A blog exploring pharmaceutical relationship marketing, emarketing and innovation with a focus on rare disorders.
The convergence of rare disease, digital communications, and pharmaceutical marketing communications

Shooting for the Moon

Posted by | 4:17pm on Wednesday, August 31, 2016

Just as JFK challenged the nation to put a man on the moon, President Obama called on the nation to harness the power of American innovation to seek new ways to prevent, diagnose and treat cancer. It appears that some folks were actually paying attention to this year’s State of the Union address because Obama’s challenge has spurred several initiatives.

Building on his 2015 Precision Medicine Initiative, Obama announced in this year’s address the creation of the National Cancer Moonshot initiative, charging Vice President Biden to lead it. Soon after, a couple billionaires announced plans to pump up cancer research in order to provide better treatments and, hopefully, a cure.

One reason for these new initiatives are the recent successes researchers had with immunotherapy treatments and each initiative is taking a different approach to accelerating the science and application of it. The National Cancer Moonshot is focusing on bring government agencies together. Cancer Moonshot 2020 is bringing researchers, biotech, pharma and tech companies together. Former New York City Mayor Michael Bloomberg and Sidney Kimmel, Jones Apparel Group founder, each donated $50 million with a dozen more supporters kicking in an additional $25 million to create the Bloomberg-Kimmel Institute for Cancer Immunotherapy. Additionally, Sean Parker, creator of Napstar, donated $250 million to form the Parker Institute for Cancer Immunotherapy.

This influx of interest in funding cancer research is encouraging for people suffering from all cancers, but especially people suffering from rare cancers. These types of cancers receive far less funding than more common cancers and are far more deadly. The Target Cancer Foundation reports that of the eight deadliest cancers (survival rate > 50%), seven are rare. For instance, only eight percent of people suffering from mesothelioma live more than 3 years and those diagnosed in stage IV have a mean survival time of one year.

However, while still in clinical trials, researchers are optimistic about immunotherapy treatments for mesothelioma patients since they have been successful with other rare cancers. And more funding for this type of research may offer new treatment approaches to everyone suffering from rare cancers that often have no cure and standard cancer treatment—chemotherapy, radiation and surgery—aren’t very effective. This is why the Mesothelioma + Asbestos Awareness Center, an online support and advocacy center, created an infographic to publicize and spread the word about a few of these initiatives. In July (cancer research month), the center teamed up with the Cancer Research Institute to inform those suffering not just from mesothelioma, but all cancers. The institute is dedicated to harnessing the immune system’s power to conquer all cancers and provide information on the potential benefits of immunotherapy.

The Rare Disease Connection
As for the rare disease community where precision medicine is essential to providing effective treatments, the increased interest, research and investments in immunotherapy treatment can only be beneficial. Providing the right treatment at the right time to the right person is essentially the definition of rare disease and immunotherapy treatments. Very few people suffer from these diseases (right person) and most current treatment options are either nonexistent or borrowed from more common diseases and not very effective, forcing scientists, researchers and physicians to search for more effective (right) treatments. The right time piece of precision medicine results in a cure or a dramatic improvement in disease course/prognosis.
On the other hand, the biological and molecular knowledge already discovered in some rare diseases is essential to researchers developing treatments for more common diseases and cancers. For example, recent research into Laron Syndrome could be applied to cancer and diabetes research. The disease causes low levels of insulin-like growth factor-1, a receptor necessary to the development, growth and survival of tumors. Patients with Laron Syndrome never develop cancer, meaning this receptor must be linked to all cancers.

National Cancer Moonshot
The National Cancer Moonshot initiative has a wider focus than immunotherapy treatment and it includes a focus on rare cancers.

This program plans to accelerate cancer research by focusing on: Prevention and cancer vaccine development; early cancer detection; cancer immunotherapy and combination therapy; genomic analysis of tumor and surrounding cells; enhanced data sharing; creation of a virtual oncology center of excellence; pediatric cancer; and a research fund to focus on high-risk, high-return cancer studies.

Several of these focus areas are already underway. For example, the National Cancer Institute and the Department of Energy have launched three pilot efforts around scientific computing. The RAS effort is developing computational approaches to enable targeted therapies. The patient-focused effort is taking experimental data from patient-derived xenografts and creating models for optimizing the best single/combination treatment for each individual. This effort may also identify new treatments. The third effort will focus on integrating cancer-care data with other data sources to improve patient outcomes.

Another program was recently started by the National Institutes for Health, 12 biopharma companies and multiple research foundations and targets rare cancers. This group plans to understand responses to cancer therapies, create clinical trials for combination therapies and develop predictive models and therapies for rare cancers.

Cancer Moonshot 2020
Focusing solely on accelerating development of genetics-informed personalized immunotherapies and making sure they become the next generation standard of care for all patient with cancer, Cancer Moonshot 2020 has set an ambitious goal.

This billion dollar initiative’s plan is to complete Phase III clinical trials for 20 tumor types in 20,000 people in order to develop effective vaccine-based immunotherapies by 2020. Its focus will be on more common-occurring cancers, but the resulting research and immunotherapies will benefit those who suffer from rare cancers.

Billionaire Patrick Soon-Shiong brought together leaders from every industry to collaborate, share information and support one another to reach this goal. The original members consist of: Celgene, Amgen, NatWorks, NatKwest, Etubics, Altor Bioscience, Precision Biologics, Johns Hopkins, Tufts, Columbia, Harvard Medical School, Jefferson University, Cancer Treatment Centers of America, Allscripts, Blackberry, Independence Blue Cross, HSBC and Bank of America. Soon-Shiong expects other organizations to join as the initiative gains traction.

Each group has a distinct role in the collaboration. Pharmaceutical and bioscience will share drug treatments and research; oncologists will recruit patients and provide treatments; insurance companies will reimburse physicians and collect date; technology companies will create a secure portal to allow collaborators to communicate and share data; and academic and other health systems will validate the science.

Some of the members are pushing the advancement of personalized immunotherapies by making sequencing more available to the general public. In January, Independence Blue Cross announced it would offer coverage for genome sequencing to its 10 million members. Sanford Health Plan soon followed. Additionally, Bank of America, Sanford Health and Phoenix Children’s Hospital, all self-insured employers, announced they would offer sequencing to their members and families. The insurers and employers plan to cover those who suffer from specific cancers, including rare cancers.

So, while researchers aren’t ready to plant a flag down on planet cancer, these initiatives, especially in personalized immunotherapies, may get them closer to one giant leap for mankind.

About Anonymous

View other posts from

Siren Interactive
  • Siren Interactive
  • Rare Disease Relationship Marketing Experts
  • 626 West Jackson Blvd, Suite 100
  • Chicago, IL 60661
  • 312.204.6700
  • 866.502.6714 (Toll Free)
  • www.sireninteractive.com