Yesterday evening Bernard Muller & Robbert Jan Stuit, both diagnosed with ALS, appeared on Dutch television. As established entrepreneurs they founded their own biotechnology company Treeway to speed up ALS research and the development of new therapies. Their website mentions “therapy developed by patients for patients“. They are not the only ones…
Especially in the area of rare diseases, patient advocacy has been an important driving force behind research and therapy development for many diseases. This advocacy has taken the shape of community building, fundraising for research, setting up and maintaining patient registries and biobanks, and even venture philanthropy.
Bernard Muller & Robbert Jan Stuit do not stop there. They see themselves as patient entrepreneurs and with their company Treeway have chosen to be in the driver seat when it comes to drug development for ALS. Their slogan “one man can and will find a way, why not be that man?” applies to more patient/parent entrepreneurs.
The most well-known is probably John Crowley. Father of 2 children diagnosed with Pompe’s disease. He joined Novazyme Pharmaceuticals in 2000 as CEO a year after the company was founded to work on a therapy for Pompe. In 2001 Novazyme was acquired by Genzyme, where until end of 2002 Crowley was in charge of the company’s global Pompe program that ultimately led to the approval of Myozyme in 2006. Watch the 4-min YouTube video below and become inspired.
Different disease, similar story. Daniel de Boer, father of a child diagnosed with Cystic Fibrosis, co-founded in 2012 ProQR Therapeutics, which since then underwent an impressive growth and currently counts about 30 people. Like John Crowley he has teamed up with Henri Termeer, former CEO of Genzyme.
Karen Aiach, mother of a child with San Filippo disease, co-founded in 2009 French biotech Lysogene, which currently focuses on an intracerebral gene therapy for San Filippo. This should pave the way for potential therapies for other life-threatening genetic disorders of the central nervous system. The company has recently successfully complete a phase I/II clinical trial. In a 6-min YouTube video Karen Aiach explains San Fillipo, the genetic therapy and what Lysogene is all about.
Interestingly, in its quest for a cure for San Filippo, Lysogene is joined by another “patient-driven” biotech on the other side of the Atlantic ocean. Phoenix Nest was co-founded in 2012 by Jill Wood and Sean Ekins together with several other parents of children diagnosed with a subtype of San Fillipo, Mucopolysaccharidosis IIIC.
What I find interesting about these entrepreneurs is that, except for John Crowley, they do not have a history within pharma. Instead they originate from the internet, maritime & oil, real estate or financial industry. It is their solid business background that has probably helped them to successfully build a team of scientific, clinical and biotech experts. Are they different from rare disease advocates that focus on peer-support, information and research support? In essence they are not. Like other rare disease advocates, they basically want to make use of their specific skills and work towards a solution for the terrible disease they have been confronted with. They just feel the urge to do that as a biotech entrepreneur.
- Importance rare disease patient advocacy recognized in scientific literature
- Start small Think big
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