PCUT's "ALS Treatment Now" / mecasermine rinfabate project continues to move forward. With the help of our regulatory, legal, and operations advisors, we are nearing the final draft of our joint venture proposal. If successful, this will be the blueprint for restarting drug manufacture and supplying an ALS-specific Phase III clinical trial. And we will have a groundbreaking model for repurposing promising drugs for deadly rare-diseases, in a fast, commercially viable way.
Rare Disease Treatment Development Gets Attention:
Right now, the need to overcome the hurdles of rare-disease drug development is becoming more widely recognized:
REAL ALS AWARENESS = Awareness for the Need for Near Term Effective Treatment:
In the last two months, several PCUT ambassadors have worked hard to clarify our message within the ALS community. These dedicated advocates are refocusing the community's attention to the need for Near Term Effective Treatment, and they are promoting an alternative to the old model that continues to focus simply on funding basic research for distantly future cures. Yet, there remains confusion in the ALS community about these two factual questions:
1) Might there be an existing drug right now that is effective at stopping or reversing ALS progression?
2) Are today's patients left without hope for treatment if we limit drug development to new treatments coming from newly funded discoveries?
The correct answer to both questions is "Yes". Indeed neither the scientific nor the medical community fully understands the mechanism of ALS, and repurposed drugs that have proven neuro-regenerative qualities and safe, stabile bio-delivery must be considered for trial when turning every stone for potentially effective treatment. Many patients --who would rather try a safe, potentially effective drug than to die with no hope of effective treatment-- have voiced their desire to try such experimental therapies.
Yet if you randomly polled a number of ALS families, I would bet that most will cite "Fundraising for a Cure" as the best thing they can do to fight ALS, despite the fact that newly funded basic research would take a minimum of 15 years to become an actual treatment, get commercially sponsored, and move through early stage clinical trial to a point where it is broadly available to ALS patients. 90% of ALS sufferers die within four years of diagnosis. Those who will only support new research are either doing bad math or they have given up on the possibility of effective treatment within current patients' lifetimes.
But there is hope for current treatment. PCUT supporters have been active on Patients Like Me and ALS-TDI forums dispelling the negative myths about mecasermine rinfabate (Iplex) that some doctors and cynical community members have spread. You know who you are, and you are a doing fantastic work for ALS patients!
ALSA Conference, Washington DC, May 9th - 11th:
Needless to say, PCUT's primary purpose was not to march to the Capitol with ALSA's request list in hand. Instead we (Gwynne Hickman and I) conducted a series of informational workshops throughout the conference days, and we attended several interesting sessions hosted by ALSA and one hosted by our friends at ALS-TDI.
It was wonderful to meet so many others who are fighting for near term ALS treatment, though the audibility and staying-power of our message was often stifled by the overwhelming tone of "more of the same" from the conference organizers and delegates. Some noticed that this year's attendees were comprised of far more social workers and far fewer patients and families than in the previous years.
During the opening session, PCUT ambassador, Tom Ohlson, brought the house down with his challenge to ALSA national leadership to start thinking creatively about patient's treatment needs. Here is an excerpt:
"...Research is why most of us are here. I know we all cling to hope, but it seems like every day is like the movie, "Groundhog Day." Every month/week/day we here about the latest, greatest breakthrough and what does it amount to? The same nothing we have had for 140 years. Frankly, many of us are tired of this emotional rollercoaster. Here is a quote from the ALSA website -- "The whole field of biomedical science is on the move as never before in the long history of medicine. I don't know what will happen over the next 20 years, but my guess is that we are on the verge of discoveries that will match the best achievement in infectious disease a generation ago." While that certainly sounds promising, the problem is that it was written 22 years ago! I think it safe to say that almost every hopeful PALS alive when that was written is now long dead. As such, ALSA needs to find a sense of urgency and do more unconventional thinking when it comes to research..."
