Each patient's battle against his or her disease is unique. Here are some patients that we've had the privilege of meeting, and their stories.
After receiving the medicine, I could see almost immediate results in my mother. However, I also wanted to better understand the impact of side effects on her too. She does exhibit many of the side effects, but her quality of life has improved. Without this medicine, she would've only lived another 1-2 months.
Date: January 2018
Medicine: Alecensa (alectinib)
In March 2014, Danny had just become a father for the second time when he was diagnosed with a rare form of cancer in his liver. A dedicated member of his local football club, Danny was asked if there was anything he'd still like to do with the time he still had. His reply? "To play a game of football with my friends. One more time." A charity match was arranged in his name. Danny played alongside his 3 year old son and their team won 4–1.
After an unsuccessful course of chemotherapy, Danny's physicians informed him that he had run out of medicines to try. They gave him few months left to live.
The thought of giving up never crossed Danny's mind. He and his wife Charlotte started searching for other options. Together they found a medicine in New York that might help, but it would cost € 300,000 and his insurance company would not pay for foreign medicines.
A skilled marketing manager, Charlotte set up a fundraising campaign to get her husband the medicine he so desperately needed. They made a list of all the people in their network who could help them, with everything from building a website and Facebook page to getting coverage in the media. Support grew, and enough money was raised for Danny to fly to New York and receive surgery and initial treatment.
TheSocialMedwork was able to import the medicine for Danny to his home country, the Netherlands. This meant that he didn't have to keep flying to New York every month. Danny passed away in 2017 after spending three more valuable years than he thought he would with his family.
Medicine: Lynparza (olaparib)
Mirjam is a dedicated sportswoman, an avid reader, director of several elementary schools, married and a mother of two boys. In March 2014, at the age of 39, she was diagnosed with B-cell non-Hodgkin lymphoma (an aggressive fast-growing cancer).
Her physician told her that 70–80% of patients respond well to conventional medicines. At first, Mirjam responded well to the chemotherapy, but this didn't last and her cancer progressed. She tried other treatments, including chemotherapy and various immunotherapies. Had the tumour been small enough she might be able to receive a stem-cell transplant, but it wasn’t, and so she was running out of options. Her physician suggested a novel immunotherapy that was not yet approved for non-Hodgkin lymphoma. They tried to join clinical trial but were unsuccessful. It looked like the end of the road for Mirjam.
Thankfully, it wasn't. Mirjam refused to give up, and after contacting patient advocates learned that the medicine she wanted so badly could be delivered to her from the US. Her physician would need to prescribe it, and she would have to pay for the medicine herself, but it was possible.
Unexpectedly, the physician wouldn't prescribe the medicine, as hadn't been approved in Europe yet. But "no" wasn't an option for Mirjam; she found another physician who was willing to prescribe the immunotherapy whilst abiding by the European regulations. Within 2 months, her sisters raised € 45,000 from friends and family. Mirjam could finally try this new treatment.
Mirjam's body responded well to the immunotherapy. Within a few days, she was feeling better. More importantly, the tumour shrank rapidly, to such an extent that she was able to qualify for a stem-cell transplant. Today Mirjam is still alive, in good health and grateful to all who helped her.
Mirjam's advice? "We're living in a fast changing world – a "no" can become a "yes" in a day. One physician said no, and whilst another said yes. Don't take no for an answer."
In July 2013, Garmt van Soest returned home from a week of kitesurfing, ready to get stuck into some new projects at work and prepare a wedding speech for one of his best friends, Paul.
Devastatingly, upon returning to the Netherlands his neurologist informed him that he had amyotrophic lateral sclerosis (ALS), also known as motor neuron disease.
With the help of his employer, Accenture, Garmt got in contact with Philips and together they started developing equipment that would enable patients to communicate with their brainwaves.
Garmt passed away in October 2017, and is dearly missed by all of his family and friends.
Medicine: Radicut (edaravone)
Hein Jamboers was diagnosed with melanoma in 2009.
He had surgery but the tumour returned; he was told he had three months to live. Hein visited other hospitals for a second opinion, but all agreed that there was nothing that could be done.
Hein started investigating treatment options himself and came across the story of a Belgian woman who had been treated with an experimental medicine; one that hadn't yet been approved in Europe.
He tracked her down, spoke to her physician, and was referred to another hospital, the Dutch Cancer Institute (NKI). He was eventually treated with the same medicine as the Belgian patient, but it had no effect on him.
Thankfully, Hein was later invited to be one of the first three patients in The Netherlands to undergo a brand new medicine – still in its early stages of development. This medicine would harvest his immune cells, culture them outside his body and then inject them back in larger numbers. At first, the side effects were severe, and he was transferred to intensive care. Miraculously however, the treatment was successful – the tumours shrank and Hein was declared cancer free six months later.
