Information Packet
70 W. Madison St.
Suite 1500
Chicago, IL60602
312-601-8856
Partnership for Cures 2006 Mission Statement
The mission of Partnership for Cures is to help funders and researchers to work together to transform the way cures are discovered. Partnership for Cures is a 501(c) (3) public charity.
Time and money are luxuries that patients battling illness do not always have, so Partnership for Cures teams up philanthropists and researchers to find new uses in new diseases for already FDA approved drugs and other therapies to quickly and inexpensively improve the quality and length of life of those who need help now.
Partnership for Cures inspires funders, researchers and research institutions to conceive and complete these direct and significant patient impact projects within a 24 month timeframe through our Two Years to Cures (2Y2C) Initiative.
This applied research uses already available drugs, knowledge and technology created as large non-profits, industry and government support long term medical research in the search of cures.
We support each of these Partnerships with rigorous business and financial management and transparency in financial and scientific reporting.
A copy of our Audited Financial Statements and our 2006 990 are available on our website at:
Board Members and their Affiliations
Directors
Chair, George N. Goldman, Asset Partners, Inc.
Margaret Christie, Golan and Christie, LLC
Judith A. Goldman, Goldman Philanthropic Partnerships
Neil Hirsch, Neil Hirsch and Assoc.
Kevin B. Krantz, JD, Stahl Cowan Crowley LLC
Mark L. Kosiek, The PrivateBank and Trust Company
Peter Kupferberg, Gofen and Glossberg
Michael Lucas, Leading Edge Investment Advisors
Robert Miller, Millco Investments
Jerry Paris, MW Management Group
Science Advisors
Chair, Alan Schwartz, MD, PhD, WashingtonUniversity
David Ginsburg, MD, Howard Hughes Medical Institute at University of Michigan
Lee Goldman M.D, ColumbiaUniversityMedicalCenter
Dr. Ross Heller, MD, MBA, University of Chicago Hospitals
Helen H. Hobbs, MD, University of TexasSouthwestern MedicalCenter
Jack Kessler, MD, Northwestern University
Greg Koretzky, MD, PhD, University of Pennsylvania
Beth McNally, MD, PhD, University of Chicago Hospitals
Gary J. Nabel M.D. Ph.D, National Institutes of Health
Fred E. Silverstein M.D., Frazier and Co.
R. Sanders Williams, MD, DukeUniversityMedicalCenter
Business Advisory Board
Rajeev Bahri, TEKchand. LLC
Steven L. Braun, CLU, Northwestern Mutual Financial Network
Christopher Lloyd, Memorial Hermann Healthcare
William Patrick Nichols, Transition Leadership International
Bruce Pivar, Waterstone Financial Services
Thomas Romano, New Century Bank
Terrell H. Sachman, AXA Advisors
Christine B. Simpson, Lanmark Group
Perry Snyderman, DLA PiperShell Vacations, LLC
Action Board
Chair, Leslie Anne Schreiber, Winger and Associates
Jennifer Adams Hurd. Windemere Nursing and RehabCenter
Josh Leventhal, Huron Consulting Group
Renee Matthews, MD, Leahy and Hoste
Julie M. Schaefer, Roche Diagnostics Corporation
Lauren Stone, National Strategy Forum
Kenlyn Vass, Cleveland Veterinary Clinic
Chairman’s Letter
I want to start out by thanking the members of our Executive, Advisory and Scientific Boards and our supporters for making our joint commitment to the Two Years To Cures mission a reality. It is with great pleasure that I recognize, in this first annual report, this important milestone – the founding of our new 501(c)(3) public charity - Partnership for Cures.
When we were “born” in 1998 as a Private Operating Foundation - Goldman Philanthropic Partnership - we could only dream about finding cures for catastrophic diseases and creating funding techniques as innovative as the cures. Over these past 8 years, we have:
- created strategic partnerships with many knowledgeable and influential institutions such as Harvard, Memorial-Sloan Kettering, Stanford, Mass General, WashingtonUniversity, Northwestern, and the Universities of Chicago, Pittsburgh, Michigan, Illinois, California, and Virginia.
- co-created the first Innovative Myeloma Cancer Research Conference with Mayo Clinic, which has developed into the premier yearly international conference on all blood cancers.
- been chosen as the new administrators of the prestigious and important Culpeper Health Care Grant programs by the Rockefeller Brothers Fund.
Most importantly we and our funding and research partners have:
- developed better treatments that are clinically helping over 100,000 worldwide patientsright now who have multiple myeloma, ALPS (autoimmune lymphoproliferative disease), traumatic brain injury and familial dysautonomia.
- found support for human clinical trials in lung cancer (Mass General) and stem cell transplants (Mayo Clinic)
- funded research projects that are one step away from clinical trial in Type I diabetes, and upcoming projects in autism, multiple sclerosis, colon cancer, Crohn’s disease and severe depression.
All of these better treatments and potential cures have testedalready existing drugs or other therapies for new uses-a fast and inexpensive way to get help to patients with disease right now as industry, government and large non-profits continue the long and expensive critical push for new drugs and other therapies to create cures.
