StemCell Therapy

As followers of this blog will know I've been blogging about Regenerative Sciences and predicting their eventual run-in with the FDA since my first post in September 2008 (Cell Therapy is Not the Practice of Medicine) and again in February 2009 (Regenexx vs the FDA 2009).  When the FDA finally proceeded with an injunction against RSI in August 2010,I helped spread the news (here).

I've watched the development of the fight between RSI and the FDA with interest.  In September 2001 I posted a rather lengthy commentary about the potential impact of the case (Potential far-reaching implications of the ongoing fight over point-of-care autologous cell therapy.

Since then I have welcomed other bloggers and commentators who are now following and commenting on the case much more closely and frequently than I including @LeighGTurner (on Twitter) and Paul Knoepfler (@PKnoepfler on Twitter and his Knoeplfer Lab Stem Cell Blog).  Recently I enjoyed being interviewed by Paul on the issue of unregulated stem cell activity and touched on the case for his blog.

Consequently I read with interest yesterday's federal court ruling upholding the FDA's injunction against RSI and the immediate commentary from the New Scientist, Stanford's Scope Blog and Knopfler's multiple posts (here and here). As a long-term follower of this case, I've been asked to comment.  Here is my brief reaction:

This is a case that was always destined for the appellate courts regardless of which way the initial court ruled.    The fact the federal court ruled in the FDA's favor certainly now sets the onus on RSI and what is anticipated to be a gamut of intervenors but taking this case to the appellate courts is what the legal team have anticipated and legal arguments designed for all along.

This is just the beginning of what will be a long and interesting battle.  The ruling was nothing more than the granting of an injunction in response to the government's motion for summary judgement.  In granting the injunction the court  agreed with the government's position that it was acting under the authority given it under the Federal Food, Drug, and Cosmetic Act, 21 U.S.C. § 321(g) but it provided little-to-no rationale for its ruling.

The court chose, in its wisdom, not to address the bulk of the RSI's legal arguments which are largely jurisdictional in nature. These are the kinds of arguments which the lower courts prefer be dealt with by appellate courts and frankly the judge did us all a favor by ruling quickly, succinctly and punting the case where we all knew it was inevitably headed.

In my opinion, other than chalking one up in the government's win column there is little to be gleaned from this ruling in terms of how RSI's arguments will be received in appellate court.  The interesting day is yet to come.

In terms of a short-term practical impact, frankly I see very little.  RSI has already ceased distributing Regenexx within the US so there will be little-to-no impact there.  As for the potential impact on other companies or clinics who might be operating on the fringes of FDA regulation within the US, I suspect it will be business as usual.

Most of the clinics/companies offering cell-based treatments/products which are arguably in contravention of FDA regulation are operating under the clear knowledge of what they are doing and where the FDA stands with respect to the treatments/products they offer and yet they persist and continue.


 For the truly fraudulent there is the risk of criminal charges and/or litigation but for those companies or practitioners who are operating in this shade of grey which are not shady (and they do exist), the  risks associated with this practice are barely higher than in the routine practice of medicine. 


In reality, with the exception of the most fraudulent examples, it takes a fair long-time for the FDA to catch up with these folks and there is good money to be made in the interim.  When they get caught, they will stop. If they've recouped their initial investment (which is nominal and the margins are high) there is very little penalty to this course of action.  Perhaps they set up shot elsewhere or simply enjoy the proceeds.  I doubt we will see much of a slow-down of this kind of activity.  Indeed it may strengthen the resolve of those committed to the cause.

In my opinion yesterday's ruling was in interesting and important milestone in a continuing evolution in the debate of how best to regulate the use of cells in treating people but I'm not sure it's the seminal pivot point that some believe.  I suspect we will not see any radical shift in terms of FDA or industry activity until (if then) the appellate courts rule.

Just my two cents....

--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

The California stem cell agency today
enjoyed substantial, if sparse, news coverage of the

“For Melissa Biliardi of Santa
Maria, the (CIRM board) vote symbolizes hope. Her son, James
Birdsall, 32, was diagnosed four years ago with Huntington's disease.
The degenerative brain disorder could prove fatal over the next 10 to
15 years. There is currently no cure or treatment, but with the
grant, UC Davis researchers hope to deliver an effective therapy in
four years.
"'This is the most hope we've ever
had for a cure or treatment,' Biliardi said.”

“California's stem cell funding
agency on Thursday approved nearly $100 million in grants for
research into heart disease, cancer and spinal cord injuries, and to
the cheers of dozens of patients and their supporters, it also
awarded money to rare but devastating diseases with no cure.”

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Directors of the California stem cell agency today referred two additional research applications for $37 million in disease team funding back to reviewers for further consideration.

Both were the subjects of appeals by researchers whose proposals were rejected by grant reviewers.