Tom and fellow PCUT ambassador John Roberts had a meeting with ALSA national president Jane Gilbert. And, in addition to the need for urgency for current patients, Tom and John both cited the need for more effective awareness campaigns. John suggested showing the anguish of real patients, not just smiling faces filled with the oblique hope for treatments that, if funded today, could not realistically become accessible treatments within current patient's lifetimes.
In his continued comments to the ALSA President, Tom said:
"....The turf battles and squabbling between organizations representing A.L.S. are not only disheartening, but counterproductive. ....Madame President, I call upon you, not your subordinates, to contact the heads of these other agencies and convene a roundtable discussion to go over common ground, points of converging interest, and the possibility of sharing rather than competing for resources. All of these agencies owe it to us, their constituents, to not work at cross-purposes, but rather in unison for the common good. In this endeavor, I call upon ALSA to take the lead."
Well said! How wonderful it would be if ALSA, as the preeminent foundation for ALS sufferers, fully represented the various needs of the patient population. Unfortunately, while praised for their palliative care services and reasonable commitment to funding basic research, the ALSA is a no-show in the area of near term potentially effective treatment options for today's patients. It is understandable that an organization must focus on what it does well, but ALSA receives charitable support from individuals and organizations that think they are supporting current ALS sufferers....not just through discount wheelchairs and VA benefits, but also through potentially effective treatment options available in time to possibly extend current sufferers' lives. We would like ALSA to recognize that they do not address this need, and to therefore resolve to support PCUT which does address it. We have an ongoing dialog with ALSA leadership, and we remain hopeful for their support.
Other Important ALS Missions:
ALS-TDI (ALS Therapy Development Institute): I have had the opportunity to learn more about this great organization over the last few months. I have met personally with Mike, Rob, Carol, and Steve, and it is refreshing to have such allies in the call for more real ALS treatments -not just more basic research. TDI assesses the highest quality treatment-ready formulations and screens them through pre-clinical animal models to tee up the best candidates for clinical trial. They have what is becoming widely recognized as the best pre-clinical model for new ALS drugs, and TDI is probably the best place to look when looking for new entrants to the clinical development process for ALS.
The problem remains, however, that clinical development for rare-diseases is often not commercially viable for private drug sponsors. We have a list of 46 rare disease treatments that have stalled before clinical trial sponsorship or shortly after beginning clinical trial. The reasons involve the lack of physiological understanding of the targeted diseases-designing a trial with the right endpoints is a roll of the dice that the drug's effectiveness will be properly measured, and so the risk of a good drug getting denied FDA approval is often too great to justify a company's investment. Of course, patients lose in this scenario, and that's why PCUT's core service is a cost-partnership model to de-risk commercial drug development. The other important difference between PCUT and ALS-TDI is that PCUT's ALS therapy is a *repurposed drug* that does not involve pre-clinical studies and Phase I and Phase II trial. Mecasermine Rinfabate was already approved for a different indication in 2005, and it showed very clean human safety in five different clinical trials between 2002-2007. This means the drug could leapfrog the early stages of clinical development for ALS and go to broad Phase III trial very soon after the supply is restored. This is a critical distinction for patients who measure their lifetimes in months and who cannot wait five years for a promising drug to get commercially sponsored and go through Phase I and Phase II before becoming accessible.
ALS Guardian Angels and Compassionate Care ALS: The wonderful people leading these organizations are dedicated to the well-being of ALS sufferers and their families. Many families are overwhelmed by the financial costs and logistic burdens of this cruel disease, and they have had no where to turn for help, until ALSGA and CCALS. Check out Stu Millheiser's mission of compassion on the ALSGA website. He is a great man.
Each of these causes is distinct and important: ALSGA and CCALS for compassionate patient support, TDI for a continued pipeline of promising ALS treatments in the future, and PCUT for immediate accessibility to safe, existing drugs that could help stop ALS progression in today's patients.
