Phase 2a CF corrector study

Galapagos doses first patient with novel CF corrector GLPG2222

Galapagos NV (Euronext & NASDAQ: GLPG) announces dosing of the first patient with cystic fibrosis (CF) Class III (F508del and a gating mutation like G551D) with novel CF corrector GLPG2222 as an add-on to Kalydeco®[1] in a Phase 2a study. Galapagos further announced the opening of an Investigational New Drug (IND) file with the US Food & Drug Administration for GLPG2222, triggering a $10 million milestone payment.
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OWN IT: Did Science Find an Answer to the Lack of Organ Donors?

As it turns out the future is now. From National Geographic:

In a remarkable—if likely controversial—feat, scientists announced today that they have created the first successful human-animal hybrids. The project proves that human cells can be introduced into a Continue reading OWN IT: Did Science Find an Answer to the Lack of Organ Donors?

Patients with Severe Chronic Rhinosinusitis Show Improvement with Verapamil Treatment

http://www.newswise.com/articles/view/668018/?sc=mwhn

A clinical trial studying the use of Verapamil (a drug currently in use for cardiovascular disease and cluster headache) in alleviating chronic rhinosinusitis (CRS) with nasal polyps revealed significant improvement in the symptoms of this subset of patients. It is the first study of its kind to explore Continue reading Patients with Severe Chronic Rhinosinusitis Show Improvement with Verapamil Treatment

Manuka honey sinus irrigation for the treatment of chronic rhinosinusitis

Manuka honey sinus irrigation for the treatment of chronic rhinosinusitis: a randomized controlled trial.

Int Forum Allergy Rhinol. 2016 Dec 9. doi: 10.1002/alr.21898.
Lee VS1, Humphreys IM1, Purcell PL1, Davis GE1.
BACKGROUND:
Manuka honey (MH) has been shown in vitro to be effective against biofilm-producing bacteria. This study assessed the effectiveness of MH for patients with active chronic rhinosinusitis (CRS) and prior sinus surgery. Continue reading Manuka honey sinus irrigation for the treatment of chronic rhinosinusitis

My Five Takeaways from #NACFC2016

I had a dream last night, and in that dream the Limp Bizkit classic song, Nookie was on repeat for some reason. I have no idea why; I haven’t heard that song in about 12 years. Needless to say I woke up pretty jacked up and motivated to get sh*t done before the Packers game this afternoon. Continue reading My Five Takeaways from #NACFC2016

Spyryx Biosciences to Present New Data at NACFC

http://www.prnewswire.com/news-releases/spyryx-biosciences-to-present-safety-stability–effectiveness-data-for-spx-101-at-the-north-american-cystic-fibrosis-conference-october-27-29-2016-orlando-fl-300350083.html

Spyryx Biosciences to Present Safety, Stability & Effectiveness Data for SPX-101 at the North American Cystic Fibrosis Conference October 27-29, 2016 Orlando, FL

Spyryx Biosciences, a clinical-stage biopharmaceutical company developing first-in-class innovative therapeutics to address severe lung diseases, will present Continue reading Spyryx Biosciences to Present New Data at NACFC

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