This article profiles Samumed, who are producing regenerative therapies based on manipulation of Wnt signaling, presently at various early stages in the pipeline. Wnt signaling is implicated in cancer, aging, and regeneration, but like many protein networks it is involved in a large number of very fundamental cellular processes, making precise control of outcomes a challenge. This is something that the Samumed researchers claim to have solved to a large enough degree to produce drugs that target this pathway, with Wnt-based regenerative treatments in the works for a range of tissues:
Samumed has raised $220 million and is halfway through raising another $100 million. The target Samumed researchers went after was obvious: a gene called Wnt, which stands for "wingless integration site," because when you knock it out in fruit flies, they never grow wings. It's a linchpin in a group of genes that control the growth of a developing fetus - whether you're a fly or a person. Together these genes are known as the Wnt pathway. Trigger the right ones and you might revive old flesh. Some cancers do their dirty work by hijacking Wnt, and blocking it might stop tumors. Most other researchers who had searched for Wnt drugs used one of biomedicine's workhorses: a cell line derived from an aborted fetus in the Netherlands in 1973. Those fetal cells are easy to use in the lab, but over the intervening decades they have become very different from normal cells in humans. The Samumed team opted to look for drug targets in colorectal cancer cells that expressed Wnt, comparing them with healthy colon cells that didn't. It took almost three years. Exactly what did they find? Samumed isn't quite saying. Normally a patent explains which chemicals a drug targets. But in 2013 the Supreme Court said that genes aren't patentable, a ruling Samumed interprets as saying the company can have its patents while keeping those biochemical pathways under wraps.
What the company will show is the animal and human data for its baldness and arthritis treatments. In mice and mini-pigs that have had hair removed, it grows back. Experiments in arthritis involve cutting the ligaments in the knees of rats so that the cartilage is destroyed. Samumed's drug regrows the cartilage, and the rats can walk again. So what happens in people? In March Samumed presented data on the use of its baldness drug, code-named SM04554, in 300 patients. Hair-loss specialists who saw the data were not blown away. Those results aren't big enough to be certain they're not occurring by chance or that men will really feel that the product is making their hair grow back. When it comes to Samumed's valuation - and medicine as a whole - the arthritis data are far more important. The largest study of Samumed's arthritis drug, SM04690, included only 60 patients. Even for small numbers the results line up alluringly: Patients who got SM04690 scored better than those on placebo on two questionnaires that measured how well they functioned and whether their pain improved. On X-rays of patients' knee joints, the space between the bones seemed to have increased, indicating cartilage might really have grown back. Still, again, even Samumed's own consultants say the data are preliminary. More proof will come from a 445-person trial that Samumed aims to complete by the end of the year.
Viewed under the microscope, Samumed looks like a company with a pair of drugs that have not been proved and, if trends in drug discovery hold true, will probably not make it to market. But its investors obviously see something far more wonderful, world-changing and potentially lucrative. If these drugs work, it becomes a better bet that some of Samumed's other medicines will work, too. There's a treatment for scarring of the lung, known as idiopathic pulmonary fibrosis. And for macular degeneration, which causes blindness.