients withdrawing from prior treatment with Tysabri (natalizumab), an Italian study shows. The study, “High-Risk PML Patients Switching from Natalizumab to Alemtuzumab: an Observational Study,” appeared in the journal Neurology and Therapy. Tysabri, an antibody with…

UCSF Neurologist Played Key Role in MS Research Turning to B-Cells, Essential Step to Ocrevus

Dr. Stephen Hauser, chair of the neurology department at the University of California San Francisco, was instrumental in the early research and later clinical trials that ultimately led to Ocrevus (ocrelizumab), the first therapy approved by the U.S. Food and Drug Administration (FDA) for both relapsing MS (RMS) and primary progressive multiple sclerosis…

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