As the patent community waits to see whether the United States Supreme Court will deal a significant, perhaps fatal blow, to the patenting of many genetic related innovations in Association of Molecular Pathology v. Myriad Genetics, announcement came this morning of a newly issued U.S. patent. This patent — U.S. Patent No. 8,399,645 — was issued to St. Jude Children’s Research Hospital. The invention relates to compositions for genetically modifying human immune cells so they can destroy some of the most common forms of cancer in children and adults.
It seems unthinkable that the Supreme Court could issue a ruling that would call into question the patent eligibility of an innovation that has the potential for curing cancer, but that is what is at stake. The Supreme Court will hear oral arguments in AMP v. Myriad on April 15, 2013, and the issue they will consider only a single question: Are human genes patentable?
While this St. Jude invention is not likely to be directly impacted by any ruling the Supreme Court makes, if the Supreme Court says that human genes are not patentable then what is to stop the march toward a ruling that says genetic modifications are likewise not patentable? Additionally, the Myriad claims relate to isolated DNA sequences, not DNA as it exists in a person or in nature. Some of St. Jude claims to this cure for cancer would seem to fall if the Myriad claims fall because they cover isolated host cells. Indeed, there is a lot at stake.
St. Jude and this Innovation
St. Jude Children’s Research Hospital is internationally recognized for its pioneering research and treatment of children with cancer and other life-threatening diseases. The hospital’s research has helped push overall survival rates for childhood cancer from less than 20 percent when the institution opened to almost 80 percent today. The research that led to this patent was supported in part by a grant from the National Institutes of Health. The patent issued today increased St. Jude’s patent portfolio to more than 100 issued United States patents.
By any measure St. Jude has been enormously successful. And those who think that government shouldn’t be funding research that results in the issuance of patents will absolutely hate the fact that the government and grants have played a role, together with the patent system, to provide the incentive and funding necessary to achieve this remarkable advance.
But what is this particular advance exactly? The invention allows human immune cells to successfully fight the cancer. “This groundbreaking invention enables human immune cells to recognize and attack certain cells that cause leukemia and lymphoma, cancers of the blood and lymphatic tissue,” said James R. Downing , M.D., St. Jude scientific director.
The invention involves genetically modifying human immune cells to enable them to manufacture a large protein molecule called a “chimeric antigen receptor” (CAR). The protein molecule is “chimeric” in that it is made from parts that do not exist together in the same molecule in nature. It is a “receptor” because a portion of it extends outside the surface of the immune cell and can receive signals from external “antigens.” An antigen is a substance capable of stimulating an immune response in the human body. The CAR invented by St. Jude fits and latches onto “CD19” antigens prevalent on the B cells that cause ALL, CLL and NHL. It then stimulates the human immune cell to attack and kill the B cells.
The patented technology represents a potentially potent new therapeutic weapon against such diseases as B-cell acute lymphoblastic leukemia (ALL), B-cell chronic lymphocytic leukemia (CLL) and B-cell non-Hodgkin lymphoma (NHL). Each year approximately 71,650 people in the United States are diagnosed with these diseases.
“This exciting invention provides a new and promising treatment option for children and adults with these life-threatening diseases and sets the stage for treating other forms of cancer with cellular immunotherapy,” Downing said. St. Jude plans to offer patent licenses to those who wish to exploit this important technology.
The Patent Claims
Of course, patent licenses are only as valuable as the claims granted, and those granted that are likely to withstand the challenge of those who seek to circumvent, as well as challenges by those who just don’t like the patent system period. The independent claims issued to St. Jude in the patent are:
1. A polynucleotide encoding a chimeric receptor comprising: (a) an extracellular ligand-binding domain comprising an anti-CD19 single chain variable fragment (scFv) domain; (b) a transmembrane domain; and (c) a cytoplasmic domain comprising a 4-1BB signaling domain and a CD3? signaling domain.
2. A vector comprising a polynucleotide encoding a chimeric receptor comprising: (a) an extracellular ligand-binding domain comprising an anti-CD19 single chain variable fragment (scFv) domain, (b) a transmembrane domain, and (c) a cytoplasmic domain comprising a 4-1BB signaling domain and a CD3? signaling domain, wherein the polynucleotide encoding the chimeric receptor is operatively linked to at least one regulatory element for expression of the chimeric receptor.
3. An isolated host cell comprising a polynucleotide encoding a chimeric receptor comprising: (a) an extracellular ligand-binding domain comprising an anti-CD19 single chain variable fragment (scFv) domain; (b) a transmembrane domain; and (c) a cytoplasmic domain comprising a 4-1BB signaling domain and a CD3? signaling domain.
As you can see, the isolated host cell claim, claim 3, could be on a very slippery slope if the Supreme Court decides that the isolation step in the Myriad patent is insufficient to establish patent eligibility.
How strange would it be if the cure for cancer has been achieved only for society to take the position that it is not patentable? If innovations of this importance are not patent eligible then why would anyone spend the amount of time and money necessary to bring them into being? Spending millions, or billions, of dollars makes no sense unless there is an opportunity to more than recoup the investment. Without patents those who spend research and development money will be handing over the invention to those who free-ride and then commercialize and distribute for less because they don’t have to recoup the research and development money.
So the next time you speak with some “enlightened” anti-patent advocate you might want to ask them what their position is on the cure for cancer. In my opinion the cure for cancer is something that we ought to be pursuing, and placing road blocks in the path of innovation at the front door by saying certain innovations are patent ineligible is ridiculous.
The anti-patent zealots will be remembered in history in the same way that we today view those who persecuted the likes of Galileo. Standing in the way of scientific advance and innovation is regressive, shortsighted and irresponsible. Only those with a special phobia against science and innovation, as well as a healthy disregard for reality, would ever stand in the way of the march of health related innovations.