EddorodzzNov 30
xeniax ⏚

Dear Fedi friends.

As some of you may know, I have a wonderful daughter Lila who had a severe form of epilepsy at the age of 5 months. This epilepsy is called West Syndrome.

We had to go through a hell of a treatment to stop her seizures. The thing that helped her was called "Synacthene Retard". It's basically ACTH, a synthetic hormone similar to adrenocorticotropic hormone produced by our anterior pituitary gland.

My daughter was one of the last kids who had a chance to get the Synacthene Retard from the lab that produced it (Alpha Sigma). This was late March 2024. And in May 2024 Alpha Sigma said they discontinued this product without ANY public explanation.

I am trying to inquire about the disappearance of Synacthene Retard from French pharmacies. I am looking for contacts of a medical journalist or an expert in pharmaceutics to help me lead this investigation. It may save lives of dozens of other kids with that suffer from West Syndrome. Thank you!

Nov 30 at 11:55amWeb
Show threadKevin Karhan Nov 30

@Xeniax Does the manufacturer at least offer licensing out the production?

  • If not I hope the regulators force them into #FRAND schemes or void any patents on that medication production process.
Show threadxeniax ⏚Nov 30
@kkarhan thanks for the question. I haven't checked yet. I will try to find out.
Show threadKevin Karhan Nov 30

@Xeniax obviously I assume they make this in like "bioreactors" with GMO'd yeast or bacteria - like most complex biological molecules like hormones and enzymes - so it does necessitate quite a buildup and a certified plant and engineers to run it, but is quite cheap to maintain a production line when adjusted for the yield.

  • But it's neither impossible nor uncommon to see that being done...

I sadly don't know any pharmacologist or contact at any pharmaceutical company to poke at and get them to look at it.

  • Tho I hope you can get some generics manufacturers that do similar products (like hormones and enzymes) to get interested in.
Show threadTrisNov 30
@Xeniax cc: @wonderfall any idea?
Show threadChris JakobsenNov 30
@Xeniax ask your national medicines agency.
Or even EMA, the European Medicines Agency.
I don't know about the legislation on compassionate use in your country.
Show threadxeniax ⏚Nov 30
@Chris thanks, I will do that. It's a great idea actually to start with the medicines agency and not with the lab
Show threadlxskllrNov 30

@Xeniax

Beth Mole from arstechnica might have some info or interest.

https://arstechnica.com/author/beth/

Author: Beth Mole

Beth is Ars Technica’s Senior Health Reporter. Beth has a Ph.D. in microbiology from the University of North Carolina at Chapel Hill and attended the Science Communication program at the University of…

Ars Technica
Show threadSebastian Crane 🏳️‍⚧️Nov 30
@Xeniax Boosted - good luck in your search. Is Lila okay now or does she still need the medication? The Wikipedia article suggests that most children 'grow out' of the condition; I hope your daughter is similarly better now.
Show threadxeniax ⏚Nov 30
@seabass thanks Sebastian. Lila is stable since April 2, 2024. She had no seizures since the treatment with Synacthene. Wikipedia explains that kids can outgrow West syndrome which is true, but unfortunately if seizures persist, the development outcomes are catastrophic (e.g. child not reaching any milestones, not seating, not walking, no speech etc). This statement about "outgrowing" is a bit misleading for people who are not familiar with West Syndrome. Also, if spasms are not stopped early on, they transform into other kinds of seizures. Most often in "Lennox-Gastaut" syndrome.
Show threadsnowyfoxNov 30
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Show threadLuuNov 30
@Xeniax
https://en.iranhormone.ir/product/synacran-tetracosactide-acetate/
Sun Pharmaceutical Industries Ltd can do this medicine too.
(Synacran) Tetracosactide acetate - Iran Hormon Pharmaceutical Company

1 mg/mL injectable suspension Each ampule contains 1mg Tetracosactide (as acetate) Pharmacologic category: An analogue of corticotropin (ACTH) Indication and administration: Diagnosis of adrenocortical insufficiency Alternative to corticosteroids in conditions such as Crohn’s disease or rheumatoid arthritis For intramuscular injection only

Iran Hormon Pharmaceutical Company
Show threadEddorodzzNov 30
@Xeniax sharing
Show threadPatrick DufourNov 30

@Xeniax I am not a medical journalist, just a psychologist! I am living in France and checked the situation of "Synacthene Retard". The article below describes the situation.