Hein has since become an advocate for melanoma patients, sharing his story in the Netherlands, overseas, and to anyone who will listen. Whilst many physicians worry about where to refer people, Hein is not. "I know how hard it is if you can't find information and so I'm happy to tell my story whenever I can. I was told I only had 3 months to live. That was in 2011. I'm still here and enjoying life to its fullest every day."
Abigail had always wanted to go to college. At 18, she was living her dream. Studying at the University of Virginia, she was an honors student and an active member of her community and church.
Devastatingly, she was diagnosed with head and neck cancer. She tried various forms of chemotherapy straight away, but they kept failing.
With no more treatments left to try, her physician suggested that she look at experimental medicines (medicines that are still under development and have yet to be tested and approved).
Based on molecular diagnostics, Abigail's physician knew of a drug that was already in development and could benefit her greatly.
She applied for clinical trials but was told she did not qualify – her cancer didn't fit the exact criteria of the trial. Her family begged different manufacturers to give her the medicine on compassionate grounds, but to no avail.
Although fighting for her own life, Abigail realised she wasn't the only one. One day she turned to her father: "Dad, if I make it, I want to devote my life to helping people with cancer and other life-threatening illnesses. Let's start working on ideas."
Together they started lobbying pharmaceutical companies and launched a media campaign that attracted a lot of attention. In June 2001, a few days before she died, Abigail spoke with a journalist: "This is not just about me. This is about so many others too."
No one knows if the medicine that Abigail was denied could have helped her. As it was at an early state of development, the side effects were unknown. This was a risk that Abigail and her family were willing to take; however, drug companies and regulatory boards denied her of the chance.
After Abigail died, her father, Frans Burroughs quit his job as an engineer and founded the Abigail Alliance For Better Access to Developmental Drugs – a lobbying group seeking to create wider access to developmental cancer drugs and other drugs for serious life-threatening illnesses. In Abigail's honour, they seek to prevent situations like hers in the future.
It was June 2010; prime minister Yukio Hatoyama of Japan just resigned; a giant earthquake hit Chile; the FIFA World Cup had just kicked-off in South Africa; and Bernard Miller was diagnosed with Amyotrophic Lateral Sclerosis (ALS).
Bernard is a successful entrepreneur. He's tall, well dressed, with salt and pepper hair and a wry grin. He loves his family and is a golf fanatic.
Before his diagnosis, Bernard ran his own company in the oil and maritime industry. When asked what he did after his diagnosis, Bernard simply smiles and shrugs: "Some people simply resign themselves to the situation, but that doesn't suit me." Instead of accepting conventional treatment plans, he decided to tackle his illness with the same entrepreneurial spirit that had served him so well in his career.
Whilst reading up on ALS and building a network within the biotech and life sciences sector, Bernard met Robbert Jan Stuit, a fellow ALS patient. They decided to approach the problem from a business perspective.
Together, they set up Project MinE, a project that studies the underlying genetic basis that plays a role in developing ALS, examining the DNA profiles of thousands of ALS patients and healthy individuals.
Around the same time, Bernard and Robert also started the biotech company Treeway, with the goal of using the data collected by MinE to help develop future therapies. Treeway investigates the disease mechanism itself and is looking for ways to slow the disease's progression.
Six years after diagnosis, Bernard is as cheerfully determined as ever: "I refuse to rest until the cause of ALS has been discovered and we can work towards finding a cure."
Medicine: Radicut (edaravone)
When the US Food and Drug Administration announced in May 2017 that a new drug had been approved for the treatment of ALS, my father and I were thrilled. He had been diagnosed with the disease a month earlier but had been turned down for exploratory testing, so this was a godsend. What he didn't realize at the time was that it would be months and months before the drug was actually available in the United States, and even more months before his insurance company would accept paying for his treatments. Anticipating this, I searched online for a foreign provider of the drug. That's when I found The SocialMedwork.
Within days I had a personal contact person there who took me through all the steps needed to obtain and pay for the drug, which came from Japan. There were slow-downs along the way such as getting the prescription from my father's doctor (persistence is needed) and getting the package through customs (patience is needed), but ultimately it took less than six weeks to get my father the drug that might help him.
The price we paid happened to be one-third of what his insurance company would have paid in the US, which made it affordable for my father to pay out-of-pocket. Coordinating with infusion nurses was also our responsibility, which added to the workload, since often it is the infusion companies that coordinate all of the work that we did ourselves. The drug did not save my father, ultimately, and he passed away at the end of December. But it 1) gave him hope and the knowledge that we were using the latest techniques to fight this horrible disease, and 2) helped him to avoid the difficulty in breathing and swallowing that most ALS patients experience in their final months. We also applied for the drug via the normal channels in the US, and have still not received an answer from Dad's insurance company.
I am so appreciative that this organization exists -- and I hope it continues to serve people who need it throughout the world!
Medicine: Radicut (edaravone)
Date: January 2018
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