Life Science Diamonds in the Rough (LSDIR)
LSDIRs are game changing advances in medicine representing fundamentally new discoveries or applications of technology.
They often arise outside the ecosystems of KOLs and marquee institutions supporting the current disease treatment paradigms.
Founders and entrepreneurs often face daunting challenges. Overcoming the faith in the status quo and investor skepticism is particularly hard if they are not well known yet in the field to which their product would be applied.
The more remarkable an innovation is, the higher the burden of proof, which often creates a Catch-22: the money to establish the necessary confirmation is available only after the confirmation has been completed.
Medicine ignored the cure to gastric ulcers in the '60s
In 2005, Barry Marshall and Robin Warren were awarded the Nobel prize in Physiology
In the words of the Nobel Committee, they were honored “for their discovery of the bacterium Helicobacter pylori and its role in gastritis and peptic ulcer disease.”
They first published their results in published in The Lancet on June 16, 1984.
John Lykoudis MD, trained in 1930s in Greece, and practiced medicine in a town of <10,000. John had peptic ulcer disease (PUD; gastric ulcers). In 1958, he treated himself for haemorrhagic gastroenteritis with antibiotics which also cured his peptic ulcer disease. He created Elgaco, an antibiotic treatment which was awarded Greek patent number 22 453 in 1961. He moved to Athens to treat more PUD patients
and cured an estimated 30,000 Greeks with gastric ulcers using Elgaco.
He attempted to publish his observations in JAMA, but his manuscript, “Ulcer of the Stomach and Duodenum”, rejected in 1966, because “[it] does not seem appropriate for our journal”
In 1967, Lykoudis succeeded in getting the attention of the Greek Prime Minister’s office. In 1967, he proposed, in essence, a phase III trial: 100 PUD patients to be treated at a State hospital by the eminent professors, 50 with conventional treatment and 50 with Elgaco but was rejected. “Their refusal to approve it is understandable, but their refusal to test it is not!”.
All pharma companies contacted including J R Geigy SA, Farbwerke Hoechst AG, Schering AG, Bayer, Leo Pharmaceutical, and Specia of Rhone Poulenc rejected licensing for lack of clinical studies and concerns about IP protection of a combination of approved medicines.
Greek medical authorities fined him for malpractice
Still happens today: treating Ulcerative colitis
Dr. Jay Pravda was an immunologist practicing in Florida. Many of his snowbird patients were coming in with poorly controlled ulcerative colitis. He reexamined the literature and published a proposal for an alternative mechanism of UC in 2005 “Radical Induction Theory”: H2O2 overproduction induces mucosal inflammation and ulcerative colitis via damage to tight junctions followed by bacteria infiltration and inflammation.
He designed a therapy and began treating patients with an MD partner who had a large gastrointestinal specialty practice in Tennessee and in 2008
Licensed the drug to a small pharma company in 2014. The initial clinical case series of 35 patients was published in 2019 showing 85% of moderate to severe UC patients saw histological remission and complete mucosal remission and 87% showed clinical remission. 97% had a clinical response.
The FDA indicated they wanted a combinatorial trial testing the individual contribution of each ingredient (4). The unwieldy clinical study meant costs exceeded financial capabilities of the small pharma company and no other investor or company was willing to underwrite the study, so program was abandoned and returned to Dr. Pravda.
Dr. Pravda was unable to secure financing for development and resorted to offering it directly to patients as a locally compounded therapy.
Founders Need Help
Academics, MDs, or other first time founders and entrepreneurs need a partner with experience beside them, as an interpreter, guide, partner, and mentor.
Are you at an institution with an entrepreneur mentoring program?
Do investors participate in campus events?
Do you have a Technology Transfer office?
Do you have a local incubator for your innovation(s)?
All of these can help.
So can having an experienced startup ally. Helping founders is in our DNA.