Venture Philanthropy in the News
Hundreds of Americans will converge on Capitol Hill Wednesday (March 2) to lobby for legislation that would make it easier to identify, develop and approve drugs for life-threatening rare diseases and pediatric cancers. The legislative push is part of a weeklong series of events that begins today (Feb. 29) with Rare Disease Day, established in 2008 to raise public awareness of rare diseases and their impact on patients and families.
Patient engagement will continue to drive both medical research and policies this year, writes FasterCures Executive Director Margaret Anderson in the Huffington Post. Among the other issues to watch this year: the drug-pricing debate, the Precision Medicine Initiative, changes in how academic research is conducted and using real-world evidence in clinical trials.
The Biotechnology Industry Organization is working with the patient advocacy group Parent Project Muscular Dystrophy to gather real-world "community-centered" patient profiles to help guide clinical studies and drug development to combat Duchenne muscular dystrophy. Recommendations on study designs and ways to use the patient data in regulatory applications will also be offered by the alliance.
The Washington Post
Frustrated that no game-changing treatments were in sight, the Cystic Fibrosis Foundation's leaders in 1999 placed what many considered a risky bet, deciding to invest millions of dollars in a small California biotech firm. The pioneering success of Robert Beall and the Cystic Fibrosis Foundation in the practice of “venture philanthropy” is prompting a growing number of nonprofit groups to explore whether they, too, might be able to benefit their patients, and bottom lines, by investing in similar ways. Dozens of organizations, from the Michael J. Fox Foundation to the Multiple Myeloma Research Foundation to the National Multiple Sclerosis Society, have embraced the approach over the past decade.
Science Translational Medicine
For most of history, patients have been the passive recipients of medical care with little or no role in research. Recently, patient participation has expanded dramatically, and today, opportunities abound to serve as active partners in defining and prioritizing research questions and solutions. FasterCures leaders explore the early foundations of patient engagement, where it occurs in the drug-development pipeline, the power of recent policy initiatives, and prospects for success in improving health outcomes.
Within eight weeks of starting the Michael J. Fox Foundation for Parkinson's Research, Deborah Brooks said the foundation wanted to fund a biotech company and were told that this “isn’t done.” She said they quickly learned that every assumption in the nonprofit world had to be challenged, including requiring that researchers share their data — something that wasn’t routinely done. They also realized that while doctors and researchers meant well, the patient’s voice was often missing from the dialogue around the disease.
The Philadelphia Inquirer
The growing movement of venture philanthropy includes nonprofits making investments that may pay dividends down the road, somewhat like what big academic institutions have been doing for years. This form of venture philanthropy garnered attention last year when the Cystic Fibrosis Foundation sold its rights to royalties from Kalydeco - a drug that helps about 4 percent of patients with the inherited disorder - for a landmark $3.3 billion. National nonprofits involved in venture philanthropy include the Multiple Myeloma Research Foundation, Muscular Dystrophy Association, and JDRF.
Crain's Cleveland Business
Don't be surprised if other local startups end up raising cash from disease foundations in the future: More foundations are expected to start thinking like investors after seeing the Cystic Fibrosis Foundation make a killing on a drug called Kalydeco. That deal is going to push even more disease foundations to think about ways to generate revenue from the drugs they help develop, according to Kristin Schneeman, program director for FasterCures, a Washington, D.C.-based nonprofit that works to improve medical research and speed the development of new therapies. “That's going to generate a lot more interest,” she said.
The Cystic Fibrosis (CF) Foundation's big win in venture philanthropy can fuel constructive competition among companies developing innovative CF drugs, benefiting both patients and the healthcare system by increasing future treatment options and reducing their cost. We should remember that the CF Foundation helped catalyze a major breakthrough in the treatment of CF. By retaining and monetizing a royalty on that first big win, the foundation now has a chance to help bring about other treatment options and the competitive pricing pressure that would come with them — providing even more benefits to patients and the healthcare system.
Gaps in biomedical research funding include cuts in federal funding that are creating serious challenges in the research community, especially academia, says FasterCures Executive Director Margaret Anderson. Venture philanthropy has filled the gap to some extent, and the model is showing what is possible in terms of drug development, but not every disease will have a robust advocacy group behind it, she says. Disease foundations do more than write checks; they also attract other capital and expertise and encourage collaboration, Anderson says.
The Diane Rehm Show
The Cystic Fibrosis Foundation raises about $130 million a year in its ongoing effort to help people who suffer with the deadly disease, but that amount seems small change in comparison with the $3.3 billion it just received related to an investment it made years ago in small drug development company. For a health-related nonprofit, $3.3 billion is a jaw-dropping amount. It gives the foundation the means to explore all kinds of new ways to help patients and to look for a cure, but the projected per patient cost of this drug, $373,000 per year, raises concerns.
The New York Times
The Cystic Fibrosis Foundation, which invested $150 million in a company developing drugs for the disease 15 years ago, stands to bring in $3.3 billion by selling the rights to future drug royalties. The move is likely to catch the attention of other nonprofits, some of which are already investing in manufacturers. "This was a dramatic example of risk-taking that has paid off in a remarkable way," said NIH Director Francis S. Collins.
