This spotlight
on cell therapy regulation will aim to provide insight into challenges, trends, developments and debates in the regulation of cell therapies aimed at regenerative medicine applications.
Heidi Simmons decided to focus
on cell therapy for treatment of blood cancers like leukemia, lymphoma and myeloma, where healthy cells are infused into patients to replenish those damaged by cancer.
Today, at 56, Atala oversees 300 researchers and support personnel, including chemists, biologists and engineers divided into different teams working
on cell therapy, a technology for what he calls partial transplants and the creation of new organs.
A concentrated source of information
on cell therapies spanning the different stages of development: including research, pre-clinical, clinical and marketed phases, being pursued by both non-profit organizations (NPO) and industries.
Not exact matches
Japanese pharma giant Takeda will buy TiGenix, a Belgian biotech focused
on stem
cell therapies, for $ 630 million in cash.
The Food and Drug Administration (FDA)
on Tuesday gave Swiss drug giant Novartis a second approval for its pioneering CAR - T cancer
therapy, which uses patients» own immune
cells (re-engineered outside the body and then replicated) to destroy blood cancers.
• Tessa Therapeutics, a Singapore - based biopharmaceutical company focusing
on T
cell therapy for solid tumors, raised $ 80 million in funding.
On the logistics end, it is easier to manufacture certain stem -
cell therapies, which will be key for human trials like the heart - muscle - regeneration program.
I admire SQZ Biotech's unique platform — they're
on the forefront of something huge that has the potential to revolutionize disease research and
cell therapy.
Basically, CAR - T
therapy involves taking a patient's own immune «killer» T -
cells, inserting new genetic code into those
cells which turn them into cancer - hunters that can home in
on malignant B -
cells (another kind of immune
cell), and then pumping these specialized leukemia - busting
cells back into the patient.
The treatment is a type of so - called CAR T -
cell therapy — taking a patient's own immune
cells, called T
cells, genetically manipulating them to attack specific proteins
on cancer, and infusing them back into the patient.
On Wednesday, the U.S. Food and Drug Administration approved Novartis» Kymriah, the first drug for a new kind of cancer treatment called CAR - T
cell therapy.
One of the top contributors to the gain was bluebird bio, which is also working
on CAR - T
cell therapy.
His research has spanned hematopoiesis, gene
therapy, stem
cell biology, genomics and cancer, consistently focusing
on bringing the very latest research advances to patients with heretofore incurable diseases.
These risks and uncertainties include: Gilead's ability to achieve its anticipated full year 2018 financial results; Gilead's ability to sustain growth in revenues for its antiviral and other programs; the risk that private and public payers may be reluctant to provide, or continue to provide, coverage or reimbursement for new products, including Vosevi, Yescarta, Epclusa, Harvoni, Genvoya, Odefsey, Descovy, Biktarvy and Vemlidy ®; austerity measures in European countries that may increase the amount of discount required
on Gilead's products; an increase in discounts, chargebacks and rebates due to ongoing contracts and future negotiations with commercial and government payers; a larger than anticipated shift in payer mix to more highly discounted payer segments and geographic regions and decreases in treatment duration; availability of funding for state AIDS Drug Assistance Programs (ADAPs); continued fluctuations in ADAP purchases driven by federal and state grant cycles which may not mirror patient demand and may cause fluctuations in Gilead's earnings; market share and price erosion caused by the introduction of generic versions of Viread and Truvada, an uncertain global macroeconomic environment; and potential amendments to the Affordable Care Act or other government action that could have the effect of lowering prices or reducing the number of insured patients; the possibility of unfavorable results from clinical trials involving investigational compounds; Gilead's ability to initiate clinical trials in its currently anticipated timeframes; the levels of inventory held by wholesalers and retailers which may cause fluctuations in Gilead's earnings; Kite's ability to develop and commercialize
cell therapies utilizing the zinc finger nuclease technology platform and realize the benefits of the Sangamo partnership; Gilead's ability to submit new drug applications for new product candidates in the timelines currently anticipated; Gilead's ability to receive regulatory approvals in a timely manner or at all, for new and current products, including Biktarvy; Gilead's ability to successfully commercialize its products, including Biktarvy; the risk that physicians and patients may not see advantages of these products over other
therapies and may therefore be reluctant to prescribe the products; Gilead's ability to successfully develop its hematology / oncology and inflammation / respiratory programs; safety and efficacy data from clinical studies may not warrant further development of Gilead's product candidates, including GS - 9620 and Yescarta in combination with Pfizer's utomilumab; Gilead's ability to pay dividends or complete its share repurchase program due to changes in its stock price, corporate or other market conditions; fluctuations in the foreign exchange rate of the U.S. dollar that may cause an unfavorable foreign currency exchange impact
on Gilead's future revenues and pre-tax earnings; and other risks identified from time to time in Gilead's reports filed with the U.S. Securities and Exchange Commission (the SEC).
The race to become the first company with a chimeric antigen receptor T -
cell (CAR - T) cancer
therapy on the market has entered its final leg, and Kite Pharma now appears to have a big advantage.
BioNTech, which has around 700 employees at sites in Germany — more than any other unlisted biotech firm in Europe — is also working
on other cancer - fighting technologies, including antibodies,
cell therapies and small molecules.
Medicine by Design brings together more than 100 high - calibre researchers in a globally competitive hub focused
on regenerative medicine and
cell therapy.
Overcoming the immense logistical challenges involved with withdrawing, shipping, modifying, returning, then reinfusing
cells for every patient treated paints a scary question mark
on the future of
cell - based cancer
therapies.
