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Tuesday, October 1, 2019

Ocular Therapeutix’s Dextenza gets permanent J-code from CMS

Ocular Therapeutix (NASDAQ:OCULjumps 10% in after-hours trading after getting a permanent J-code for Dextenza 0.4 mg for intracanalicular use from the Centers for Medicare and Medicaid Services.
The J-code becomes effective today and will replace the previously issued C-code, which became effective July 1, 2019. The company will retain transitional pass-through status granted for Dextenza from CMS.
“J-codes are more widely recognized by commercial insurance and Medicare Advantage and Part B plans, and physician familiarity with J-codes should allow for a simpler and more convenient reimbursement process that we believe will further contribute to DEXTENZA’s commercial potential,” said Ocular President and CEO Anthony Mattessich.
https://seekingalpha.com/news/3503121-ocular-therapeutixs-dextenza-gets-permanent-j-code-cms

CymaBay presenting two abstracts at Liver Meeting

CymaBay Therapeutics (NASDAQ:CBAY) will present two abstracts related to its seladelpar program for treating primary biliary cholangitis at The Liver Meeting in November.
Seladelpar is a “potent and selective peroxisome proliferator-activated receptor delta agonist” that’s in a global phase 3 registration study for treating PBC, as well as phase 2 studies for treating nonalcoholic steatohepatitis and primary sclerosing cholangitis.
In a dataset covering PBC patients with cirrhosis, seladelpar exposures were comparable to non-cirrhotic PBC patients over 12 weeks.
The Liver Meeting takes place in Boston from Nov. 8-12.
https://seekingalpha.com/news/3503128-cymabay-presenting-two-abstracts-liver-meeting

Aimmune +11.6% on positive allergist survey

Piper Jaffray says a new proprietary allergist survey shows “massive upside” to sales potential and expectations for Aimmune’s (NASDAQ:AIMT) peanut allergy drug.
Raymond James says the survey showed awareness and willingness to prescribe Palforzia.
Key quote: “While many have equated the opinions of a few high profile critics with the overall market view, bears would do well to pay attention to this feedback.”
AIMT shares are up 11.6% after hours to $22.85.
https://seekingalpha.com/news/3503130-aimmune-plus-11_6-percent-positive-allergist-survey

Using a Fitbit and music to counteract insomnia

Lots of people like to listen to music at bedtime. With the advent of the portable music player and in-ear headphones, this phenomenon has become widespread. Of course, music can help improve our state of mind and perhaps even help those who suffer from insomnia to get to sleep. The downside is that once you have fallen asleep the music will keep playing and this might have detrimental effects on how deeply you sleep afterwards and perhaps even cause issues in terms of damage to hearing.
Research published in the International Journal of Medical Engineering and Informatics offers a novel solution to defeating insomnia with but without the risk to one’s . Shriram Vasudevan, Ikram Shah, Sriharsha Patallapalli, S. Karthikeyan, S. Subhash Chandran, and U. Adithya Bharadwaj of Amrita University, Coimbatore, India are the team behind the new approach. Their wearable, Fitbit, based system allows one to nod off while listening to music but once one has actually fallen asleep the music is muted. This team says means there should be no disturbance of normal sleep patterns caused by the music continuing to play and no risk to hearing.
Fundamentally, their monitors the data from the Fitbit and calculates when the person has most likely fallen to sleep so that the music can be muted without disturbing them. Of course, many sleepers set a timer on their music to switch it off within a few minutes or an hour or so, but that only has benefits if one has actually fallen asleep. The monitored approach means the music only stops once the user is fast asleep.

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Watching music move through the brain

Study identifies brain protein that could put the brakes on Alzheimer’s

University of California, Irvine biologists blazing new approaches to studying Alzheimer’s disease have made a major finding on combating inflammation linked to the disease. The School of Biological Sciences researchers’ discovery about the role of a protein called TOM-1 heralds a shift toward examining the molecular underpinnings of Alzheimer’s processes.
Their study appears in the Proceedings of the National Academy of Sciences.
“Scientists have known for a long time that inflammation is a driver of Alzheimer’s , but inflammation is complex and involves many factors,” said School of Biological Sciences Dean Frank LaFerla, Ph.D., whose laboratory conducted the research. “That’s why we decided to look at TOM-1.
The protein helps to regulate a key component of the inflammatory response. “We were interested in TOM-1 because its levels are low in the Alzheimer’s brain and in the brains of Alzheimer’s rodent models,” said Alessandra Martini, Ph.D., the paper’s first author and a postdoctoral researcher who worked with LaFerla. “However, its specific role in the disease has largely been unexplored.”
The scientists discovered that reducing the amount of TOM-1 in Alzheimer’s rodent models increased pathology, which included increased , and exacerbated cognitive problems associated with the disease. Restoring TOM-1 levels reversed those effects.
“You can think of TOM-1 as being like the brakes of a car, and the brakes aren’t working for people with Alzheimer’s,” LaFerla said. “This research shows that fixing the brakes at the could provide an entirely new therapeutic avenue. With millions of people affected by Alzheimers and the numbers growing, we must research a diverse portfolio of approaches so we can one day vanquish this terrible disease.”