In the process, we have created a powerful example of new ways that potential cures are found and funded – and have seen many of the techniques that we pioneered being used. Our original vision is being accomplished - by us on an individualized disease/philanthropist basis – and by others, who are able to address the huge, third-world needs.
I am pleased to announce that our efforts will have its first “offspring”– with the formation in 2007 of The Rockford, Illinois Chapter of Partnership for Cures. We wish them every success – and we will support them in every way possible.
George N. Goldman
Chairman of the Board
Partnership for Cures
Two Years To Cures Research Program
Partnership for Cures created the Two Years to CuresInitiative(2Y2C) to bring funders, researchers and institutions together to drive better treatments and cures to patients with disease right now.
The Two Years to CuresInitiative drives better treatments and cures to patients by testing already approved drugs and other therapies for new uses. Groups of donors fund these clinical trials, most of which cost under $250,000 and are completed in two years or less with a potential direct and significant outcome for patients with disease right now. Many of these Two Years to Cures projects are translation of a drug from one disease to another based on new knowledge about either the drug or the disease; some are testing combinations of existing drugs for synergistic effects, much like the lung cancer clinical project that spun off of the study PFC donors funded at Mass General; some are moving salvage therapies from end of treatment forward; some projects test non-drug or alternative medicine therapies; and some are based on anecdotal successes or observations from clinical practice. They can be preventive, diagnostic, predictive or therapeutic in nature.
An incredible amount of scientific knowledge, technology and research skill exists that could impact patients. The missing pieces are the inspiration and funding to get it started and the management to get it finished. Partnership for Cures (PFC) and its funder partners bring that motivation, financial support and business rigor to help researchers and institutions drive better treatments and cures to patients in two years or less.
Two years ago a group of Partnership for Cures funding partners provided $35,000 to a young researcher who translated an existing FDA approved cancer drug to create the first functional treatment for children with a rare fatal blood cell disease called ALPS. Today, these children finally have a safe and relatively inexpensive treatment option that creates a good quality life and the hope to live more healthfully for many years.
Government, academia, industry and large disease specific non-profits play a vital role training researchers, creating cutting edge knowledge about diseases, formulating new drugs and improving technology. However, their structures and missions do not typically harness the available resources to impact patients quickly.
The Two Years to CuresInitiative unites all of the critical partners and resources to produce research that can impact patients in the next two years:
- Up to the minute scientific discoveries about diseases and drugs
- FDA approved drugs and other therapies with new potential uses
- Researchers with brilliant ideas that can be tested quickly
- Passionate funders impacting the lives of the suffering
- Research institutions that provide additional research funds
- Patients and clinicians who inspire and participate in the research
- Lay and science volunteers who review and rank the research
- Partnership for Cures business rigor to compress timelines, leverage investments, report widely and provide complete transparency
In 2006 the FDA only approved 17 new drugs. Why?
First, it takes a long time and costs a lot of money to bring a drug to market, so drug companies are very careful to pick only those drugs that have the potential to help them offset those costs. They can’t afford to seek approval for a drug that they know will work if there are only a few thousand patients that can benefit from it-they would have to charge more than the market will accept, so they can’t make their investment back.
Second, between the strict regulatory processes designed to protect the public and the high cost of litigation when something goes wrong, drug companies have to be very wary of which drugs they bring to market. Last year more than 50 drugs were refused approval at the last step of the billion dollar process, adding to the overall cost of drugs and eliminating potential cures for patients.
Until we change the regulatory, pricing, litigation and reward structure for bringing new drugs to market we will continue to limit the availability of drugs that can help patients with disease right now!
There are over 4000 safe and effective FDA approved drugs being prescribed for patients, many with the potential to treat other diseases. Research to find this out takes far less time and cost than to create a new drug. So where is this kind of research?
First, there is often no profit potential in it. Most of these drugs are off patent. Adding a new small patient population to a low cost drug won’t make a profit for any drug company.
Second, this is a dead end for a researcher. They depend on a long stream of grants to support their careers. It is difficult for them to interrupt that stream for 1-2 years to work on drug translation. Clinician-scientists are the only ones who take on this kind of project.
Private philanthropy is the only viable funder for this kind of research. The good news is that researchers are willing to partner with philanthropists to undertake this research, and the legal and regulatory rules allow, even encourage, this research to take place.
Drug translation can have an almost immediate and significant impact on patients who have disease right now!
Media contact:
Dr. Bruce Bloom
312-601-8856
For Immediate Release
July 1, 2006
Partnership for Cures Funding Partners Create
Effective New Treatment for Orphan Disease
Chicago— A group of tactical philanthropists who support Partnership for Cures (PFC), a public charity dedicated to accelerating treatments for life-threatening diseases, has funded a project at Children’s Hospital of Philadelphia that created a breakthrough treatment for children and young adults with the rare devastating disease Autoimmune Lymphoproliferative Syndrome (ALPS). ALPS is considered an “orphan” disease, since it affects less than 200,000 Americans.
