Interesting email thread - FA/ Ataxia info. share (with video)

To ■■■■■■■■■■■■■■■■■■■■■■■■ Today at 12:16

Well said Jen, my thoughts exactly. When I think of the thousands of £££’s raised by my friends and family to help find a cure, and this is what we get!!!

I saw a printed T with the letters FUFA on the front. I think we should print some with FU HORIZON instead.

Chris Polhill.

From: ■■■■■■■■■■■■■■■■■■■■■■■■ [mailto:■■■■■■■■■■■■■■■■■■■■■■■■]
Sent: 03 December 2016 03:33
To: internaf ■■■■■■■■■■■■■■■■■■■■■■■■
Subject: Re: [internaf] Friedreich Ataxia Scientific News: Wednesday, November 30, 2016

So we are raising money to fund research to develop drugs we can’t afford?

From: internaf ■■■■■■■■■■■■■■■■■■■■■■■■ on behalf of internaf ■■■■■■■■■■■■■■■■■■■■■■■■
Reply-To: internaf ■■■■■■■■■■■■■■■■■■■■■■■■
Date: Friday, 2 December 2016 at 8:54 pm
To: internaf ■■■■■■■■■■■■■■■■■■■■■■■■
Subject: Re: [internaf] Friedreich Ataxia Scientific News: Wednesday, November 30, 2016

They would probably end up being very expensive, but the problem here is very different and less ethically reprehensible. They are new drugs, which are not on the market, they have never before been tested in humans. They have to overcome a long and expensive process from the discovery of their properties until their approval, the risk of failure and losing all the money invested is very high.

With the Nicotinamide it would probably happen the same as with the Thiamine, it is not patentable. The process of go from research to clinical use in FA is paid by patients association (basically Ataxia UK), probably this development will not be in the hands of pharmaceutical companies, as “there is no millionary business”.

But it is not gold all that shines, generally speaking, in most cases the discovery of the possible curative properties and its possible therapeutic use is made thanks to the basic research that is often paid by public money or by groups of patients, as per example FARA, Ataxia UK, and other patient organizations, this means that laboratories take advantage of a work that we have paid for ourselves (directly or through our taxes), but this has no impact on the sale price.

We need the pharmaceutical companies to get a cure: and they know it!

I advise you to watch this video, it exposes very well those problems.

Medical Research: Houston, we have a problem…

Juan Carlos

2016-12-02 9:56 GMT+01:00 ‘Paul Konanz’ ■■■■■■■■■■■■■■■■■■■ [internaf] ■■■■■■■■■■■■■■■■■■■■■■■■:

Yes to EPI-743 and RTA-408 for the lock. I’m not sure about Nicotinamide.

Pricing for any of them is probably going to be “high” because there’s only 6 -7 thousand FA’ers in the USA and 15 – 20 thousand in the world. The millions of dollars they invested to develop the drug now needs to come back in income.


From: ■■■■■■■■■■■■■■■■■■■■■■■■ [mailto:■■■■■■■■■■■■■■■■■■■■■■■■]
Sent: Friday, December 02, 2016 12:43 AM
To: internaf ■■■■■■■■■■■■■■■■■■■■■■■■
Subject: Re: [internaf] Friedreich Ataxia Scientific News: Wednesday, November 30, 2016

Will the same apply to epi743, nicotinamide or rta408?

From: internaf ■■■■■■■■■■■■■■■■■■■■■■■■ on behalf of internaf ■■■■■■■■■■■■■■■■■■■■■■■■
Reply-To: internaf ■■■■■■■■■■■■■■■■■■■■■■■■
Date: Thursday, 1 December 2016 at 1:10 pm
To: internaf ■■■■■■■■■■■■■■■■■■■■■■■■
Subject: RE: [internaf] Friedreich Ataxia Scientific News: Wednesday, November 30, 2016

In the USA not likely. Horizon will have a lock on this application for some time to come and that price tag is already established in their other applications.


On Thursday, 1 December 2016 11:39 AM, “Tom Sathre ■■■■■■■■■■■■■ [internaf]” ■■■■■■■■■■■■■■■■■■■■■■■■ wrote:


Won’t the price come down as more drug manufacturers chase those (high$) prices?


On 11/30/2016 2:48 PM, Ross Ashley ■■■■■■■■■■■■■■■■■ [internaf] wrote:

The price for Actimmune is $45000 USD per month, yes that’s the MONTHLY cost. Here in British Columbia, it’s more cost effective to hire 24 hr care. ;

That’s a drug that was developed in the early 90’s by another company. The technology has been sold several times, each time the price has increased. Horizon Pharmaceuticals is just the latest corporation to get their hands on the tech.

FARS scores decreased by an average of almost 5 after a 12 week study. As you know, FARS has a maximum score of 159, higher is worse. So, I’m not planning o n robing a bank every month or so to pay for this.

On Wed, Nov 30, 2016 at 12:00 PM, Tom Sathre ■■■■■■■■■■■■■ [internaf] ■■■■■■■■■■■■■■■■■■■■■■■■ wrote:

I think we ataxians and our clinicians finally have a “treatment” in the marketplace!

Could you, please, ask Dr. Collins about this?


On 11/30/2016 10:12 AM, Juan Carlos ■■■■■■■■■■■■■■■■■■■■ [internaf] wrote:

Launches of First Therapies Approved for Spinal Muscular Atrophy and Friedreich’s Ataxia Will Revolutionize Treatment and Drive Growth of These Rare Disease Markets

BURLINGTON, Mass., Oct. 25, 2016 /PRNewswire/ – Decision Resources Group finds that the anticipated launches of the first therapies for the treatment of spinal muscular atrophy (SMA) or Friedreich’s ataxia (FA) will transform treatment of these diseases and lead to their markets expa nding dramatically.
Following the anticipated label expansion of Horizon Pharma’s Actimmune (interferon gamma-1b), the FA market is forecasted to grow significantly over the next ten years. However, interviewed neurologists’ perceived limitations of Actimmune, including its unclear mechanism of action, variable effect on frataxin protein levels, and modest preservation of neurological function, could constrain its uptake and allow competitors to challenge Actimmune’s position.
According to interviewed experts, gene therapy will also transform the FA treatment landscape, possibly negating the need for drug treatment. However, gene therapies being developed by Agilis Biotherapeutics, Pfizer, and RaNA Therapeutics are in preclinical testing and unlikely to launch during the study period.
“Despite the lack of competing brands entering the market in the near term, Actimmune’s high U.S. cost may be an obstacle for its rapid adoption among patients with FA. If Actimmune can show that it delays disease progression or improves neurological function over a year or more, prescribers, patients, and payers are likely to accept its very high price.”