Failing Patent System Destroys Chances of Revolutionary Stem Cell Drug from Reaching Market

Several years ago, a development stage pharmaceutical lab had produced a small molecule that could induce stem cell development at the site of injury in adults and trigger the regeneration of biological tissue and re-gaining of full functionality with little to no side effects. In other words, heart attack or stroke patients could be administered a drug that would revive their damaged tissue and save them from a lifetime of suffering. Unfortunately, this drug will never reach the market and help the millions of people suffering from a variety of disorders such as heart disease, strokes, or arthritis.

Recently, I had the opportunity to speak with a patent attorney specializing in biopharmaceutical patent acquisition and management who disclosed very disheartening news. After an in-depth conversation regarding the pharmaceutical industry and the manner in which new medications and therapies are brought to market, it was revealed that there exist a myriad of treatments shown to be very effective that, unfortunately, never reach the consumer. The most interesting patent the firm managed was for a small molecule that could induce autologous stem cell development (meaning, it is obtained from the same individual, not transplanted from someone else) at the site of acute disease or trauma to aid in the regeneration of tissue. This small molecule was called GM1485.

Before I describe the incredible therapeutic potential of this drug, I want to share what the attorney had told me about why this drug will never help a single person.

Current patent laws impose regulations which create a small window of opportunity for bio-medical treatments to reach the market. If a treatment does not get FDA approval within this small window of opportunity, then the treatment and its patents are doomed to spend the rest of their days in some storage unit. I’ll explain why that is in just a moment.

The failing patent system in the U.S. creates an unfair situation for the suffering patients and their families. Highly effective treatments fall through the cracks and never see the light of day, while other ineffective treatments get approval. Some might consider this to be a conspiracy, and I won’t argue for or against that, but the governing bodies that impose patent regulations are to blame. The patent system is currently destroying all chances of various highly effective pharmaceutical treatments to reach market. These regulations need to be reassessed and the period of exclusivity which is granted for each patent needs to change, especially for health care related treatments. It is because of these laws, which I’ll quickly explain later, that pharmaceutical companies won’t support the approval of a stem cell inducing drug with the potential to regenerate tissue in patients suffering from a wide variety of diseases or injuries.

Typically, when a new drug is discovered or developed, the research lab conducts pre-clinical trials in laboratory animals to validate the drug’s therapeutic potential. These labs don’t have the resources to apply for FDA approval since it costs upwards of $2 billion to go through the entire process. Once the data suggests that the drug is effective, patents are filed and the data is verified through countless experiments. After the patents have been granted and the years of testing suggest that the drug works, larger pharmaceutical companies purchase or license the patents and begin the long, laborious, and costly procedures of getting FDA clearance.

Since it takes several years and billions of dollars for pharmaceutical companies to go through the necessary hoops to get FDA approval, they typically purchase the rights for drugs that have at least 7 years of exclusivity remaining on the patents. The patent attorney I spoke with mentioned that by the time a drug is approved by the FDA, there is usually only 2-4 years of protection left on the patent. That is the window of opportunity for the pharmaceutical company to recuperate the billions of dollars it invested to get the drug approved and sold to the consumer. Once the patent expires, generic forms of the drug can be produced.

Unfortunately for suffering patients and GliaMed Inc., the development-stage biopharmaceutical lab that owns the rights to GM1485, pharmaceutical companies will NOT purchase the rights to this drug and fight for FDA approval because the patents are going to expire in a few years and won’t provide the pharmaceutical company with enough time to recuperate the enormous sums of money that would be invested in the process. And so, this drug will spend the rest of its days in some refrigerator in a lab.

I would now like to share some of the promising data gathered from the animals studies. It’s important to note that any given drug that is tested in animals will always have somewhat of a different interaction in a human body.

In pre-clinical trials it was found that GM1485 instructs the cells at the site of injury to release various bio-chemicals that instruct nearby cells to convert back into a stem cell and regenerate the surrounding tissue, without ANY surgical transplantation or other chemical induction. GM1485 belongs to a class of compounds termed Regenerative Immunophilin Ligands or RILs. Treatment with GM1485 induced the localized expression of transcription factors characteristic of pluripotent stem cells at the site of the acute disease or trauma. It was shown that these stem cells can then be reprogrammed by local signals to become neurons, cardiac myocytes, chondrocytes, blood vessels or other cell types, which then contributed to fully differentiated tissues.

I have summarized some of the important therapeutic applications of GM1485 below.

GliaMed Inc. demonstrated in laboratory and animal studies that GM1485 can:

Be ingested orally or applied topically, much like a cream, to regenerate tissues of the heart, brain, skin and more.

Promote the regeneration of all components of the skin after injury, including nerves, hair follicles, blood vessels, dermis and epidermis with minimal scarring.

Promote the regeneration of cardiac tissue when administered orally for 30 days following a heart attack.

Promote the regeneration of neural tissue following a stroke and regaining of functionality associated with that brain region.

Promote the synthesis of cartilage that could help arthritis patients and athletes with severe cartilage damage.

Used as a topical agent, it regenerates tissue without the scarring and loss of functionality typically experienced after a wound is repaired. Furthermore, this regeneration keeps the aesthetic appearance of the original tissue. In animal studies, mice treated with GM1485 fully developed all the complex skin structures in less than half of the time of untreated wounds. The photographs from below were from a swine study demonstrating the accelerated healing of a wound and the regeneration of hair follicles.

In addition to the accelerated wound closure, the GM1485 product has the potential to reduce or eliminate scarring, a potential life saver for burn victims. Most importantly, it is a topical product administered like a cream without any injections or surgical procedures.

The scarring of damaged tissue following a heart attack contributes to the dysfunction of the heart in patients. In an animal study, GliaMed Inc. demonstrated that once-daily treatment of GM1485 resulted in 100% survival and near complete regeneration of heart tissue following a heart attack. Animals not treated with GM1485 had extensive scarring of the heart.

In another animal study it was shown that the administration of GM1485 induced the regeneration of brain tissue at the site of injury. Rather then developing scar tissue which is often the case, the brain tissue regenerated itself. If administered shortly after a stroke, it has the potential to revive functionality in patients.

GliaMed Inc. also demonstrated that GM1485 induces the biosynthesis of human cartilage in the petri-dish, potentially helping arthritis patients and athletes with damaged joint cartilage. Furthermore, mice with holes punched in their ear regenerated full composite tissue after being treated with GM1485.

Although this drug can benefit millions of people around the world with very little side effects, it will forever be stored in a refrigerator. The sad reality is that the patent system in the U.S. does not actually promote innovation and progression. It continues to hinder the advancement of medical technologies shown to have amazing potential to change our lives and the way we view disease or injury.

One way to fix this problem would be to have a publicly funded organization pick up these drugs with expired patents and use public funds to fight for FDA approval. If approved, generic versions can be manufactured by other pharmaceutical companies and the public organization can write it off in their books as a billion dollar loss. I know it sounds crazy, but crazier things have happened.