Monday, November 12, 2018

Combating Stem Cell Snake Oil: A Primer From a California Researcher

Hundreds of dubious, unregulated stem cell clinics exist throughout the country with the most in California. Desperate people seeking help have been maltreated and fleeced. What to do?

UC Davis stem cell research Paul Knoepfler has produced a useful guide to reporting dubious
activities along with a list of state and federal agencies that could have a role.

In an item on his blog last week, Knoepfler wrote,
"I would emphasize concrete reasons for concern such as the use of an unapproved stem cell drug product by the clinic, a physician practicing outside their area of expertise so putting their patients at risk, false marketing, and potential or documented (if they’ve already happened) patient harms.

"For some clinics that aren’t led by physicians, I would also emphasize the risks of non-physicians such as chiropractors or Ph.D.s doing procedures for which they aren’t trained or licensed. I think patients (or people communicating on their behalf) making complaints about clinics and their personnel will have the greatest impact."
Knoepfler continued,
"We can make a difference by pushing back on the worst clinics. Of course, not every action by those of us in the stem cell arena who are concerned about predatory clinics will hit a bullseye to make real change, but sometimes it has happened in the past and will happen again in the future too."
I would add that the activities and stories that emerge from these dubious enterprises damage the reputation of the field as a whole. Indeed, when I talk to folks in the general public, the clinics' offerings are the most often mentioned reference they have.

As California's stem cell agency moves closer to seeking more billions from voters in 2020, fraudulent stem cell activity could create much confusion about the legitimacy of the entire field. Supporters might want to keep that in mind. But critics should as well. Fleecing the public with unproven therapies is unhealthy for those who are swindled but also for society in general.
Sphere: Related Content

Thursday, November 08, 2018

California's Stem Cell Agency Opening Door to Pumping More Millions into the Sizzling Gene Therapy Market

Gene therapy graphic from FDA
The California stem cell agency today crossed a key threshold into the "somewhat breathtaking" and potentially multibillion-dollar world of gene therapy, a field that it has skirted previously. 

The action came when a key panel of the agency's governing board this morning approved its first-ever procedure for awarding millions of dollars for research not connected to stem cells. 

"This is where the (regenerative) science is going," said Jeff Sheehy, chair of the Science Subcommittee of the board of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known. 

Under Prop. 71, the ballot initiative that created the $3 billion agency in 2004, CIRM is limited to financing research involving stem cells in some fashion. However, a provision of the measure also allows the support of a "vital research oppportunity" under certain conditions. Today's action formalizes the process for making that finding. 

CIRM said in in a document that it is "vital" that the agency expand its horizons.
"CIRM has supported projects that combine stem cell and gene therapy technologies, such as the gene-corrected stem cell transplants at UCLA that essentially cured 5-year old Evangelina Padilla Vaccaro and several CAR-T cell approaches using stem memory T cells that aim to tackle various cancers. 
"The support of stem cell research that contributes to these treatments is and will continue to be the core of CIRM funding. However, treatment opportunities in regenerative medicine that utilize gene therapy technologies but not necessarily stem cells are also valuable and worthy of pursuit."
The agency's statement continued,
"The field of regenerative medicine brings together technologies that include stem cells, gene therapy, and tissue engineering that in many cases combine to produce a therapeutic product. In some cases, one technology leads the way. For CIRM and the patients it aims to serve, it is vital to support technologies which prove to be highly complementary and augmenting to stem cells, such as gene therapy."
Several directors noted during today's meeting that the change could lead to less cash for purely stem cell projects. 

The field of gene therapy has attracted widespread interest within the regenerative medicine industry and among federal medical regulators. Scott Gottlieb, commissioner of the Food and Drug Administration, earlier this year announced steps to speed development of the therapies. 

"'The pace of progress in gene therapy has been somewhat breathtaking,' he (Gottlieb) said, with more than 500 experimental drugs now in development. 'The promise is becoming very much a reality.'"
In April, Novartis AG ponied up $8.7 billion to buy AveXis Inc., of Illinois to strength its efforts to produce a marketable gene therapy.