Since PSMA's limited
on normal
cells, the
therapy should hammer away at tumors while letting healthy tissues do their job.
«The event, the fourth of its kind, seeks to raise global awareness and create a forum for collaboration around the wide array of powerful and promising
cell therapies, gene
therapies, and immunotherapies emerging from medical institutions around the world, as well as the impact new technology will have
on humanity and society,» a press release by the Cure Foundation explains (h / t Christian Post).
They rule out selective abortion of defective fetuses, and they focus our attention
on therapies aimed at somatic
cells rather than germ
cells.
Still, scientists are hopeful that someday adult patients will routinely be able to receive
cell therapies based
on cord blood stem
cells.
We are identifying and supporting work
on the technologies of the future: in advanced manufacturing; renewable energy;
cell therapy; interconnected digital; intelligent transport; and next generation computing.
Currently, most cancer
therapy relies
on frontal assaults
on malignant
cells.
A transformative cancer
therapy based
on modified immune
cells has lured doctors, companies, and patients alike, but many are hitting a frustrating roadblock: generating enough of these chimeric antigen receptor (CAR)- T
cells to meet surging demand.
On its own, this immune response had no immediate effect in the fight against the utilized breast tumors, but in combination with the ADC it proved itself effective in attacking cancer
cells in mice, resulting in the complete cure of the majority of mice receiving the combination
therapy.
Seattle Children's Hospital in Washington recently broke ground
on a building that in a few years will triple or quadruple its
cell therapy capacity, now about 10 batches of CAR - T
cells a month.
Geron was bigger and better funded than ACT, and it was the first company to be approved by the US Food and Drug Administration (FDA) to test a
therapy in humans based
on embryonic stem (ES)
cells.
They isolated blood
cells from HIV - positive patients
on antiretroviral
therapy and at different stages of disease progression, as well as
cells from non-infected individuals.
While the combination
therapies block off the principal pathway that cancer
cells use to fuel their growth, the
cells come to bypass this blockade and, like vehicles
on a detour route, make use of additional pathways to continue growing and spreading.
Based
on results of the current study described in a report online June 18 in the journal Cancer
Cell, Johns Hopkins researchers say they are planning a phase I clinical trial to test the paclitaxel - fostamatinib combination
therapy in patients with recurrent advanced ovarian cancer.
«Current
therapies in clinical trials are focused
on targeting genetic changes in tumors and helping to boost one's immune system to fight the cancer
cells.
«Cultural revolution in the study of the gut microbiome: Human gut -
on - a-chip technology used to co-culture gut microbiome, human intestinal
cells could lead to new
therapies for inflammatory bowel diseases.»
Advocates of biomedical research, professional societies, and patient groups had mobilized to plead for a more careful review, warning that this bill's language was so vague it might hinder research
on human stem
cells and new medical
therapies.
The Wyss team believes the ability of the human gut -
on - a-chip to culture the microbiome with human gut
cells also holds promise for the field of precision medicine, where a patient's own
cells and gut microbiota could one day be cultured inside a gut -
on - a-chip for testing different
therapies and identifying an individualized treatment strategy.
Rubinstein said checkpoint
therapies work by cutting the brake cables
on the white bloods
cells that are inherently able to kill tumor
cells.
However, there is still limited data
on the short and long - term effects of administering stem
cell therapy in chronic respiratory disease.
Patients with metastatic non-small
cell lung cancer will always progress after chemotherapy, so most patients go
on to be treated with immunotherapy, a type of
therapy that uses the body's immune system to fight cancer.
All the monkeys had been put
on immunosuppressive
therapy to prevent rejection of the transplanted human
cells.
On 13 April, the Texas Medical Board voted to accept rules intended to give doctors «a reasonable and responsible degree of latitude» in using experimental stem
cell therapies.
«If cancer recurs, it may be because the
therapy didn't hit key mature
cells that take
on stem
cell - like behavior.
«The results show we can now produce the number of
cells needed for human
therapy and get formation of new heart muscle
on a scale that is relevant to improving the function of the human heart,» Laflamme said.
Although no clinical trials involving
therapies derived from iPS
cells are
on the books, researchers are currently testing drugs
on them.
We have the gene and stem -
cell therapies to do it now — if only we dare use them
on unborn babies
Researchers such as geneticist Richard King of the University of Minnesota and
cell biologist Vitali Alexeev of Thomas Jefferson University are working
on gene
therapies or drugs that would fix albinism - causing mutations.
«The next step of progress is to use this knowledge to decipher what potential impact the genetic variants can have
on the health of individuals, when we conduct health - related research,» added Professor Michael Pepper, Unit Director of the South African Medical Research Council's (SAMRC) Stem
Cell Research and
Therapy Unit, Faculty of Health Sciences at the University of Pretoria.
The Sartorius & Science Prize for Regenerative Medicine &
Cell Therapy is an annual prize geared toward researchers focused on basic or translational research that advances regenerative medicine and cell ther
Cell Therapy is an annual prize geared toward researchers focused on basic or translational research that advances regenerative medicine and cell t
Therapy is an annual prize geared toward researchers focused
on basic or translational research that advances regenerative medicine and
cell ther
cell therapytherapy.
It could be a key step
on the road to
therapies which protect beta
cells or encourage them to regenerate.
EMD Serono, Kirschbaum says, «focuses
on the development of targeted cancer
therapies on three therapeutic platforms: targeting the tumor
cell, the tumor environment, and the immune system.»