TL;DR:Â MyCART-01 is an investigational CAR-T therapy trial. A presentation in Vapi showcased a response rate approaching 90% based on approximately 25 patients. Site-level information provided for regulatory verification accounts for 39 patients and classifies their latest reported outcomes as 14 deaths, 11 relapses, 13 remissions and one unstated outcome. These figures require independent verification and do not establish that the therapy caused every death. They do, however, demand an urgent reconciliation of the denominator, data cut-off, durability, patient selection and a reported CAR-T reinfusion.
Disclosure and purpose of this post
I am posting anonymously because this matter involves professionally sensitive information and potentially serious consequences for Indian patients.
MyCART-01âCTRI/2024/10/074924âis registered as a Phase I/II, single-arm, open-label, multicentre study of an investigational CAR-T therapy for patients with relapsed or refractory B-cell malignancies.
What was reportedly presented in Vapi
At a presentation held in Vapi, healthcare professionals were reportedly shown MyCART-01 results covering approximately 25 patients.
A response rate approaching 90% was prominently showcased, accompanied by claims relating to improved activation, expansion, durability and resistance to T-cell exhaustion.
The site-level information available to me accounts for 39 patients and presents a substantially more concerning picture.
Site-level outcomes requiring verification
The information provided to me classifies the latest reported status of the patients as follows:
- AIIMS, New Delhi â 9 patients:Â 3 deaths, 3 relapses and 3 remissions.
- Sarvodaya Hospital, Faridabad â 9 patients:Â 3 deaths, 3 relapses and 3 remissions.
- Rajiv Gandhi Cancer Institute, New Delhi â 7 patients:Â 1 death, 3 relapses and 3 remissions.
- Apollo Cancer Centre, Chennai â 1 patient:Â outcome not stated.
- VIMS â 7 patients:Â 4 deaths, 1 relapse and 2 remissions.
- Jaslok Hospital, Mumbai â 3 patients:Â no reported deaths, 1 relapse and 2 remissions.
- Apollo Hospital, Gandhinagar â 3 patients:Â 3 deaths and no reported remissions.
Taken together, this information accounts for 39 patients: 14 deaths, 11 relapses, 13 remissions and one unstated outcome.
Which 25 patients were includedâand what happened to the other 14?
The sponsor must answer:
- Which exact 25 patients were included in the Vapi presentation?
- Why were the remaining 14 patients not presented?
- Was a different data cut-off applied?
- Were patients who died, relapsed, discontinued early or became unevaluable excluded?
- Were only infused or response-evaluable patients counted?
- Were initial responses shown without disclosing subsequent relapse or mortality?
- How many responses remained ongoing at three, six and twelve months?
- Were ALL and NHL patients combined despite being biologically and clinically different diseases?
- Were patients treated after the presentationâs data cut-off, or had they already entered the trial pathway?
- Was the near-90% calculation based on the intention-to-treat, enrolled, infused or response-evaluable population?
The public trial target of 41 does not itself prove that all 39 patients described above were infused. That is why the sponsor must publish the complete patient flow rather than leaving outsiders to speculate.
A transparent disposition table should show:
Referred â screened â eligible â enrolled â leukapheresed â successfully manufactured â infused â reinfused, if applicable â response-evaluable â relapsed â deceased â in continuing remission.
Every exclusion from the efficacy denominator should have a documented reason.
Reported CAR-T reinfusion at VIMS
A particularly serious report concerns a patient treated at VIMS.
According to the information provided to me, the patient relapsed after the initial MyCART-01 infusion and was reportedly given a further CAR-T reinfusion in an attempt to achieve remission.
If confirmed, this is not a minor procedural detail. CDSCO must determine:
- Whether reinfusion was permitted under the approved clinical-trial protocol.
- Whether prospective Ethics Committee and CDSCO approval was obtained.
- Whether the patient provided specific informed consent for the additional infusion.
- What clinical evidence and rationale supported the decision.
- Whether the reinfusion was reported as a protocol deviation, if required.
- Whether all toxicities following both infusions were captured in the safety dataset.
