Sm04554

[nameless]

I'm sure neither side explicitly stated that was the reason for the second trial - but Samumed undoubtedly knows it's likely to be passed (industry/biotech is one of the major drivers for this act). So, in the event that it does get passed, why not run a relatively small and quick trial to acquire the necessary biomarker data that would lead to earlier approval. They're not really losing any time and the additional cost could be well worth it.

Given that the drug appears safe - and presumably effective - the FDA won't say no to this. More data in terms of patient outcomes and the drug's mechanism of action is never a bad thing. I'm sure the FDA would happily approve the trial on that basis (while at the same time, knowing in the back of their minds that the major reason Samumed probably wants the trial - although not explicitly stating it - is to potentially avoid a Phase 3 trial and pursue earlier approval in the event that the act is approved).

You know, I was thinking the exact same thing you posted but I thought that other posters would say that it sounds far-fetched. I swear it. I was thinking that Samumed may have pushed for the study because they believe that the 21st Century Cures Act might pass and they want to get biomarkers so they can eliminate the need for a phase 3 study, but they probably told the FDA that they wanted to gather more biomarker data since wnt mediation is kind of new, and deep down inside the FDA knows that Samumed is really thinking ahead to the possibility that the 21st Century Cures Act could be passed.

I swear I was thinking this when I typed my earlier post the FDA would not allow a study based on a pending ACT that isn't law yet. Since I was thinking the same thing you stated that means I agree with your idea. I think that is what happened between the FDA and Samumed.

[hellouser]

On second thought I doubt if the FDA would allow a human study (putting
humans at risk) that would satisfy the requirements of a pending legal bill
that is not yet law.

Then you'll NEVER know if it works. The whole point of finding if drugs work is to test on humans.

[nameless]

We need to contact our US senators and tell them to approve the 21st Century Cures Act in its' entirety, especially the part of the Act that allows drug companies to use biomarkers in lieu of phase 3 studies. We all need to jam their phone lines and send them emails.

[TravisB]

This might be a dumb question but why should we be excited about this?

Are there any signs that it might work? Any preliminary results?

Also, how it's supposed to work? Will it be able to regrow full head of hair? Or maintain only?

I wasn't following Sm04554 topic

[JayM]

This might be a dumb question but why should we be excited about this?

Are there any signs that it might work? Any preliminary results?

Also, how it's supposed to work? Will it be able to regrow full head of hair? Or maintain only?

I wasn't following Sm04554 topic

Then please read the topic. I know its a lot of pages but they answer your questions. Well apart from what it will be able to do. Who even knows.

[nameless]

Then you'll NEVER know if it works. The whole point of finding if drugs work is to test on humans.

Hellouser I don't think you understood my statement.

In any case, they already know if it works. They learnt whether or not it works from the initial phase 2 study. It's pretty obvious that it works because they're investing more money into it - moving it to an extra phase 2 means they're spending more money. It didn't die after the initial phase 2 and that should tell you something.

[nameless]

This might be a dumb question but why should we be excited about this?

Are there any signs that it might work? Any preliminary results?

Also, how it's supposed to work? Will it be able to regrow full head of hair? Or maintain only?

I wasn't following Sm04554 topic

Read the posts like Jay said.

[macbeth81]

We need to contact our US senators and tell them to approve the 21st Century Cures Act in its' entirety, especially the part of the Act that allows drug companies to use biomarkers in lieu of phase 3 studies. We all need to jam their phone lines and send them emails.

This article makes it sound like the Senate is making their own bill based off of the 21st Century Cures Act and will not vote on the House passed bill. It states they won't be ready until next year, but the older article states by Thanksgiving. Not sure which is accurate but sounds typical of our Senate.

http://morningconsult.com/2015/07/the-senates-cures-bill-is-coming-next-year/

http://www.nationaljournal.com/health-care/storm-clouds-ahead-for-21st-century-cures-bill-20150710

[It's2014ComeOnAlready]

This article makes it sound like the Senate is making their own bill based off of the 21st Century Cures Act and will not vote on the House passed bill. It states they won't be ready until next year, but the older article states by Thanksgiving. Not sure which is accurate but sounds typical of our Senate.

http://morningconsult.com/2015/07/the-senates-cures-bill-is-coming-next-year/

http://www.nationaljournal.com/healt...-bill-20150710

It should essentially be the same, and I know that the creator of the bill (Fred Upton) is working on it with the senate. Also, it's very likely the bill should be passed or at least voted on before the end of the year, because of 2016 being an election year.