Children with this disease lead difficult and shortened lives. ALPS causes anemia and increased infections, so most of these children spent 5-10 days in the hospital each month receiving transfusions and IV antibiotics. Though they have normal intelligence, physical capabilities and communication skills, they are often years behind in school because of their chronic hospitalization. They take large amounts of steroids and other drugs that have significant side effects and don’t prolong life. Few live beyond their twenties.
Until now. This discovery, using a relatively safe drug already on the market, has created a low cost, low side effect treatment for these children. The first children on the regimen have been hospitalization free since early 2006 and are off all of their other medicines with the huge side effects. They are, for the first time, leading normal childhood lives. And their lifespan is projected to double.
Orphan disease patients are often children. These patients are usually the last in line to get new treatments because the bulk of research funding goes towards diseases that affect large percentages of the population. Physicians and researchers are learning new things about these orphan diseases everyday that create opportunities for better treatments and cures, but few financial resources are available to test these new opportunities. This funding gap lets strategic funders have a huge impact on children who are suffering right now.
Judy Goldman is a cancer patient who wants to give back. “I have multiple myeloma, also an orphan disease, and have been blessed by being in remission. It’s important to me to do something to advance research on rare diseases, especially diseases that affect kids,” she says. “When Partnership for Cures searched out this project for our foundation, my husband George and I jumped at the chance to quickly help kids who have a life-threatening disease.”
Dr. David Teachey cares for patients and does research. “My ALPSpatients have a disease that causes their white blood cells to build up rather than die off naturally. It’s very painful and often fatal. Many of my patients die in their teens. Based on some very new information, I came up with an idea that a drug called Rapamycin, which is a pretty safe drug used for cancer, might help my patients, but I needed proof. I looked around for a long time but could not find funding from government or other conventional sources. Partnership for Cures found a funding partner for me,” he says. “They helped me fund a research plan when no one else would or could. Less than two years after I started, I had the evidence that Rapamycin significantly reduced ALPSin mice and began to use it in my patients. I love doing research, but it’s all about the patients and not wanting to see them suffer. One patient has been on Rapamycin for months now, with excellent results. This is case where a small amount of money went a long way.”
Judy says Partnership for Cures helped her target her resources to orphan disease research that had a chance to work. “Others might not have funded research that could benefit just a handful of patients, she says, “but I say look at what Dr. Teachey has accomplished by focusing his research on finding a treatment for his patients. These children now have a chance at a healthier life. Their families get to watch them grow up instead of watching them suffer. How often do you get that kind of return on your investment in just two years?”
The mission of Partnership for Cures is to help funders and researchers
to work together to transform the way cures are discovered.
For information contact Dr. Bruce E. Bloom, 312-601-8856,
Sample Program Appeal: in cases where we have a cure in sight
but not a significant donor
Responding to an Urgent Need-Find a Medically Sound Autism Therapy
Issue
Autism patients and their families do not have access to safe, inexpensive, medically sound therapies substantiated by reliable and valid scientific research.
Autism, now called Autistic Spectrum Disorder (ASD), is a grouping of widely varying symptoms in patients with special medical, communication, behavioral, social and cognitive issues. ASD is a complex mixture of genetic, environmental, inflammatory, immunological, and other factors that science has not fully unraveled. Patients diagnosed with ASD often have autoimmune disorders, allergies and nerve inflammation that scientists have shown results from underlying immune system defects. ASD can be devastating to the family and the patient, leading to desperate searches for solutions. While communication and behavioral strategies slowly help many ASD patients over time, families urgently search for safe and sound medically based treatments that might be transformational. This search has led to the development and marketing of a number of controversial treatments that clinicians claim are medically sound, but have not been subjected to objective scientific testing.
A Breakthrough Potential Solution
Clinically test a safe and low cost drug that: (a) has already been FDA approved; (b) has a scientific basis for why it might treat the defective immune system issues of ASD patients and; (c) reportedly creates significant behavioral improvement when taken by ASD patients
Plain Language Summary: Scientists have discovered that some ASD patients may have a defect in their body’s immune system, which can cause significant brain cell inflammation. A low cost drug (ACTOS) that is currently being used to treat type II diabetes is known to help the body control defective immune cells. Some thoughtful ASD physicians wondered if ACTOS might therefore have beneficial effects in ASD patients. FDA rules allowed these physicians to use this drug for a non-approved use, as long as the drug appeared safe to use and they obtained the patient’s and parents’ consent beforehand. These physicians treated carefully selected ASD patients with ACTOS for long enough to conclude that they see significant behavioral changes in about 75% of the patients. This anecdotal information has been collected on about 300 ASD patients treated with ACTOS. The physicians claim most of these ASD patients showed good clinical and laboratory improvement with few, if any, side effects. However, this kind of anecdotal information is not considered scientifically valid by most physicians for them to begin to prescribe this drug for their ASD patients. It is therefore critically important to scientifically and rapidly test this promising treatment so that sound medical decisions can be made jointly by physicians, patients and family members.