Connect with us

Health

Biogen trial of ALS drug fails main goal, but company says data are encouraging

Published

 on

A late-stage trial of Biogen Inc’s experimental treatment for an inherited form of Amyotrophic Lateral Sclerosis (ALS) failed to reach its main goal, but secondary measures and biomarkers showed favorable trends, the company said on Sunday.

Biogen will engage with regulators and other stakeholders “to understand the meaningfulness of this data and potential paths forward,” Toby Ferguson, head of the neuromuscular development unit at Biogen, told Reuters. The company is treating trial patients in a follow-on study and recently launched a Phase 3 trial of the drug, tofersen, in patients who are not yet experiencing ALS symptoms.

Tofersen, administered directly into the spinal canal each month, is designed to suppress the production of SOD1, a protein that can accumulate to toxic levels in ALS patients with mutations in a specific gene. Around 2% of ALS cases are believed to be caused by the genetic mutation.

After 28 weeks of treatment, the 108-patient trial showed a 1.2-point difference on a scale evaluating functional status for patients with fast-progressing ALS who were given tofersen compared to placebo patients, which was not statistically significant. In the group of patients with slower-progressing disease, the difference was 1.4 points.

An improvement of at least 2 points would be clinically meaningful, Guggenheim Partners said in a recent research report.

Biogen also detailed results from secondary trial endpoints, including breathing ability and muscular strength, indicating that patients treated with tofersen fared better than placebo patients, and that placebo patients switched to the drug in the extension phase of the study experienced similar gains.

“Despite the fact that there was no statistically significant difference in the primary endpoint, there is a clinical signal here,” said Dr. Timothy Miller, the study’s lead investigator and ALS Center Director at Washington University School of Medicine, St. Louis. He presented the tofersen data at the annual meeting of the American Neurological Association.

The trial also showed that patients given tofersen had lower levels of SOD1 protein compared to placebo patients, as well as lower levels of plasma neurofilament light chain, a potential marker of nerve cell degeneration.

“That suggests that there is an effect on clinical function of the person,” Dr. Miller said.

Most side effects in trial patients were mild to moderate, including headache and back pain, but two patients experienced spinal cord inflammation, and 5.6% of tofersen patients dropped out of the study.

Globally, around 168,000 people have ALS, a fatal neurological disorder also known as Lou Gehrig’s disease. Around 10% of cases are linked to genetic mutations, including SOD1.

Biogen, which licensed tofersen from Ionis Pharmaceuticals Inc, now plans to open early access to the drug to all patients with SOD1-associated ALS. In countries allowing such programs, patients can access a medicine free of charge before it is licensed commercially.

Shares of Biogen have fallen by about a third after hitting $414 in early June on the U.S. Food and Drug Administration’s controversial approval of Alzheimer‘s drug Aduhelm. The agency’s decision to approve the drug based on evidence that it removes protein plaques associated with the brain-wasting disease, rather than proof that it improves cognition, led to a backlash that has curtailed use of the medication while Medicare, the U.S. health plan for seniors, works to develop payment terms.

 

(Reporting By Deena Beasley; Editing by Andrea Ricci)

Health

'I was shocked': Mother, child mistakenly given COVID-19 vaccine instead of flu shot – Vancouver Is Awesome

Published

 on


WINNIPEG — A Manitoba mother says a routine appointment for her and her three-year-old to get flu shots ended in frustration and mixed messages after they were each mistakenly given an adult dose of a COVID-19 vaccine. 

Jenna Bardarson is calling for policy changes at the province’s vaccination centres to make sure that doesn’t happen to another family. 

The shots were administered on Nov. 24 at the Keystone Centre in Brandon.

Bardarson says that shortly after she and her daughter, Dali, got their shots, the health worker who had given them excused herself to speak with a supervisor. When the worker returned, she told them she had made a mistake and given them both the adult Pfizer-BioNTech vaccine. 

“I was shocked. I didn’t know what to say. My immediate concerns were, of course, would my daughter be OK and also who could I speak to about this,” Bardarson said in online social media messages Friday to The Canadian Press.

Once she got home, Bardarson made multiple calls to different departments with the regional medical authority, hoping to speak with someone about the error and her concerns, she said.

She said no one was able to provide her with the answers or information she needed. “The conversations with various Prairie Mountain Health members have been frustrating, to say the least.”

Bardarson said she already had two doses of a COVID-19 vaccine and was due for her booster shot next month. Her daughter is too young to be eligible.

Health Canada last month approved a pediatric version of the Pfizer shot for children ages five to 11, but it has not yet approved a vaccine for those under five. 

Bardarson said she and her daughter had headaches and sore arms the following day. Her daughter had no appetite and was throwing up.

Manitoba Health confirmed the mistake in a statement and said staff from Prairie Mountain have reached out to the mother to discuss what happened as well as to provide an update on an investigation.

