Bradley J. Fikes Oct. 3, 2015
http://www.sandiegouniontribune.com/news/2015/oct/03/stem-cell-meeting-mesa-2016/
It’s been a long wait for patients hoping for stem cell science to be turned into new treatments. After years of frustration at the incremental progress, stem cell therapies at last are streaming into clinical trials -- in San Diego, nationwide and across the world.
In early October, leaders in this transformation will give a progress report and look ahead in the biotech hub of Torrey Pines Mesa, a major center of stem cell research.
The annual Stem Cell Meeting on the Mesa provides a three-day look at regenerative medicine from the scientific and business perspectives. And as in recent meetings, momentum on the business side continues to accelerate. Pharmaceutical companies and biotech investors who had previously dismissed the sector as unready are marching in.
Highlights include a progress report from UC San Diego’s new Sanford Stem Cell Clinical Center, which is testing three stem cell therapies. Gene therapy for cancer and other diseases has turned white-hot with a boost from stem cell technology. And the public is invited to a forum on whether regenerative medicine is at last fulfilling its promise.
The meeting takes place amid a trend of converging fields that promise to give stem cell therapy a huge boost in the next decade, said veteran biomedical industry observer John McCamant.
This marriage of technologies will make it easier to create more durable matches of transplanted tissue derived from stem cells with patients, he said.
“It’s like we had all these pieces, and now they’re coming together,” said McCamant, who publishes the Berkeley-based Medical Technology Stock Letter.
Most important is the nascent ability to selectively turn down the immune system to prevent rejection or amp it up to fight cancer.
Immune matching is the last hurdle to overcome for successful stem cell therapy. Scientists are getting more proficient at maturing stem cells into the kinds of functional tissue that could, for example, relieve symptoms of Parkinson’s disease or repair spinal cord injury. But if the immune system rejects it, the transplant won’t work.
To minimize immune rejection, the cells would be grown from the patient to be treated, or autologous. But for cost reasons, most of these programs use stem cells taken from other people, called allogeneic.
Taming immune response
The allogeneic tissue can function normally and repair damage, but if the body’s immune system rejects it, the therapy won’t work. Suppressing the immune system to prevent rejection exposes the patient to the risk of infections. And that’s where advances in fine-tuning the immune system will greatly benefit regenerative medicine, McCamant said.
This progress is now taking place in treating graft vs. host disease, a complication of an immune mismatch between transplanted organs and the patients who receive them. There is no cure. It’s usually treated with immune-suppressing steroids, which often don’t work. Experimental treatments promise to change that bleak picture for the better.
“These are potentially taking us into hundreds of thousands of transplants, as opposed to just the few we use today,” McCamant said.
This is already happening. In 2012, Osiris Therapeutics received Canadian approval of Prochymal, a stem cell product to control the disease in children that’s in U.S. testing. Prochymal contains mesenchymal stem cells from adult donors. These cells repair damage and turn down the immune response to the transplant.
The former CEO of Osiris Therapeutics, C. Randal Mills, now leads California’s stem cell program, the California Institute for Regenerative Medicine. CIRM is one of the meeting’s organizers, and Mills will introduce two panels.
For cancer, turning up the immune response is what’s wanted. New experimental therapies have shown strong preliminary indications of success. Patients with resistant blood cancers have responded to therapy with immune cells called T cells that have been genetically modified to recognize the cancer cells as foreign. This CAR T cell therapy, which is sometimes used with stem cell transplants, is also being explored for solid tumors.
Selectively modulating the immune response is directly applicable to transplants of tissues grown from stem cells, McCamant said. It will make it feasible for replacement tissues to be grown in large quantities and supplied “off the shelf” as needed.
Home grown, nationwide reach
Stem Cell Meeting on the Mesa started 10 years ago as a one-day purely local event, said Larry Goldstein, who leads UC San Diego’s Sanford Stem Cell Clinical Center. But the region’s expertise attracted scientists from outside, and business interest. Five years ago the event expanded to add a business-focused partnership forum.
Today, scientists and business leaders come from around the country and elsewhere, said Goldstein and Morrie Ruffin, managing director of the Alliance for Regenerative Medicine. The event is organized by the Sanford Consortium for Regenerative Medicine in La Jolla; along with the California Institute for Regenerative Medicine, the state’s stem cell agency; and the Alliance for Regenerative Medicine, based in Washington, D.C.
“This has become known in the sector as the ‘Mesa meeting,’ which is now recognized as a major event in the regenerative medicine, cell and gene therapy space,” Ruffin said.
But it’s still in many ways a local meeting, Goldstein said.
“So we’ll talk about the clinical trials going on at the Sanford Stem Cell Clinical Center, and there will be sessions on some of the basic science that’s going on around here.”
The clinical center is conducting four trials of experimental therapies derived from stem cell research. They can be found at j:mp/sccctrials.
One uses a monoclonal antibody drug to attack cancer stem cells that cause chronic lymphocytic leukemia. The drug was developed with funding from the California Institute for Regenerative Medicine, and named cirmtuzumab in tribute. Another trial treats chronic heart failure; the two others treat spinal cord injury and type 1 diabetes.