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Potential target for diabetes-associated Alzheimer’s disease

More information: Alessandra Cadete Martini et al. Amyloid-beta impairs TOM1-mediated IL-1R1 signaling, Proceedings of the National Academy of Sciences (2019). DOI: 10.1073/pnas.1914088116

New protocol allows prescribing anticancer meds outside approved use

A large team of researchers affiliated with institutions across the Netherlands has begun what they call a Drug Rediscovery protocol—a clinical trial of sorts that involves giving cancer patients anticancer drugs that are not typically used for their type of cancer. In their paper published in the journal Nature, the group describes their protocol and its purpose.
Doctors who treat patients know that sometimes drugs developed for treating one type of cancer can effectively treat other types of cancer—but a system for identifying which drugs those might be has not been developed. In this new effort, the doctors and researchers on the project are seeking to remedy that problem. They have set up a protocol whereby cancer patients who are out of options are given a chance to try other drugs not approved for their type of cancer. They report that initial results are promising—up to one-third of patients given alternative remedies have seen some improvement from them—and two are in remission.
Such a protocol is possible because of the way modern cancer drugs are developed—most are precision anti-cancer therapies directed against a specific DNA mutation in a tumor. Thus, to choose an alternative drug, a doctor working under the Drug Rediscovery protocol would look up a drug that has been approved for treating a similar type of mutation. As an example, many drugs have been developed and approved for treating that have been designed to target mutations that result in overproduction of HER2. If a patient in the program has a cancer type that also results in overproduction of HER2, it might be wise to allow them to try it as well.
The researchers in the program note that very little information is available for doctors trying to treat dying with drugs outside of approved lists. They believe that a protocol such as theirs will generate results that can be used by other doctors. If a patient experiences good results with an alternative drug, it goes into the database. If none of the patients given a certain see any improvement, then the team can cross it off a list of possible therapies for a certain kind of cancer.
The researchers report that the protocol now has over 1000 patients, and they hope to expand it by connecting with similar programs taking place in other countries. They note that as more data goes into the database, the better the database will become at providing reasonable alternatives for cancer doctors.

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Zhang group identifies gene that may make TNBC cells vulnerable to existing

More information: D. L. van der Velden et al. The Drug Rediscovery protocol facilitates the expanded use of existing anticancer drugs, Nature (2019). DOI: 10.1038/s41586-019-1600-x

Childhood TB shot may offer long-term protection from lung cancer

A tuberculosis vaccine commonly used in other parts of the world might reduce a person’s risk of developing lung cancer if given early in childhood, a six-decade-long study reports.
The Bacille Calmette-Guerin (BCG) is the only vaccine approved for preventing tuberculosis (TB)—a potentially fatal infectious disease that typically attacks the lungs. Because TB risk is low in the United States, the vaccine isn’t often given to American children, according to the U.S. Centers for Disease Control and Prevention.
But the new study suggests the vaccine may have some positive side effects.
“BCG-vaccinated participants had a significant 2.5-fold lower rate of cancer,” said study senior author Dr. Naomi Aronson, director of infectious diseases at Uniformed Services University in Bethesda, Md.
She said lower lung cancer rates persisted in those who received the vaccine no matter where they lived, and whether they smoked, drank alcohol or had tuberculosis.
Aronson said BCG affects the immune system somehow and may provide even more benefit in the lungs.
The initial study was conducted in 3,000 American Indian and Alaska Native children in the 1930s. If the findings are confirmed in different groups, Aronson said the use of BCG vaccine in childhood “might be considered for risk reduction for lung cancer over a lifetime.”
Dr. Len Lichtenfeld, interim chief medical officer of the American Cancer Society, reviewed the study and called the findings fascinating. “And you rarely see this duration of follow-up,” he added. “The authors went to great lengths to validate their information.”
But, he said, it’s unlikely that BCG will be used for lung cancer prevention. While the study found a statistically significant reduction in the rate of lung cancer, the actual number of cases was very low. Just 42 people in the study were diagnosed with lung cancer.
There’s also a serious, ongoing shortage of BCG vaccine that would limit any such efforts, Lichtenfeld said. The vaccine is an for a certain type of bladder cancer, and doctors find it hard to get enough for that purpose.
In addition, the BCG vaccine has been tested as a treatment in a number of other cancers with mixed results. In some cases, it looked as if lesions had shrunk, but the vaccine didn’t prolong survival, he explained.
Plus, Lichtenfeld said, there’s a very effective way to prevent many cases of lung cancer—don’t smoke. And, if you do, quit. “Tobacco causes most, but not all lung cancers. Not smoking helps prevent many cancers,” he said.
The initial study was conducted between 1935 and 1938. About 3,000 children from nine American Indian and Alaska Native tribes at multiple U.S. sites were randomly given the BCG vaccine or a placebo.
None of the youngsters had had tuberculosis. They were vaccinated between 5 and 11 years of age, with a median age of 8 years. Half were younger when they got the shot, half were older.
From 1992 to 1998, researchers reviewed health information from the trial participants.
There was no statistically significant differences in overall cancer rates between the vaccine and placebo groups. But the odds of lung were significantly lower, the study found.
Researchers noted that is a leading cause of death for Alaska Natives and Native Americans.
The study was published Sept. 25 in the journal JAMA Open.
https://medicalxpress.com/news/2019-10-childhood-tb-shot-long-term-lung.html