One was from Timothy Hoey of OncoMed Pharmaceuticals in Redwood City, who sought $20 million.  The other was from Henry Klassen of UC Irvine, who sought $17 million. (See here and here for their appeals.)

The  board began the day by directing staff to come back to the board in early September. But with the large number of grants to be reassessed, it was acknowledged some might not be acted on until the board's meeting in late October.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Directors of the California stem cell
agency approved an additional
$20 million disease team grant today before adjourning their
meeting. The grant brought the disease team round to a total of $151 million.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Directors of the California stem cell
agency today approved $151 million in research awards aimed at
commercializing stem cell research and pushing therapies into
clinical treatment.


Patients and researchers cheered when the action was announced. 


The awards of up to $20 million each
were ratified by CIRM's governing board, which added two to the
six applications approved by reviewers. The original
six totalled $113 million. Directors budgeted $243 million
for today's round.


Five of the applications involving
appeals were sent back by the board for more review.
(See here, here and here.)
They will be considered again in early September or October.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Andy McMahon Harvard photo

The governing board of the California stem cell agency this afternoon approved a $5.7 million grant to lure a Harvard researcher to the University of Southern California in Los Angeles.

The scientist is Andrew McMahon, who serves on the executive committee of the Harvard Stem Cell Institute. CIRM officials said McMahon is ready to begin his work immediately at USC.

McMahon won stellar reviews from CIRM's grant reviewers who said he was an “exceptional scientist and one of the leading young developmental biologists.” Reviewers gave his proposal a score of 90 and, in summary, said,

“Major strengths include the candidate's exceptional productivity and contributions to the fields of mammalian embryology and kidney development, the significance and potential of the research program, the PI's proven leadership capabilities, and the outstanding institutional commitment.”

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

The California stem cell agency today
cleared the way for approval of an $18 million grant to develop a new
cell-based therapy for treatment of ALS.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Two California stem cell firms today
won a reprieve from rejection in their bids for $40 million in
funding from the California stem cell agency.

“We have a fiduciary responsibility
to select the best science.”

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Directors of the California stem cell
agency today deferred action on a $20 million proposal that was
rejected by its grant reviewers and sent it back for more consideration. 

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

California stem cell agency chairman J.T. Thomas has raised the possibility of sending some of the disease team applications back for additional review if the board feels that is necessary to consider new information and resolve scientific disputes. 

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

The California stem cell agency will continue to be funded with short-term borrowing -- commercial paper -- provided through the state treasurer's office, J.T. Thomas, chairman of the agency's governing board, said today. 

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Today's session of the governing board of the $3 billion California stem cell agency has begun. The major item on the agenda is a $243 million grant round that has triggered a record pace for appeals by rejected applicants. At the request of the California Stem Cell Report, the agency has provided the conflict of interest list used by the agency to determine which directors will not be allowed today to vote or participate in the discussion of specific applications.  The list can be found below. Conflict of Interest List  -- CIRM Directors Meeting 7-26-12ound below.  

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

As followers of this blog will know I've been blogging about Regenerative Sciences and predicting their eventual run-in with the FDA since my first post in September 2008 (Cell Therapy is Not the Practice of Medicine) and again in February 2009 (Regenexx vs the FDA 2009).  When the FDA finally proceeded with an injunction against RSI in August 2010,I helped spread the news (here).

I've watched the development of the fight between RSI and the FDA with interest.  In September 2001 I posted a rather lengthy commentary about the potential impact of the case (Potential far-reaching implications of the ongoing fight over point-of-care autologous cell therapy.

Since then I have welcomed other bloggers and commentators who are now following and commenting on the case much more closely and frequently than I including @LeighGTurner (on Twitter) and Paul Knoepfler (@PKnoepfler on Twitter and his Knoeplfer Lab Stem Cell Blog).  Recently I enjoyed being interviewed by Paul on the issue of unregulated stem cell activity and touched on the case for his blog.

Consequently I read with interest yesterday's federal court ruling upholding the FDA's injunction against RSI and the immediate commentary from the New Scientist, Stanford's Scope Blog and Knopfler's multiple posts (here and here). As a long-term follower of this case, I've been asked to comment.  Here is my brief reaction:

This is a case that was always destined for the appellate courts regardless of which way the initial court ruled.    The fact the federal court ruled in the FDA's favor certainly now sets the onus on RSI and what is anticipated to be a gamut of intervenors but taking this case to the appellate courts is what the legal team have anticipated and legal arguments designed for all along.

This is just the beginning of what will be a long and interesting battle.  The ruling was nothing more than the granting of an injunction in response to the government's motion for summary judgement.  In granting the injunction the court  agreed with the government's position that it was acting under the authority given it under the Federal Food, Drug, and Cosmetic Act, 21 U.S.C. § 321(g) but it provided little-to-no rationale for its ruling.