All but the heart failure trial are safety studies. That trial, in Phase 3, uses precursor cells derived from bone marrow injected into the damaged heart. The cells release growth chemicals expected to cause regeneration of heart muscle and reduce scarring.
Along with the scientific talks and partnership presentations from biotech companies, the meeting includes keynote speeches from researchers of unusual interest, Goldstein said. One will be delivered by a UC Los Angeles researcher who will discuss treating Severe Combined Immune Deficiency, or “bubble boy” disease with genetic editing technology.
Basic science will be well-represented at the scientific symposium, held on Friday. It includes presentations on growing neurons; genetically engineering blood cells to fight cancer and other diseases, and bioengineering for regenerating the heart.
Asked to name his favorite part of the meeting, Goldstein said there was no one thing.
“I like it all, he said. “I’m curious about everything.”
Business advances
Stem cell therapy has in recent years converged with gene therapy, the field of correcting genetic defects. Gene therapy will be more prominent this year than in previous meetings, Ruffin said.
The first gene therapy product approved was Glybera. Made by the Dutch company UniQure, Glybera is approved in Europe to treat exceptionally severe cases of familial lipoprotein lipase deficiency, an enzyme disease that impairs fat digestion. These patients suffer from severe pancreatitis despite adhering to a fat-restricted diet.
Glybera carries a gene that provides a more powerful version of the enzyme, delivered through a non-replicating virus injected into muscle tissue in the legs.
Jörn Aldag, uniQure’s CEO, will discuss Glybera at the meeting.
Such progress is reflected in the increased attendance at the business side of the meeting, said Don Gibbons, a spokesman for CIRM and head of the education and communications committee for the Alliance for Regenerative Medicine.
When the business sessions were added five years ago, it was probably too early, Gibbons said.
“The technologies probably weren’t quite ripe enough to truly interest big pharma or VCs (venture capitalists) in taking them to the next stage,” Gibbons said. “But we knew that we were almost there. By the second year, the partnering forum had some robustness to it, and in the last couple of years, the partnering forum has really been exciting.
“If you look at the program for this year, it’s really a maturing technology that’s getting itself ready for commercialized products,” he said.
As as sign of that maturation, a fair number of the business sections are less than exciting, Gibbons said. The starry-eyed vision of scientists and patients has been reinforced with more prosaic matters such as manufacturing.
“These talk about technology and process issues breakthroughs that you need to address if you’re going scale things up,” he said. “And so they’re kind of mundane and boring, but they’re the things you need to address.”
http://www.sandiegouniontribune.com/news/2015/oct/03/stem-cell-meeting-mesa-2016/
It’s been a long wait for patients hoping for stem cell science to be turned into new treatments. After years of frustration at the incremental progress, stem cell therapies at last are streaming into clinical trials -- in San Diego, nationwide and across the world.
In early October, leaders in this transformation will give a progress report and look ahead in the biotech hub of Torrey Pines Mesa, a major center of stem cell research.
The annual Stem Cell Meeting on the Mesa provides a three-day look at regenerative medicine from the scientific and business perspectives. And as in recent meetings, momentum on the business side continues to accelerate. Pharmaceutical companies and biotech investors who had previously dismissed the sector as unready are marching in.
Highlights include a progress report from UC San Diego’s new Sanford Stem Cell Clinical Center, which is testing three stem cell therapies. Gene therapy for cancer and other diseases has turned white-hot with a boost from stem cell technology. And the public is invited to a forum on whether regenerative medicine is at last fulfilling its promise.
The meeting takes place amid a trend of converging fields that promise to give stem cell therapy a huge boost in the next decade, said veteran biomedical industry observer John McCamant.
This marriage of technologies will make it easier to create more durable matches of transplanted tissue derived from stem cells with patients, he said.
“It’s like we had all these pieces, and now they’re coming together,” said McCamant, who publishes the Berkeley-based Medical Technology Stock Letter.
Most important is the nascent ability to selectively turn down the immune system to prevent rejection or amp it up to fight cancer.
Immune matching is the last hurdle to overcome for successful stem cell therapy. Scientists are getting more proficient at maturing stem cells into the kinds of functional tissue that could, for example, relieve symptoms of Parkinson’s disease or repair spinal cord injury. But if the immune system rejects it, the transplant won’t work.
To minimize immune rejection, the cells would be grown from the patient to be treated, or autologous. But for cost reasons, most of these programs use stem cells taken from other people, called allogeneic.
Taming immune response
The allogeneic tissue can function normally and repair damage, but if the body’s immune system rejects it, the therapy won’t work. Suppressing the immune system to prevent rejection exposes the patient to the risk of infections. And that’s where advances in fine-tuning the immune system will greatly benefit regenerative medicine, McCamant said.
This progress is now taking place in treating graft vs. host disease, a complication of an immune mismatch between transplanted organs and the patients who receive them. There is no cure. It’s usually treated with immune-suppressing steroids, which often don’t work. Experimental treatments promise to change that bleak picture for the better.
