The Mission of Making Immunotherapy Accessible and Affordable
Gene and cell therapy are taking center stage in the treatment of cancer. Not only do more people with cancer respond to such advanced therapies, a higher percentage are experience complete remission. The excitement is more than justified since, as Carl June, one of the leaders in immunotherapy notes, “potentially everyone can benefit from gene modified T cells and there’s no danger of graft versus host disease because it’s not a transplant from another person.”
But the barriers to making immunotherapy widely available are great. It consists of a very expensive process of taking a patient’s T-Cell, separating them from other cells and then reprogramming them in a highly controlled laboratory. Additionally producing the reprogrammed cells is itself. time-consuming, costly, and prone to any number of potential manufacturing errors.
The average cost of the therapy and its administration can be as much as $500,000. While even at that level, immunotherapies are worth it, the expense and time required will limit access for years to come.
Or maybe not. Celularity, a company founded by Robert Hariri, one of the pioneers in the development of stem cell therapy has been conducting pre-clinical and clinical evaluation of fully allogeneic, off-the-shelf cell therapy product (PNK-007) on hematological cancers and solid tumors.
The results of the research on PNK-007 (to be released at 2019 American Association for Cancer Research (AACR) Annual Meeting being held from March 29 — April 3, 2019 in Atlanta, Georgia
will be first ever clinical findings.
Additionally, two other companies, Celyad and Allogene, are evaluating allogeneic cells in treating cancer. launched Celularity a company with a mission to make regenerative medicine as affordable and as convenient as the polio vaccine.
The ability to use cells from a broad population eliminates on barrier to making immunotherapy widely available. CAR-Ts are expensive and difficult to make because it requires using one’s own cells and then engineering them to avoid an immune response that shuts downs every organ.
Allogeneic cells have also been off-limits because — much like donated bone marrow transplants — cells from another body will be viewed as invaders by our immune system.
Democratizing cell therapy for cancer requires a reliable and safe source of cells that can be mass produced and used off the shelf in any physician’s office. Celularity for example manufactures stem cells from the placenta, which, as Hariri has demonstrated, is the only source of non-programmable stem cells that overcome the immune system’s lethal resistance.
The Celularity study, if successful, will be a major milestone of a quest to for an affordable source of cell therapies, a mission was born in the biohazard bin of Columbia Presbyterian’s delivery room nearly 20 years ago. A neurosurgeon at the time, Hariri was frustrated that he could patch people back up but not make them whole.
He was rooting through blood-smeared smocks, gauze pads and half eaten pizza slices to retrieve a handful of the placentas that were discarded after birth. The day before he had been looking at the ultrasound of his daughter and it struck him that the placenta had developed more rapidly than the embryo. And as a neurosurgeon he saw how the open spine sutures of spina bifida babies were healed without scars just by placing the infant back into the mother’s placenta. What was in the placenta that protected his daughter and made a surgical gash in a yet born infant disappear?
After a few experiments he discovered placental stem cells were an inexhaustible source of healing and protective power. He founded then launched — Anthrogenesis — (which became Celgene Cellular Therapeutics in 2002) to show that placental stem cells were not just the best source of such material, but that it was the best source of therapeutics bar none. As Hariri notes, “We showed that placental cells can be produced in such quantities and consistency that doctors could use them the same way they prescribe small molecules or biologic agents
At Celularity Hariri’s research has consisted of building CAR-Ts and then studying how they stacked up against placental-derived stem cells is delivering cancer-fighting instructions to the immune system. As Hariri told me: “That was the premise behind our searching the placenta for these cells, and it served as the basis for our product development and, ultimately, clinical development, which has shown that allogeneic transplant is accepted without problems.”
Now at Celularity he is poised to manufacture stem cells that as Hariri told me, can be “easily deployed, readily adoptable like a medicine, and could integrate into the existing healthcare system, where practitioners are most comfortable with traditional pharmaceuticals.”
We should remember that when penicillin was first developed, it cost nearly $200,000 (in today’s dollars) to treat one patient. The inability to mass produce the antibiotic was the limiting factor. Similarly, if Celularity and other startups can make cell therapy as ubiquitous as penicillin, it will transform our world in the same way, allowing millions, if not billions, of people, to live longer free from disease and disability.
