PALSFirst Campaign Leader Dies Friday from ALS

Cheryl Anne Beede-Sweeney, courageous wife, daughter, sister, and mother of three passed away Friday, July 3, 2015 in Duluth, Minnesota after a long battle with Amyotrophic Lateral Sclerosis (ALS)/Motor Neuron Disease (MND).  

ALS, also known as Lou Gehrig's Disease is a fatal disease that rips away your ability to move, eat, speak, and breathe. Every 90 minutes someone is diagnosed with ALS and another dies.  There is no cure for ALS.  Only one drug on the market exists that prolongs life on average only three months.  Like many people with ALS, Cheryl did not qualify to participate in clinical trials due to current lengthy diagnostic criteria and strict inclusion/exclusion criteria.  Years were spent trying to access BrainStorm Cell Inc.'s NurOwn stem cell treatment via the U.S. FDA Expanded Access Program, but BrainStorm Cell Inc. denied her access to this potentially life saving treatment, thus was the birth of the PALSFirst Campaign.   

Cheryl truly was the heart and soul of the PALSFirst Campaign.  She was a natural leader, advocate, and faith-based healer.  Her smile, continuous love for others, and hope that there would be a cure someday for ALS filled others with joy.   More than anything, Cheryl wished for others suffering from ALS to have the opportunity to try new treatments through clinical trial redesign, U.S. FDA Accelerated Access and Expanded Access Programs, and policy reform.  Cheryl's presence will be greatly missed, but her legacy will live on with the PALSFirst Campaign and all those who continue to fight for access to treatments.  

Cheryl Beede Sweeney's Obituary

What ALS Can Learn from the Cancer Experience: To Move Forward We Must Look Back

To move forward, we need to reflect on the past.  The Spanish philosopher, essayist, poet and novelist George Santayana once famously said, “Those who cannot remember the past are condemned to repeat it.”  Not knowing your history can indeed have devastating consequences both in repeating mistakes but also not capitalizing on opportunities.

 

The Cancer Experience teaches us that developing successful treatments is not easy.  There are a lot of hurdles that must be overcome including technological, financial, political and socio-cultural.  Complicated diseases need a full-forced commitment of funding and political will.  It took decades, billions of dollars, and strong political commitment to fund cancer scientists and medical doctors whose research efforts helped lay the foundation for advancements in medical procedures, radiation therapy, chemotherapies, hormone and immune therapies that we benefit from today.     

This was made possible through huge financial investments not just from private donations, but also sizable public funding.  Mary Lasker and Dr. Sidney Farber spearheaded the private-public funding effort in the 1960s, which resulted in enactment of the 1971 Cancer Act, and billions of dollars in funding towards the National Cancer Institute.  The Ice Bucket Challenge was a major breakthrough in private funding for research, now we need equal commitment for public funding to support non-profit universities, teaching hospitals, institutes and government agencies.  Places such as the Robert Packard Center for ALS Research at Johns Hopkins, National Institutes of Neurological Disorders and Stroke, and ALS Treatment Development Institute. 

The Cancer Experience teaches us that it is important for grant dollars to be flexible and support a diverse workforce of researchers.  The most innovative, game changing cancer therapies came from the minds of new doctors and researchers in the field, people with fresh eyes, willing to take more risks and think out of the box.  People like Dr. Bernard Fisher who in 1958 went against an almost 70 year practice of the radical mastectomy and proved through randomized control trials that women who underwent breast conserving surgery also known as the lumpectomy had the same survival rates as women who underwent a radical mastectomy.  People like Dr. Sidney Farber who in 1947 went against the medical community by giving children suffering with leukemia the experimental chemotherapeutic, aminopterin.  The standard at the time was to make children comfortable until their inevitable death.  Aminopterin allowed some of the children to go into remission.  Had these medical renegades not been hired and given resources, evidence-based practices would not have been developed, advancements in treatment would have been lost.

Today, the lack of research dollars for the biomedical sciences has stymied advancements.  In order for new scientists and medical doctors to even be considered in the applicant pool of 300 or more people vying for the same job, they must already have funding.  To get funding, scientists and medical doctors must be affiliated with a university or a government facility.  A catch-22 occurs, resulting in small numbers of scientists and medical doctors, experienced but not necessarily game-changers; all competing for finite resources.  A substantial amount of grants require a significant amount of preliminary data generated by the Principal Investigator (lead scientist) and proof of success.  Therefore, high-risk projects that may challenge current paradigms or lead to new therapeutics are less likely to be funded compared to “inside the box” ideas.  The ALS Community must identify the Dr. Sidney Farbers and Mary Laskers and pave the way for real commitment through policy change and private-public funding.

What ALS Can Learn from the Cancer Experience: Introduction

It was 1969 and President Nixon declared a War on Cancer.  Despite being in the midst of a recession caused by the Vietnam War, billions of dollars were poured into cancer research and a new era in cancer’s history started.  Cancer was once a disease scientists knew little about, a disease no one wanted to discuss became a national priority.

This change didn’t come about suddenly.  As a field of science it has span time in memoriam, from early Egyptian papyrus that documented some of the earliest cases of breast cancer, to the invention of early treatments such as the radical mastectomy and radiation therapy of the 1890s, to the diverse chemotherapies, hormone therapy and immune therapy of today.  This success story has multiple heroes.  First there were the dedicated scientists with an insatiable appetite for discovery, conviction to eliminate human suffering, and defiance against the status quo.  Next on the scene were the entrepreneurs and visionaries that spearheaded private and public funding efforts, followed by American citizens who both suffered from the disease and watched their friends and family members die of the disease until a boiling point was reached and they said ‘We’re not going to take it anymore.”

The history, science, medicine, and politics of cancer were beautifully summarized in Siddhartha Mukherjee’s book, The Emperor of all Maladies: A Biography of Cancer, recently turned into a PBS documentary film by Director Ken Burns.  Most striking about this film is the parallels to other major diseases and public health funded projects in U.S. history; parallels that the ALS/MND Community can learn from and apply to advance ALS science, treatments, and access to experimental drugs and therapies outside of clinical trials.  

This post is one of a five part series on lessons learned from The Cancer Experience:

• Introduction
• To Move Forward We Must Look Back
• Identifyand Support Champions and Change Agents
• Bypassing Clinical Trials, A Cautionary Tale
• American Public Can be a Powerful Change Agent