Well, it's early days for CRISPR and the other gene therapies. Those of us who lived through the go-go days of biopharma remember what happened to the much-vaunted monoclonal antibody therapies. More heat than light.
Marton, I meant there were many stumbles before MoA therapies became viable. Lots of companies went bankrupt trying to prove the technology. I think it will be a similar trajectory for gene therapies. 35 years ago, first FDA approval...years of research before that. Colour me gently skeptical.
I remember, years ago, listening to a lecture from Novartis about Glivec. It seems that it was intended to help prolong life but in some circumstances it actually cured the person.
The lecturer (involved in the development of the drug) cried.
People go on about "big pharma" as though it's this monoloth, forgetting that it is made up of people who are just as likely to get cancer as any of us.
A lot of the initial research that leads to product development, clinical trials and release of a usable drug is actually carried out by university labs, sometimes funded by big pharma but often funded by charities, especially in the U.K.
"Those of us who lived through the go-go days of biopharma remember what happened to the much-vaunted monoclonal antibody therapies. More heat than light"
I'm extremely grateful for the development of monoclonal antibodies - because of Herceptin I've lived for 16 years beyond a diagnosis of aggressive breast cancer.
Cherub, that's the happy end-point of a difficult history. The first MoAb companies went bankrupt. The technology was almost shelved because of all the problems.
From NIH:
The first licenced monoclonal antibody was Orthoclone OKT3 (muromonab-CD3) which was approved in 1986 for use in preventing kidney transplant rejection [7]. It is a monoclonal mouse IgG2a antibody whose cognate antigen is CD3. It works by binding to and blocking the effects of CD3 expressed on T-lymphocytes. However, its use was limited to acute cases due to reported side-effects (e.g. human anti-mouse antibody response) [8]. This is representative of the relative lack of early clinical and commercial success of monoclonal antibodies. A major stumbling block was the fact that the production of early monoclonal antibodies was limited by whether or not there was a suitable myeloma cell line available (usually mouse or rat).
Just sayin'...1975 (first MoAb) to approval took a looong time. I expect other genetic tinkering (like CRISPR) to have a similar trajectory.
“CRISPR is becoming a mainstream methodology used in many cancer biology studies because of the convenience of the technique,” said Jerry Li, M.D., Ph.D., of NCI’s Division of Cancer Biology.
Bossybaby, that info is really interesting, I had no idea Monoclonal Antibody development went as far back as the the mid 80s, I thought it was more of a 90s thing. At the of my diagnosis a relative of mine who was a Professor and Medical Director of a cancer centre in Scotland put me on to a number of studies to read on Herceptin. I'm the sort of person who likes to be informed about things and it took the fear of what I was facing up to away.
I had horrendous side effects from the chemo drug Taxotere but knew I had to keep going with it - other ladies I knew who were experiencing the same refused their last cycle.