“Patient safety is a critical aspect of all health-care services in Manitoba. We are constantly reviewing our processes to ensure that our systems support our staff in preventing errors,” it said.

“In this case … our team reviewed the existing processes to make adjustments that would help avoid a similar error from occurring in the future.”

Bardarson said the health region has not provided her with updated information on the investigation and would not discuss any consequences the health worker may have faced. 

Manitoba Health said no further action would be taken against the worker, because she immediately recognized the error and told a supervisor. 

For Bardarson, that’s not enough.

“I by no means want her fired; however, there should be some sort of measures in place for harm reduction.”

Bardarson suggested taking away the worker’s injection privileges or enhanced supervision during vaccinations. 

She said she would also like to see areas at vaccination centres separated by vaccine types, instead of having different vaccines offered in the same booth.

Manitoba Health could not say if others have been given a COVID-19 vaccine by mistake, but acknowledged that medication errors, although rare, do occur. It added that Bardarson was provided with information about the risks of the COVID-19 vaccine, which in this case it says are low.

Health Canada said it is not in charge of immunization monitoring and could not comment on whether similar mistakes have occurred in other parts of the country.

This report by The Canadian Press was first published Dec. 3, 2021.

___

The story was produced with the financial assistance of the Facebook and Canadian Press News Fellowship. 

Brittany Hobson, The Canadian Press


Adblock test (Why?)



Source link

Continue Reading

Health

Vaccine makers could make Omicron-specific booster, says Fauci

Published

 on

COVID-19 vaccine makers have contingency plans to deal with the Omicron variant that include a combination vaccine against the original version and the variant as well as a variant-specific booster dose, a top U.S. health official said on Friday.

The U.S. government is working with Moderna, Pfizer, and J&J on multiple contingency plans, infectious disease expert Anthony Fauci told reporters at a White House briefing.

“One is to rev up the production of the vaccines that they already have. The next is to make, for example, a bivalent, where you have the vaccine against both the ancestral strain and the new variant, and the other is to make a variant-specific boost,” said Fauci.

“They are now assuming they may have to do that and are being prepared for that,” he added.

Data from a National Institutes of Health study strongly suggest that existing boosters provide cross protection against a number of variants, including Omicron, Fauci said.

“Although we haven’t proven it yet, there’s every reason to believe that if you get vaccinated and boosted that you would have at least some degree of cross protection, very likely against severe disease, even against the Omicron variant.”

The U.S. Centers for Disease Control and Prevention is working with local authorities to investigate suspect cases of the Omicron variant in states other than those where cases have already been reported, Director Rochelle Walensky said at the briefing.

There have been cases of Omicron detected in about 40 countries, she said, but the Delta variant remains the dominant strain in the United States.

“I know that the news is focused on Omicron. But we should remember that 99.9% of cases in the country right now are from the Delta variant. Delta continues to drive cases across the country, especially in those who are unvaccinated,” she said.

(Reporting by Ahmed Aboulenein and Jeff Mason; Additional reporting by Doina Chiacu; Editing by Rosalba O’Brien and Dan Grebler)

Continue Reading

Health

FDA adds strict safety warnings on arthritis drugs from Pfizer, AbbVie and Lilly

Published

 on

The U.S. health regulator has added its strictest warning to the labels of drugs from Pfizer, Eli Lilly and AbbVie belonging to a class of anti-inflammatory treatments called JAK inhibitors, citing risk of serious health issues and death in patients 50 and over, the drugmakers said on Friday.

The addition of the warning on the labels follows the agency’s review of Pfizer’s Xeljanz after initial results from a February trial showed an increased risk of serious heart-related problems and cancer in some patients being treated with the drug.

Xeljanz, which brought in worldwide sales of $2.44 billion for Pfizer in 2020, is approved in the United States for the treatment of conditions including rheumatoid arthritis, psoriatic arthritis and ulcerative colitis – an inflammatory bowel disease.

AbbVie’s rheumatoid arthritis drug Rinvoq and Pfizer’s Xeljanz are now recommended for use only in patients, who have had inadequate response or intolerance to one or more TNF blockers, which are another class of drugs used against inflammatory conditions.

The Food and Drug Administration’s boxed warnings on the labels of Rinvoq , Xeljanz and Lilly’s Olumiant flags the risk of cardiovascular death and stroke in high-risk patients who are aged 50 and above, and are current or past smokers.

Additional information about the risk of some types of cancer and death was also added to their labels.

The marketing applications for Rinvoq’s expanded use in atopic dermatitis, psoriatic arthritis, ankylosing spondylitis and ulcerative colitis remain under review by the FDA, AbbVie said.

 

(Reporting by Manojna Maddipatla in Bengaluru; Editing by Anil D’Silva and Krishna Chandra Eluri)

Continue Reading

Trending