Announcing New Clinical Trial Tools From The CFF

Today, we are thrilled to announce that we have launched three new clinical trial tools to better inform our community about the significance of clinical research and help people with CF find the trials that are right for them. We hope that these Continue reading Announcing New Clinical Trial Tools From The CFF

ChemDiv Announces New Drug Discovery Collaboration Agreement with Cystic Fibrosis Foundation Therapeutics, Inc.

http://www.prnewswire.com/news-releases/chemdiv-announces-new-drug-discovery-collaboration-agreement-with-cystic-fibrosis-foundation-therapeutics-inc-300276512.html

Today, ChemDiv, a fully integrated target-to-market Contract Research Organization (CRO) headquartered in San Diego, announced that it has entered Continue reading ChemDiv Announces New Drug Discovery Collaboration Agreement with Cystic Fibrosis Foundation Therapeutics, Inc.

Scripps study could impact future treatment of organ transplant patients

http://www.cbs8.com/story/31476725/scripps-study-could-impact-future-treatment-of-organ-transplant-patients

Posted: Mar 15, 2016 3:59 PM EDT Updated: Mar 15, 2016 3:59 PM EDT

LA JOLLA (CNS) – A study released Tuesday by scientists at The Scripps Research Institute in La Jolla could lead to major changes in the way patients with transplanted organs are treated in the future to prevent rejection.

In the study published in the American Journal of Transplantation, the researchers analyzed 234 kidney transplant biopsies and discovered that around 80 percent of the genes expressed in cases of early, acute rejection were also present in instances of chronic, much later rejections.

Acute and chronic rejections were thought to be separate conditions, but now appear to be difference stages along the same arc, the scientists say. About half of kidney transplants are rejected within 10 years, forcing patients onto dialysis, according to TSRI.

“For our transplant population, this is a major new understanding of the molecular basis of immune rejection that challenges the field to reconsider its current paradigms and has multiple immediate and actionable therapy implications for patients,”said Dr. Daniel Salomon, director of the Laboratory for Functional Genomics at TSRI. “The insights here most likely apply to liver, heart and lung transplants, too.”

He said the research shows that almost all transplant organ failure is due to inadequate suppression of the immune system, so that post-transplant patients can potentially be treated with the same therapies in order to prevent rejection.

“The new view that emerges from this research is that almost all transplant organ failure is due to inadequate immunosuppression, and with that understanding comes a potential for a major change in the practice of post- transplant drug therapy,”said Salomon, who is also medical program director of the Scripps Center for Organ Transplantation.

The researchers said more frequent biopsies could catch the body’s rejection of transplanted kidneys earlier than they are now.

Their study found a kind of kidney damage and scarring called interstitial fibrosis and tubular atrophy that could be a clue of approaching kidney rejection. Previous studies found that the presence of IFTA and inflammation — as seen under a light microscope — correlated with an increased risk of rejection. But IFTA on its own has been seen as evidence of a past injury, not active rejection, and is rarely treated.

“There was injury and inflammation there, just like in acute rejection patients — we just weren’t able to see it with the light microscope,”said Brian Modena, the first author of the study. “If you catch that early, you might potentially prevent chronic rejection. That would be a hugely positive benefit for our patients.”

TSRI reported that genetic expression profiling also proved to be a good tool for detecting subclinical acute rejection, which is active in about 20 percent of transplant patients in their first year, but impossible to suspect or diagnose until progression to clinical rejection.

Numerous other researchers took part in the study, including representatives of the Mayo Clinic, Northwestern Comprehensive Transplant Center in Chicago, and the University of Michigan. The study was funded by the National Institutes of Health.

Continue reading Scripps study could impact future treatment of organ transplant patients

Ask Your Questions to CF Roundtable

Do you have questions for our CF Roundtable columnists?

The US Adult CF Assn (USACFA) publishes CF Roundtable. USACFA’s directors consist of all adults with CF. Some of our columnists, although not directors, Continue reading Ask Your Questions to CF Roundtable

UI strikes agreement with Pfizer Inc. to develop potential cystic fibrosis gene therapy

The University of Iowa Research Foundation recently finalized a license and sponsored research agreement with Pfizer Inc. to support the development of potential gene therapies for cystic fibrosis (CF) by the laboratories of the Continue reading UI strikes agreement with Pfizer Inc. to develop potential cystic fibrosis gene therapy

In Cystic Fibrosis-Related Inflammation, Link Found to Specific Immune Cells and a Protein

http://lungdiseasenews.com/2016/01/18/13492/

In a new study, researchers at the University of North Carolina discovered that alveolar macrophages — specific immune cells — and a protein called XBP-1 are responsible for the exacerbated Continue reading In Cystic Fibrosis-Related Inflammation, Link Found to Specific Immune Cells and a Protein

Nivalis’ N91115 Gets Orphan Drug Status for Cystic Fibrosis

Nivalis Therapeutics, Inc. announced that the FDA has granted orphan drug designation to its lead candidate, N91115, for the treatment of patients suffering from cystic fibrosis. The stock surged 15.5% on the news. Continue reading Nivalis’ N91115 Gets Orphan Drug Status for Cystic Fibrosis

Why Do Some Infections Persist? Blame Bacterial Socialism, Says New Study

http://www.newswise.com/articles/why-do-some-infections-persist-blame-bacterial-socialism-says-new-study

New research to be published January 13 in the journal Scientific Reports shows that some bacterial cultures adopt an all-for-one/one-for-all strategy that would make a socialist proud Continue reading Why Do Some Infections Persist? Blame Bacterial Socialism, Says New Study

CF Researchers Identify Signaling Networks Controlling Mutant CFTR Protein Folding

http://cysticfibrosisnewstoday.com/2015/12/29/signaling-networks-controlling-mutant-cftr-protein-folding-identified/

 

From Cystic Fibrosis News Today
By Magdalena Kegel

A new study published in the journal e-LIFE has taken a novel approach to looking at deficits in protein folding associated with mutations in the CF transmembrane conductance regulator Continue reading CF Researchers Identify Signaling Networks Controlling Mutant CFTR Protein Folding

Gene therapy: A promising candidate for cystic fibrosis treatment

An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Continue reading Gene therapy: A promising candidate for cystic fibrosis treatment