In late July, the US FDA accepted a Biologics License Application (BLA) from Mesoblast Limited. The BLA was resubmitted for the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD).
If approved, Mesoblast’s “RYONCIL” will be the first allogenic, off-the-shelf cellular medicine in the US, as well as the first cell therapy for children up to 18 years old with the condition.
Mesoblast CEO, Dr. Silviu Itescu said, “We are pleased that FDA has accepted our BLA resubmission for review and look forward to the potential approval of RYONCIL for children with SR-aGVHD.”
Of the application’s acceptance, Dr. Wee Kiat Tan, co-CEO of CytoMed Therapeutics (NASDAQ: GDTC), a pre-clinical biotechnology company focusing on developing off-the-shelf, cell-based immunotherapy cancer treatments, said…
“This is a great advancement for allogeneic cell based therapy as once the approval is obtained, this means that the regulatory and approval pathway has been trailblazed.
“All other allogeneic cell based therapy, including CytoMed’s allogeneic gamma delta T cell cancer therapy can follow this regulatory pathway to bring potentially affordable cell therapy to the masses.”
The FDA has considered Mesoblast’s resubmission to be a complete response to prior inquiries arising from its last submission, and Mesoblast anticipates a decision on or before the FDA’s Prescription Drug User Fee Act (PDUFA) goal date of January 7, 2025.
Read the full article, HERE
Or watch CytoMed Therapeutics’ (NASDAQ:GDTC) latest investor presentation, HERE
Clinical Trials Arena, a leading source of data journalism on clinical trials and operations, has said clinical trials for chimeric antigen receptor (CAR)-T therapies are expected to reach all-time highs this year.
According to the publication, clinical trials of CAR-T therapies last peaked in 2021 at 272, with GlobalData’s Clinical Trials Database now showing, that before the end of 2024, clinical trials for CAR-T therapies will surpass that previous one-year record high.
While the US Food and Drug Administration (FDA) has already approved CAR-T therapies from two companies, Novartis and Kite Pharma, insurance companies and government backed insurance may still be reluctant to cover CAR-T therapy treatment.
And the reason being, the costs per patient can reach millions of dollars.
However promising and/or effective the already FDA approved therapies may be, unless the costs associated with creating and administering these therapies fall drastically, the pool of cancer patients who can afford treatment (and insurers who cover it) may continue to remain relatively small.
However, CytoMed Therapeutics (NASDAQ: GDTC), a pre-clinical CAR-T cell therapy company may have a novel, off-the-shelf approach that could, in fact, bring the costs down significantly.
Dr. Wee Kiat Tan, co-CEO of CytoMed Therapeutics said of the company’s technology, “Unlike most CAR-T therapies now which are personalized, CytoMed’s allogeneic, “off-the-shelf” gamma delta T cell therapy can be used for patients without need to match donor and recipient…
“Hence production of drug can be scaled up greatly to bring down cost. Furthermore, the therapy can potentially target many different indications, and can be used by patients of different cancers, further enabling access to the masses.”
Additionally, Dr. Tan says, “CytoMed hopes to begin its own clinical trials later this year, by winter 2024.”
Read more of the Clinical Trials Arena article, HERE
Or, see CytoMed Therapeutics’ latest investor presentation, HERE
Back in November of 2023, the US Food and Drug Association (FDA) issued a warning about the potential risks of secondary cancer occurrences, particularly blood cancers, that may be associated in patients who have undergone CAR-T cell therapies.
Its warning read, “FDA Investigating Serious Risk of T-cell Malignancy Following BCMA-Directed or CD19-Directed Autologous Chimeric Antigen Receptor (CAR) T cell Immunotherapies.”
However, a recent, and large study conducted by Stanford Medicine, at Stanford Health Care, has shown the risk of secondary cancers associated with CAR-T cell therapies may, in fact, be quite low.
The study of over 700 patients revealed that the risk of secondary cancers, after three years of therapy, is about 6.5%. Further, the only fatal secondary cancer researchers found was in a patient that likely suffered immunosuppression caused by the therapy, but not by the CAR-T cells themselves.
In this case, according to Stanford Medicine, “The compromised immune systems allowed pre-existing, but not previously detected, cancer cells to grow explosively in the patient.”
Of the roughly 6.5% of patients who did have a secondary occurrence of cancer, analysis found there was no evidence that T cells responsible for the secondary cancer were those engineered for the CAR-T cell therapy.
Dr. Wee Kiat Tan, co-CEO of CytoMed Therapeutics (NASDAQ: GDTC), said of the Stanford study, “The long term follow-up study of patient who received CAR-T treatment has shown that it is relatively safe long term, and has not led to very significant cancer risk.”
Further, Dr. Tan said, “CytoMed employs non-viral, non-gene integrating methods to equip our gamma delta T cells with a CAR. Since there is no gene integration, the genome is unchanged which further decrease the chances of secondary cancers.”
Read more about the Stanford Medicine study, HERE
Or, see CytoMed Therapeutics’ latest investor presentation, HERE