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PCUT Newsletter
April 2010 Newsletter

Dear Friends and Partners,

We are making progress! Of course, for those living in the burning house of rare quick-killer diseases, the work to achieve effective treatment can never be done fast enough. Be assured that I and the other partners will remain undeterred in our mission to restore availability of promising treatments for desperately ill patients who have yet to be offered any hope for overcoming their conditions.

Our Mission:

PCUT is a nonprofit, virtual production company for developmental drugs that have clear lifesaving potential. We restart the manufacture of medically sound therapies that, due to economic reasons, now sit on the shelf. The lack of access to developmental therapies is especially acute in rare terminal disorders, where the faster the disorder typically kills, the less audible the patient’s voice is. Although patients of many rare diseases are underserved by traditional therapy development, we must focus on one at a time, and we are excited to undertake a promising ALS (Lou Gehrig’s Disease) treatment as our pilot project. What makes this opportunity special is that the drug already exists and could be made available to patients in much less time than would therapies that are still in basic research and early clinical trial.

Mecasermine Rinfabate (rhIGF-1/IGFBP3) was FDA approved for growth hormone insensitivity disorders while trademarked as Iplex in 2006. In peer reviewed clinical trials on over 120 patients, the drug made good on its promise to deliver IGF-1’s restorative qualities to the body in a safer and more stable form than that of earlier approved free IGF-1 therapies. In fact, no serious adverse side effects have been attributed to the drug’s use in the reports of any of the trials. Its potential for ALS is that the IGF-1 has long shown neurotrophic (restorative and protective) effects in motor neurons, and the IGF-1/IGFBP3 complex is an effective delivery mechanism to mediate that action. Furthermore, the careful observations of clinical investigators in the ongoing U.S. Investigational New Drug (IND) program demonstrated efficacy on an aggregate level with varying measures of improvement in each individual patient. We also have some medical testimonies from an investigator of the Italian IND program, and that doctor reported favorable results in multiple ALS patients. For a disease about which so little is known and for which all approved therapies are ineffective at slowing progression, the safety and efficacy data we have for Mecasermine Rinfabate clearly merit further human trial.

But facing legal and financial challenges, the maker of Iplex stopped all production of the drug in 2009. No other supply exists, and remaining inventories for ongoing IND programs are expected to run out by the end of 2010. PCUT is now making progress in bringing back this safe drug for ALS patients who decide to pursue it in trial programs. More on our Website



Updates:

1) Advisory Committee:

Several key advisors have joined our cause in the last two months. PCUT is now working with a leading research clinic in San Francisco in both a clinical and medical advisory capacity. We also have both an experienced gene-expression laboratory director and a science officer in the field of human growth factor delivery. Several biotech consultants and regulatory affairs experts are donating their time to this project as well.

2) Some Basics:

501c3 Recognition was achieved in November thanks to our great lawyers and thanks to testimony from one of our financial partners.

We enjoy ongoing relationships with some central figures at FDA and Senate HELP committee who understand the need for expedited therapy development for patients of rare “immediately life-threatening diseases.”

Our website is complete ( www.PCUT.org ), and so is our secure donation gateway, provided by Network for Good.

We have formed developmental partnerships with National Organization for Rare Disorders, Abigail Alliance for Better Access to Developmental Drugs, Kakkis Every Life Foundation, Hannah’s Hope Fund, Orphan Biotec AG, and Partnership for Cures. Click here for links to these wonderful organizations.

3) Current Work:

Right now we are working with our advisory team on structuring proposals to four companies involved in the manufacture of mecasermine rinfabate. Our nonprofit plan covers all phases of licensing, manufacture, clinical trial and commercialization. And the needs of desperately ill patients are the highest priority. We will re-engage our FDA contacts when the manufacturing proposals are accepted.

The good people at ALS Worldwide have released the interim report of the ongoing Investigational New Drug study (IND) of ten U.S. patients. While human safety had already been clearly established in earlier non-ALS clinical trials, the ALS-specific IND investigation provides important demonstration of ALS patients’ slowdown of symptom progression. The report is written with exacting scientific discipline, and tracks the most relevant ALS biomarkers in use today. The cumulative body of evidence is compelling for the efforts at achieving broader human trial access.



Calendar

April 12th: PCUT has tentatively agreed to join the NORD Task Force on Orphan Drug Development, which convenes in April.

May 6th: I (Jess) have a speaking engagement with the Chartered Financial Analyst society of San Francisco, and I will discuss nonprofit application of investment careers. I intend to raise awareness for the PCUT model.

May 9th-11th: ALSA National Conference. Join us for our four informational workshops! The first one, “Myths and Reality about Mecasermine Rinfabate” will take place at 9:30pm Sunday May 9th, in the lobby of the JW Marriot Hotel, Washington DC. The other three are set at convenient times throughout the 3-day Conference: Two on Monday, and one on Tuesday. Our conferences are not on ALSA's official schedule, so please contact us directly for more information.

May 18th: NORD's Partners in Progress event. We have been invited, and we will attend if the partnership and fundraising opportunities appear great enough to merit the trip.

Fall/Spring: A feature length documentary film began last year and includes substantial coverage of PCUT’s work to restore Mecasermine Rinfabate for ALS patients. We are one of three stories highlighting how patient advocates can take direct action for therapeutic solutions. I agreed to participate because visibility and clarity of message are pivotal in our fight for today's patients.



We Need Your Help

This letter is a call to action as much as it is a progress update. We have a shoestring budget, which directly affects our ability to move quickly on our initiatives for patients.

Here are some ways to help us:

1) Donate:

Most of the funding for PCUT has been from my personal savings, and we simply cannot continue to operate this way. We are a 100% volunteer organization and we urgently need your financial contribution. Even a $25 donation is significant.

If you, a loved one, or a friend is suffering from ALS or another rare disorder, or if you want to help bring potentially life-extending therapies to these people, make yourself a partner today!


2) Electronic Networking:

Take a few minutes to add your name to our Facebook Fanpage, “PCUT.ORG”, and Facebook “Cause”. Share your interest in PCUT with your friends on other online communities like Patients Like Me, and ALS-TDI. Put our website link (www.PCUT.org ) on your personal page and on other ALS group pages. You may also want to forward this newsletter to others. Part of our mission is to share with other patients and patient’s loved ones the legitimate hope for near-term therapy.

3) Leadership:

Share your professional skills with us! There are many projects we need help with, especially in the areas of IP law, venture finance, clinical data analysis, and grant writing. If you are thinking of getting more active in this cause, please contact us at [e-mail], or check the Volunteer section of our website.



If this letter was valuable to you, please sign up at the bottom of this page to ensure you receive future newsletters.

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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