Anyone seeking re-election will want their name on that bill. It is extremely popular among constituents.

[nameless]

This article makes it sound like the Senate is making their own bill based off of the 21st Century Cures Act and will not vote on the House passed bill. It states they won't be ready until next year, but the older article states by Thanksgiving. Not sure which is accurate but sounds typical of our Senate.

http://morningconsult.com/2015/07/the-senates-cures-bill-is-coming-next-year/

http://www.nationaljournal.com/healt...-bill-20150710

They're dragging their feet. We need them to vote on this thing quick.

[It's2014ComeOnAlready]

The more I think about it, the more the use of biomarkers for phase 3 trials makes terrific sense. Most drugs never even reach phase 3. If they've shown definite safety and effectiveness in phases 1 & 2, and if there are biomarker(s) that can determine whether or not a drug is actually having an effect on its target, then that is much more common sense than what the current rules are.

Phase 3 trials are the most expensive to run due to their size, duration etc. If a company cannot continue to fund a drug that is safe, effective, and could otherwise prove that it is a targeted therapy, then all the FDA is doing in this case is stifling innovation.

For these reasons, I also believe a drug like setipiprant, which has already had a phase 3, will also be sped up. They would only need to determine whether or not the drug is reducing PGD2 levels in the scalp, along with scalp biopsies to determine the change in the number of hairs in an anagen state.

I don't believe in God, but God bless the politicians, medical professionals, and venture capitalists who are responsible for this bill lol

[JayM]

Is there a date it has to be passed into law/not passed by?

[lacazette]

Look at this:

Angela Christiano, Ph.D.

Hair-Loss Treatment with Hair-Loss Gene Promotes New Hair Growth, Regulates Male-Pattern Baldness

The discovery of the APCDD1 gene, which causes hair loss, has potential for future male-pattern baldness treatments, and for regulating male and female hair growth, density of hair, and hair graying.

the APCDD1 gene inhibits the Wnt signaling pathway

Laboratory researchers have targeted this Wnt pathway to turn on or off hair growth in mice, but until now, the pathway did not appear to be involved in human hair loss. Their findings provide evidence that hair-growth patterns in humans and in mice are similar, and they suggest for the first time that manipulating the Wnt pathway may have an effect on human hair-follicle growth.

Unlike commonly available hair loss treatments that involve blocking hormonal pathways, treatments involving modulation of the APCDD1 gene and the Wnt pathway would be non-hormonal, which may enable many more people suffering from hair loss to receive such therapies

Advantages:
•Surgical treatment options for hair loss require multiple costly procedures. This technology represents a way to modulate hair growth without the need of surgery as APCDD1 represents a potential drug target.
•Targeting APCDD1 will not only prevent further hair loss but has the potential to grow new hair, in contrast to current medical alternatives.
"

http://innovation.columbia.edu/technologies/2419_hair-loss-treatment-with-hair-loss-gene-promotes-new-hair-growth-regulates-male-pattern-baldness-and-graying-hair

So it's seems there's two ways: inhibit APCDD1
or upregulate the pathway that is inhibited by this gene ( samumed )

Samumed patented their Wnt pathway activators in 2011 http://www.google.com.na/patents/EP2605652A1?cl=un

[seanway]

So how to fight APCDD1 ? sm04554 ?

[lacazette]

It's been 4 years, so I think she didn't found for the moment a topically safely way to inhibit this gene with a molecule

Whereas samumed have the safely molecule to activate the wnt pathway

So yes I think SM04554 will make our Wnt signaling pathway come back to his normal activity. The gene inhibit the pathway but we will upregulate this pathway daily. So I really think that SM will stop (or slow for years) the hair loss, and even more activation will give regrowth. But abberant activation give skin cancer, so they have to be careful.
But our Wnt signaling pathway is inhibited right now, so we just need an upregulation that lead to his normal activity, so it's far from cancer aberrant activity i think