What Each of Us Can Do for Patient Treatment:
We still need your help in the outreach effort for Near Term ALS Treatment Options. Share with your friends the need for real ALS awareness. Challenge those who simply want to throw more resources at basic research. Visit www.PCUT.org, and our pages on Facebook and Causes.com.
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Thanks for spending this time with me. Please check in with us and let us know how we are doing.
Sincerely,
Jess Rabourn
PCUT
PCUT
PCUT is a 501c3 nonprofit company whose mission is to use innovative business models to restore the commercial viability of safe, potentially effective treatments for rare diseases. Its beneficiaries are sufferers of rare, life threatening disorders who seek more treatment options in the near term. PCUT's pilot project is the manufacture and clinical trial of mecasermine rinfabate (IGF-1/IGFBP3) for use against ALS.
Other Important ALS Missions:
ALS-TDI (ALS Therapy Development Institute): I have had the opportunity to learn more about this great organization over the last few months. I have met personally with Mike, Rob, Carol, and Steve, and it is refreshing to have such allies in the call for more real ALS treatments -not just more basic research. TDI assesses the highest quality treatment-ready formulations and screens them through pre-clinical animal models to tee up the best candidates for clinical trial. They have what is becoming widely recognized as the best pre-clinical model for new ALS drugs, and TDI is probably the best place to look when looking for new entrants to the clinical development process for ALS.
The problem remains, however, that clinical development for rare-diseases is often not commercially viable for private drug sponsors. We have a list of 46 rare disease treatments that have stalled before clinical trial sponsorship or shortly after beginning clinical trial. The reasons involve the lack of physiological understanding of the targeted diseases-designing a trial with the right endpoints is a roll of the dice that the drug's effectiveness will be properly measured, and so the risk of a good drug getting denied FDA approval is often too great to justify a company's investment. Of course, patients lose in this scenario, and that's why PCUT's core service is a cost-partnership model to de-risk commercial drug development. The other important difference between PCUT and ALS-TDI is that PCUT's ALS therapy is a *repurposed drug* that does not involve pre-clinical studies and Phase I and Phase II trial. Mecasermine Rinfabate was already approved for a different indication in 2005, and it showed very clean human safety in five different clinical trials between 2002-2007. This means the drug could leapfrog the early stages of clinical development for ALS and go to broad Phase III trial very soon after the supply is restored. This is a critical distinction for patients who measure their lifetimes in months and who cannot wait five years for a promising drug to get commercially sponsored and go through Phase I and Phase II before becoming accessible.
ALS Guardian Angels and Compassionate Care ALS: The wonderful people leading these organizations are dedicated to the well-being of ALS sufferers and their families. Many families are overwhelmed by the financial costs and logistic burdens of this cruel disease, and they have had no where to turn for help, until ALSGA and CCALS. Check out Stu Millheiser's mission of compassion on the ALSGA website. He is a great man.
Each of these causes is distinct and important: ALSGA and CCALS for compassionate patient support, TDI for a continued pipeline of promising ALS treatments in the future, and PCUT for immediate accessibility to safe, existing drugs that could help stop ALS progression in today's patients.
What Each of Us Can Do for Patient Treatment:
We still need your help in the outreach effort for Near Term ALS Treatment Options. Share with your friends the need for real ALS awareness. Challenge those who simply want to throw more resources at basic research. Visit www.PCUT.org, and our pages on Facebook and Causes.com.
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Thank you for being part of this partnership. As Margaret Mead reminded us, "Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has."
Jess Rabourn
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PCUT is a 501c3 nonprofit company whose mission is to use innovative business models to restore the commercial viability of safe, potentially effective treatments for rare diseases.Its beneficiaries are sufferers of rare, life threatening disorders who seek more treatment options in the near term.PCUT's pilot project is the manufacture and clinical trial of mecasermine rinfabate (IGF-1/IGFBP3) for use against ALS.