- Whether any response after reinfusion was counted in the headline efficacy analysis.
- Whether the Vapi presentation disclosed that any patient had received more than one CAR-T infusion.
Reinfusion is not automatically unethical or non-compliant. It may be permissible if protocol-authorised, appropriately approved, specifically consented to and transparently reported.
If those safeguards were absent, however, the episode could represent significant non-compliance with the approved protocol and applicable ethical protections.
Was the trial population unusually favourable?
The registered eligibility criteria permit patients with an ECOG performance status of 0â1.
However, the information reported to me indicates that the overwhelming majority of enrolled patients were ECOG 0. I have also been informed that some patients who appeared to satisfy the protocol criteria were nevertheless not taken forward.
If confirmed, this could produce a study population materially fitter than the broader relapsed or refractory population for whom the therapy may ultimately be considered.
A predominance of ECOG 0 patients does not itself prove improper selection. Investigators may have legitimate clinical reasons for declining individual patients. But in a small, uncontrolled trial, selection can materially influence treatment completion, safety and response rates.
CDSCO should therefore audit:
- The complete referral and screening logs.
- Baseline ECOG distribution.
- Disease burden and major prognostic factors.
- Reasons for every screen failure and post-screening exclusion.
- Decisions made by any central screening committee.
- Patients who underwent leukapheresis but were not infused.
- Manufacturing failures or out-of-specification products.
- Whether exclusion criteria were applied consistently across centres.
If higher-risk but protocol-eligible patients were systematically excluded, results from a highly selected cohort must not be presented as though they establish performance in the broader Indian population.
The MyCART-01 construct requires greater scrutiny
MyCART-01 has reportedly been presented as a âthird-generationâ CAR-T containing both CD28 and 4-1BB intracellular costimulatory domains, with claims concerning improved activation, expansion, durability and resistance to T-cell exhaustion.
Calling a construct âthird-generationâ does not clinically validate it.
In the FDA-approved product labels I reviewed:
- Axicabtagene ciloleucel uses CD28-associated intracellular signalling.
- Tisagenlecleucel uses a 4-1BB intracellular costimulatory domain.
- Lisocabtagene maraleucel uses a 4-1BB costimulatory domain. Its CD28 transmembrane region must not be confused with a second CD28 intracellular costimulatory domain.
I have not identified an FDA-approved CAR-T product containing both CD28 and 4-1BB intracellular costimulatory domains in the same CAR construct. If such an approved product exists, I welcome a link to its regulatory label.
The absence of an approved precedent does not prove that MyCART-01 is unsafe or ineffective. Novel architecture can be valuable. But combining costimulatory domains can alter T-cell activation, expansion, persistence, cytokine production, exhaustion and toxicity. Claims of superiority must therefore be established through robust nonclinical evidence, cellular-kinetic data, transparent safety reporting and durable clinical outcomes.
A novel investigational architecture cannot be converted into a commercially validated platform merely through an early response percentage from a few dozen selected patients.
Indian patients must not become a substitute for global evidence that does not yet exist.
This is not opposition to Indian innovation
Indian innovation deserves rigorous science, complete evidence and public trust.
What it cannot demand is that Indian patients accept a lower evidentiary standard merely because a product is indigenous, novel or described as âthird-generation.â
A near-90% response rate is an extraordinary claim. When site-level information describes 14 deaths, 11 relapses and only 13 remissions among 39 patients, that claim demands urgent and independent verification.
If the information in this post is wrong, a source-data review will clear the product, reassure patients and strengthen confidence in Indiaâs regulatory system.
If it is correct, identifying the problem before wider patient exposure may prevent avoidable harm.
CDSCO must establish the complete truth before further regulatory or commercial progression.
Corrections and responses
I welcome an evidence-based public response from Micro CRISPR, the participating investigators or CDSCO.
If documentary evidence demonstrates that any figure or statement in this post is inaccurate, I will correct it visibly and preserve the correction history.
Please do not identify, contact or speculate about individual patients or their families. This post concerns clinical-trial transparency and regulatory verification. It is not medical advice and should not influence an individual treatment decision without consultation with the treating medical team.
