Submitted by Jade Morgan as part of our contributors program.
One of the world’s wealthiest biotechnology executives is leaving a gift to the world. He wants to give the world a solution to cancer.
He is working on Factor 5A- a protein at the wellhead of all cancer genes that controls cell death. When Factor 5A is not working properly, cells refuse to die and continuously duplicate. As everyone knows, cancer is caused by “rogue cells” that duplicate too much and form tumors. The solution is for Factor 5A to tell the cell to die.
If we tell cancer cells to die, we cure cancer.
This is the story of the final gift to the world of a doctor named Harlan Waksal. He made his fortune selling a company he founded for $6.5 billion to pharmaceutical giant Eli Lilly (NYSE: LLY). He is now the Chairman of the Board at Senesco Technologies (OTCQB: SNTI), a company that has been researching Factor 5A since the late 1990s.
With hundreds of millions in personal wealth, Dr. Waksal needs nothing. He has already proven his knowledge in science, drug development, clinical practice and corporate leadership. So if you think it matters whether or not you invest in Senesco after reading this story, you are missing the point entirely. This article is not concerned with the stock price of Senesco. Dr. Waksal could fund the entire company—personally—for the foreseeable future. Senesco stock is not the point.
This story is about a whole new way of treating cancer. If there is one protein that can tell all cancer cells to die, it is Factor 5A. This is the story of Factor 5A and why one of the world’s most successful doctors is so excited about it.
Spoiler alert: three humans have already received the treatment. Results to follow.
Dr. Waksal’s Early Years
Harlan Waksal immigrated to Ohio from Europe with his family in the 1950s. His family had suffered during the early 1990s. Germans subjected his mother to the brutalities of the Auschwitz concentration camp. His father fought for the Polish underground. His brother supported the family in the United States by working in the scrap metal business.
Soon-to-be-Dr. Waksal was interested in studying science. He earned a Bachelor of Arts in biology and philosophy from Oberlin College in 1975 and an MD from Tufts University School of Medicine in 1979. Within five years, Dr. Waksal and his brother had co-founded a biotechnology firm. They attracted some legendary investors, including Tom Rosse, who provided an initial $250,000 for a business plan.
It was in meeting with potential investors on a business trip to Denver that Dr. Waksal and his brother decided to name the firm “ImClone Systems,” amalgamating the facets of science they wanted to pursue at the company: immunology, DNA cloning and medical information systems.
Dr. Waksal and his brother ran ImClone Systems as a holding company for scientists who wanted to advance their research in immunology-based diagnostics and infectious diseases. A few years later, they began to actively seek an Initial Public Offering (IPO) of stock in order to fund the development of vaccines for the Hepatitis B and AIDS viruses. Timing, however, was not on their side during 1987. The stock market crashed in October of that year, ultimately forcing the brothers to shelve their plans.
The stock market recovered over the next four years, and Dr. Waksal was able to witness the IPO of ImClone at $14 per share. Its stock price subsequently climbed as high as $27 before falling out of favor with investors when it failed to develop a vaccine for Hepatitis B and AIDS.
Dr. Waksal Turns to Cancer Research and Gains a Fortune
ImClone struggled throughout the early 1990s. In 1995, however, things began to look up for the company. Dr. Waksal and his brother decided to back the initiatives of Dr. John Mendelson at the University of California at San Diego who developed a cancer drug called Erbitux. In the spring of 1995, Erbitux shrank cancerous tumors in two patients with head and neck cancer. ImClone’s stock price quickly rallied on the news, and Dr. Waksal and his brother received licensing offers alongside takeover bids. They closed on a co-marketing agreement with German drug maker Merck for European rights.
By 1999, ImClone’s Erbitux had successfully treated colon cancer. Soon, the FDA granted Erbitux fast-track status as an advanced treatment of colon cancer, and by 2001, Erbitux was treating pancreatic cancer. With a rallying stock price alongside the success of Erbitux, ImClone filed for final drug approval from the FDA.
By September of that year, Bristol-Myers Squibb (NYSE: BMY) offered $2 billion for a 40% share of the profits in Erbitux and another $1 billion for 19.9% of ImClone’s stock.
