Clinical TrialsLiving With CFNews & EventsRespiratory Disease

New drug (Thymosin alpha 1) discovery: potential for Cystic Fibrosis treatment

According to an article published on www.sciencedaily.com, researchers have discovered a potential new drug to treat and stop the progression of cystic fibrosis. While many other promising drugs are in the pipeline, we here at cysticfibrosis.com will be keeping a keen eye on further developments of this medication.

Read the original article here on www.sciencedaily.com/releases/2017/04/170410124023

According to the article, Thymosin alpha 1 (Tα1) is a novel therapeutic single molecule-based therapy that could correct genetic and tissue defects and also significantly reduce inflammation seen in cystic fibrosis patients.

Quoted:

“Cystic fibrosis is the result of mutations in the gene encoding the protein called the cystic fibrosis transmembrane conductance regulator (CFTR), which is important to maintain chloride-channel activity affecting the salt and water balance in the lungs. This mutation results in a misfolded CFTR protein, and its premature degradation leads to impaired chloride permeability and persistent lung inflammation.”

“Goldstein and co-authors Luigina Romani, Ph.D., M.D., at the University of Perugia and Enrico Garaci, M.D., at the University of Rome “San Raffaele,” both in Italy, published their results in Nature Medicine. They report that Tα1, a synthetic version of a naturally occurring peptide first isolated from the thymus, corrects the multiple tissue defects found in the lungs and small intestines in a mouse model of cystic fibrosis, as well as the defects in the CFTR seen in cells isolated from cystic fibrosis patients. Tα1 not only significantly reduces the inflammation seen in cystic fibrosis, but also increases CFTR maturation, stability, and activity. Due to this two-pronged action, Tα1 offers a strong potential to be a single-molecule therapeutic agent to treat and stop the progression of cystic fibrosis.”

Tα1 is not currently available in the U.S. but it has been approved for clinical use for over 15 years in 35 countries in the treatment of other medical conditions (not CF) such as viral infections, immunodeficiency diseases, malignancies, and HIV/AIDS. According to the original article, it appears to have a good safety profile.

A variation of this research was quoted on https://cysticfibrosisnewstoday.com/2017/04/12/study-shows-molecule-reverses-cystic-fibrosis-tissue-defects-in-mice/

******* Edited – opinion of blog editor at cysticfibrosis.com *******

We are always excited when we hear news on potential treatment discoveries for Cystic Fibrosis, which is why we share them with the community. However it should be clearly noted that this is very recent news (less than a week old); the research was performed by an isolated team; and no information whatsoever about clinical trials are mentioned. Even if the medication meets the claims made in the article, the reality is that it would first need to undergo rigorous testing and would not be available as a treatment option for a very long time. However we do remain hopeful about its potential, and will keep you posted if we learn anything more. We will also attempt to contact the researchers.

8 Comments

  1. Kari
    April 17, 2017 at 9:50 pm — Reply

    Will this drug treat all CF mutations?

  2. Eileen
    April 18, 2017 at 12:37 pm — Reply

    Will it come to us my grandson is 10 and has cf would love to see him well

  3. Kathi Grant
    April 18, 2017 at 1:27 pm — Reply

    Has this drug been successful against the Delta F 508 mutation?

  4. Marilyn Rogers
    April 18, 2017 at 5:50 pm — Reply

    Do we know when (– medication brand name removed –) will be available here in the U.S.? Praying soon. My grandchildren Ivy 6, and Jude 2 , along with all who suffer, NEED it! I love them with all my heart. Want them to live long, healthier, and happy lives! Thank you for giving us hope!

  5. Paul Merritt
    April 18, 2017 at 9:03 pm — Reply

    Is it possible to start trials for FDA approval here in the US? And could it be fast tracked since it is already used as treatment in other countries?

    • Hessam
      April 19, 2017 at 2:27 am — Reply

      (– medication brand name removed –) has not been tested nor approved by any institute as being a help for CF, not even close to clinical trial. Only Doctor Allen Goldstein and a Italian doctor had some tests on it without any further discoveries. There is no assurance of the drug working on people with CF. I am a 35 year old with CF as soon as I found out I called the company that makes the drug and asked about it and they said the only answer of the accuracy of this article can be answered by the 2 doctors that have done the research. If anyone has any more information on it please share.

    • April 19, 2017 at 3:58 pm — Reply

      Hi Paul, just to clarify, it is not being used in other countries to treat CF, it is a medication that is at the moment used for treatment of different diseases altogether.

  6. April 19, 2017 at 3:54 pm — Reply

    Hi everyone, thank you for all the comments and questions, and thank you to Hessam for your valuable and proactive input. We unfortunately do not have any further information other than the very preliminary news that we shared, so we cannot answer any of the raised questions. We have updated this article to hopefully more accurately reflect that all claims are quoted from the article, and that this discovery should merely be viewed as a potential. While the medication itself is used to treat other conditions, it is NOT currently used for CF treatment. Thank you and let us know if you should learn anything more.

Leave a reply

Your email address will not be published. Required fields are marked *

Previous post

Indrepta C Lottery Winners

Next post

Full-Fat Fermented Dairy May Cause Less Inflammation Than Regular Dairy