Gottlieb has said that he expects the FDA to approve 40 gene therapies by 2022 and possibly a cure for sickle cell anemia within 10 years. 

CIRM's new gene therapy policy will go before the full CIRM board one week from today where it is expected to be approved. Additional information on the policy can be found on the Science Subcommittee agenda.  Sphere: Related Content

Tuesday, November 06, 2018

An FDA High Sign for a California Stem Cell Agency Bet: San Diego Biotech Business Earns FDA Speed-Up Approval

A San Diego firm backed by nearly $24 million from the California stem cell agency scored this week with a special designation from the federal government that could help speed approval of its therapy for a type of blood cancer. 

The firm is Poseida Therapeutics, Inc., which is testing the safety of a therapy for a type of blood cancer in a phase one clinical trial. 

The firm announced yesterday that the Food and Drug Administration (FDA) had granted Regenerative Medicine Advanced Therapy (RMAT) status for the adult stem cell-connected treatment. The designation is intended to expedite development of the multiple myeloma therapy. 

Eric Ostertag, CEO of the firm, said in a news release, that Poseida's potential treatment, P-BCMA-101, "is the first anti-BCMA CAR-T therapy to receive RMAT designation from the FDA and underscores the urgent need for new treatment options for multiple myeloma." 

He continued, 
“Initial Phase 1 data presented at the CAR-TCR Summit earlier this year included encouraging response rates and safety data, including meaningful responses in a heavily pretreated population...."
Ostertag said the firm expects to have more data by the end of the year. 

The stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), has invested in 49 clinical trials. Five have garnered RMAT designation, which can significantly speed development of a commercial product. As of September, the FDA had granted only 24 RMAT designations nationally. 

Last Sepetember, Geoff Lomax, CIRM senior officer for medical affairs and strategic centers, wrote in cell&gene that the two-year-old, RMAT program streamlines therapy development by enabling possible priority FDA review and accelerated federal approval. 

Longitude Capital of Menlo Park, Ca., is a major investor in the firm, pumping in tens of millions of dollars.   Sphere: Related Content

Thursday, November 01, 2018

California Stem Cell Agency Moving to Expand its Reach into Big Market for Gene Therapy

The definition of gene therapy under proposed changes for
 research funding by the California stem cell agency. CIRM chart
California's $3 billion stem cell research program is set to add gene therapy -- minus stem cells -- as research that it will be able to legally finance. 

The change comes as the stem cell agency is looking to generate results that are likely to resonate with voters in November 2020 who may be asked to provide an additional $5 billion in funding for the program. The agency expects to run out of cash by the end of next year. 

Gene therapy has received considerable attention in the last few years. Yesterday, Orchard 
Therapeutics, a British gene therapy firm that has links to CIRM (also see here) and research by Donald Kohn of UCLA, raised $200 million in an initial stock offering. The company said in its prospectus that the total market potential "in the diseases areas underlying our five lead programs could be greater than $2 billion annually."

Donald Kohn, UCLA photo
Kohn has received nearly $31 million in backing from the stem cell agency, which is formally known as the California Institute for Regenerative Medicine (CIRM).

The Science Subcommittee of the agency is expected to approve extension of its gene therapy efforts next Wednesday in a teleconference meeting that will be available globally through the Internet. 

Under the provisions of the ballot measure that created the agency in 2004, the agency is limited in scope. But exceptions are possible if a finding is made that a "vital research opportunity" exists. 

Next week's meeting is expected to formalize the process of making that determination in regards to gene therapy that does not involve stem cells. It will require a 2/3 vote of CIRM's grant review group, among other things. 

Members of the public can participate in the hearing remotely via the Internet or at locations in Oakland, San Francisco, La Jolla, Riverside and Napa. Directions can be found on the agenda. Sphere: Related Content