Disease foundations often give money to startups working on treatments that might help the people they advocate for. But it's not too often you'll see a nonprofit foundation join with a venture firm to create their own company, as JDRF and PureTech Ventures are doing today. Boston-based PureTech has secured a $5 million investment from New York-based JDRF (formerly known as the Juvenile Diabetes Research Foundation) to spawn T1D Innovations.
The Wall Street Journal
When it comes to developing new drugs, pharmaceutical companies and federal agencies have always called the shots. Now patients and their families want a turn. Parent Project Muscular Dystrophy, an advocacy group founded by family members frustrated by a lack of research on Duchenne muscular dystrophy, initiated and wrote a draft guidance for pharmaceutical companies trying to develop drugs to treat the fatal condition.
Accelerator programs and incubators are growing rapidly in number within the healthcare industry, with most replicating standard tech incubator models. But one organization has worked to redefine what an accelerator program can look like in the health space by joining one of the country's largest and most influential associations in its landmark effort to court healthcare innovation. Dr. Ross Tonkens, a cardiologist and chief medical officer in Cary, N.C. has directed the creation of the Science and Technology Accelerator Program inside the American Heart Association, that targets and supports ground-breaking ideas from residents to senior clinicians.
In founding (and funding) the Melanoma Research Alliance (MRA), Debra and Leon Black have effected not only more research and knowledge, but viable change. The MRA, in other words, isn't merely funding research; it's birthing a movement. All of which makes the MRA a very compelling model for philanthropic foundations.
Applying to a nonprofit or foundation for a basic research grant in your area of study can be an effective way to gain visibility and credibility with the organization; building these early connections can be of great use for developing and funding a startup in the future. Much like the other organizations that you are accustomed to applying to for grant funding, these organizations will put out requests for proposals for basic research and translational development projects, and they have links and program coordinators listed and accessible through their Web sites. Many of these groups can be accessed through FasterCures, who has The Research Acceleration and Innovation Network (TRAIN), which lists profiles for 55 organizations that provide $600 million in medical research grants annually.
Now, 17 years later, Kathy Giusti has made unprecedented progress against the cancer through the Multiple Myeloma Research Foundation (MMRF), the organization she founded in 1998. To date, the MMRF has raised $250 million in research money. In addition to funding 350 grants, a huge chunk of the money was used to build the first and largest multi-center tissue bank, which now has access to 4,000 bone marrow samples. With more than two decades of experience in the pharmaceutical industry, Giusti and her organization have spearheaded 47 trials of 24 different drugs and helped introduce six Food and Drug Administration-approved drugs for myeloma.
The Wall Street Journal
For six years, The Wall Street Journal followed a group of parents and scientists seeking a treatment for a rare and fatal genetic disease that strikes primarily children. Their collaboration accelerated development of a promising drug and, along the way, pushed the boundaries of medical research itself.
The Wall Street Journal
Patients with rare and deadly diseases are getting a powerful new boost. Cancer foundations and other nonprofit patient groups - including the Leukemia & Lymphoma Society, Multiple Myeloma Research Foundation, and Michael J. Fox Foundation for Parkinson's Research - are investing tens of millions of dollars to build genetic databases in an effort to speed drug development and jumpstart clinical trials. The databases are designed to collect DNA and other information from patients with hard-to-treat diseases.
In this commentary, Kathy Giusti, founder and CEO of the Multiple Myeloma Research Foundation describes how the foundation has developed innovative, collaborative business models to reshape the R&D enterprise with the single-minded focus of accelerating the development of new treatments for patients to extend their lives and lead to a cure.
Kathy Giusti founded the Multiple Myeloma Research Foundation after being diagnosed with the disease in 1996. As head of worldwide operations at a major pharma company at the time, she was horrified by the lack of drugs in the pipeline for her deadly "orphan" cancer.
Non-profit, patient-focused foundations are becoming an increasingly visible force in early-stage biotech research. Washington, D.C.-based FasterCures, which is an advocate for accelerating research, lists more than 50 nonprofits that are currently working with companies on research projects. They range from large organizations such as the New York-based Juvenile Diabetes Research Foundation and the Multiple Myeloma Research Foundation in Norwalk, CT, to smaller groups, such as Parent Project Muscular Dystrophy in Hackensack, NJ.
Kalydeco, for cystic fibrosis, is a triumph of genetics and drug development, the first medicine to directly affect the genetic defect that causes the disease. Kalydeco would probably not exist were it not for the Cystic Fibrosis Foundation, which funded its early development at Vertex and gets a royalty on the drug. This success paved the way for other disease foundations including the Michael J. Fox Foundation, Myelin Repair Foundation, and the Multiple Myeloma Research Foundation.
Seven years after he was diagnosed with Parkinson's disease, actor Michael J. Fox decided to start a foundation to push for more research into the disease. Now the Michael J. Fox Foundation (MJFF) for Parkinson's Research is playing a leading role in funding the development of new drugs – it is the sole backer of a test for a Parkinson's vaccine – and in working with big pharmaceutical companies like Sanofi and Bristol-Myers Squibb to keep experimental medicines moving through the pipeline. This video highlights discussions with MJFF and two other patient groups: the Multiple Myeloma Research Foundation and Myelin Repair Foundation.