Bottom-line: the product is hardly available in France and Europe. In order to maintain product availability for as long as possible, the prescription and dispensing of SYNACTHENE and SYNACTHENE RETARD must be strictly reserved for its indications in cases deemed a priority.

<https://www.sfendocrino.org/rupture-de-stock-de-synacthene/#:~:text=En%20raison%20de%20difficult%C3%A9s%20d'approvisionnement%2C,de%20SYNACTHENE%20RETARD%201%20mg>

Rupture de stock de SYNACTHENE - Société Française d'Endocrinologie

  L’ANSM a été informée par le laboratoire Sigma-Tau que la rupture de stock de SYNACTHENE 0,25 mg/ml solution injectable, annoncée début avril aux professionnels de santé, est effective en ville et imminente à l’hôpital, tandis que la distribution de SYNACTHENE RETARD 1mg/ml suspension injectable est limitée à l’hôpital pour les seules indications jugées prioritaires […]

Société Française d'Endocrinologie
Show threadxeniax ⏚Nov 30
@pdufour Merci beaucoup pour l'article Patrick. Je sais que Synacthene est devenu difficile voire impossible à trouver en Europe, pas qu'en France. Le problème avec cette formulation de ne le prescrire qu'en cas jugés prioritaires est que cela devient une raison pour les neuropédiatres d'arrêter de le prescrire alors qu'il est très efficace là où les autres traitements échouent. Les neuropédiares commencent à prescrire que de l'hydrocortisone qui est plus accessible mais peu efficace. C'est un serpent qui se mord la queue. C'est pour cela qu'il faut rendre le synacthene plus facilement accessible...
Show threadBenjohnNov 30

@Xeniax I'm glad you found a helpful treatment.

I'm not an expert, but I understand (might be wrong) that part of patent law is that patent holders are required to license a patent if they do not wish to exploit it directly. You can't "block" use of a patent because you hold it.

So, perhaps the patent holder could be persuaded to license it?

I hope you find a solution!

Show threadxeniax ⏚Nov 30
@benjohn thank you! Thats precious
Show threadBenjohnNov 30

@Xeniax Apparently this is the case in the UK (and perhaps other places) – https://www.legislation.gov.uk/ukpga/1977/37/section/48 … but it may not be in the US (and perhaps other places) https://en.wikipedia.org/wiki/Compulsory_license

I con't know how this would be resolved internationally!

Patents Act 1977

An Act to establish a new law of patents applicable to future patents and applications for patents; to amend the law of patents applicable to existing patents and applications for patents; to give effect to certain international conventions on patents; and for connected purposes.

Show threadPostcardsFromParadiseNov 30

@Xeniax

It seems that the formal name of the drug is Tetracosactide.

Show threadAcratopègeNov 30
@Xeniax @vivet_lili a peut-être une piste 🤔 🤷
Show threadVulcanTouristNov 30

@Xeniax

You know likely reason why it was discontinued: no profit in a drug needed by a small minority. It's the same reason my wife had no treatment opportunities for her very rare autoimmune disease that affects only hundreds of people globally.

We don't need to privatize government agencies. We need to publicize Big Pharma and other corporations in control of the means of (life-saving) production.

Show threadxeniax ⏚Nov 30
@VulcanTourist agree... I am so sorry you and your wife had this experience.
Show threadBakersRelayNov 30
@Xeniax Promoting to #Epilepsy #WestSyndrome
Show threadGlobalement inoffensiveNov 30

@Xeniax
No explanation on the ANSM website.

But synacthene (without "retard") is still available.

France is experiencing A LOT of availability problems from several years.

Show threadxeniax ⏚Nov 30
@Galamadryade the other form of synacthene is not as efficient because it doesn't have the same release mechanism... synacthene retard is stimulating the production of ACTH throughout the day, progressively, while the normal synacthene has a momentary action.
Show threadGoGhostlyDec 1
@Xeniax an experienced doctor with big pharmaceutical connections can probably guess why
Show threadxeniax ⏚Dec 1
@GoGhostly I will try to ask our neuropediatrician, but she is so hard to get! Hyper busy and also not willing to discuss research side of things...
Show threadGoGhostlyDec 1
@Xeniax I think that the unwillingness to discuss the research is very telling
Often, doctors will not tell other doctors about pharmaceutical companies trying to keep ppl sick because it is very demoralizing