“These are potentially taking us into hundreds of thousands of transplants, as opposed to just the few we use today,” McCamant said.
This is already happening. In 2012, Osiris Therapeutics received Canadian approval of Prochymal, a stem cell product to control the disease in children that’s in U.S. testing. Prochymal contains mesenchymal stem cells from adult donors. These cells repair damage and turn down the immune response to the transplant.
The former CEO of Osiris Therapeutics, C. Randal Mills, now leads California’s stem cell program, the California Institute for Regenerative Medicine. CIRM is one of the meeting’s organizers, and Mills will introduce two panels.
For cancer, turning up the immune response is what’s wanted. New experimental therapies have shown strong preliminary indications of success. Patients with resistant blood cancers have responded to therapy with immune cells called T cells that have been genetically modified to recognize the cancer cells as foreign. This CAR T cell therapy, which is sometimes used with stem cell transplants, is also being explored for solid tumors.
Selectively modulating the immune response is directly applicable to transplants of tissues grown from stem cells, McCamant said. It will make it feasible for replacement tissues to be grown in large quantities and supplied “off the shelf” as needed.
Home grown, nationwide reach
Stem Cell Meeting on the Mesa started 10 years ago as a one-day purely local event, said Larry Goldstein, who leads UC San Diego’s Sanford Stem Cell Clinical Center. But the region’s expertise attracted scientists from outside, and business interest. Five years ago the event expanded to add a business-focused partnership forum.
Today, scientists and business leaders come from around the country and elsewhere, said Goldstein and Morrie Ruffin, managing director of the Alliance for Regenerative Medicine. The event is organized by the Sanford Consortium for Regenerative Medicine in La Jolla; along with the California Institute for Regenerative Medicine, the state’s stem cell agency; and the Alliance for Regenerative Medicine, based in Washington, D.C.
“This has become known in the sector as the ‘Mesa meeting,’ which is now recognized as a major event in the regenerative medicine, cell and gene therapy space,” Ruffin said.
But it’s still in many ways a local meeting, Goldstein said.
“So we’ll talk about the clinical trials going on at the Sanford Stem Cell Clinical Center, and there will be sessions on some of the basic science that’s going on around here.”
The clinical center is conducting four trials of experimental therapies derived from stem cell research. They can be found at j:mp/sccctrials.
One uses a monoclonal antibody drug to attack cancer stem cells that cause chronic lymphocytic leukemia. The drug was developed with funding from the California Institute for Regenerative Medicine, and named cirmtuzumab in tribute. Another trial treats chronic heart failure; the two others treat spinal cord injury and type 1 diabetes.
All but the heart failure trial are safety studies. That trial, in Phase 3, uses precursor cells derived from bone marrow injected into the damaged heart. The cells release growth chemicals expected to cause regeneration of heart muscle and reduce scarring.
Along with the scientific talks and partnership presentations from biotech companies, the meeting includes keynote speeches from researchers of unusual interest, Goldstein said. One will be delivered by a UC Los Angeles researcher who will discuss treating Severe Combined Immune Deficiency, or “bubble boy” disease with genetic editing technology.
Basic science will be well-represented at the scientific symposium, held on Friday. It includes presentations on growing neurons; genetically engineering blood cells to fight cancer and other diseases, and bioengineering for regenerating the heart.
Asked to name his favorite part of the meeting, Goldstein said there was no one thing.
“I like it all, he said. “I’m curious about everything.”
Business advances
Stem cell therapy has in recent years converged with gene therapy, the field of correcting genetic defects. Gene therapy will be more prominent this year than in previous meetings, Ruffin said.
The first gene therapy product approved was Glybera. Made by the Dutch company UniQure, Glybera is approved in Europe to treat exceptionally severe cases of familial lipoprotein lipase deficiency, an enzyme disease that impairs fat digestion. These patients suffer from severe pancreatitis despite adhering to a fat-restricted diet.
Glybera carries a gene that provides a more powerful version of the enzyme, delivered through a non-replicating virus injected into muscle tissue in the legs.
Jörn Aldag, uniQure’s CEO, will discuss Glybera at the meeting.
Such progress is reflected in the increased attendance at the business side of the meeting, said Don Gibbons, a spokesman for CIRM and head of the education and communications committee for the Alliance for Regenerative Medicine.
When the business sessions were added five years ago, it was probably too early, Gibbons said.
“The technologies probably weren’t quite ripe enough to truly interest big pharma or VCs (venture capitalists) in taking them to the next stage,” Gibbons said. “But we knew that we were almost there. By the second year, the partnering forum had some robustness to it, and in the last couple of years, the partnering forum has really been exciting.
“If you look at the program for this year, it’s really a maturing technology that’s getting itself ready for commercialized products,” he said.
As as sign of that maturation, a fair number of the business sections are less than exciting, Gibbons said. The starry-eyed vision of scientists and patients has been reinforced with more prosaic matters such as manufacturing.
“These talk about technology and process issues breakthroughs that you need to address if you’re going scale things up,” he said. “And so they’re kind of mundane and boring, but they’re the things you need to address.”