Dr. Waksal’s high from the Bristol-Myer’s deal was temporary. While he was on vacation in Colorado, he received a call on Christmas Day from a Bristol-Myers official that the FDA’s approval of Erbitux seemed less likely. He called his brother the following morning, and the two of them attempted to counteract the FDA’s decision not to approve the drug. Ultimately, the FDA rejected Erbitux, and ImClone’s stock plummeted.
Nevertheless, over the next few years, ImClone developed other technologies and rallied back to all-time highs. In October 2008, Eli Lilly bought ImClone for $6.5 billion. The deal created one of the largest oncology franchises in the biopharmaceutical industry, and Dr. Waksal personally profited several hundred million dollars.
Dr. Waksal Joins Senesco Technologies
In October 2008—the same month in which Dr. Waksal made his fortune—he joined the board of Senesco. Dr. Waksal was intrigued by the genetic focus of Senesco’s cancer treatment, which had been in development for almost two decades by that point. Dr. Waksal became one of the two board members who put up $11.5 million to fund an early-stage study in humans to test whether or not inducing cell death via a protein called Factor 5A could treat multiple myeloma cancer.
By March 2010, the FDA also granted Senesco its prestigious Orphan Drug Status after its successful trials in plants, rodents and primates. Senesco then began enrolling three late-stage multiple myeloma cancer victims who had failed to respond to two prevailing market treatments (in other words, very sick people). By 2012, Senesco administered its genetically-driven therapy treatment targeting Factor 5A, called SNS01-T, into the first three humans.
The treatment targets Factor 5A in B-cell cancers via intravenous infusions and was later expanded to diffuse large B-cell lymphoma (DLBCL) and mantle cell lymphoma (MCL). Senesco’s SNS01-T’s trial for humans involves cohorts that will receive progressively stronger doses. The first cohort of three cancer victims—already complete—began at a very low dose of 1 milligram per patient. (This was roughly 1/30th of the ideal dose of the serum.) Patients were dosed twice weekly for six weeks. The second cohort of Senesco’s SNS01-T’s trial in humans is now open for enrollment and begins at a higher dosage level.
Again, the first cohort of the Phase 1b/2a clinical trial of SNS01-T was complete as of August 2012, at which time to company revealed that SNS01-T was well-tolerated in all three participants. More importantly, two of the three patients achieved “stable disease,” and one remained stable four weeks beyond the end of the dosing period.
Incredible Success, Even at 1/30th the Ideal Dose
The “stable disease” outcome was remarkable, and not only due to the fact that the three victims were late-stage (almost terminally ill) patients who had failed to respond to not one but two prevailing market treatments. When added to the miniscule dosage level—1/30th of the ideal amount—to see any progress would have been encouraging, but to see two patients stabilize? Dr. Waksal was astounded.
If the treatment were to ultimately gain FDA approval, it would be administered at much higher potency and, therefore, kill cancer cells far more effectively. Even so, three terminally ill patients were able to see substantial improvement with only a tiny dose.
After a Lifetime of Cancer Research, Dr. Waksal Might Have Found the Cure
Dr. Waksal is indisputably one of the most successful executives in the cancer treatment industry. His fortune comes from properly identifying cancer treatments that work, and he has been rewarded with hundreds of millions for his accuracy. He now believes that Senesco’s Factor 5A is the next big thing.
The thing about Factor 5A is that it functions in all types of cancers, in both animals and plants (yes, plants get cancer too). When the Factor 5A-related genetic switch is in the “pro-living” position, cancer cells over-duplicate and form tumors. When switched to the “pro-death” position, cancer cells will die. Senesco’s treatment is therefore incredibly broad. If approved for multiple myeloma, Senesco would rapidly seek approval for other types of cancer.
The summary is simple. Dr. Waksal is working on a cancer treatment that stabilized patients at just 1/30th the ideal dosage on late-stage patients who failed to respond to two other FDA-approved treatments.
If you had sold your company for $6.5 billion, on what project would you be working? Dr. Waksal is at Senesco, trying to give the world a solution to cancer.