The court chose, in its wisdom, not to address the bulk of the RSI's legal arguments which are largely jurisdictional in nature. These are the kinds of arguments which the lower courts prefer be dealt with by appellate courts and frankly the judge did us all a favor by ruling quickly, succinctly and punting the case where we all knew it was inevitably headed.

In my opinion, other than chalking one up in the government's win column there is little to be gleaned from this ruling in terms of how RSI's arguments will be received in appellate court.  The interesting day is yet to come.

In terms of a short-term practical impact, frankly I see very little.  RSI has already ceased distributing Regenexx within the US so there will be little-to-no impact there.  As for the potential impact on other companies or clinics who might be operating on the fringes of FDA regulation within the US, I suspect it will be business as usual.

Most of the clinics/companies offering cell-based treatments/products which are arguably in contravention of FDA regulation are operating under the clear knowledge of what they are doing and where the FDA stands with respect to the treatments/products they offer and yet they persist and continue.


 For the truly fraudulent there is the risk of criminal charges and/or litigation but for those companies or practitioners who are operating in this shade of grey which are not shady (and they do exist), the  risks associated with this practice are barely higher than in the routine practice of medicine. 


In reality, with the exception of the most fraudulent examples, it takes a fair long-time for the FDA to catch up with these folks and there is good money to be made in the interim.  When they get caught, they will stop. If they've recouped their initial investment (which is nominal and the margins are high) there is very little penalty to this course of action.  Perhaps they set up shot elsewhere or simply enjoy the proceeds.  I doubt we will see much of a slow-down of this kind of activity.  Indeed it may strengthen the resolve of those committed to the cause.

In my opinion yesterday's ruling was in interesting and important milestone in a continuing evolution in the debate of how best to regulate the use of cells in treating people but I'm not sure it's the seminal pivot point that some believe.  I suspect we will not see any radical shift in terms of FDA or industry activity until (if then) the appellate courts rule.

Just my two cents....

--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

The California stem cell agency today
enjoyed substantial, if sparse, news coverage of the

“For Melissa Biliardi of Santa
Maria, the (CIRM board) vote symbolizes hope. Her son, James
Birdsall, 32, was diagnosed four years ago with Huntington's disease.
The degenerative brain disorder could prove fatal over the next 10 to
15 years. There is currently no cure or treatment, but with the
grant, UC Davis researchers hope to deliver an effective therapy in
four years.
"'This is the most hope we've ever
had for a cure or treatment,' Biliardi said.”

“California's stem cell funding
agency on Thursday approved nearly $100 million in grants for
research into heart disease, cancer and spinal cord injuries, and to
the cheers of dozens of patients and their supporters, it also
awarded money to rare but devastating diseases with no cure.”

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Directors of the California stem cell agency today referred two additional research applications for $37 million in disease team funding back to reviewers for further consideration.

Both were the subjects of appeals by researchers whose proposals were rejected by grant reviewers.

One was from Timothy Hoey of OncoMed Pharmaceuticals in Redwood City, who sought $20 million.  The other was from Henry Klassen of UC Irvine, who sought $17 million. (See here and here for their appeals.)

The  board began the day by directing staff to come back to the board in early September. But with the large number of grants to be reassessed, it was acknowledged some might not be acted on until the board's meeting in late October.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Directors of the California stem cell
agency approved an additional
$20 million disease team grant today before adjourning their
meeting. The grant brought the disease team round to a total of $151 million.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Directors of the California stem cell
agency today approved $151 million in research awards aimed at
commercializing stem cell research and pushing therapies into
clinical treatment.


Patients and researchers cheered when the action was announced. 


The awards of up to $20 million each
were ratified by CIRM's governing board, which added two to the
six applications approved by reviewers. The original
six totalled $113 million. Directors budgeted $243 million
for today's round.


Five of the applications involving
appeals were sent back by the board for more review.
(See here, here and here.)
They will be considered again in early September or October.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Andy McMahon Harvard photo

The governing board of the California stem cell agency this afternoon approved a $5.7 million grant to lure a Harvard researcher to the University of Southern California in Los Angeles.

The scientist is Andrew McMahon, who serves on the executive committee of the Harvard Stem Cell Institute. CIRM officials said McMahon is ready to begin his work immediately at USC.

McMahon won stellar reviews from CIRM's grant reviewers who said he was an “exceptional scientist and one of the leading young developmental biologists.” Reviewers gave his proposal a score of 90 and, in summary, said,

“Major strengths include the candidate's exceptional productivity and contributions to the fields of mammalian embryology and kidney development, the significance and potential of the research program, the PI's proven leadership capabilities, and the outstanding institutional commitment.”

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

The California stem cell agency today
cleared the way for approval of an $18 million grant to develop a new
cell-based therapy for treatment of ALS.

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

Two California stem cell firms today
won a reprieve from rejection in their bids for $40 million in
funding from the California stem cell agency.

“We have a fiduciary responsibility
to select the best science.”

Source:
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss